一项双臂2期临床试验的结果，该试验使用移植后环磷酰胺预防单倍体相合供者和错配无关供者造血干细胞移植中的移植物抗宿主病。
Results of a 2-arm, phase 2 clinical trial using post-transplantation cyclophosphamide for the prevention of graft-versus-host disease in haploidentical donor and mismatched unrelated donor hematopoietic stem cell transplantation.
作者信息
Gaballa Sameh, Ge Isabell, El Fakih Riad, Brammer Jonathan E, Kongtim Piyanuch, Tomuleasa Ciprian, Wang Sa A, Lee Dean, Petropoulos Demetrios, Cao Kai, Rondon Gabriela, Chen Julianne, Hammerstrom Aimee, Lombardi Lindsey, Alatrash Gheath, Korbling Martin, Oran Betul, Kebriaei Partow, Ahmed Sairah, Shah Nina, Rezvani Katayoun, Marin David, Bashir Qaiser, Alousi Amin, Nieto Yago, Qazilbash Muzaffar, Hosing Chitra, Popat Uday, Shpall Elizabeth J, Khouri Issa, Champlin Richard E, Ciurea Stefan O
机构信息
Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.
University Medical Center Freiburg, Freiburg, Germany.
出版信息
Cancer. 2016 Nov 15;122(21):3316-3326. doi: 10.1002/cncr.30180. Epub 2016 Jul 12.
BACKGROUND
High-dose, post-transplantation cyclophosphamide (PTCy) to prevent graft-versus-host disease (GVHD) has improved outcomes in haploidentical (HAPLO) stem cell transplantation (SCT). However, it remains unclear whether this strategy is effective in SCT from 1-antigen human leukocyte antigen (HLA)-mismatched unrelated donors (9/10 MUD) and how the outcomes of these patients compare with those of haploidentical transplantation recipients.
METHODS
A parallel, 2-arm, nonrandomized phase 2 clinical trial was conducted of melphalan-based reduced-intensity conditioning with PTCy, tacrolimus, and mycophenolate mofetil to prevent GVHD in patients with high-risk hematologic malignancies who underwent HAPLO (n = 60) or 9/10 MUD (n = 46) SCT.
RESULTS
The 1-year overall and progression-free survival rates were 70% and 60%, respectively, in the HAPLO arm and 60% and 47%, respectively, in the 9/10 MUD arm. The day +100 cumulative incidence of grade II to IV acute GVHD and grade III to IV acute GVHD was 28% and 3%, respectively, in the HAPLO arm and 33% and 13%, respectively, in the 9/10 MUD arm. The 2-year cumulative incidence of chronic GVHD was 24% in the HAPLO arm and 19% in the 9/10 MUD arm. The 1-year cumulative incidence of nonrelapse mortality was 21% in the HAPLO arm and 31% in the 9/10 MUD arm, and the 1-year relapse rate was 19% in the HAPLO arm and 25% in the 9/10 MUD arm.
CONCLUSIONS
Although this was a nonrandomized study and could not serve as a direct comparison between the 2 groups, the authors conclude that PTCy-based GVHD prophylaxis is effective for both HAPLO and 9/10 MUD SCTs. Prospective randomized trials will be required to compare the efficacies of alternative donor options for patients lacking HLA-matched donors. Cancer 2016;122:3316-3326. © 2016 American Cancer Society.
背景
移植后高剂量环磷酰胺（PTCy）用于预防移植物抗宿主病（GVHD）已改善了单倍体（HAPLO）干细胞移植（SCT）的疗效。然而，尚不清楚该策略在1抗原人类白细胞抗原（HLA）不匹配的无关供者（9/10 MUD）的SCT中是否有效，以及这些患者的结局与单倍体移植受者的结局相比如何。
方法
进行了一项平行、双臂、非随机的2期临床试验，对接受HAPLO（n = 60）或9/10 MUD（n = 46）SCT的高危血液系统恶性肿瘤患者采用基于美法仑的减低强度预处理联合PTCy、他克莫司和霉酚酸酯来预防GVHD。
结果
HAPLO组1年总生存率和无进展生存率分别为70%和60%，9/10 MUD组分别为60%和47%。HAPLO组II至IV级急性GVHD和III至IV级急性GVHD的+100天累积发生率分别为28%和3%，9/10 MUD组分别为33%和13%。HAPLO组慢性GVHD的2年累积发生率为24%，9/10 MUD组为19%。HAPLO组非复发死亡率的1年累积发生率为21%，9/10 MUD组为31%，HAPLO组1年复发率为19%，9/10 MUD组为25%。
结论
尽管这是一项非随机研究，不能作为两组之间的直接比较，但作者得出结论，基于PTCy的GVHD预防对HAPLO和9/10 MUD的SCT均有效。对于缺乏HLA匹配供者的患者，需要进行前瞻性随机试验以比较不同供者选择的疗效。《癌症》2016年；122：3316 - 3326。©2016美国癌症协会
Zotero 插件