Lancet Infect Dis

Background: Maribavir was approved by the European Medicines Agency in 2022 for the treatment of refractory or resistant cytomegalovirus (CMV) infections in adults after solid-organ transplantation or haematopoietic cell transplantation (HCT). However, real-world data in HCT recipients remain scarce. We aimed to report the clinical experience with maribavir in the HCT setting.

Methods: We did a multicentre retrospective registry study in adult and paediatric HCT patients who received maribavir for clinically significant CMV infection (CMV infection requiring treatment or CMV disease) at 37 EBMT centres across 15 countries in Europe after maribavir approval. To be included, patients had to have at least 12 weeks of follow-up. The primary objective was response to maribavir treatment at 12 weeks, which included CMV resolution and treatment failure; secondary endpoints included reasons for discontinuation, overall survival, and non-relapse mortality at 12 weeks.

Findings: Patients who underwent transplantation from March 30, 2021, to Oct 9, 2024, and received maribavir treatment between Dec 16, 2022, and Jan 17, 2025, were included. A total of 126 courses were provided for 118 patients (109 adults aged >18 years and nine children aged ≤18 years); 109 (87%) of 126 courses were given for pre-emptive treatment and 17 (13%) were given for CMV disease. Maribavir was given for refractory or resistant CMV infection or disease in 64 (51%) of 126 courses (mainly refractory CMV infection; 46 [37%] courses) and due to toxicities in 62 (49%) courses (mainly neutropenia; 31 [25%] courses), and most often used as second-line or third-line therapy (101 [80%] of 126 courses). CMV resolution was reached in 88 (81%) of 109 courses after pre-emptive treatment and in 12 (71%) of 17 courses after treatment for CMV disease. Maribavir discontinuation due to adverse events was infrequent (two [2%] of 126 courses). A CMV disease developed in five (5%) of 109 pre-emptive treatment courses. At 12 weeks, overall survival was 82% (95% CI 75-89), and non-relapse mortality was 15% (95% CI 9-22).

Interpretation: The findings of this large real-world study confirm that maribavir is an effective and well tolerated option for CMV infection and disease in HCT recipients.

Funding: None.