Romiplostim for Aplastic Anemia

This trial examines the effectiveness of a drug called romiplostim for individuals with severe aplastic anemia (SAA), a condition where the bone marrow produces insufficient blood cells. Participants are divided into two groups: one group, consisting of those untreated previously, will receive romiplostim with immunosuppressive therapy; the other group, comprising individuals whose previous treatments failed, will receive romiplostim alone. This trial may suit children or young adults with severe aplastic anemia, whether they are new to treatment or have tried other treatments without success. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

The trial requires that you stop taking certain medications like eltrombopag, avatrombopag, lusutrombopag, and androgens before participating. If you've stopped androgen therapy for more than 2 weeks, you may be eligible to join the study.

Research has shown that romiplostim has been tested for safety in people with aplastic anemia (AA). One study found that romiplostim, used alone, was safe starting at doses of 20 micrograms per kilogram each week. It worked quickly in those whose aplastic anemia did not respond to other treatments, with most patients tolerating the treatment well and experiencing no serious side effects.

Romiplostim is unique because it boosts the production of platelets by mimicking the natural hormone thrombopoietin, which is a different approach compared to traditional treatments for aplastic anemia that often involve immunosuppressive therapy or bone marrow transplants. Researchers are excited because Romiplostim offers a potential new way to help patients who are either new to treatment or have not responded to previous therapies. By directly stimulating platelet production, Romiplostim could reduce the need for transfusions and improve blood counts more effectively than existing options.

In this trial, participants with severe aplastic anemia (SAA) will receive treatments tailored to their condition. Research has shown that romiplostim, administered alone to participants in Cohort B, can effectively treat severe aplastic anemia, particularly in patients unresponsive to other treatments. Approximately 70% of patients have experienced improvements in blood counts with this treatment. Participants in Cohort A will receive romiplostim combined with immunosuppressive therapy (IST). Studies have shown that this combination yields promising results, with response rates between 70% and 95%. This suggests that romiplostim can enhance blood cell production, crucial for managing aplastic anemia. However, researchers continue to study the long-term effectiveness and safety beyond one year.¹⁶⁷⁸⁹