Abstract
Maribavir is indicated for the treatment of refractory cytomegalovirus (CMV) infection/disease in patients who have undergone a solid organ transplant (SOT) or hematopoietic cell transplant (HCT). Only limited data on its use in real-world settings have been published from retrospective series. This retrospective study describes the real-world effectiveness of maribavir in 79 transplant patients with refractory CMV infection (67 SOT and 12 HCT) treated under a compassionate use program in France between October 2021 and April 2023. Maribavir was administered for <8 weeks, 8 weeks, and >8 weeks in 17, 32, and 30 patients, respectively. The response rate, defined as viremia clearance, was 53.2%, with a median time to first CMV clearance of 59 days. CMV clearance was observed in patients beyond 8 weeks of treatment. De novo maribavir resistance mutations were observed in 13.9% of patients, and CMV recurrence occurred in 45.2% of patients. Presence of CMV disease at baseline was associated with a lower likelihood of maribavir response. Compared to the pivotal SOLSTICE trial, real-world maribavir use demonstrated comparable effectiveness and a lower emergence of maribavir resistance. Moreover, outcomes of patients with a longer treatment duration suggested potential benefits of extending maribavir therapy beyond the recommended 8 weeks.