动员的血液或骨髓移植患者的长期结果和晚期效应：一项随机试验。

Long-term outcome and late effects in patients transplanted with mobilised blood or bone marrow: a randomised trial.

机构信息

Hematology and Stem Cell Transplantation, Asklepios Hospital St Georg, Hamburg, Germany.

出版信息

Lancet Oncol. 2010 Apr;11(4):331-8. doi: 10.1016/S1470-2045(09)70352-3. Epub 2010 Jan 30.

DOI:10.1016/S1470-2045(09)70352-3

PMID:20117965

Abstract

BACKGROUND

Most allogeneic haematopoietic stem cell transplants now use peripheral blood progenitor cell transplantation (PBPCT) instead of bone-marrow transplantation (BMT). Long-term data on outcome and late effects of PBPCT compared with BMT are scarce. Here we present long-term data from a randomised study comparing PBPCT with BMT.

METHODS

Between February, 1995, and September, 1999, 329 patients with leukaemia received either PBPCT (n=163) or BMT (n=166) from HLA-identical sibling donors after central randomisation accounting for stratification criteria. Follow-up data were collected via questionnaires from 87% (176 of 202; 84 PBPCT, 92 BMT) patients who survived for more than 3 years (median of 9.3 years) after transplantation. Efficacy analyses included all patients who received treatment. This study is registered with ClinicalTrials.gov, number NCT01020175.

FINDINGS

10-year overall survival was 49.1% for patients who underwent PBPCT and 56.5% for patients who underwent BMT (HR 0.83, 95% CI 0.60-1.15; p=0.27). Leukaemia-free survival was 28.3% with BMT versus 13.0% with PBPCT (0.61, CI 0.32-1.16; p=0.12) for acute lymphoblastic leukaemia; 62.3% with BMT versus 47.1% with PBPCT for acute myeloid leukaemia (0.67, 0.39-1.16; p=0.16); and 40.2% with BMT versus 48.5% with PBPCT for chronic myeloid leukaemia (1.12, 0.73-1.74; p=0.60). More patients developed chronic graft-versus-host disease after PBPCT (n=56, 73%) than after BMT (n=46, 56%; p=0.021), with more frequent involvement of skin, liver, and oral mucosa, and more patients who underwent PBPCT needed immunosuppressive treatment 5 years after transplantation (n=20, 26%) than patients who had BMT (n=10, 12%; p=0.024). Nonetheless, there was no difference in performance status, return to work, incidence of bronchiolitis obliterans, and haematopoietic function between the two groups. 14 cases of secondary malignancies occurred (five after BMT, nine after PBPCT), resulting in a cumulative incidence of 3% and 7% after BMT and PBPCT (p=0.17), respectively.

INTERPRETATION

More than 9 years after transplantation, overall and leukaemia-free survival remain similar in patients who underwent BMT and PBPCT. Differences in the incidence of chronic graft-versus-host disease and the duration of immunosuppression exist, but do not affect survival, general health status, or late events.

FUNDING

No external funding was received.

摘要

背景

目前大多数异基因造血干细胞移植都使用外周血造血干细胞移植（PBPCT）代替骨髓移植（BMT）。与 BMT 相比，PBPCT 的长期结果和晚期效应的长期数据仍然很少。在此，我们从一项比较 PBPCT 与 BMT 的随机研究中提供长期数据。

方法

1995 年 2 月至 1999 年 9 月，329 例白血病患者在中央随机分组中接受了 HLA 完全匹配的同胞供体的 PBPCT（n=163）或 BMT（n=166），并进行了分层。对移植后生存时间超过 3 年（中位时间为 9.3 年）的 87%（176 例中有 176 例；84 例 PBPCT，92 例 BMT）的患者通过问卷调查收集随访数据。疗效分析包括所有接受治疗的患者。本研究在 ClinicalTrials.gov 注册，编号为 NCT01020175。

结果

接受 PBPCT 的患者 10 年总生存率为 49.1%，接受 BMT 的患者为 56.5%（HR 0.83，95%CI 0.60-1.15；p=0.27）。接受 BMT 的患者无白血病生存率为 28.3%，而接受 PBPCT 的患者为 13.0%（0.61，0.32-1.16；p=0.12），用于急性淋巴细胞白血病；接受 BMT 的患者急性髓细胞白血病的无白血病生存率为 62.3%，而接受 PBPCT 的患者为 47.1%（0.67，0.39-1.16；p=0.16）；接受 BMT 的患者慢性髓细胞白血病的无白血病生存率为 40.2%，而接受 PBPCT 的患者为 48.5%（1.12，0.73-1.74；p=0.60）。接受 PBPCT 的患者慢性移植物抗宿主病的发生率（n=56，73%）高于接受 BMT 的患者（n=46，56%；p=0.021），皮肤、肝脏和口腔黏膜受累更为频繁，且接受 PBPCT 的患者在移植后 5 年需要免疫抑制治疗的比例（n=20，26%）高于接受 BMT 的患者（n=10，12%；p=0.024）。但是，两组之间的表现状态、重返工作、细支气管炎闭塞的发生率和造血功能均无差异。发生 14 例继发性恶性肿瘤（BMT 后 5 例，PBPCT 后 9 例），导致 BMT 和 PBPCT 后累积发生率分别为 3%和 7%（p=0.17）。