PDSA E-News: October 23, 2015

ITP & PLATELET DISORDERS RESEARCH & TREATMENTS

Higher Platelet Counts and Fewer IVIg Infusions for ITP Patients Receiving Romiplostim (Nplate®)

Thrombopoietin receptor agonists (TPOs) are the newest treatments approved for chronic ITP patients.  How romiplostim (Nplate®), one of the TPOs, has impacted patient treatment, especially in use of intravenous immunoglobulin (IVIg) has not been evaluated outside of clinical trials.  This Canadian retrospective cohort study of adult ITP patients treated with romiplostim compared IVIg use, clinical outcomes, and cost before and after treatment with romiplostim.  Twenty-nine patients (median age of 54) with ITP received romiplostim.  Patients on average tried two prior ITP treatments, including splenectomy, before receiving romiplostim.  Median platelet counts were 23,000 before romiplostim and 124,000 after romiplostim.

Patients on average had received two IVIg infusions per year prior to romiplostim treatment and then 0.7 per year after treatment.  Nineteen patients discontinued the romiplostim during follow up because of a lack of response.  There were no significant differences in healthcare costs before and after romiplostim when additional treatments, nursing resources, and hospitalizations were taken into consideration.  The researchers concluded romiplostim led to improved platelet counts and fewer IVIg infusions for most ITP patients.  Also, in practice, romiplostim was not continued long term.

Zeller MP, Heddle NM, Kelton JG, et. al.  “Effect of a thrombopoietin receptor agonist on use of intravenous immune globulin in patients with immune thrombocytopenia.”  Abstract, Transfusion. 2015 Sept 24, doi 10.1111/trf.13336 (Epub ahead of print).
http://www.ncbi.nlm.nih.gov/pubmed/26400824

Approval Granted to Protalex for Phase Ib Trial of ITP Therapy PRTX-100

Biopharmaceutical company, Protalex, Inc. has gained approval from the French National Agency for Medicines and Health Products (ANSM) for the company’s clinical trial application to start a Phase Ib study of their drug PRTX-100 in adults with persistent/chronic ITP.  The ANSM approval authorizes Protalex to begin the study (PRTX-100-203) at several locations in France.  Protalex also announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending PRTX-100 for designation as an orphan medicine for treating ITP. 

PRTX-100 is a new generation immunomodulatory therapy being investigated for treating autoimmune diseases like rheumatoid arthritis and ITP.  It is a highly purified form of Staphylococcal protein A (bacterial protein), shown to affect immune function in prior studies.  PRTX-100, at very low concentrations, has the ability to bind to human B-lymphocytes and macrophages, which modulates immune processes.  The 203 Study and positive COMP opinion follow the company’s earlier announced initiation of a US Phase I/II clinical trial in ITP adults and US Food and Drug Administration (FDA) Orphan Drug Designation for PRTX-100 to treat ITP.  The French study will evaluate safety and efficacy of PRTX-100 in a range of doses.  Along with the US-based 202 Study this research will provide important data to support further development plans in ITP treatment.  The Vice President-ITP Programs for Protalex said, “We believe PRTX-100 represents a promising new approach for the treatment of ITP.”

“Protalex Announces Regulatory Approval to Initiate Phase 1b Trial of PRTX-100 to Treat Immune Thrombocytopenia in France.”  Protalex Press Release, Oct. 5, 2015, reported online in Business Wire.
http://www.businesswire.com/news/home/20151005005303/en/Protalex-Announces-Regulatory-Approval-Initiate-Phase-Ib 

HOSPITALS, INSURANCE & MEDICAL CARE

Improving Diagnosis in Health Care

The National Academies of Sciences, Engineering, and Medicine have released a new report on advancing the way clinicians diagnose and treat patients.  Diagnostics can help a patient in providing more information about the disease or disorder to the doctor and forming a plan of action for treatment; diagnostic errors, however, can seriously hinder good health care.  This report analyzes the current state of Diagnostics and proposes several suggestions for improving this vital aspect of patient care. 

Diagnostic error is defined as “the failure to (a) establish an accurate and timely explanation of the patient’s health problem(s) or (b) communicate that explanation to the patient.”  The most important part of this definition is its focus and emphasis on the patient’s point of view.  Diagnostic errors can result from a variety of issues, such as insufficient communication between patient and doctors and a work culture that discourages transparency. 

Ultimately, the report suggests the following recommendations be made to diagnostic procedures:

1. Encourage teamwork in diagnostics between clinicians and patients.
2. Improve education and training in diagnostic procedures.
3. Facilitate a work culture that enhances support for diagnostics.
4. Improve health technology in order to further educate both patients and clinicians on diagnostics.
5. Develop a medical liability system that promotes improved diagnosis through learning from diagnostic errors.
6. Design payment plans and funding for research which support and further enhance diagnostics.

National Academies of Sciences, Engineering, and Medicine.  2015.  Improving diagnosis in health care. Washington, DC: The National Academies Press.
http://iom.nationalacademies.org/~/media/Files/Report%20Files/2015/Improving-Diagnosis/DiagnosticError_ReportBrief.pdf

GENERAL HEALTH & MEDICINE

Controlling Inflammation Reduces Chronic Disease Risk

Researchers have discovered the relationship between nutrition and inflammation in chronic diseases.  Controlling the body’s inflammatory response, which plays an important role in disease development, has continued to develop as an important preventative and therapeutic target.

Experts have discovered that the presence of inflammation is significantly influenced by nutrition, which can also assist in diminishing the risk of chronic disease.  It is important to note that sporadic inflammation is a normal and functioning factor in immune defense, however, common and chronic inflammation is a serious issue in chronic disease.  Therefore, the preventative effects of specific aspects of nutrition have been demonstrated to be an effective method in controlling inflammation, and thus reducing risk of chronic disease.

As the study demonstrates, an inadequate or extreme inflammatory response may be a result of a deficiency or excess of specific nutrients such as “folate, vitamin B12, vitamin B6, vitamin 1, vitamin E, or zinc.”  A diet rich in fats and sugars often leads to inflammation following a meal (post-prandial inflammation).  Unfortunately, the combination of nutrient deficiency and overconsumption of unhealthy foods is exemplary of the Western-style diet.  This has been correlated to the escalation of chronic immune disorders and diseases such as allergies, food allergies, and obesity.  Ultimately, proper maintenance and observance of nutrition can lead to a reduction in these inflammation causing health issues, resulting in a lowered risk for chronic illness.

Anne M. Minihane, et al.  “Low-grade inflammation, diet composition and health: current research evidence and its translation.”  British Journal of Nutrition 2015; published online.
http://journals.cambridge.org/action/displayAbstract?fromPage=online&aid=9888262&fileId=S0007114515002093

CLINICAL TRIALS

Are you living with ITP?

If you or someone you know is living with ITP and are 18 years of age or older, this clinical trial may be for you. There is a clinical trial being conducted by Bristol-Myers Squibb with an investigational drug for the treatment of adult ITP (either persistent or chronic). Have you previously received one or more prior therapies? Have you initially responded to at least one therapy if so – you may be eligible? This study has sites in the US, Canada and Europe.

For a complete site list, visit MyITPStudy.com

Is your current ITP treatment working for you?

Rigel Pharmaceuticals Inc. is conducting two Phase 3 clinical studies with fostamatinib, an investigational drug for the treatment of patients with persistent or chronic ITP.  If you are at least 18 years of age, have had a diagnosis of ITP for at least 3 months, and have previously received at least 1 typical regimen for the treatment of ITP — you may be eligible. There are clinical trial sites already open in the U.S., Canada, Europe and Australia.

For a complete site list, please visit -  http://tinyurl.com/RigelPhase3ITP.

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