Department of Hematology, Oslo University Hospital, Oslo, Norway; Institute for Clinical Medicine, University of Oslo, Oslo, Norway.
Department of Hematology, Oslo University Hospital, Oslo, Norway; Institute for Clinical Medicine, University of Oslo, Oslo, Norway.
Transplant Cell Ther. 2023 Jun;29(6):364.e1-364.e11. doi: 10.1016/j.jtct.2023.02.023. Epub 2023 Mar 4.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the sole curative option for many patients diagnosed with hematologic malignancies. A major obstacle is graft-versus-host disease (GVHD), causing significant morbidity and mortality. Extracorporeal photopheresis (ECP) is an increasingly applied treatment for GVHD, owing in part to its favorable safety profile. In contrast, reports on the use of ECP to prevent GVHD are rare, and randomized controlled trials (RCTs) are lacking. We conducted an RCT to assess whether ECP applied post-transplantation could prevent the development of GVHD within the first year of transplantation. We enrolled 157 patients (age 18 to 74 years) with a hematologic malignancy undergoing their first allo-HSCT, randomized as 76 to the intervention group and 81 to the control group. ECP was initiated directly on engraftment and was planned twice weekly for 2 weeks, then once weekly for 4 weeks. GVHD, relapse, and death were analyzed by Cox regression analysis. During the first year, 45 patients in the intervention group and 52 control patients developed GVHD (hazard ratio [HR], .82; 95% confidence interval [CI], .55 to 1.22; P = .32). There were no differences in acute or chronic GVHD or its organ distribution in this intention-to-treat RCT. A per-protocol analysis revealed a significant difference in GVHD between the intervention group (per-protocol; n = 39 of 76) and the control group (n = 77), 46% versus 68%, respectively (HR, .47; 95% CI, .27 to .80; P = .006). Relapse occurred in 15 patients in the intervention group and in 11 control patients (HR, 1.38; 95% CI, .64 to 3.01; P = .42). GVHD-free relapse-free survival, event-free survival, overall survival, and nonrelapse mortality did not differ significantly between the 2 study groups. There also was no significant difference in immune reconstitution between the 2 groups. This first intention-to-treat RCT investigating ECP as GVHD prophylaxis in allo-HSCT for hematologic malignancy does not support the use of ECP as an adjunct to standard drug-based GVHD prophylaxis.
异基因造血干细胞移植（allo-HSCT）是许多血液系统恶性肿瘤患者的唯一治愈选择。主要障碍是移植物抗宿主病（GVHD），导致发病率和死亡率显著增加。体外光化学疗法（ECP）是一种越来越应用于 GVHD 的治疗方法，部分原因是其良好的安全性。相比之下，关于 ECP 预防 GVHD 的报告很少，并且缺乏随机对照试验（RCT）。我们进行了一项 RCT，以评估移植后立即应用 ECP 是否可以预防移植后第一年 GVHD 的发展。我们招募了 157 名年龄在 18 至 74 岁之间患有血液系统恶性肿瘤并接受首次 allo-HSCT 的患者，随机分为 76 名干预组和 81 名对照组。ECP 在植入后立即开始，计划每周两次进行 2 周，然后每周一次进行 4 周。通过 Cox 回归分析分析 GVHD、复发和死亡。在第一年，干预组的 45 名患者和对照组的 52 名患者发生 GVHD（风险比[HR]，0.82；95%置信区间[CI]，0.55 至 1.22；P=0.32）。在这项意向治疗 RCT 中，急性或慢性 GVHD 或其器官分布没有差异。方案分析显示，干预组（方案；n=76 名患者中的 39 名）与对照组（n=81 名患者中的 77 名）之间存在 GVHD 差异有统计学意义，分别为 46%和 68%（HR，0.47；95%CI，0.27 至 0.80；P=0.006）。干预组的 15 名患者和对照组的 11 名患者发生复发（HR，1.38；95%CI，0.64 至 3.01；P=0.42）。GVHD 无复发生存率、无事件生存率、总生存率和非复发死亡率在两组之间无显著差异。两组之间的免疫重建也没有显著差异。这项首次调查 allo-HSCT 中 ECP 作为 GVHD 预防的意向治疗 RCT 不支持将 ECP 作为标准药物 GVHD 预防的辅助治疗。
