Abstract
Objective: To evaluate the real-world effectiveness and safety of maribavir for treating CMV infection in hematopoietic stem cell transplant (HSCT) patients.
Methods: This was a dual-center, retrospective, controlled cohort study. Patients who received maribavir 400 mg twice daily (BID) for CMV infection post-HSCT at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences and the Chinese PLA General Hospital between June 1, 2024, and July 31, 2025, were included. Patients receiving other first-line antiviral therapies (predominantly foscarnet-based) in 2023 served as controls. The primary endpoint was CMV clearance rate. Confirmed viremia clearance was defined as plasma CMV DNA level below the lower limit of quantification in two consecutive post-baseline samples, separated by ≥5 days
Results: A total of 62 patients were enrolled from both centers.58.06% (36/62) were male, with a median age of 42 years (range: 15-66). 74.19% (46/62) received haploidentical transplants. The median time to CMV infection post-transplant was 116 days (range: 14-394). Nineteen patients developed infection while on CMV prophylaxis. Fourteen patients developed CMV disease. Thirteen had recurrent CMV infection, ten had refractory infection, and sixteen were intolerant to prior antiviral therapy. The median baseline viral load was 3044 copies/mL (range: 421-60,700). The median duration of maribavir treatment was 16 days (range: 2-108). The median time to initial viral load decline was 5 days (range: 1-32). Excluding two patients who died prior to viral clearance, the CMV clearance rate was 96.77% (60/62). The median time to confirmed viral clearance was 7 days (range: 1-42). CMV recurrence occurred in 11.67% (7/60) of patients after stopping maribavir. All patients tolerated maribavir treatment. No treatment-related adverse events were observed during therapy.
Nineteen patients received first-line maribavir for post-HSCT CMV infection, while 20 control patients received other first-line regimens (12 predominantly foscarnet; 8 received combination therapy including ganciclovir or letermovir). Baseline characteristics were balanced between groups: Maribavir group: 73.68% male (14/19), median age 43 years (range 21-66), 84.42% alternative donor transplants (16/19), 21.05% breakthrough infection during prophylaxis (4/19). Control group: 70.0% male (14/20, P=0.798), median age 43 years (range 17-59, P=0.339), 85.0% alternative donors (17/20, P=0.946), 35.0% breakthrough infection (7/20, P=0.333). Median time to infection post-HSCT was similar (139 vs 118 days, P=0.748). Median time from treatment initiation to plasma CMV DNA below the treatment threshold was 5 vs 6.5 days (P=0.238). The CMV clearance rate was 94.74% (18/19) with maribavir versus 100% in controls (P=0.299). Median time to viral clearance was shorter in the maribavir group (7 days, range 2-20) compared to controls (12 days, range 1-27), although this difference was not statistically significant (P=0.251). CMV recurrence rates were 5.26% (1/19) and 10.0% (2/20), respectively (P=0.579).
Conclusion: In real-world, maribavir demonstrated rapid, effective, and safe clearance of CMV viremia in HSCT recipients. The comparative cohort study demonstrates comparable CMV clearance rates between first-line maribavir and conventional antiviral regimens in HSCT recipients. Notably, maribavir was associated with a clinically relevant 5-day reduction in median time to viral clearance (7 vs 12 days).
