Hôpital Saint-Louis, APHP, Service d'hématologie greffe, Paris, France.
Université de Paris, Epidemiology and Statistics Research Center (CRESS), Institut National de la Santé et de la Recherche Médicale (INSERM), Institut National de la Recherche Agronomique (INRA), Paris, France.
Bone Marrow Transplant. 2021 Aug;56(8):1888-1899. doi: 10.1038/s41409-021-01252-7. Epub 2021 Mar 25.
This multicenter prospective phase 2 trial analyzed disease-free survival (DFS) in myelofibrosis patients receiving ruxolitinib for 6 months before transplantation. Seventy-six patients were recruited. Age-adjusted dynamic international prognostic scoring system was intermediate-1, intermediate-2, and high in 27 (36%), 31 (41%), and 18 (24%) patients. All patients received ruxolitinib from inclusion to conditioning regimen (fludarabine-melphalan) or to progression. A donor was found in 64 patients: 18 HLA-matched sibling donor (MSD), 32 HLA-matched unrelated (UD10/10), and 14 HLA mismatched unrelated donor (UD9/10. Among 64 patients with a donor, 20 (31%) achieved a partial response before transplantation and 59 (92%) could be transplanted after ruxolitinib therapy (18/18 MSD, 30/21 UD10/10, 11/34 UD9/10), of whom 19 (32%) were splenectomized. Overall survival from inclusion was 68% at 12 months. One-year DFS after transplantation was 55%: 83%, 40%, and 34% after MSD, UD10/10 or UD9/10, respectively. Cumulative incidence of grade 2-4 acute graft-versus-host disease (GVHD) was 66% and non-relapse-mortality was 42% at 12 months. Short course of ruxolitinib before transplantation is followed by a high rate of transplantation. With the platform used in this protocol, outcome was much better in patients transplanted with HLA-matched sibling donor as compared to unrelated donor.
这项多中心前瞻性 2 期试验分析了接受鲁索利替尼治疗 6 个月后进行移植的骨髓纤维化患者的无病生存（DFS）。共招募了 76 名患者。年龄调整后的动态国际预后评分系统在 27 名（36%）、31 名（41%）和 18 名（24%）患者中为中-1、中-2 和高。所有患者从纳入到预处理方案（氟达拉滨-马法兰）或进展都接受了鲁索利替尼治疗。在 64 名有供体的患者中，发现了 18 名 HLA 匹配的同胞供体（MSD）、32 名 HLA 匹配的无关供体（UD10/10）和 14 名 HLA 错配的无关供体（UD9/10）。在 64 名有供体的患者中，20 名（31%）在移植前达到部分缓解，59 名（92%）可以在鲁索利替尼治疗后进行移植（18/18 MSD、30/21 UD10/10、11/34 UD9/10），其中 19 名（32%）进行了脾切除术。从纳入到 12 个月的总生存率为 68%。移植后 1 年的 DFS 为 55%：分别为 MSD、UD10/10 或 UD9/10 后的 83%、40%和 34%。12 个月时，2-4 级急性移植物抗宿主病（GVHD）累积发生率为 66%，非复发死亡率为 42%。
在这项方案中使用的平台上，与无关供体相比，接受 HLA 匹配的同胞供体移植的患者的结果要好得多。
