Luznik Leo, O'Donnell Paul V, Symons Heather J, Chen Allen R, Leffell M Susan, Zahurak Marianna, Gooley Ted A, Piantadosi Steve, Kaup Michele, Ambinder Richard F, Huff Carol Ann, Matsui William, Bolaños-Meade Javier, Borrello Ivan, Powell Jonathan D, Harrington Elizabeth, Warnock Sandy, Flowers Mary, Brodsky Robert A, Sandmaier Brenda M, Storb Rainer F, Jones Richard J, Fuchs Ephraim J
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA.
Biol Blood Marrow Transplant. 2008 Jun;14(6):641-50. doi: 10.1016/j.bbmt.2008.03.005.
We evaluated the safety and efficacy of high-dose, posttransplantation cyclophosphamide (Cy) to prevent graft rejection and graft-versus-host disease (GVHD) after outpatient nonmyeloablative conditioning and T cell-replete bone marrow transplantation from partially HLA-mismatched (haploidentical) related donors. Patients with advanced hematologic malignancies (n = 67) or paroxysmal nocturnal hemoglobinuria (n = 1) received Cy 50 mg/kg i.v. on day 3 (n = 28) or on days 3 and 4 (n = 40) after transplantation. The median times to neutrophil (>500/microL) and platelet recovery (>20,000/microL) were 15 and 24 days, respectively. Graft failure occurred in 9 of 66 (13%) evaluable patients, and was fatal in 1. The cumulative incidences of grades II-IV and grades III-IV acute (aGVHD) by day 200 were 34% and 6%, respectively. There was a trend toward a lower risk of extensive chronic GVHD (cGVHD) among recipients of 2 versus 1 dose of posttransplantation Cy (P = .05), the only difference between these groups. The cumulative incidences of nonrelapse mortality (NRM) and relapse at 1 year were 15% and 51%, respectively. Actuarial overall survival (OS) and event-free survival (EFS) at 2 years after transplantation were 36% and 26%, respectively. Patients with lymphoid malignancies had an improved EFS compared to those with myelogenous malignancies (P = .02). Nonmyeloablative HLA-haploidentical BMT with posttransplantation Cy is associated with acceptable rates of fatal graft failure and severe aGVHD or cGVHD.
我们评估了高剂量移植后环磷酰胺(Cy)在门诊非清髓性预处理及来自部分HLA配型不合(单倍型相合)相关供者的T细胞充足型骨髓移植后预防移植物排斥和移植物抗宿主病(GVHD)的安全性和有效性。67例晚期血液系统恶性肿瘤患者或1例阵发性睡眠性血红蛋白尿患者在移植后第3天(n = 28)或第3天和第4天(n = 40)接受静脉注射50mg/kg的Cy。中性粒细胞(>500/μL)和血小板恢复(>20,000/μL)的中位时间分别为15天和24天。66例可评估患者中有9例(13%)发生移植物衰竭,其中1例死亡。至第200天,II-IV级和III-IV级急性移植物抗宿主病(aGVHD)的累积发生率分别为34%和6%。接受2剂与1剂移植后Cy的受者发生广泛性慢性移植物抗宿主病(cGVHD)的风险有降低趋势(P = 0.05),这是两组之间唯一的差异。1年时非复发死亡率(NRM)和复发的累积发生率分别为15%和51%。移植后2年的精算总生存率(OS)和无事件生存率(EFS)分别为36%和26%。与髓系恶性肿瘤患者相比,淋巴系恶性肿瘤患者的EFS有所改善(P = 0.02)。非清髓性HLA单倍型相合骨髓移植联合移植后Cy与可接受的致命性移植物衰竭及严重aGVHD或cGVHD发生率相关。