Successful Treatment of an Infant With Severe Combined Immunodeficiency and Ganciclovir-Resistant Cytomegalovirus Disease With Maribavir Followed By Subsequent Graft Recovery

Background: Refractory and resistant cytomegalovirus infection occurring in young infants has few effective treatments. Observations: A 2-month-old infant with severe combined immunodeficiency experienced graft failure following umbilical cord hematopoietic stem cell transplant due to cytomegalovirus (CMV) infection. Following treatment with foscarnet, ganciclovir, and viral-specific cytotoxic T-lymphocyte therapy, ganciclovir resistance developed, viremia was persistent, and CMV disease developed. Maribavir was added with resolution of CMV disease and improvement in the viral load. Evaluation for repeat transplant revealed late engraftment, which ultimately led to immunologic recovery. Conclusions: Maribavir is a novel antiviral therapy for refractory CMV infection, including in young infants.