BREAKING: Isatuximab Shows Promise in Addressing Red Cell Aplasia Following Stem Cell Transplants, Pioneering Clinical Trial Underway
[City, State] – A novel approach utilizing the anti-CD38 agent isatuximab is demonstrating potential in treating delayed red cell engraftment – a serious immune-mediated complication following allogeneic hematopoietic cell transplantation. A recently published case report, and the subsequent clinical trial it spurred, offer a potential new avenue for patients unresponsive to standard therapies.
Mary Naufal, PharmD, MS, BCOP, detailed the case and the rationale behind using isatuximab in a recent interview with Pharmacy Times. Delayed red cell engraftment falls under the umbrella of immune cytopenias, characterized by immune destruction of differentiated blood cells, impacting both red blood cells and platelets. The incidence of these cytopenias post-transplant ranges from 20% to 40%, and is heightened in patients undergoing allogeneic transplants, reduced-intensity conditioning, or those with ABO mismatches.
“The therapies that we use for patients who develop this complication have largely been extrapolated from the non-transplant setting,” explained Naufal. “The standard of care is usually high-dose steroids; though, those come with meaningful side effects and further immunosuppress already immunocompromised patients.” Other common treatments include IVIG, erythropoietin, calcineurin inhibitors, and B-cell targeted agents like bortezomib and anti-CD20 agents.
The case report focused on a patient experiencing pure red cell aplasia after transplant who had failed to respond to other treatments. Initially, the patient was denied insurance coverage for daratumumab, another CD38-targeted antibody with existing (though limited) evidence of success in this setting. “We landed on [isatuximab] as an option because our patient was initially denied insurance coverage for daratumumab,” Naufal stated. The drug was ultimately obtained through the manufacturer’s care assist program.
Following a multiple myeloma governance regimen – weekly dosing for four weeks, then every two weeks – the patient required eight doses of isatuximab before achieving transfusion independence and demonstrating increased erythroid precursors. To date, this is believed to be the frist published case report detailing the use of isatuximab in this specific context.
The positive outcome from this single case served as a catalyst for a prospective clinical trial, now fully enrolled, led by one of Naufal’s colleagues. “I think our accomplished outcome for this patient, combined with the lack of prospective studies…motivated one of our physicians to take the lead in opening a study to look at this prospectively,” Naufal said.”This case served as an incentive to explore the therapy in a prospective fashion so that the data becomes more reliable.” Data analysis and publication of the trial results are anticipated in the future.
