Aplastic anemia is a rare bone marrow failure disorder where the body stops making enough blood cells. About 600 to 900 people are diagnosed in the U.S. each year. Current treatment includes immunosuppressive therapy — usually horse or rabbit ATG plus cyclosporine — or stem cell transplant for eligible patients.
What's actually going on in research
Trials are testing eltrombopag, a platelet-boosting drug now FDA-approved as add-on therapy, in various combinations to improve response rates. Researchers are studying thrombopoietin receptor agonists, new conditioning regimens for transplant, and treatments for inherited bone marrow failure syndromes that can present like aplastic anemia. Some studies focus on reducing transplant complications and preventing relapse after immunosuppression.
Combination immunosuppression
Trials are testing whether adding eltrombopag to standard ATG plus cyclosporine increases the number of people who recover blood counts. Early results suggest higher response rates with the three-drug combination.
Reduced-intensity transplant
Studies are refining conditioning regimens before stem cell transplant to reduce complications like graft-versus-host disease. Newer protocols aim to expand transplant access for older patients and those without matched siblings.
Relapse prevention
Researchers are testing whether extending eltrombopag treatment after initial response can prevent the 30-40% of patients who relapse after stopping immunosuppression. Trials compare different durations and dosing strategies.
What to know before you search
Eligibility typically depends on disease severity, prior treatments, time since diagnosis, donor availability for transplant trials, and whether the aplastic anemia is acquired or inherited.
What types of trials are currently open
- Immunosuppression trials — Testing combinations of ATG, cyclosporine, and eltrombopag to improve how many people respond and how completely blood counts recover.
- Transplant trials — Studying different conditioning regimens and graft-versus-host disease prevention strategies, especially for older patients or those without matched donors.
- Maintenance trials — Testing whether continuing treatment after initial response prevents relapse, particularly with eltrombopag or other thrombopoietin receptor agonists.
- Biomarker studies — Looking at genetic markers and immune patterns to predict who will respond to immunosuppression versus needing transplant.
- Inherited syndromes — Studies of telomere biology disorders and Fanconi anemia, which can appear similar to acquired aplastic anemia but need different treatment.
Recently added Aplastic Anemia trials
Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic Anemia
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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