Michonneau David, Malard Florent, Le Grand Sophie, Magro Leonardo, D'Aveni Maud, Tudesq Jean Jacques, Villate Alban, Meunier Mathieu, Maillard Natacha, Castilla-Llorente Cristina, Marçais Ambroise, Cabrera Quentin, Huynh Anne, Menard Anne-Lise, Forcade Edouard, Labussière-Wallet Hélène, Raus Nicole, Loschi Michael
Hematology and transplantation unit, Saint Louis hospital, Paris, France.
INSERM UMR1342 Saint Louis Research Institute, Paris Cité University, IHU Thema2 Leukemia Institute Paris Saint Louis, Paris, France.
Bone Marrow Transplant. 2025 Apr 1. doi: 10.1038/s41409-025-02554-w.
Chronic graft versus host disease is a major cause of morbidity after allogeneic haematopoietic cell transplantation. Belumosudil has recently been approved for the treatment of cGVHD refractory after two lines of treatment. However, few data are available to evaluate its efficacy and safety in real life. 68 patients with cGVHD received belumosudil through a compassionate access program in France. The median follow-up was 337 days from belumosudil initiation. Eighty-two percent of patients had severe cGVHD with a median of three organs involved. Patients had received a median of three prior treatment lines. Median treatment duration was 251 days. The best overall response rate (ORR) was 57.3%, including 14.7% complete remission (CR) and 42.6% partial response (PR). The ORR at three and six months was 47% and 45.6%, respectively. Liver and mouth involvement showed the highest response rates (72.7% and 70.4%), while lung involvement had the lowest (17.2%). Median failure-free survival (FFS) was not reached, with 6- and 12-month FFS rates of 89.1% and 80.4%, respectively. Nine patients died, mainly from GVHD (n = 5). Ten adverse events were reported, leading to treatment discontinuation in three cases. These results support the efficacy and safety of belumosudil in refractory cGVHD.
慢性移植物抗宿主病是异基因造血细胞移植后发病的主要原因。贝利莫司最近已被批准用于治疗经两线治疗后难治的慢性移植物抗宿主病（cGVHD）。然而，在现实生活中评估其疗效和安全性的数据很少。68例cGVHD患者通过法国的一项同情用药计划接受了贝利莫司治疗。从开始使用贝利莫司起，中位随访时间为337天。82%的患者患有重度cGVHD，累及器官的中位数为3个。患者接受的先前治疗线数中位数为3条。中位治疗持续时间为251天。最佳总体缓解率（ORR）为57.3%，包括14.7%的完全缓解（CR）和42.6%的部分缓解（PR）。3个月和6个月时的ORR分别为47%和45.6%。肝脏和口腔受累的缓解率最高（分别为72.7%和70.4%），而肺部受累的缓解率最低（17.2%）。未达到中位无失败生存期（FFS），6个月和12个月的FFS率分别为89.1%和80.4%。9例患者死亡，主要死于移植物抗宿主病（n = 5）。报告了10起不良事件，3例导致治疗中断。这些结果支持贝利莫司在难治性cGVHD中的疗效和安全性。
