PDSA e-News: November 26, 2019

Dr. James BusselImmune Thrombocytopenia: Are we stuck in the mud or is there light at the end of the tunnel?

PDSA Medical Advisor Dr. James Bussel was interviewed by Medpage Today about an article he co-authored with fellow pediatric hematologist Dr. Oriana Miltiadous. The article is called “Immune Thrombocytopenia (ITP): Are we stuck in the mud or is there light at the end of the tunnel?” and was accepted for publication in the summer through Clinical Hematology International. While currently in press, the article can be found online using the link below. The premise of the article is to acknowledge how research in the area of ITP has not grown as fast compared to other diseases, such as leukemia, over the last thirty years. Within the article, Dr. Bussel and Dr. Miltiadous offer possible explanations for this, and discuss three main characteristics specific to ITP that make it difficult for quicker advancements to happen. One characteristic discussed about ITP is that it is a heterogeneous disease. Not everyone with ITP developed the disease due to the same underlying etiology, not everyone with ITP will be affected in the same way, and not everyone with ITP will respond to the same treatments. Some will go into remission sporadically, others will not. The second characteristic reviewed is that due to a low risk for death and serious bleeding complications with ITP, studying this disease is lower on the priority list for hematologists and oncologists compared to their more high-risk demanding oncology cases. A third characteristic highlighted included a lack of advancements to diagnose ITP. It is not clear that everyone with ITP actually has the same disease since there is no single test routinely administered to rule out secondary causes of a low platelet count and there is no single genetic anomaly that can be tested for to establish someone has ITP definitively. ITP is a diagnosis of exclusion, unlike specific types of leukemia and other diseases where it is clear everyone with that diagnosis has complications arising from the same underlying reason, even if the disease shows variable expressivity.

Dr. Bussel’s interview: https://www.medpagetoday.com/resource-centers/chronic-immune-thrombocytopenia-meeting-challenge/slow-progress-itp-interview-james-bussel-md/2662

Online Article: https://www.atlantis-press.com/journals/chi/125914914


Doctor examining childClinical trial for early use of eltrombopag in pediatric patients with ITP

Thrombopoietin receptor agonists (such as romiplostim, eltrombopag, and avatrombopag) are primarily used as second-line therapies in the treatment of ITP when first line therapies have failed. At Baylor College of Medicine, pediatric hematologist Dr. Jenny Despotovic is leading a multi-center clinical trial where pediatric participants with ITP are being given eltrombopag (Promacta/Revolade) as first-line therapy within the first three months following their diagnosis. With platelet count monitoring and a record of bleeding events, it is hoped this study will lead to better way to treat ITP within the pediatric community and fewer children will go on to develop chronic ITP. Eltrombopag stimulates the bone marrow to produce more platelets and has been shown to have a positive effect on the immune system. In other studies, a significant number of adults using eltrombopag have gone into remission after dealing with low platelet counts for over twenty years.


Comment from PDSA medical advisors:

Essentially, there is consensus among all guidelines that deal with the management of ITP in children, and that is to reserve treatment to situations in which there is severe bleeding or prior to procedures. The reasons for this decision are several-fold. ITP remits spontaneously in most children, life-threatening bleeding is rare, therapies such as corticosteroids and IVIg may have significant acute side-effects and immunosuppression, and splenectomy might increase the risk of serious infection in the absence of a fully mature immune system. However, we may be on the verge of seeing a change in the “watch-and-wait” approach and the reasons for this are also based on several considerations. ITP does not remit in all children, others require medical interventions in the interim, and concerns about the risk of bleeding can inhibit participation in athletic and social activities and contribute to patient and family anxiety. It has also become clear that thrombopoietin receptor agonists (TPO-RA) are effective in over 80% of adults with ITP and long-term safety data has largely mitigated concerns about potentially increased risk of thromboembolism and accentuation of bone marrow reticulin formation. This article by Dr. Despotovic describes the opening of a clinical trial of one such agent, eltrombopag, in children with ITP of less than 3 months duration. The results of published trials in children with chronic ITP make it highly likely this approach will raise platelet counts and, hopefully, reduce bleeding and the need for acute interventions and thereby improve quality of life. Long-term follow-up of treated children will be critical to ensure that the safety profile is the same as in adults with ITP.


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