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一项前瞻性研究比较了接受清髓性或强度降低性预处理的成人髓系恶性肿瘤患者同种异体移植的结局和与健康相关的生活质量。

A prospective study comparing the outcomes and health-related quality of life in adult patients with myeloid malignancies undergoing allogeneic transplantation using myeloablative or reduced-intensity conditioning.

机构信息

Blood and Marrow Transplant Program, Princess Margaret Hospital, University of Toronto, Ontario, Canada.

出版信息

Biol Blood Marrow Transplant. 2012 Jan;18(1):113-24. doi: 10.1016/j.bbmt.2011.05.022. Epub 2011 Jun 12.

Abstract

We compared the outcomes including health-related quality of life (HRQOL) in adult patients undergoing allogeneic transplantation using myeloablative conditioning (MAC) or reduced-intensity conditioning (RIC). This outcome study was a nonrandomized, prospective, observational noninferiority study, and primarily designed to determine whether RIC was as effective as MAC for myeloid malignancies. Comprehensive longitudinal assessment of HRQOL was done at baseline, day 30, day 100, day 180, and day 365 using validated instruments. A total of 115 patients (MAC, 51; RIC, 64) participated in this study. Of these 115 patients, 105 (91%) participated for HRQOL assessments. The main indication for HCT was acute myeloid leukemia (72%). Except age (median 41 vs 59 years, P < .0001), baseline characteristics were similar in patients undergoing MAC and RIC, respectively. Progression-free survival (PFS) at 1 year was 59% (SE = 7%) and 53% (SE = 6%) for the patients undergoing MAC and RIC, respectively (90% confidence interval [CI] -9% to +21%, P = .53). No significant difference in overall survival (OS), cumulative incidents of acute and chronic graft-versus-host disease (aGVHD, cGVHD), nonrelapse mortality (NRM) or relapse was observed in the 2 cohorts. The trajectory of decline and recovery of HRQOL was similar between the 2 cohorts. We conclude that clinical outcomes and HRQOL in patients with myeloid malignancies undergoing RIC are not inferior to MAC at 1 year.

摘要

我们比较了接受清髓性(MAC)或减低强度(RIC)预处理的异体移植成年患者的临床结局,包括健康相关的生活质量(HRQOL)。这是一项非随机、前瞻性、观察性非劣效性研究,主要旨在确定 RIC 对髓系恶性肿瘤是否与 MAC 同样有效。采用经过验证的量表,在基线、第 30 天、第 100 天、第 180 天和第 365 天进行全面的 HRQOL 纵向评估。共有 115 例患者(MAC,51 例;RIC,64 例)参与了这项研究。在这 115 例患者中,有 105 例(91%)患者接受了 HRQOL 评估。HCT 的主要适应证是急性髓系白血病(72%)。除年龄(中位数分别为 41 岁和 59 岁,P<0.0001)外,MAC 和 RIC 组患者的基线特征相似。MAC 和 RIC 组患者的 1 年无进展生存率(PFS)分别为 59%(SE=7%)和 53%(SE=6%)(90%置信区间[CI]:-9%至+21%,P=0.53)。两组患者在总生存率(OS)、急性和慢性移植物抗宿主病(aGVHD、cGVHD)、非复发死亡率(NRM)或复发的累积发生率方面均无显著差异。两组患者 HRQOL 下降和恢复的轨迹相似。我们的结论是,在 1 年时,RIC 组患者的临床结局和 HRQOL 与 MAC 组无差异。

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