Aplastic anemia is a rare bone marrow failure disorder where the body stops making enough blood cells. About 500-1,000 Americans are diagnosed each year. Standard treatment includes immunosuppressive therapy or stem cell transplant, and most patients now survive long-term with appropriate treatment.
What's actually going on in research
Trials are testing eltrombopag and other thrombopoietin receptor agonists to boost blood counts, refined immunosuppressive combinations, gene therapy for severe cases, and new approaches to prevent transplant rejection. Researchers are studying why some patients relapse after initial response and how to identify the best treatment path earlier.
Thrombopoietin receptor agonists
Eltrombopag, FDA-approved for aplastic anemia in 2014, helps stimulate blood cell production and is now being combined with standard immunosuppression. Trials are testing whether adding it upfront improves outcomes and reduces relapse rates.
Improved immunosuppression
Studies are testing optimized combinations of antithymocyte globulin and cyclosporine, along with new agents that may work faster or with fewer side effects. The goal is higher response rates and longer remissions without transplant.
Gene therapy approaches
Researchers are exploring gene-modified stem cell transplants for patients without matched donors. Early work focuses on severe cases where standard options have failed.
What to know before you search
Eligibility typically depends on disease severity, prior treatments, blood counts, transfusion needs, and whether a matched donor is available for transplant trials.
What types of trials are currently open
- Immunosuppression trials — Testing combinations of drugs that suppress the immune attack on bone marrow, often comparing standard therapy to regimens that add eltrombopag or other agents.
- Transplant trials — Studies of stem cell transplant from matched or partially-matched donors, testing ways to reduce rejection and graft-versus-host disease.
- Eltrombopag trials — Testing thrombopoietin receptor agonists alone or in combination with immunosuppression to improve blood counts and reduce transfusion needs.
- Relapse prevention trials — Testing maintenance treatments for patients who responded to initial therapy but remain at risk for disease returning.
- Biomarker studies — Following patients to identify genetic or immune markers that predict response to treatment or risk of relapse.
Recently added Aplastic Anemia trials
Take an immune therapy for severe aplastic anemia that hasn't responded to other treatments
This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) \<30 × 10\^9/L and/or hemoglobin (HGB) \<90 g/L at enrollment. If the phase I results demonstrate an acceptable safety profile and allow determination of the maximum tolerated dose (MTD), the phase II part will be initiated directly to evaluate the efficacy of isatuximab.
Complete MRI scans to help detect oral cancer early
This study being done to learn more about the use of medical Magnetic Resonance Imaging (mMRI) and dedicated dental MRI (ddMRI) as a non-invasive diagnosing tool when evaluating potential oral cancerous and precancerous lesions in Fanconi Anemia patients.
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