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挽救性单倍体相合移植后环磷酰胺治疗移植物失败患者的结果:代表法语国家骨髓移植和细胞治疗学会的报告。

Outcomes of Salvage Haploidentical Transplant with Post-Transplant Cyclophosphamide for Rescuing Graft Failure Patients: a Report on Behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy.

机构信息

Bone Marrow Transplant Unit, Saint Louis Hospital, Paris, France.

Statistics and Epidemiology Department, Saint Louis Hospital, Paris, France.

出版信息

Biol Blood Marrow Transplant. 2019 Sep;25(9):1798-1802. doi: 10.1016/j.bbmt.2019.05.013. Epub 2019 May 24.

Abstract

Prognosis of patients with graft failure is dismal, and retransplantation is the sole option for long-term survival. To address the interest of haploidentical transplantation as a salvage option in this context, we analyzed data from 24 patients with graft failure or loss retransplanted with a haploidentical donor who received post-transplant cyclophosphamide (PTCy) as graft-versus-host disease prophylaxis (GVHD). Fludarabine-based reduced-intensity conditioning was used in 23 patients and the Baltimore regimen in 14 patients. The median delay between previous and salvage transplantation for graft failure was 63 days (range, 39 to 98). In addition to PTCy, all patients received cyclosporine, and 22 patients also received mycophenolate mofetil for GVHD prophylaxis. With a median follow-up of 353 days (range, 16 to 2010), 1-year overall survival (OS) was 56% (95% confidence interval, 38% to 81%). Transplant complications accounted for 80% of deaths. The cumulative incidence of neutrophil engraftment at day +30 was 79%. Cumulative incidence of grades II to IV acute GVHD at day 100 was 14%, and 1-year cumulative incidence of chronic GVHD was 31%. One-year cumulative incidence of relapse was 13%. Stem cell source did not impact on engraftment, GVHD, relapse, or OS. Salvage haploidentical transplant with PTCy for rescuing graft failure patients leads to an acceptable 1-year OS and might be a valid option in this poor situation.

摘要

移植失败患者的预后较差,而再次移植是长期生存的唯一选择。为了解haploidentical 移植作为这种情况下挽救方案的应用情况,我们分析了 24 例因移植物衰竭或丢失而接受haploidentical 供者移植且接受移植后环磷酰胺(PTCy)作为移植物抗宿主病(GVHD)预防的患者的数据。23 例患者接受了氟达拉滨为基础的减低强度预处理方案,14 例患者接受了巴尔的摩方案。上次移植和挽救性移植之间因移植物衰竭的中位延迟为 63 天(范围 39 至 98 天)。除了 PTCy 外,所有患者均接受了环孢素治疗,22 例患者还接受了霉酚酸酯预防 GVHD。中位随访 353 天(范围 16 至 2010 天),1 年总生存率(OS)为 56%(95%置信区间,38%至 81%)。移植并发症占死亡的 80%。+30 天中性粒细胞植入的累积发生率为 79%。100 天的 II 至 IV 级急性 GVHD 的累积发生率为 14%,1 年慢性 GVHD 的累积发生率为 31%。1 年的累积复发率为 13%。干细胞来源未影响植入、GVHD、复发或 OS。对于挽救移植失败患者,用 PTCy 进行挽救性 haploidentical 移植可获得可接受的 1 年 OS,在这种不良情况下可能是一种有效的选择。

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