Innovative ITP Research from the 2019 American Society of Hematology (ASH) Annual Meeting

This year’s 2019 ASH meeting, held December 7-10 in Orlando (Florida), attracted over 30,000 clinicians, scientists, pharmaceutical company personnel and even investment analysts worldwide to share ground-breaking research in the field of hematology. The conference included the annual ITP breakfast meeting, coordinated by PDSA and four of PDSA’s Medical Advisors, Dr. James Bussel, Dr. Nichola Cooper, Dr. Michele Lambert and Dr. John Semple. Thirteen hematology experts were invited to present their leading-edge ITP research. In this issue of the e-news, we report on experimental therapies for ITP for which updates on or new clinical trials were presented. Watch for additional ASH reports in the 2019 winter issue of The Platelet News, the PDSA quarterly newsletter.

The ASH abstract numbers are shown in parentheses. You can search on the number and read the complete abstract at: https://ashpublications.org/blood/issue/134/Supplement_1.

Abstracts 1 and 2 describe a novel mechanism to increase the platelet count in ITP that also might have application to other diseases. These two studies evaluate a novel method to treat ITP using molecules that target and bind to the so-called neonatal Fc receptor (FcRn). The drugs are designed to increase IgG clearance by blocking normal IgG recycling in the body. IgG antibodies normally may remain in the blood for thirty days but, when recycling is blocked, they may only last about five days. This is important because when you lower all IgG molecules, you also lower IgG antibodies to platelets. These IgG anti-platelet antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Therefore, it would be expected that lowering IgG levels will equally lower IgG anti-platelet antibody levels which will increase platelet count in patients with ITP.

CONTENTS:

• Phase 2 Study of Efgartigimod, a Novel FcRn Antagonist, in Adult Patients with Primary Immune Thrombocytopenia (ITP) (128022)
• Rozanolixizumab, an Anti-FcRn Antibody: Final Results from a Phase 2, Multiple-Dose Study in Patients with Primary Immune Thrombocytopenia (129839)
• Inhibition of the Classical Pathway of Complement with Sutimlimab in Chronic Immune Thrombocytopenic (ITP) Patients without Adequate Response to Two or More Prior Therapies (122210)

 
CONTENTS:

• Immune Thrombocytopenia: Are we stuck in the mud or is there light at the end of the tunnel?
• Clinical trial for early use of eltrombopag in pediatric patients with ITP

 
CONTENTS:

• Splenectomy in the context of new medical therapies for ITP
• Nplate® (romiplostim) now approved for earlier use in adults with immune thrombocytopenia

 
CONTENTS:

• Prognostic factors for Immune Thrombocytopenia remission after laparoscopic splenectomy
• Comparison of first-line treatments for newly diagnosed Immune Thrombocytopenia

 
CONTENTS:

• Physicians’ Perceptions on Causes of Secondary ITP And the Leading Causes of Misdiagnosis in Both Primary and Secondary ITP
• Retrospective Study Examines The Treatment Results of Sequentially Switching Thrombopoietic Receptor-Agonists In Immune Thrombocytopenia Patients

 
CONTENTS:

• Long-term overuse of corticosteroids in the real-world treatment of patients with immune thrombocytopenia
• ITP patient perceptions on splenectomy outcomes

 
CONTENTS:

• Patient and Physician Perceptions on Treatment Approaches
• FDA approves new drug “Doptelet” for treatment of chronic ITP

 
CONTENTS:

• Characteristics, Risk Factors, and Management of Venous Thromboembolism in ITP
• Prospective Study Examines the Variability of Second-Line Treatment Outcomes In Children With ITP

 
CONTENTS:

• High-Dose Dexamethasone Displays The Ability To Reduce Elevated Interleukin-16 Levels In Adult Immune Thrombocytopenia Patients
• An Open-Label Phase 3 Extension Study Examines The Effects Of Long-term Fostamatinib (TavalisseTM) Treatment On Adult ITP Patients

 
CONTENTS:

• Increased Monitoring Should Be Utilized When Receiving Immune Checkpoint Inhibitors
• Posttransfusion Purpura with Antibodies Against Human Platelet Antigen-4a following Checkpoint Inhibitor Therapy: a Case Report and Review of the Literature

 
CONTENTS:

• Retrospective Study Finds That The Combination Therapy of Rituximab and Cyclophosphamide Displays Increased Therapeutic Effects Compared To Each Drugs Individual Therapeutic Effects on Patients Suffering From Refractory ITP
• Chidamide Study Displays Potential For Novel Therapies and Multiple Pathways For ITP Intervention

 
CONTENTS:

• FDA Approves Romiplostim (N-Plate) as a Treatment Option for Pediatric Patients with Immune Thrombocytopenia
• Berberine May Offer ITP Patients Suffering from Corticosteroid Resistance Induced by Gut Microbiota Imbalance a New Option for Therapy