This e-newsletter is a monthly publication of the Platelet Disorder Support Association. The information in this newsletter is for educational purposes only. For advice on your unique medical condition, please consult a health care professional.
- Eltrombopag (Promacta): After 5 Years, Positive Results
- Romiplostim (Nplate) Safety: Studies Summarized
- Romiplostim (Nplate) in Children: Long-Term Results
- Eltrombopag (Promacta): Children Improve Quality-of-Life
The ASH abstract numbers and titles are listed below the summaries. You can search on the number and read the abstracts at https://ash.confex.com/ash/2012/webprogram/start.html (after you agree with the terms). See http://www.hematology.org/Meetings/Annual-Meeting/ for additional meeting information.
More ASH information will be summarized in the next two issues of The Platelet News. For information on receiving that 28-page newsletter see http://www.pdsa.org/index.php/products-a-publications/newsletters
Participants in prior studies of eltrombopag were given the opportunity to continue taking the pills to help determine the long-term efficacy and side effects of the treatment. Of the people in the extended study, some treated for more than 5 years, 85% achieved a platelet count of 50,000/uL or greater at least once, and 62% had a platelet count of 50,000/uL or greater for at least half the time. The most common side effect was a headache.
2198 "Long-Term Safety and Efficacy of Eltrombopag in Adults with Chronic Immune Thrombocytopenia (ITP): Report of up to 5.5 Years of Treatment in EXTEND"
Researchers summarized the detail data from 13 romiplostim studies published between 2002 and 2011 and compared the adverse events of those taking romiplostim with a control group receiving a placebo or usual treatments for ITP. Of the 1059 patients evaluated, more than 90% in both groups reported adverse events. The most common adverse event was headache. The romiplostim group had more bone pain and nausea. The placebo/usual care group reported more bleeding and treatment-related adverse events.
2185 "Integrated Analysis of Long Term Safety in Patients (pts) with Chronic Immune Thrombocytopenia (ITP) Treated with Romiplostim"
ROMIPLOSTIM (NPLATE) IN CHILDREN: LONG-TERM RESULTS
Researchers reported results of an extension to the romiplostim (Nplate) clinical trial in children with severe, chronic ITP. The participants, treated for more than 2 years, responded well, with usual platelet counts equal to or above 50,000/uL. The treatment was generally well-tolerated, although some of the participants needed rescue therapy for bleeding events. The study is ongoing.
621 "Long-Term Use of Open-Label Romiplostim in Children with Chronic/Refractory Immune Thrombocytopenia (ITP)"
Children with chronic ITP often have a reduced quality-of-life due to activity restrictions, fears, and the consequences of treatment. In addition to measuring the platelet count for children enrolled in a clinical trial of eltrombopag, researchers asked the older children and parents of the younger children to complete a quality-of-life questionnaire before treatment and as treatment progressed. The preliminary results show that treatment with eltrombopag improved the quality-of-life for almost all the clinical trial participants.
2197 "Health-Related Quality of Life in Children with Chronic Immune Thrombocytopenia Treated with Eltrombopag in the PETIT Study"
Note: For more information on eltrombopag and romiplostim see:
Results for an initial safety and dosing study for a new treatment for ITP, SM101, had very positive results. About 75% of the participants receiving the highest dose responded to a single course of treatment at 20 days and the response lasted at least 3 months after stopping the treatment. SM101 works differently from other treatments for ITP. It blocks the action of immune complexes, thereby reducing the autoimmune response and associated inflammation. SuppreMol, the German manufacturer of SM101, plans additional clinical studies in the US and Europe in 2013.
3388 "Interim Results From a Phase Ib/IIa Clinical Trial with the Soluble Fc-Gamma IIb Receptor SM101 for the Treatment of Primary Immune Thrombocytopenia" Poster:
SuppreMol press release. "SuppreMol's SM101 shows a sustained clinical activity and a favorable safety profile in Primary Immune Thrombocytopenia (ITP) patients.":
Veltuzumab (anti-CD20) is designed to deplete B-cells, a type of white blood cell. It works in the same manner as rituximab but differs in some structures and functions. It is given as an injection under the skin, not as an infusion. Of the 40 participants in the veltuzumab clinical trial, 68% had some response. The response was independent of the dose, the dosing schedule, or the length of time someone had ITP. There were no serious side effects or safety concerns.
622 "Phase I/II Study of Monotherapy with Subcutaneous Injections of Low-Dose Anti-CD20 Veltuzumab in Relapsed Immune Thrombocytopenia"
Lifestyle, genetics, and/or environmental exposure may influence the cause and course of ITP, but little is known about the specifics. PDSA is funding a research study to help define which factors are most important. A preliminary questionnaire was given to 109 children and adults. Some initial results: the most frequent hazardous substances contacted were cleaning supplies and chlorinated water; more than half of respondents reported more stress than usual; nearly one-half reported an infection prior to diagnosis. The researchers are comparing the survey results with treatment response for the initial group and refining the questionnaire in anticipation of giving it to a much larger population of ITP patients.
2239 "A Survey of the Etiology of Immune Thrombocytopenia (ITP)"
Oxidative stress (more reactive oxygen than the body can dismantle) is associated with many diseases. Researchers analyzed the markers of oxidative stress in children with ITP and matched healthy controls. They found that all children with ITP had more oxidative stress than their healthy counterparts. Those children whose ITP continued for a longer time had more markers of oxidative stress than the children who recovered. These results confirm prior findings that oxidative stress is associated with ITP and anti-oxidant therapies may be a helpful treatment option.
3319 "The Role of Oxidative Stress in Pediatric Immune Thrombocytopenia"
Note: Many lifestyle changes, such as mind/body interventions and diet, can reduce the amount of oxidative stress in children and adults. See the complementary treatments section of the PDSA web site for information:
This e-newsletter is published by the Platelet Disorder Support Association, 133 Rollins Avenue, Suite 5, Rockville, MD 20852, phone 1-87-Platelet, fax: 301-770-6638, web: http://www.pdsa.org, e-mail: email@example.com