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年轻慢性淋巴细胞白血病患者的 HLA 全相合同胞异基因骨髓移植。欧洲血液和骨髓移植组及国际骨髓移植登记处。

HLA-identical sibling bone marrow transplantation in younger patients with chronic lymphocytic leukemia. European Group for Blood and Marrow Transplantation and the International Bone Marrow Transplant Registry.

作者信息

Michallet M, Archimbaud E, Bandini G, Rowlings P A, Deeg H J, Gahrton G, Montserrat E, Rozman C, Gratwohl A, Gale R P

机构信息

Edouard Herriot Hospital, Lyon, France.

出版信息

Ann Intern Med. 1996 Feb 1;124(3):311-5. doi: 10.7326/0003-4819-124-3-199602010-00005.

Abstract

OBJECTIVE

To characterize in detail the outcomes of HLA-identical sibling bone marrow transplantation for chronic lymphocytic leukemia (CLL) in patients younger than 60 years of age.

DESIGN

Retrospective cohort study.

SETTING

30 centers for bone marrow transplantation worldwide, which reported data on outcome of HLA-identical sibling bone marrow transplantation for CLL to the European Group for Blood and Marrow Transplantation or the International Bone Marrow Transplant Registry between 1984 and 1992.

PATIENTS

54 patients diagnosed with CLL (median age, 41 years; range, 21 to 58 years). The median interval from diagnosis to transplantation was 37 months (range, 5 to 130 months). At the time of transplantation, 3 patients were at Rai stage 0; 10 were at stage 1; 10 were at stage 2; 7 were at stage 3; and 22 were at stage 4.

INTERVENTION

Transplant regimens varied. Most patients received high-dose cyclophosphamide and total body irradiation, followed by infusion of bone marrow from an HLA-identical sibling. After transplantation, immune suppression with cyclosporine or methotrexate or both was generally used to prevent graft-versus-host disease.

MEASUREMENTS

The primary outcome was survival. We also studied hematologic remission, defined as normalization of the leukocyte count, hemoglobin level, and platelet count, and absence of lymphadenopathy and splenomegaly.

RESULTS

38 patients (70%) achieved hematologic remission. Twenty-four (44%) remain alive a median of 27 months (range, 5 to 80 months) after transplantation. Three-year survival probability was 46% (95% CI, 32% to 60%). Three patients who received transplants at Rai stage 0 remain alive 21, 32, and 45 months after transplantation. Three-year survival probabilities were as follows: 68% (CI, 38% to 98%) in 10 patients who received transplants at Rai stage 1, 30% (CI, 2% to 58%) in 10 patients who received transplants at Rai stage 2, 57% (CI, 21% to 93%) in 7 patients who received transplants at Rai stage 3, and 34% (CI, 12% to 56%) in 22 patients who received transplants at Rai stage 4 CLL. Five patients (9%) died of progressive leukemia and 25 (46%) of treatment-related complications.

CONCLUSIONS

Bone marrow transplants from HLA-identical siblings can result in hematologic remission and survival in persons with CLL, but it is uncertain how these results compare with those of conventional therapies.

摘要

目的

详细描述60岁以下慢性淋巴细胞白血病(CLL)患者接受 HLA 相同同胞骨髓移植的结果。

设计

回顾性队列研究。

地点

全球30个骨髓移植中心,这些中心在1984年至1992年间向欧洲血液和骨髓移植组或国际骨髓移植登记处报告了 HLA 相同同胞骨髓移植治疗CLL的结果数据。

患者

54例诊断为CLL的患者(中位年龄41岁;范围21至58岁)。从诊断到移植的中位间隔时间为37个月(范围5至130个月)。移植时,3例患者处于 Rai 分期0期;10例处于1期;10例处于2期;7例处于3期;22例处于4期。

干预

移植方案各不相同。大多数患者接受大剂量环磷酰胺和全身照射,随后输注来自 HLA 相同同胞的骨髓。移植后,通常使用环孢素或甲氨蝶呤或两者进行免疫抑制以预防移植物抗宿主病。

测量指标

主要结局为生存情况。我们还研究了血液学缓解情况,定义为白细胞计数、血红蛋白水平和血小板计数恢复正常,且无淋巴结病和脾肿大。

结果

38例患者(70%)实现血液学缓解。24例(44%)在移植后中位生存27个月(范围5至80个月)。三年生存概率为46%(95%可信区间,32%至60%)。3例在 Rai 分期0期接受移植的患者在移植后21、32和45个月仍存活。三年生存概率如下:10例在 Rai 分期1期接受移植的患者中为68%(可信区间,38%至98%),10例在 Rai 分期2期接受移植的患者中为30%(可信区间,2%至58%),7例在 Rai 分期3期接受移植的患者中为57%(可信区间,21%至93%),22例在 Rai 分期4期CLL接受移植的患者中为34%(可信区间,12%至56%)。5例患者(9%)死于进行性白血病,25例(46%)死于治疗相关并发症。

结论

来自 HLA 相同同胞的骨髓移植可使CLL患者实现血液学缓解并生存,但这些结果与传统疗法相比如何尚不确定。

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