The UCLA Health patient was diagnosed with a platelet condition that puts him at high risk of bruising and bleeding
"Sei Nagashima was an ebullient 4-year-old enjoying his first day of preschool in Porter Ranch when a plastic swing hit him lightly on his left eye. By the time his parents arrived to pick him up, the scratch had become a bruise that soon blossomed into a black eye."
- From uclahealth.org. Written by Martha Groves. Photo courtesy of Taku Nagashima.
"After a 19-year-old man developed "a strange rash on his arms and legs" and noticed blood in his urine, he learned that a platelet disorder was preventing his blood from clotting—and tests revealed a surprising trigger."
- From advisory.com. Lisa Sanders writes for the New York Times Magazine.
"For the past year I religiously text my husband, Shea, every Friday afternoon. There’s nothing unusual in that, but the weekly message contains just a number, no words. Triple digits and we’re happy; single digits and it’s time to get family in to mind the children and pack a bag for hospital.
The number I text is my platelet count. I am one of approximately 1,000 people in Ireland with ITP (immune thrombocytopenia), a rare blood disorder in which the immune system mistakenly attacks its own platelets. It can occur in pregnancy, follow a virus, vaccination or certain medications, but for most people the cause is unknown."
-By Karen Tomkins for The Irish Times. Image courtesy of Karen Tomkins and The Irish Times.
The blood drives are especially important because Daniel, as well as his father, relies on blood products every two weeks to survive.
“The importance of donating blood is critical in saving lives. Daniel would not have survived if it wasn’t for blood and plasma donors,” his mother, Dawn, told TAPinto Bordentown in November. “We started Daniel’s Fight because he coded waiting for blood. They did not have a match at the time. So now we hold blood drives every 3 to 4 months.”
- By Kristin Antonello. Photo courtesy "Daniel's Fight"
"For decades, McMaster University in Hamilton, Ont., has been a hub for research on blood and its diseases — known as hematology — but in recent weeks it has taken on an even more prominent role in the field: working to identify the rare blood-clotting syndrome linked to certain COVID-19 vaccines.
The lab, a small space on the third floor of the university hospital, is the only one in Canada with the equipment and expertise to test for the syndrome, known as vaccine-induced prothrombotic immune thrombocytopenia, or VIPIT."
- From cbc.ca. Image copyright and courtesy of McMaster University.
Sobi™, a North American affiliate of the international biopharmaceutical company Swedish Orphan Biovitrum AB (publ) (Sobi®), dedicated to rare diseases, in partnership with the Platelet Disorder Support Association (www.pdsa.org PDSA), a leading immune thrombocytopenia (ITP) advocacy organization in North America, today announced the launch of florio®ITP, an app for patients living with ITP. florio ITP is a digital diary mobile app developed by Florio GmbH, a Sobi company, in collaboration with ITP patients and healthcare professionals. The florio ITP app is intended to help support patients with ITP with a simple tool that allows them to organize, track and record their health and treatment information. It is available for free download for iOS (Apple) and Android mobile phone users.
- From Sobi Press Release
The Platelet Disorder Support Association is proud to announce that its strong financial health and ongoing accountability and transparency have earned a 100/100 rating from Charity Navigator’s new Encompass Rating System. This score designates PDSA as an official “Give with Confidence” charity, indicating that our organization is using its donations effectively based on Charity Navigator’s criteria. Charity Navigator is America’s largest and mostutilized independent charity evaluator.
"Gregory Michael, MD, a 56-year-old ob/gyn in private practice in Florida, died from complications of immune thrombocytopenia (ITP), according to a Facebook post written by his wife, Heidi Neckelmann.
Public health officials at the state and federal level are continuing to investigate Michael's case in relation to the COVID-19 injection, but vaccine experts and hematologists have emphasized that this case is extremely rare, and the coincidental timing of ITP and vaccination doesn't demonstrate causality."
- Amanda D'Ambrosio, Staff Writer, MedPage Today. Photo courtesy of the family. From Facebook.
"ITP's link to COVID gained a media spotlight earlier this month after the Miami obstetrician, Gregory Michael, MD, developed ITP days after getting the Pfizer COVID vaccine. In early January, after 2 weeks in the ICU, Michael died of a hemorrhagic stroke caused by the low platelet count.
Pfizer said in a statement that they are "actively investigating" the case, "but we don't believe at this time that there is any direct connection to the vaccine." Other experts have said the timing, particularly in a relatively young and healthy man, means a link to the vaccine is possible or even likely, but final results won't be known until the US Centers for Disease Control and Prevention finishes its investigation."
- By Donavyn Coffey, Medscape.com
"Anti-vaccine groups are exploiting the suffering and death of people who happen to fall ill after receiving a COVID-19 shot, threatening to undermine the largest vaccination campaign in U.S. history.
In some cases, anti-vaccine activists are fabricating stories of deaths that never occurred."
- By Liz Szabo | Kaiser Health News/Tribune Content Agency
"Kayleigh was diagnosed with hypothyroidism and then hyperthyroidism, which are prevalent for Down syndrome individuals. She also had sleep apnea and immune thrombocytopenia — or ITP — a condition in which the blood doesn’t clot normally.
When Kayleigh reached about 215 pounds, doctors gave Williamson the grim warning that things would not end well for her daughter.
“It dawned on me I didn't want my daughter laying in a hospital bed and me apologizing for her for something I could have prevented with a lifestyle change,” said Williamson.
The mother and daughter made health their main mission. The two joined Weight Watchers, and Williamson, who had just finished her first half marathon, encouraged Kayleigh to start running with her."
- By Travis Recek, Spectrum News 1. Photo courtesy Spectrum News 1.
Rigel Pharmaceuticals, Inc. and Medison Pharma today announced that Health Canada has approved the new drug submission (NDS) for TAVALISSE® (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to other treatments.
"This approval of TAVALISSE provides ITP patients and physicians in Canada with a new oral treatment option, the only therapy to address the underlying platelet destruction that causes ITP," said Raul Rodriguez, Rigel's president and CEO. "With Medison as our collaborative partner, we believe TAVALISSE is well positioned for commercial success in the Canadian market."
In October 2019, Rigel entered into exclusive license agreements with Medison to commercialize TAVALISSE in Canada and Israel. With the approval from Health Canada, Medison intends to launch TAVALISSE in Canada in Q1 2021. In Israel, a decision on the new drug application (NDA) is anticipated during Q2 2021.
- From Rigel Press Release
COVID-19 continues to present a prolonged, global public health emergency, but PPTA remains steadfastly committed to its mission to promote the availability of — and access to — safe and effective plasma protein therapies for all patients. PPTA member companies operate more than 1,000 Source plasma donation centers in the U.S. and four countries in the European Union and manufacture plasma-derived therapies that treat patients with serious, rare, and often genetic diseases and disorders, including: primary immune deficiencies, bleeding disorders, Alpha-1 Antitrypsin deficiency, hereditary angioedema, and certain neurological conditions. These conditions are often only treated with therapies produced from plasma.
There is an urgent need for plasma donations.
- From PPTA Statement
"Laura Hamilton was diagnosed with a bleeding disorder after finding huge bruises.
The 38-year-old TV presenter – who has kids Rocco, five, and Tahlia, four, with husband Alex – was suddenly covered in huge bruises after welcoming her second child, which led to a terrifying diagnosis.
She told the Daily Mirror newspaper: "It was terrifying. I had been on quite a strict diet. I'd cut out sugar and carbs and I was the slimmest I'd been. I was exercising loads and felt in really good shape.
"When Tahlia was about seven months old, I was due to drive to Portugal to film a fitness app, but a few days before I was due to go I started noticing all this bruising coming out on my legs."
Eventually, her mother-in-law suggested she should see a doctor, because she suspected the bruising could be caused by a blood disorder."
- From thelist.co.uk. Photo courtesy laurahamiltonofficial.com.
PPTA has seen plasma donations fall this year. This decline is caused by the ongoing COVID-19 pandemic and the uncertainty felt by plasma donors. Declines in donations have the potential to restrict patients’ access to plasma derived therapies, or PDTs. Amy Efantis, PPTA, President & CEO stated that “Given the length of time to manufacture PDTs, without the needed raw material, it may become challenging at some point to supply enough PDTs to meet rare disease patients clinical needs.” Standard plasma collection remains critically important to support the needs of individuals with rare and chronic diseases. It is encouraging to see an increased awareness about the role convalescent plasma can play as a potential treatment for COVID-19 and we hope this message has a similar effect for standard plasma collection which is needed for patients with rare and chronic diseases every day.
- From PPTA press release
"A cheap and widely available drug can help save the lives of patients seriously ill with coronavirus.
The low-dose steroid treatment dexamethasone is a major breakthrough in the fight against the deadly virus, UK experts say.
The drug is part of the world's biggest trial testing existing treatments to see if they also work for coronavirus.
It cut the risk of death by a third for patients on ventilators. For those on oxygen, it cut deaths by a fifth."
- By Michelle Roberts - Health editor, BBC News online
"According to the Platelet Disorder Support Association, there is no added risk for patients with low platelets to be infected by the virus.
There is also no evidence that platelet production would decrease.
On the group’s website, medical experts say, 'There is no literature to support the virus would suppress platelet production any more than influenza, but some patients seem to be more sensitive to systemic viral infections than others in general.'”
- By Karen Zatkulak for News13 WLOS
The risk of systemic lupus erythematosus (SLE) was higher for patients with idiopathic thrombocytopenic purpura (ITP) in a report published in the Annals of Rheumatic Diseases.
Patient data for this retrospective cohort analysis were examined from the National Health Insurance Research Database in Taiwan. There were 723 patients with ITP included in the analysis and 14,460 patients who did not have ITP and represented the control group. The researchers analyzed patient records to determine if there was a relationship between ITP and an incident diagnosis of SLE.
- Article by Vicki Moore, PhD for HematologyAdvisor.com. Photo courtesy of Hematology Advisor.com.
Combination treatment with thrombopoietin receptor agonists (TPO-RA), immunosuppressants, and intravenous immunoglobulin G (IVIG) appears to be efficacious in the management of patients with severe refractory immune thrombocytopenia (ITP), according to a study published in the British Journal of Haematology.
Researchers evaluated the combination of ciclosporin A and mycophenolate mofetil (CSA/MMF; to inhibit T-cell activation), romiplostim or eltrombopag (to stimulate platelet production), and IVIG (to inhibit autoantibody-mediated platelet destruction) as a way to target 3 different mechanisms of ITP pathology.
- Article by Lauren Dembeck, PhD for HematologyAdvisor.com. Photo courtesy of HematologyAdvisor.com.
COVID-19 didn’t stop a birthday celebration for one special teen in Kalispell on Tuesday.
Cars and fire trucks lined up at Woodland Park and headed over to Nathan Brewer’s house for a birthday parade.
“Today, Kalispell came together to show my 16-year-old son their love and support,” Nathan Brewer’s mother, Nicole Cooper Christensen, said.
Brewer has a rare blood disorder and his treatment recently failed.
- By Vanessa Perez, NBC Montana. Photo by NBC Montana.
Osaka, JAPAN, and King of Prussia, PA, USA
Partnership brings together world-leading plasma companies to focus on developing and delivering a hyperimmune immunoglobulin in the global fight against COVID-19
April 6, 2020
Biotest, BPL, LFB, and Octapharma have joined an alliance formed by CSL Behring and Takeda Pharmaceutical Company Limited to develop a potential plasma-derived therapy for treating COVID-19. The alliance will begin immediately with the investigational development of one, unbranded anti-SARS-CoV-2 polyclonal hyperimmune immunoglobulin medicine with the potential to treat individuals with serious complications from COVID-19.
“Unprecedented times call for bold moves,” said Julie Kim, President of Plasma-Derived Therapies Business Unit, Takeda. “We collectively agree that by collaborating and bringing industry resources together, we could accelerate bringing a potential therapy to market as well as increase the potential supply. We invite companies and institutions focusing on plasma to support or join our alliance.”
“Leaders lead during uncertainty. There is no question that we are all experiencing the impact of COVID-19,” said Bill Mezzanotte, CSL Behring’s Executive Vice President and Head of Research and Development. “This effort aims to accelerate a reliable, scalable and sustainable option for caregivers to treat patients suffering from the impact of COVID-19. In addition to pooling industry resources, we will also collaborate with government and academic efforts as a single alliance whenever we can, including important activities like clinical trials. This will make it more efficient in these hectic times for these stakeholders as well.”
The collaboration will leverage leading-edge expertise and work that the companies already have underway. Experts from the alliance will begin collaborating across key aspects such as plasma collections, clinical trial development and manufacturing. Further companies and institutions may join the alliance as well.
Developing a hyperimmune will require plasma donation from many individuals who have fully recovered from COVID-19, and whose blood contains antibodies that can fight the novel coronavirus. Once collected, the “convalescent” plasma would then be transported to manufacturing facilities where it undergoes proprietary processing, including effective virus inactivation and removal processes, and then is purified into the product.
Individuals interested in donating plasma can visit this link to find the nearest licensed plasma collection center to their location.
- From press release
Abbey died on Jan. 12 after a sudden and brief battle with idiopathic thrombocytopenic purpura, an autoimmune blood disorder in which the immune system attacks the blood platelets. Because platelets help stop bleeding, it can result in easy bruising, bleeding gums and internal bleeding.
After her death, Abbey’s kidneys went to two people. Her pancreas went to one of the kidney recipients. Her liver has been donated for research.
- By Maggie Gilroy, Binghamton Press & Sun-Bulletin. USA Today Network. Photo courtesy of the family.
Researchers have homed in on the cells that cause an autoimmune disease to arise in the body.
The lack of understanding of the root cause of autoimmune disease means that treatment for autoimmune conditions is mostly symptomatic. Most clinicians aim at reducing the level of immune cell activation and controlling inflammation in other organs such as the joints, kidneys, and skin. None of the current treatments addresses the course of the disease directly.
The problem was with the extreme rarity of the rogue cells in a blood sample. Often, they make up less than 0.25 percent or 1 in 400. The most sophisticated analyses of these cells to date have yielded only an estimated average of the enormous variety of different immune cells in the sample. The current study thus switched to sequencing the genome of each cell in the sample.
- By Dr. Liji Thomas, MD. Image Credit: Dr Ofir Shein-Lumbroso.
A significant number of pediatric patients with immune thrombocytopenia (ITP) have difficulties with fatigue, emotional and behavioral symptoms, and executive functioning, with each having an effect on quality of life (QoL), according to a paper published in the British Journal of Haematology.
Fatigue, minor cognitive impairment, and overall reduction in health-related QoL are concerns in adults with ITP, but no studies have previously measured emotional and behavioral issues among pediatric patients. Therefore, pediatric patients are generally treated with a “watch and wait” approach.
- By Jonathan Goodman, MPhil for Hematology Advisor. Photo courtesty of pixabay.
Doctors diagnosed Bridget Fox, 6, with Immune thrombocytopenia purpura, or ITP, about a year ago during a kindergarten physical. The disorder causes excessive bleeding and bruising.
"She's had eight different blood transfusions. One of them was a platelet transfusion. Then she received medication that has blood products in it," said Tabitha Fox, Bridget's mom.
- By Kelsey Dickeson for KSNB News. Photo courtesy KSNB and Tabitha Fox.
Dova Pharmaceuticals, Inc., a wholly owned subsidiary of Swedish Orphan Biovitrum AB (Sobi™) has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for avatrombopag for the potential treament of Chemotherapy-Induced Thrombocytopenia (CIT). Enrollment remains ongoing for the phase 3 clinical study for the treatment of patients with CIT.
CIT, a potentially serious complication of chemotherapy, results in low platelet levels and can lead to chemotherapy dose reductions, chemotherapy dose delays, or changes to chemotherapy regimens. For cancer patients receiving chemotherapy with curative intent, alterations in their chemotherapy regimen due to low platelets may compromise their long-term outcomes. Approximately 10 percent of US cancer patients per year experience CIT2 which may require chemotherapy regimen modifications. Currently there are no approved treatments available for CIT in the US or EU.
- From SOBI Press Release
Evidence-Based Guidelines to Drive Improved Care for Patients with ITP
Published on: December 03, 2019
(Washington, DC, Dec. 3, 2019) — Today, ASH published new state-of-the art guidelines on the treatment of immune thrombocytopenia (ITP), a rare blood disorder characterized by a decrease in the number of platelets, the part of the blood needed for normal clotting. The guidelines are published in the journal Blood Advances.
The 2019 ASH Clinical Practice Guidelines on ITP, developed in partnership with the University of Oklahoma Health Sciences Center, synthesize all available evidence about existing treatments to offer recommendations for disease management in both children and adults.
- From the American Society of Hematology
Over the last decade, there have been numerous developments and changes in treatment practices for the management of patients with immune thrombocytopenia (ITP). This article is an update of the International Consensus Report published in 2010. A critical review was performed to identify all relevant articles published between 2009 and 2018. An expert panel screened, reviewed, and graded the studies and formulated the updated consensus recommendations based on the new data. The final document provides consensus recommendations on the diagnosis and management of ITP in adults, during pregnancy, and in children, as well as quality-of-life considerations.
- From the American Society of Hematology
Patients with steroid-refractory immune thrombocytopenia (ITP) who received rituximab after splenectomy derived superior benefit compared with those given rituximab before splenectomy, according to William A. Hammond, MD, FACP, Division of Hematology and Medical Oncology, Mayo Clinic, Jacksonville, Florida, and colleagues.
To assess the effect treatment sequence has on time to relapse, Dr Hammond et al evaluated freedom from relapse (FFR) and response rates after rituximab or splenectomy as single or sequential interventions in 218 patients with steroid-refractory ITP.
All patients in the study underwent treatment with splenectomy or rituximab from January 1, 2002, through December 31, 2015, at Mayo Clinic.
- By Hina Porcelli for Oncology Learning Network
Former Pakistan prime minister Nawaz Sharif continues to remain critical and his platelet count has dropped again even as doctors are trying to reduce the steroid dose being administered to the ailing leader.
"Former PM NawazSharif remains critical. The treating doctors tried to reduce the Steroids dose being given to him but unfortunately resulted in a drop in Platelet count again. The cause needs to be diagnosed & established without delay," Sharif's personal physician Adnan Khan tweeted on Saturday.
"Severe existent co-morbidities has added to the seriousness of the nature of the critical illness, where a very delicate balance has to be maintained between coagulation & anti-coagulation to sustain fragile unstable health status," he said in a follow-up tweet.
Earlier on Tuesday, the doctor said that Sharif's health was in a critical condition and fighting a battle for survival.
- From Asian News International. Photo courtesy of Wikipedia.com.
Without access to eltrombopag, Gellein continues to take other medications and suffer from side-effects of anxiety, depression, weakness, nausea and dizziness. She is admitted to hospital several times each year for emergency treatment, which is common for patients with uncontrolled ITP.
She wants people to know the ripple effect it has caused on those around her, and desperately wants her life back.
"My life could have been so much different with these drugs," said Gellein. "Something needs to change, if not for me, for the hundreds of young people being diagnosed with ITP in Canada every year."
- By Dr. Meera Dalal for CBC News. Photo courtesy of Chrystal Gellein.
Sharif, 69, is being treated at a hospital in Lahore, where medical tests suggest he has a low count of blood platelets.
Jailed former Pakistani Prime Minister Nawaz Sharif is suffering from a serious immune disorder, his party leaders say, accusing the government of not providing him adequate medical care.
Sharif, 69, is being treated at a government-run hospital in the eastern city of Lahore, where medical tests suggest he has a dangerously low count of blood platelets, Talal Chaudhry, a senior leader of Sharif's PML-N party, told Al Jazeera on Friday.
"It is acute immune thrombocytopenic purpura, a bleeding disorder, in which the immune system destroys platelets," the doctor told Dawn, speaking on condition of anonymity. "We are hopeful that his condition will improve in a few days."
- by Asad Hashim for aljazeera.com. Photo courtesy of Wikipedia.
Amgen today announced that the U.S. Food and Drug Administration (FDA) approved Amgen's Supplemental Biologics License Application (sBLA) for Nplate® (romiplostim) to include new data in its U.S. prescribing information showing sustained platelet responses in adults with immune thrombocytopenia (ITP), a rare, serious autoimmune disease characterized by low platelet counts. The updated indication expands treatment with Nplate to newly diagnosed and persistent adult ITP patients who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. In December of last year, the FDA approved another sBLA for Nplate in the treatment of pediatric patients with ITP.
"These new data are the first of their kind to prospectively examine treatment-free remission as an outcome for patients with ITP. Thirty-two percent of patients who received Nplate soon after an insufficient response to the first course of steroids maintained platelet response for at least six months without Nplate or any other ITP therapy," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "This approval will provide patients the opportunity to receive Nplate earlier in the course of their disease, potentially reducing their need for prolonged steroid use.
- From Amgen press release
Rigel Pharmaceuticals, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a positive trend vote on the Marketing Authorization Application (MAA) for fostamatinib disodium hexahydrate (fostamatinib). The indication for the positive trend vote is for the treatment of chronic immune thrombocytopenia in adult patients who are refractory to other treatments.
"During the EMA review process for fostamatinib in adult chronic ITP, we have had very constructive interactions with the committee," said Raul Rodriguez, president and CEO of Rigel. "We are pleased with this positive trend vote from the CHMP this week, which brings us one step closer to potentially providing a new therapeutic option for a patient population that has a clear unmet clinical need."
- From Rigel press release
To date, the Phase 1/2 trial of PRN1008 has enrolled 26 adult patients who have had two platelet counts < 30,000/µL within 15 days prior to treatment. Oral PRN1008 starting doses were 200mg once daily, 400mg once daily, 300mg twice daily, and 400mg twice daily, with intra-patient dose escalation allowed every four weeks, with the trial having a current median treatment duration of 12.7 weeks (range 0.14 to 39.71).
Of the 26 patients enrolled to date, 39 percent (80% confidence interval (CI) 27.3, 51.0) achieved the trial’s primary endpoint of ≥ 2 consecutive platelet counts of ≥ 50,000/µL, separated by at least five days, and increased by ≥ 20,000/µL from baseline without requiring rescue medication. In addition, 46 percent (80% CI 34.2, 58.5) of enrolled patients achieved any 2 platelet counts ≥ 50,000/µL.
- From Principia press release.
When Nowak's platelets get low, hemorrhaging occurs in the form of dark red, rash-like blotches all over his legs, arms, chest and back. Plus, he has dealt with leg pain this season from compartment syndrome that developed in the spring.
Despite this, he has run cross country and track, and swam all four years at Wheeler. In making it to the top of Mount LeConte, he proved something to himself.
“It was definitely one of the hardest things I've ever done,” Nowak said. “I've been through so much with ITP, and I think it can just symbolize how if you keep pushing through things, you can make it and keep doing what you want to do.”
- By Robbie Weinstein for nwitimes.com. Photo by Robbie Weinstein.
The immune system is meant to protect the body from foreign invaders. A rare condition called immune thrombocytopenia (ITP) occurs when the body’s defense system becomes confused and targets healthy blood cells. The body starts destroying its own platelets, leading to bleeding. The severity can range from simple nosebleeds to life-threatening bleeding in the brain or gastrointestinal tract. Baylor College of Medicine researchers at Texas Children’s Hospital are working to find out if a drug used for the chronic form of ITP could be used early in the disease course and improve outcomes.
- By Molly Chiu for bcm.edu (Baylor College of Medicine). Photo courtesy of Wikipedia Commons.
THIS 14-YEAR-OLD HAS AN INCURABLE DISEASE. BUT HE SMILES THROUGH IT ALL.
Sean was diagnosed in second grade with chronic ITP, or immune thrombocytopenia, a disorder in which the immune system mistakenly attacks platelets —the cells that help blood to clot. If the platelet count dips too low, it can lead to bruising and excessive bleeding.
But there he is smiling through treatment after treatment, too many to count, including four chemotherapy sessions late last year.
Smiling through the frustration of sometimes being isolated from other kids while he fights an incurable disease.
Smiling through the fear that he might be deprived of his biggest love: soccer.
- By Jon Spencer, Mansfield News Journal. Photo courtesy of the Mansfield News Journal and the Putt Family.
FDA monitoring confirms that, despite increased supply of immune globulin (IG) products in recent years, the demand for IG product has also increased over that same time and there is an ongoing shortage of Immune Globulin (Intravenous) (IGIV) and Immune Globulin (Subcutaneous) (IGSC) products in the United States. This shortage could impact patient care.
In the setting of the increased demand for IG, other factors that ordinarily might not impact availability may have a greater effect. These could include uneven product distribution across different localities, logistics of contractual obligations, production delays, and other factors.
- From FDA.gov
I live in a town of fewer than 35,000 people with only two aged hematologists about to retire, five general surgeons, and one hospital. My splenectomy was my surgeon’s third. Before surgery, we tried just about every chemical therapy it was reasonable to try. I was refractory to them all. And yet, still, I had to ask to be sent 244 miles away for a referral to another hematologist because the two in my small town were not up-to-date on my disease and had the mistaken belief that there was a high risk of bone marrow fibrosis with the latest medications. This research is more than 10 years old. But for most people with my diagnosis, it’s easier to just throw some prednisone at them and call it a day.
- By Colin Justin for TheMighty.com
Given recent reports regarding the availability of various immunoglobulin (IG) products that are administered intravenously (IVIG) or
subcutaneously (SCIG), PPTA is sharing Manufacturer Toll-Free Numbers (MTFNs) currently in place. We will update this list as necessary.
The available MTFNs are intended to enable health care professionals to inquire about specific brands of IG therapy for their patients. The
MTFNs are for health care professionals because when an incoming call is received, the company representative may require specific clinical
information that only the treating physician or health care provider can furnish. If the manufacturer and health care professional are able to
meet the patient’s clinical need, the caller may be required to provide their DEA Registration Number and specific billing and shipping
information.
- From PPTA
For the last eight years, Leslie MacGregor from Newton, Massachusetts, has been getting intravenous infusions every month. The treatment is called immunoglobulin, and it bolsters her defective immune system with concentrated, infection-fighting antibodies from healthy donors.
Without it, she would get serious infections of her lungs or skin, bad enough to land in the hospital. She would also suffer autoimmune attacks that cause agonizing nerve pain. When they flare up, she says, "it makes me feel like my feet and legs are being dipped in boiling oil."
MacGregor's latest infusion was scheduled for the end of June at Massachusetts General Hospital. Five days before, her doctor called and told her that she would only be able to get half her usual dose, and that her next six months of scheduled infusions were all canceled.
- By Carey Goldberg for 90.9FM, WBUR
Although upfront management of immune thrombocytopenia (ITP) in adults has not changed much over the years, advances in the treatment of refractory ITP have some clinicians looking for guidance. In 2018, the FDA approved fostamatinib (Tavalisse) as a second-line treatment for adults with chronic ITP, making it the first new drug marketed for adults with ITP since approvals of romiplostim (Nplate) and eltrombopag (Promacta) a decade earlier. The FDA is also considering a request to expand indications for avatrombopag (Doptelet) to include refractory ITP in adults, with a decision expected by June 30, 2019.
- By Christin L. Melton, ELS
The Living Rare, Living Stronger NORD Patient & Family Forum was held on June 21-23 in Houston. More than 500 people attended the weekend event, which was filled with educational opportunities, as well as plenty of sharing of stories, networking and bonding, and fun.
PDSA was highlighted at the event as the recipient of the 2019 NORD Abbey S. Meyers Leadership Award for the organization’s impact in the fight against rare diseases. “It is truly an honor to be recognized by NORD and the 280 rare disease organizations they represent,” said Caroline Kruse, President and CEO of PDSA. “We are grateful for our partnership with NORD, which has enabled us to advance our mission and make a difference in the lives of ITP patients through our ITP Natural History Study Registry and research program.”
The FDA expanded the approval of avatrombopag to include treatment of adults with chronic immune thrombocytopenia who had insufficient response to previous treatment.
Avatrombopag (Doptelet, Dova Pharmaceuticals) — an oral thrombopoietin receptor agonist administered with food — already had been approved for treatment of thrombocytopenia among adults with chronic liver disease who are scheduled to undergo a procedure.
The FDA based the chronic immune thrombocytopenia (ITP) indication in part on results of a phase 3 study that showed avatrombopag administration resulted in a platelet count of at least 50,000 per µL at day 8 of therapy for most patients with chronic immune thrombocytopenia. The agent appeared superior to placebo for maintaining platelet counts in the target range during the 6-month target period.
- From Healio.com
Hospitals are scary places, especially for kids. Ella Casano knows that all too well: Five years ago, the 12-year-old from Fairfield, Connecticut, was diagnosed with a rare autoimmune disease called Idiopathic Thrombocytopenia Purpura (ITP).
Some children outgrow ITP but Ella hasn't been so lucky. So every eight weeks she has to get an IV infusion, an experience that filled her with anxiety.
"When I had my first infusion, I was surprised and a little bit intimidated by the look [and] the amount of tubing and medical equipment on my IV pole," she wrote on her website. "As I saw more and more children experiencing the same feelings, I became more interested in creating a friendlier experience for young IV patients."
So she cut up a stuffed animal, grabbed a hot-glue gun and created what would become the first "Medi Teddy," a stuffed animal pouch that covers and conceals an IV bag.
- By Daniel Avery for Newsweek.com. Photo courtesy of Meg Casano.
Hematology analyzers help providers diagnose certain blood disorders and assess appropriate treatment options
The U.S. Food and Drug Administration is alerting hospitals, laboratories and providers of a Class 1 recall of Beckman Coulter DxH 800, DxH 600 and DxH 900 hematology analyzers—devices that run blood tests to help providers diagnose diseases and conditions such as anemia (low red blood cell or hemoglobin count), infections, blood clotting problems, blood cancers and immune system disorders. This is an update to an urgent medical device correction letter first issued by the company in 2018, after the company received complaints of inaccurate blood platelet counts.
Hematology analyzers run diagnostic tests that count the number of different types of red and white blood cells, platelets, hemoglobin (oxygen levels) and hematocrit (volume of red blood cells in blood) levels in a blood sample. These diagnostic tests are often performed as part of routine patient check-ups and are commonly used as part of pre-surgical laboratory patient assessments to help providers assess if patients are suitable and healthy for surgery. They are also used to determine whether a patient with a very low platelet count needs platelet transfusion or to evaluate for potential bleeding and bruising disorders. Platelets help blood to clot, so a patient with a very low platelet count (severe thrombocytopenia) may be at an increased risk for life-threatening bleeding. The recall is related to the devices' platelet analyzing function. Beckman Coulter has not received complaints that this issue impacts other reported parameters, including white blood cell count, white blood cell differential, red blood count or hemoglobin tests.
- From U.S. Food and Drug Administration Press Release. Photo courtesy of Beckman Coulter.
Udom had been hospitalized over the weekend, receiving treatment for a blood disorder called immune thrombocytopenic purpura (ITP) characterized by a decrease in platelets in the blood. She left the hospital Sunday, and a day later became the eighth girl in state history to shoot a 67 or better at the state championships.
Udom reached the 17th tee tied for the lead. She pulled a hybrid out of her bag, looking to find the fairway on the par-5 and give herself a chance at birdie.
- By Rene Ferran for The Oregonian/OregonLive. Photos by Taylor Balkom, for The Oregonian/OregonLive.
A Madison-area business executive decided to change his hairdo to benefit a kid diagnosed with an autoimmune disease.
Cayden Krueger was diagnosed with immune thrombocytopenia purpura when he was 6 years old. Patients with ITP have too few platelets in their blood, resulting in easy bleeding and bruising. Cayden has been spreading awareness of ITP through fundraising and by having a purple mohawk.
When John Stevenson, U.S. Cellular's senior director of financial services, found out about Cayden, he decided to challenge his employees and real estate teams to raise money for Cayden's Pump It Up for Platelets Fundraiser.
- From channel3000.com. Photo courtesy of the Krueger family.
May 11, 2019
Novartis today announced a voluntary recall of three lots of Promacta (eltrombopag) 12.5 mg for oral suspension to the consumer level. The oral suspension lots are being recalled because of a risk of potential peanut flour contamination that occurred at a third-party contract manufacturing site.
Promacta tablets in 12.5 mg, 25 mg, 50 mg and 75 mg strengths are not impacted by this recall and are not manufactured in the same facility.
- From Novartis Press Release
“The worst day of my life, the worst day of my wife’s life, was that day,” said David Watkins, Maya’s father. “I can’t even begin to tell you how horrible it was. It was a big scare, and the bone marrow biopsies were not easy for Maya. They are extremely painful. And because her bones are so dense as a collegiate athlete, they couldn’t go deep enough punching through the hip, so they had to actually do it twice. It was brutal.”
Thankfully, the biopsy came back negative for cancer.
“She handled it all with grace,” Boise State beach volleyball coach Allison Buck said. “She had a great attitude about it and I can’t say enough about how much strength she’s shown.”
- By Rachel Roberts, Idaho Statesman. Photo courtesy of David Watkins.
Different antiphospholipid antibody (APA) profiles in patients with immune thrombocytopenia (ITP) may affect individual response to ITP-specific treatments, according to a study published in Hematology (2019;24[1]:134-138).
“The relevance of detecting antibodies against anticardiolipin, β2-glycoprotein I (β2gpI) or lupus anticoagulant (LA), collectively called antiphospholipid autoantibodies (APA), in subjects with…ITP…is still a debated issue,” explained investigator Fabrizio Vianello, MD, Hematology and Clinical Immunology Unit, Department of Medicine, Padua University School of Medicine, Italy, and colleagues.
“In particular, whether APA profile may affect the clinical course of ITP is unknown,” they added.
- By Janelle Bradley for Oncology Learning Network
"The Smiths are one of four families with children who attend Loyola Catholic School in Mankato who came together Sunday for Chillin’ for a Cure; raising awareness, support and research for rare diseases with a bake sale, vendors, a chili cook-off and bingo. Each family chose an organization to highlight diseases they had experienced personally.
Despite the subzero temperatures Sunday, over 300 people showed up to show solidarity and support. Odegard said several people approached him to share their own personal stories of coping with rare diseases."
- By Dan Greenwood, The Free Press serving South-Central Minnesota. Photo by Dan Greenwood.
"Taylor Hardin had big plans for her junior year of high school.
A stand-out basketball player, a few of her goals included scoring her 1,000th career point and seeing her team go to state.
Both of those things happened, but it wasn't without a lot of struggle and hardship.
In September 2018 Taylor, 16, went in for a regular check-up and unexpectedly her pediatrician decided to do blood work. Those tests revealed that Taylor had idiopathic thrombocytopenic purpura (ITP), a blood disorder that leads to bleeding and excessive bruising due to low levels of platelets, the cells that help blood clot."
- By Ashley Gardner, Texarkana Gazette. Photo by Hunt Mercier, Texarkana Gazette.
The Platelet Disorder Support Association has been selected as the recipient of the 2019 NORD Abbey S. Meyers Leadership Award for the organization’s impact in the fight against rare diseases. “It is truly an honor to be recognized by NORD and the 280 rare disease organizations they represent,” said Caroline Kruse, President and CEO of PDSA. “We are grateful for our partnership with NORD, which has enabled us to advance our mission and make a difference in the lives of ITP patients through our ITP Natural History Study Registry and research program.”
PDSA will be honored at the NORD 2019 Rare Impact Awards, taking place Saturday, June 22 at Space Center Houston. “There are 25-30 million Americans living with rare diseases today, and more than half of them are children,” said Peter L. Saltonstall, President and CEO of NORD. “Thankfully, there are also people, organizations and companies doing extraordinary work to help improve and save their lives. It is our privilege to honor them for their stellar contributions to the community, and our pleasure to do so in a truly out-of-this-world setting.”
This year’s Rare Impact Awards event will be presented as part of the 2019 Living Rare, Living Stronger NORD Patient and Family Forum, which will bring the rare community together with physicians, medical students and health care professionals for a program of learning, sharing and connecting in Houston, TX.
About the 2019 Rare Impact Awards: https://rarediseases.org/nord-announces-honorees-2019-rare-impact-awards/
Cayden was surrounded by family and friends Saturday morning when he received the check, including his best friend from school.
"It means more to me than anything, knowing that we have 18 people here with us today, all family," said Cayden's mom, Jenni Krueger.
Professional dancer Derek Hough also helped present the check, and he said meeting kids like Cayden is why he loves being part of this.
"The secret to living is giving, and they really embody that, and it's really, really beautiful to see that and to see how they're turning sort of a typical bad situation but turning it into something good and to help their community in some way," Hough said.
- By Sanika Bhargaw, WMTV
Together Jeff and Kristen put on a fundraiser last year in honor of Marci Mayhew, who is the facilitator of the Clare, MI support group and a good friend of Kristen Blackburn, and ended up raising over $2,000 for PDSA.
"Jeff has been responsible for several fundraisers and special events at Sunset Shores, including a recent benefit for patients with ITP (Immune thrombocytopenia), a rare blood disorder that causes uncontrolled and wide-spread bruising. The event raised funds for the PDSA, (Platelet Disorder Support Association)."
- By Pat Maurer, Correspondent for Clare County Review
A Townview Elementary third grader will get the trip of a lifetime when his family goes to Disney World and Universal Studios in Florida on March 4-8, courtesy of the Kids Wish Network.
The student was awarded with the trip because of his efforts to spread awareness for his health condition, Idiopathic Thrombocytopenic Purpura (ITP).
Cayden Krueger, 8, is the son Jenni Krueger and Bob Krueger; and brother to Cody, 18, Casie, 17 and Cora, 5.
He learned about the trip during a Super Bowl party on Sunday. The Kids Wish Network had sent him a box from the Green Bay Packers filled with a football, shirt, hat, stuffed dog, flag, stickers and card from Aaron Rodgers as well as Rodgers' photo and autograph. He opened it and was thrilled with the many contents.
- By Hillary Gavan, Beloit Daily News
Cayden Krueger was surprised with a trip to Disney World for his efforts to bring awareness to a rare disease.
Cooper Higgins, 7, battling rare blood disorder known as idiopathic thrombocytopenic purpura
Cooper stopped playing hockey and had to be home-schooled because of his condition. A simple nose bleed could be fatal and even a minor bump on the head could lead to internal bleeding in the brain.
Over the next year, the family cycled through medication and treatments, trying anything they could to make a difference. But nothing worked longer than a few weeks and often the side effects were even worse.
Kelly estimates they have spent thousands of dollars on medicines. However, they finally found something that is working … at least for the time being.
- By Eric Bowling, The Westlock News. Photo courtesy of the family.
With a smile that lights up her eyes and a giggle that is a gift from God, 4-year-old Makayla Willett has some of Fort Wayne’s toughest and strongest athletes willing to do just about anything for her. She also might be more resilient than all of them.
Makayla’s dad is Fort Wayne Komets athletic trainer Matt Willett so she’s around the rink and the team frequently, but one place the players never expected to find her was at Lutheran Children’s Hospital. When the team made their recent annual Christmas visit, Makayla and her mom and dad were there for what to them has become a normal, weekly visit, but it’s something that would terrify any other parents.
- From News-Sentinel.com. Courtesy photo.
Most pediatric patients with immune thrombocytopenia (ITP) had rapid and durable platelet responses when treated with the thrombopoietin fusion protein analog romiplostim (Nplate), according to integrated data from 5 clinical trials that were presented at the 2018 ASH Annual Meeting.
Nearly 90% of 282 evaluable patients had platelet responses, which occurred after a median treatment duration of 6 weeks. Patients remained in response for a median of 76% of months during follow-up.
- From OncLive
"Immune thrombocytopenia is different in children than in adults, including the fact that it often resolves on its own
While immune thrombocytopenia (ITP) can occur in both adults and children, what makes pediatric ITP most distinct from adult ITP is the high rate of children who improve on their own, noted James Bussel, MD, of Weill Cornell Medical College in New York City.
According to the Platelet Disorder Support Association, ITP affects at least 3,000 children annually in the U.S. -- most between the ages of 2 and 6. And like adults, children will demonstrate the same symptoms of ITP -- the bruises and/or small purple or red spots on the skin called petechiae, as well as nosebleeds, bleeding gums, and blood in the urine or stool.
It is unclear why some children get ITP, but it seems to occur most often in previously healthy children a few days after they've come down with a minor viral infection. According to this study, there was a history of previous infection in 60% of pediatric cases."
- by Mike Bassett, Contributing Writer, MedPage Today
The Platelet Disorder Support Association (PDSA) Board of Directors is pleased to announce that Executive Director Caroline Kruse has been named President and Chief Executive Officer. Kruse, who has been Executive Director of PDSA since January 2009, will serve in the dual role focusing on the core functions of the organization including strategy, finance, board management, fundraising, and program delivery.
Under Caroline’s dedicated leadership, PDSA has developed new relationships with key disease stakeholders and government agencies and strengthened existing relationships with partners and supporters. Her focus on mission has resulted in a greater emphasis on advocacy, expanding programs for children, teens and families affected by ITP, increasing education initiatives and advancing research to improve the quality of life for those living with immune thrombocytopenia (ITP) and other platelet disorders.
“Under Caroline’s leadership PDSA has become the go to source for patients, families and caregivers living with ITP. In addition, PDSA has become highly respected by the medical community and is a model for this type of patient support operation. Becoming President and CEO is in recognition of her exceptional leadership over the past ten years as Executive Director,” said Peter Pruitt, chairman of the board of directors for PDSA.
Prior to her appointment as Executive Director, Caroline was PDSA’s first Director of Public Relations. Before joining the PDSA staff, she served for two years as a PDSA board member, the second year as Vice Chair. Caroline co-founded the first local ITP support group in the country in Cleveland, Ohio in 2003, and still serves as its facilitator. She brings a personal perspective to PDSA as a patient with two rare diseases, including ITP, so she knows firsthand the physical and emotional struggles ITP patients face daily.
“I am honored to be appointed President and CEO of PDSA,” said Kruse. “It is a privilege to lead an organization that has such a meaningful impact on the patient community we serve. I am excited about the important work ahead as we continue to collaborate with researchers, clinicians and industry, building upon our progress to lessen the burden of living with ITP and other platelet disorders, and empower patients.”
Prior to joining PDSA, Caroline worked as a broadcasting professional for over 20 years, working on-air and behind-the-scenes at WCPN, Cleveland’s National Public Radio affiliate, and at FOX and CBS TV affiliate stations. She is the creator, producer and co-host of the nationally syndicated talk radio program, “Family Matters.” Caroline is the recipient of numerous broadcasting and journalism honors, including Ohio Excellence in Journalism Awards, Cleveland A.I.R. Awards from the March of Dimes, Distinction in Media Excellence Awards, and Cleveland Communicators’ Award from Women in Communications. Her award-winning programs focused primarily on health and family issues.
Kruse is a graduate of Cleveland State University, where she earned a B.A. degree in Communications.
The Platelet Disorder Support Association, a 501 (c) (3) nonprofit corporation, was patient-founded in 1998 to educate and empower those with immune thrombocytopenia and other platelet disorders. Today, PDSA is a powerful force serving and unifying the global ITP community of patients, practitioners, caregivers, advocates and key disease stakeholders. We build awareness, educate the global community, and provide critical connections and resources that empower patients to take charge of their disease and encourage practitioners to exercise patient-centered medical care. https://pdsa.org/
Principia Biopharma Inc., a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology, today announced that PRN1008, an oral, reversible covalent Bruton’s Tyrosine Kinase (BTK) inhibitor, has been granted orphan-drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with immune thrombocytopenic purpura (ITP). Principia is currently conducting a Phase 2 clinical trial to evaluate the safety and efficacy of PRN1008 in patients with ITP. Principia previously received orphan-drug designation for PRN1008 by the FDA for the treatment of pemphigus vulgaris in June 2017.
“The FDA granting orphan-drug designation is an important milestone as we continue to develop PRN1008 for ITP patients in need,” said Martin Babler, Chief Executive Officer of Principia. “We believe that PRN1008 has the potential to modulate the immune system’s attack on platelets, the underlying cause of ITP, and to be an effective oral therapy to reduce the untoward effects often seen with more invasive or broader immune suppression.”
- From Principia Biopharma Inc. press release
On October 15, 2018, PDSA President & CEO, Caroline Kruse joined this panel discussion moderated by Christopher P. Austin, M.D., Director, NCATS, NIH at the NORD Rare Diseases & Orphan Products Breakthrough Summit. The panel discussed innovative collaborations and how the role of patients, caregivers and patient organizations has become instrumental in helping to advance research and development of therapies.
"Dr. Jenny Despotovic, director of the Immune Hematology program at the Texas Children’s Hematology Center, served as editor of the recently published textbook Immune Hematology: Diagnosis and Management of Autoimmune Cytopenias. The textbook provides a concise yet comprehensive overview of the most common autoimmune cytopenias affecting adults and children.
This is the first textbook dedicated exclusively to immune cytopenias. Despotovic worked with several of her colleagues to develop, write and edit the content in the textbook, which had over 2,000 downloads in the first two months since publication."
- From Texas Children's Hospital "Connect News"
First SYK inhibitor for the treatment of adult chronic ITP
SOUTH SAN FRANCISCO, Calif., Oct. 11, 2018 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for fostamatinib in adult chronic immune thrombocytopenia. The validation was received on October 4, 2018 and initiated the MAA review process. The company anticipates a decision from the Committee on Human Medicinal Products by the fourth quarter of 2019. Currently, fostamatinib is commercially available in the U.S. under the brand name TAVALISSE™ (fostamatinib disodium hexahydrate), which is the first and only SYK inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment.
- From Rigel Press Release
"Charlie Clarke and his Mum Carrie, from Wrexham, are raising awareness of ITP (Immune Thrombocytopenia) by taking part in the Rare Aware photography project by Mold-based organisation Same but Different.
"It’s important to us that there is more awareness, which is why we wanted to take part in the Rare Aware campaign.
"Knowledge is power. The more people that know about ITP and other rare conditions, the better."
- By Steve Craddock, Chief Reporter, The Leader. Photo Courtesy Ceridwen Hughes.
"Sara’s lab results revealed abnormal blood levels, including a platelet count near 11,000. Platelets help stop bleeding by clotting; a normal person’s platelet count can range from 150,000 to 400,000. In most cases, surgeons won’t perform surgery on someone with a platelet count under 50,000. At 11,000, Sara was at risk for serious internal bleeding.
Our attending hematologist, Dr. Jenny Despotovic, eventually diagnosed Sara with immune thrombocytopenic purpura (ITP), a rare autoimmune blood disorder in which the body’s immune system attacks and destroys platelets. Dr. Despotovic recommended an immediate blood transfusion and steroids as a first line of treatment. We were hopeful when Sara’s platelet count rebounded initially, but she required another blood transfusion and round of steroids when her count plummeted a few months later. Though this helped stabilize Sara, her platelet count dropped to under 10,000 by late July."
- By Mark and Sara Ciesielski, patient family. Photo courtesy of Paul Kuntz
In 2015, at the age of 20, Jessica was diagnosed with a second autoimmune disease known as ITP. Immune thrombocytopenia is a rare bleeding disorder characterized by a low amount of platelets in the blood, according to the ITP foundation.
“If you have one autoimmune disease, you are generally more susceptible to others,” she said.
Jessica had to undergo chemotherapy treatments to get her platelets up.
Her platelet count is somewhere between 9 and 12, she said. “Human beings should be at about a 300 platelet count.”
“Since my body attacks my liver, they have me on immune suppressants,” Jessica said. “When I get sick, it is worse. It’s never a simple cold. I get hospitalized.”
- By Michele Lutes for The Signal Santa Clarita Valley. Photo by Dan Watson, The Signal.
"The European Commission has authorised Cablivi (caplacizumab) as the first therapeutic specifically indicated for the treatment of adults experiencing an episode of aTTP.
The condition is a life-threatening, autoimmune-based blood clotting disorder characterised by extensive clot formation in small blood vessels throughout the body, leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction), ischemia and widespread organ damage."
- By Selina McKee for Pharma Times Online.
"Platelets are blood cells that help with clotting, and it is essential to maintain their levels. However, some people have thrombocytopenia, or a low platelet count, which means they have to find ways to increase their levels. Eating certain foods may help increase a person's platelet count naturally.
Foods to eat to increase platelet count include:
Avoiding specific products, such as alcohol and the artificial sweetener aspartame, can also help to increase platelet count."
- By Jayne Leonard for Medical News Today.
"It's called the Survivorship program. Susan visits the Roger Maris Cancer Center four days a week, providing an escape for patients receiving treatments. Which is how she met 10-year-old Megan Hillman.
"Megan and I fell in love right away and the minute I met Megan I knew I had someone really special," said Susan Kolstad.
These two spend hours together creating beautiful arts and crafts while Megan receives her treatments.
"My spleen is enlarged and my spleen is eating up my platelets," explained Megan Hillman.
Megan has a disorder called ITP, it's not cancer, but it does require her to get regular treatments. Something that would be pretty dang scary if Susan wasn't there."
- By Tyler Ziegler for WDAY News. Photo courtesy WDAY News.
"The FDA has accepted Novartis’ supplemental New Drug Application (sNDA) and granted Priority Review designation to Promacta (eltrombopag) in combination with standard immunosuppressive therapy (IST) for first-line treatment of severe aplastic anaemia (SAA), a rare and serious blood condition.
Priority Review designation is used for treatments that address a serious or life-threatening disease or condition and, if approved, would provide a significant improvement in treatment safety or efficacy."
- By Linda Banks, pharmaphorum.com.
Results of Two Phase 3, Randomized, Placebo‐Controlled Trials
"The primary endpoint was stable response (platelets ≥50,000/μL at ≥4 of 6 biweekly visits, weeks 14‐24, without rescue therapy). Baseline median platelet count was 16,000/μL; median duration of ITP was 8.5 years. Stable responses occurred in 18% of patients on fostamatinib vs. 2% on placebo (P=0.0003). Overall responses (defined retrospectively as ≥1 platelet count ≥50,000/μL within the first 12 weeks on treatment) occurred in 43% of patients on fostamatinib vs. 14% on placebo (P=0.0006). Median time to response was 15 days (on 100mg bid), and 83% responded within 8 weeks."
- From Wiley Online Library
"She went to urgent care, thinking it would be a quick visit, but doctors kept her in the hospital for 43 days because she had immune thrombocytopenia purpura (ITP) - a condition characterized by low levels of platelets, blood cells that prevents bleeding.
'This is a very serious condition,' she said. 'If you were to just slightly bump your head you can have internal bleeding.'
During that period of time, doctors performed a series of tests and treatments, including 50 blood transfusions, steroids, and a splenectomy, a procedure that removes the spleen."
- By Jaleesa Baulkman for Dailymail.com.uk. Photo courtesy of Emily Dumler.
"Allegheny County has paid a $35,000 settlement to parents who say a UPMC doctor misdiagnosed their infant son's bruises as abuse and not a medical condition.
According to the complaint, filed in March in federal court against the county and the doctor, the 6-month-old baby's pediatrician noticed four bruises on him during a July 2015 wellness check."
- by Keith Hodan, Trib Total Media. Photo courtesy - By Dllu - Own work, CC BY-SA 4.0, https://commons.wikimedia.org/w/index.php?curid=47851338
"Long-term eltrombopag demonstrated efficacy in restoring platelet counts and decreasing risk of bleeding in patients with chronic/persistent immune thrombocytopenia (ITP) lasting more than 6 months, according to a study published in Blood.
Previous studies demonstrated that eltrombopag treatment for 6 months or less effectively increased platelet counts and reduced bleeding in patients with chronic ITP. The purpose of this analysis was to evaluate the safety and efficacy of long term therapy with eltrombopag."
- By James Nam, PharmD. Photo courtesy OncologyNurseAdvisor.com.
"Remigio Pereira reveals he’s on the mend after spending two weeks in hospital receiving treatment “for my ITP episode.”
In a Facebook post he wrote on Monday, the former singer for “The Tenors” opens up about what he’s been going through due to his ITP (Idiopathic thrombocytopenic purpura), a bleeding disorder in which the immune system destroys platelets and prevents blood from clotting.
“I’m so sorry I haven’t had the energy [to] write lately… I have been wanting to thank you all for your well wishes, but my health hasn’t been so co-operative,” writes Pereira."
- By Brent Furdyk, for etcanada.com. Photo courtesy Remigio Pereira/Twitter.
"On May 27th, 2016 Parker was a happy and healthy seven-year-old that was awarded an overall achievement award and scholarship at her dance recital.
“Exactly one week later at 11 p.m. she was being rushed from the Lacombe Hospital to the Stollery Children’s Hospital in Edmonton in an ambulance,” said Richer.
Some of her symptoms at the time included fever, a bleeding nose and bruising.
“It happened so fast and you don’t even realize it at the time but if we wouldn’t have had blood at the hospital when she arrived there she wouldn’t have survived a couple of more hours,” said mom, Carrie Ann."
- By Carlie Connolly for Red Deer Express
"argenx, a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, today announced that it has recruited 50% of the immune thrombocytopenia (ITP) patients in the Phase 2 proof-of-concept study of ARGX-113.
“We are pleased to have reached the 50% recruitment milestone in this study, which is enrolling on schedule,” commented Nicolas Leupin, Chief Medical Officer of argenx. “We remain on track, and look forward to presenting top-line data from the ARGX-113 clinical trial in ITP in the second half of 2018, which will be our second indication with Phase 2 top-line data from ARGX-113.”"
- From argenx press release
"Pregame festivities will also honor Cayden Krueger from Beloit, Wis., as the “Shopko Kick-Off Kid.” The 7-year-old has ITP disease, a platelet disorder, and founded a charity 5K walk in his hometown to raise money for the Platelet Disorder Support Association."
- From Packers.com. Photo courtesy of Wikipedia.com
"The aim of the present study was to compare response rate between patients, underwent splenectomy vs. rituximab as second-line therapy. Adult patients diagnosed with ITP who did not respond to corticosteroids or relapsed during the period 1990-2014 were included in a quasi-experimental study. Categorical variables were compared using Fisher exact test. Response to treatment was compared using logistic regression. Data were analyzed using SAS V9.2. One-hundred and forty-three patients with ITP were identified through medical records. Of 62 patients treated..."
- From the NIH - PubMed
"Novartis Korea said Tuesday additional data analysis from its EXTEND clinical trial for Revolade (ingredient: eltrombopag) proved it safe and effective for those 65 years and older with idiopathic thrombocytopenic purpura (ITP).
The sub-set study findings were presented at the first International Congress of BMT symposium held in Seoul on Aug. 26.
Revolade (known as Promacta in the U.S.) was developed by GlaxoSmithKline (GSK) and transferred to Novartis노바티스 when it acquired the former’s oncology portfolio."
- By Marian Chu, Korea Biomedical Review
"Protalex, Inc., a clinical-stage biopharmaceutical company, today announced that following a planned interim analysis of data from the second dose cohort of its European Phase 1b study of PRTX-100 in adults with persistent/chronic Immune Thrombocytopenia (ITP) (PRTX-100-203 Study), the Company has initiated enrollment in the third cohort of this dose-escalating study. The first patient in the third cohort was recently dosed at 12.0 µg/kg, double that of the second dose cohort of 6.0 µg/kg. PRTX-100 has been granted Orphan Drug Designation in the U.S. and in Europe for the treatment of ITP."
- From Protalex press release
"The exact pathogenesis of ITP is unknown; potential causes range from inflammatory responses and reactions to single nucleotide polymorphisms that cause defective genomic changes.
Some disease states may cause substitution of nucleotides in the human genome, leading to an imbalance of T-helper-1 (Th1) and Th2 cells. Th1/Th2 imbalance may lead to an overproduction of inflammatory cytokines that can cause conditions such as ITP."
- By James Nam, PharmD for Oncology Nurse Advisor.
"The mother of a 4-year-old boy called the nurse line because her son had what she described as an itchy, red rash on his arms and back since the night before. After a full triage, she was given instructions for symptomatic care and follow-up with the pediatrician the next day. The mother called back later that day because the patient had developed the same rash everywhere and some new bruising on his arms and legs after playing on the trampoline in the yard. She was directed to have her child evaluated in the emergency department."
- By Barrett, Whitney MD, Emergency Medicine News. Photo courtesy of http://journals.lww.com.
"An energetic little girl has been told she can no longer play with her friends – because one small cut or bruise could kill her.
Skyla Budding, from Merthyr Tydfil, suffers with a rare disorder called idiopathic thrombocytopenic purpura (ITP) which means her blood doesn’t clot and leads to excessive bleeding.
Her mum Sarah Markham says she’s had no choice but to stop the active five-year-old from enjoying some of her favourite hobbies.
“It was really important that Skyla didn’t do anything that would cause her to bruise or to cut herself,” she explained.
“So even when we weren’t in hospital having treatment, she still couldn’t do any of the things that she loves to do like riding on her bike or jumping on the trampoline."
- By Hayley Richardson, thesun.co.uk. Photo courtesy Sarah Markham.
"Protalex, Inc., a clinical-stage biopharmaceutical company, today announced that following completion of a planned interim analysis of safety and efficacy data from the second dose cohort, the Company is escalating the dose of PRTX-100 in its U.S. Phase 1/2 study of PRTX-100 in adults with persistent/chronic Immune Thrombocytopenia (ITP) (PRTX-100-202 Study). One of the three patients treated in the second dose cohort achieved a protocol defined platelet response. Treatment of the first patient in the third cohort at a dose of 6.0 micrograms/kg, double that of the second dose cohort of 3.0 micrograms/kg, is expected soon."
- From Protalex Press Release
"Scientists have long believed that in ITP, the body’s attacks on platelets originated mainly in the spleen mediated by autoantibodies, and in about 70 percent of ITP cases, immune suppression and removal of the spleen controls the condition. But in the remaining 30 percent of patients, symptoms persist.
In this new study, OMRF scientist Lijun Xia, M.D., Ph.D., who holds the Merrick Foundation Chair in Biomedical Research at OMRF and heads the foundation’s Cardiovascular Biology Research Program, and his team used an experimental model with platelets that carried an aberrant version of O-glycans, sugars that the body produces. Specialized cells in the liver quickly cleaned up these platelets as abnormal cells—resulting in a drop in platelet counts.
“Until now, the spleen has been considered the major site for platelet clearance,” said Xia. “But these glycans-deficient platelets exhibited a shorter lifespan and were being cleared from the liver instead.” "
- From omrf.org
"The U.S. Food and Drug Administration (FDA) filed Rigel Pharmaceuticals, Inc.’s New Drug Application for the use of Tavalisse (fostamatinib disodium) for the treatment of chronic immune thrombocytopenia (ITP).
Tavalisse, an oral investigational drug candidate designed to inhibit SYK kinase, a key enzyme in the immune process that leads to platelet destruction in ITP. It was previously granted Orphan Drug designation for treatment of the same condition in April.
ITP is a bleeding disorder in which blood doesn't clot as it does typically due to an unusually low number of platelets or thrombocytes. It’s almost always chronic in adults, but is often acute and self-limited in children. Symptoms include excessive bruising and bleeding.
'The FDA acceptance for filing of our NDA is an exciting milestone for Rigel," said Raul Rodriguez, Rigel's president and chief executive officer. "If approved, we believe Tavalisse will provide a new treatment option for patients with chronic or persistent ITP. We look forward to working closely with the FDA as they review our submission.'"
- By Mathew Shanley, Rare Disease Report
"Researchers at Weill Cornell Medicine have discovered an innovative method to make an unlimited supply of healthy blood cells from the readily available cells that line blood vessels. This achievement marks the first time any research group has generated such blood-forming stem cells.
“This is a game-changing breakthrough that brings us closer not only to treat blood disorders, but also deciphering the complex biology of stem-cell self-renewal machinery,” said senior author Dr. Shahin Rafii, director of the Ansary Stem Cell Institute, chief of the Division of Regenerative Medicine and the Arthur B. Belfer Professor at Weill Cornell Medicine."
- By Geri Clark. Photo courtesy of Dr. Raphael Lis.
"Whilst studying for a degree in Stage Management at the Bristol Old Vic Theatre School Megan is aiming to raise £100,000 to support her consultant Dr Charlotte Bradbury and her research team at Bristol Haematology and Oncology Centre in their study of ITP. The team are developing a tracer and blood scanning techniques that will enable them to identify suitable and effective treatments for the individual patients."
- By Selina Cuff, for http://www.chewvalleygazette.co.uk
"In a major six-year trial involving over 20,000 women in 21 countries, researchers showed that tranexamic acid, a little-known blood-clotter invented in the 1950s, reduced maternal bleeding deaths by a third if it was given within three hours. It costs less than $2 a dose and does not require refrigeration."
- By Donald G. McNeil Jr. for the NY Times.
"The name Tavalisse (pronounced - ta vah lees') was developed based on the FDA's Guidance for Industry, Contents of a Complete Submission for the Evaluation of Proprietary Names. Created with the help of experts in this field, the proprietary name development included input from prescribers, pharmacists, linguists and employees to create a unique and approachable name.
Earlier this month, the company announced that it submitted a New Drug Application (NDA) to the FDA for Tavalisse in patients with chronic and persistent immune thrombocytopenia (ITP). The FDA previously granted Orphan Drug designation to Tavalisse for the treatment of patients with ITP."
- From Rigel Pharmaceuticals, Inc. press release.
"Heparin is a natural anticoagulant that derives its name from the tissue it was first isolated from: hepar in Greek means liver. Listed in the WHO Model List of Essential Medicines, heparin has stood the test of time as one of the oldest drugs still employed in clinical treatment to reduce clotting. However, heparin use is not without consequence as its usage can result in an auto-immune clotting disorder known as heparin-induced thrombocytopenia (HIT). Interestingly, HIT can persist even after heparin has cleared from the body – a mystery that the CBR researchers aimed to solve."
- By Bryan Lin, PhD Candidate at Pryzdial Lab for the University of British Columbia
"FWGBD is very pleased to report that our founding President, Dr. Andra H. James (Andi), has been awarded the HTRS (Hemostasis and Thrombosis Research Society) Lifetime Achievement Award, at their 2017 Scientific Symposium on April 8, 2017.
Andi is an Ob/Gyn with a singular grasp of benign hematology. Her understanding of, and concern about bleeding and clotting disorders in women was informed and enhanced by her Fellowship in Maternal-Fetal Medicine at Duke, where she spent three years in the laboratory of Dr. Russell Ware; her Ob/Gyn residency at the University of North Carolina-Chapel Hill, incubator of advances in knowledge about hemophilia and hemostasis; by her graduate training in Public Health at Johns Hopkins; and by having been a nurse-midwife at the start of her career."
- From the Foundation for Women & Girls with Blood Disorders
"Scientists from George Mason University recently isolated a substance in the blood of a Komodo dragon that appeared to have powerful germ-killing abilities.
Inspired by the discovery, they created a similar chemical in the lab and dubbed it DRGN-1.
Tests on mice that were given skin wounds infected with two types of bacteria showed that DRGN-1 had three valuable properties: It punched holes in the outer membranes of both gram-negative and gram-positive bacteria, it dissolved the biofilms that glue bacteria together, and it sped skin healing."
- By Donald G. McNeil Jr. for the NY Times.
"Donald Arnold, associate professor of medicine, is helping jump start the Platelet Disorder Support Association (PDSA) in Canada, a not-for-profit patient advocacy group that has been very active in the U.S. It aims to improve the lives of people with ITP and other platelet disorders through education, advocacy, research and support.
Arnold, a hematologist, is a medical advisor for the PDSA, which is holding its first meeting April 29 in Montreal. This meeting is geared for ITP patients, caregivers and the medical community."
- By McMaster University