Thrombocytopenia means having too few platelets, the blood cells that stop bleeding. It can be mild and discovered by accident, or severe enough to cause spontaneous bruising and dangerous bleeding. Treatment depends on the cause — autoimmune attack, bone marrow disease, medication side effect, or inherited disorder.
What's actually going on in research
Trials are testing thrombopoietin receptor agonists that stimulate platelet production, antibody therapies for immune thrombocytopenia (ITP), treatments for chemotherapy-induced low platelets, and gene therapies for inherited platelet disorders. Researchers are also studying how to predict bleeding risk and avoid unnecessary transfusions.
TPO receptor agonists
Drugs like romiplostim and eltrombopag boost platelet production and are now standard for ITP. Trials are testing newer versions with fewer side effects and studying their use in other causes of low platelets.
Fostamatinib for ITP
This pill blocks an enzyme involved in platelet destruction and was FDA-approved in 2018 for ITP. Ongoing studies are exploring its use in other autoimmune causes of thrombocytopenia.
Gene therapy
Early trials are testing gene therapy for inherited platelet disorders like congenital amegakaryocytic thrombocytopenia. The goal is to correct the genetic defect and restore normal platelet production.
What to know before you search
Eligibility typically depends on platelet count, cause of thrombocytopenia (autoimmune, drug-induced, bone marrow disease, inherited), prior treatments, and bleeding history.
What types of trials are currently open
- Treatment trials — Testing new drugs that either boost platelet production or reduce immune destruction of platelets.
- ITP trials — Studies focused on immune thrombocytopenia, testing antibodies, immune modulators, or new versions of TPO agonists.
- Chemotherapy support trials — Testing treatments to prevent or treat low platelets caused by cancer chemotherapy.
- Gene therapy trials — Early studies of gene correction for inherited platelet disorders, typically in children.
- Bleeding prediction studies — Research to better predict who will bleed and who can safely avoid treatment despite low platelet counts.
Recently added Thrombocytopenia trials
Take a steroid and platelet-boosting medication for 12 weeks
This prospective, single-arm, multicenter, exploratory study will enroll 38 adults patients with newly diagnosed primary immune thrombocytopenia (ITP). Patients will receive standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA) as initial therapy During the core treatment phase (Weeks 1-12). Corticosteroids will be tapered and discontinued within 8 weeks, whereas TPO-RA treatment will continue for 12 weeks. For patients with treatment failure( defined as platelet count \< 30 × 10\^9/L or less than 2-fold increase of baseline platelet count or bleeding) ,a sequential multitarget combination strategy will be explored in subsequent treatment phases. Specifically, patients with treatment failure after 2 weeks of initial therapy (Weeks 3-12), will receive the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody（mAb） as sequential combination therapy. During the core treatment follow-up phase (24 weeks), patients with treatment failure will enter the exploratory treatment phase (Weeks 13-36) . Patients who received multi-target drug therapy during the core treatment period will switch to an alternative TPO-RA with cross-administered rituximab and anti-CD38 mAb, while those who did not will receive sequential rituximab or anti-CD38 mAb. Finally, patients will enter the safety follow up period (4 weeks, weeks 37-40).
Share your treatment preferences in a survey
This study aims to evaluate treatment preferences of patients with Primary Immune Thrombocytopenia (ITP) in the United States (US) by conducting a preference elicitation survey.
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