Anjali Sharathkumar, MD, Shares Romiplostim Outcomes in Severe Aplastic Anemia With MDS

Dr. Sharathkumar, of Carver College of Medicine at University of Iowa, met with Heme Today at the 2024 American Society of Pediatric Hematology/Oncology Conference to share findings from her paper presentation titled, “Romiplostim for Treating Children With Severe Aplastic Anemia and Myelodysplastic Syndromes [MDS]”.

Dr. Sharathkumar explained the motivation for the trial of romiplostim in children with severe aplastic anemia while she was evaluating romiplostim for immune thrombocytopenia in a phase III study. She noted that several studies suggested that thrombopoietin mimetics, such as romiplostim, “actually induce hematopoietic stem cells and cause the expansion of the hematopoietic stem cell pool.”

Simultaneously, she received a handful of new pediatric patients with aplastic anemia who were unable to receive standard of care due to the lack of appropriate donors. Dr. Sharathkumar and colleagues offered off-label romiplostim. “To our surprise, our first patient responded very well and then the second patient responded,” she stated.

Following the promising results, Dr. Sharathkumar received funding and evaluated romiplostim in a new interventional cohort. She described that the preliminary analysis showed a “phenomenally high success rate,” with romiplostim in children with severe aplastic anemia.

In wrapping up our interview, Dr. Sharathkumar also covered the long-term follow-up results and safety findings from the paper presentation.

Related: What Are the Late Effects, Quality of Life in Patients With Severe Aplastic Anemia After HSCT?