Immunosuppressants can be used to treat chronic graft-versus-host disease (cGVHD) following allogeneic hematopoietic cell transplantation. However, real-world retrospective studies on practical regimens and health outcomes remain limited. To identify challenges associated with systemic treatment of cGVHD, we analyzed anonymized Japanese medical insurance claims data. Among 1090 patients with cGVHD, 81% received systemic treatment with immunosuppressive drugs. The most common initial regimen was a combination of tacrolimus and steroids. Second-line treatment was provided in 44% of patients including the addition of steroids, calcineurin inhibitors, or mycophenolate mofetil, or switching to another drug class. Third-line treatment was provided in 23% and steroid monotherapy was the most commonly used. Immunosuppressive treatment was temporarily discontinued in 79% of patients but was re-initiated in approximately a half of them. Young recipients (< 15 years) and umbilical cord blood recipients had lower rates of treatment re-initiation and conferred lower mortality. Various complications were observed after transplantation but most of them gradually resolved, with the exception of chronic bone, eye, and respiratory system disorders. The immunocompromised state of these patients was also reflected in the frequent use of anti-infective agents. The adverse effects associated with long-term steroid use pose a clinical challenge, highlighting the need for alternative treatments.
Shindo et al. (2026) studied this question.