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
rhTPO for Thrombocytopenia in Chronic Liver Disease Patients Undergoing Elective Invasive Procedures.
This is a global, multicenter, randomized, double-blind, placebo-controlled study using recombinant human thrombopoietin stimulating factor ( rhTPO) to treat adults with Chronic liver disease-related thrombocytopenia (CLDT). The study will evaluate rhTPO efficacy in elevating platelet level and its safety in the treatment of thrombocytopenia associated with liver disease prior to an elective procedure to reduce pre-procedural platelet transfusion needs and the requirements for rescue treatment for bleeding.
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol. Since constitutional bone marrow failure syndromes (CMF) are rare genetic disorders characterized by a variable and incompletely defined clinical phenotype, a partially unknown genetic etiology, and pathogenetic mechanisms that are only partly understood, we hypothesize that a number of patients currently diagnosed with idiopathic acquired aplastic anemia (AAA) or idiopathic single-lineage/multilineage bone marrow failure (BMF) may have been misdiagnosed and may actually have an underlying CMF. Therefore, our primary objective is to identify CMF underlying BMF and vice versa, by defining well-characterized patient cohorts based on clinical and family history, clinical manifestations, and immunological findings; identifying disease-specific parameters and biomarkers; and providing disease-specific guidelines to facilitate early diagnosis and, consequently, enable timely and appropriate treatment. In this context, the use of appropriate artificial intelligence (AI) tools is likely to improve the diagnostic accuracy of this approach.
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