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
Aplastic Anemia Life-Course Cohort Study
To establish a longitudinal cohort spanning the entire care continuum of acquired aplastic anemia (AA), from diagnosis and treatment through long-term follow-up, integrating routinely collected clinical data with biospecimens obtained in accordance with applicable ethical and regulatory requirements. The cohort will support observational studies of the natural history of AA; treatment effectiveness and safety; the evolution of clonal hematopoiesis and clonal T-cell populations; immune reconstitution following immunosuppressive therapy (IST); the incidence of platelet transfusion refractoriness and its association with response to IST; and hematopoietic and immune reconstitution after transplantation. Ultimately, the cohort aims to generate real-world evidence to inform clinical practice and reduce the burden of AA.
Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi Anemia
Finding effective ways to slow down or prevent the development of cancer in people with Fanconi anemia (FA) is a critical medical priority. Individuals with FA are at dramatically higher risk of developing aggressive cancers, particularly of the head and neck, and these cancers are difficult to treat with standard therapies such as surgery, chemotherapy or radiation. Because options for treatment are limited and outcomes are often poor, prevention strategies are urgently needed. In this study, the investigators will test two widely available drugs, metformin and naproxen, to evaluate whether they can lower the risk of cancer in FA patients. Metformin is commonly prescribed for type 2 diabetes, while naproxen is a nonsteroidal anti-inflammatory drug (NSAID) frequently used for pain and inflammation. Both have shown potential in previous research to influence biological pathways linked to cancer development. By repurposing these well-known drugs, this trial aims to explore a safe, practical approach to cancer prevention in this vulnerable population. The study, conducted at the Rockefeller University and the University of Minnesota, has several goals. First, the investigators will carefully assess the safety and tolerability of metformin and naproxen when used in participants with FA. Because these individuals can be more susceptible to metabolic disorders like diabetes compared to the general population, and may have genitourinary abnormalities related to their underlying condition, monitoring for side effects and adverse events is especially important. Second, investigators will evaluate whether intervention affects visible or microscopic changes in the mouth, where cancers often arise. This will involve performing small biopsies of oral tissue to look for cancerous or pre-cancerous lesions. Third, investigators will investigate early molecular changes using an exploratory less invasive technique: collecting cells with a soft brush, similar to a toothbrush. These samples will allow the investigators to analyze biomarkers-biological signals that can indicate increased or reduced cancer risk-before and after intervention. Together, these approaches will provide information on whether metformin or naproxen are safe and well-tolerated in the FA population. As an additional outcome, by combining direct tissue analysis with biomarker studies, the investigators aim to detect subtle changes that precede cancer, giving us an early indication of whether the drugs are working as preventive agents. Although this is an early-phase study, its potential significance is considerable. Demonstrating that well-tolerated, inexpensive, and widely accessible drugs may have potential to reduce cancer risk would represent a major advance for FA patients, who currently face limited options for prevention and treatment. This work may also offer insights relevant to broader cancer prevention efforts in other high-risk groups. Ultimately, our study seeks to take an important step toward changing the outlook for people with Fanconi anemia by moving the focus from late-stage treatment toward early prevention.
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