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儿童和青少年费城阳性急性淋巴细胞白血病的异基因造血干细胞移植:意大利儿科血液学和肿瘤学协会(AIEOP)的回顾性多中心研究。

Allogeneic hematopoietic stem cell transplantation for Philadelphia-positive acute lymphoblastic leukemia in children and adolescents: a retrospective multicenter study of the Italian Association of Pediatric Hematology and Oncology (AIEOP).

机构信息

Oncoematologia Pediatrica e Centro Trapianti, Ospedale Infantile Regina Margherita, Piazza Polonia 94, Turin, Italy.

出版信息

Biol Blood Marrow Transplant. 2012 Jun;18(6):852-60. doi: 10.1016/j.bbmt.2011.10.015. Epub 2011 Oct 20.

Abstract

Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL) still represents a major challenge. We report the experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP) with allogeneic hematopoietic stem cell transplantation (HSCT) in children with Ph+ ALL from 1990 to 2008. Sixty-nine patients received HSCT from either a related (37, 54%) or an unrelated (32, 46%) donor. Twenty-five patients (36%) underwent transplantation before 2000 and 44 (64%) after 2000. Twenty-three patients (33%) received Imatinib mesylate treatment before HSCT and seven (10%) after HSCT. After a median follow-up of 56 months, the overall survival (OS) probability was 51% (95% confidence interval [CI], 38-63), the leukemia-free survival (LFS) was 47% (95% CI, 34-59), transplantation-related mortality (TRM) was 17% (95% CI, 10-30), and relapse incidence (RI) was 36% (95% CI, 26-50). Transplantation in first complete remission, female gender, and lower WBC count at diagnosis were associated with a better LFS in both univariate and multivariate analyses. Patients with p210 transcript had a trend for a worse prognosis compared with those who had the p190 transcript. Our series confirms the role of HSCT in the eradication of Ph+ ALL. Early HSCT is recommended once morphologic remission is obtained.

摘要

费城阳性急性淋巴细胞白血病(Ph+ ALL)仍然是一个重大挑战。我们报告了意大利儿科血液学和肿瘤学协会(AIEOP)从 1990 年到 2008 年在 Ph+ ALL 儿童中进行异基因造血干细胞移植(HSCT)的经验。69 例患者接受了来自亲缘(37 例,54%)或无关供体(32 例,46%)的 HSCT。25 例(36%)在 2000 年之前接受移植,44 例(64%)在 2000 年之后接受移植。23 例(33%)在 HSCT 前接受甲磺酸伊马替尼治疗,7 例(10%)在 HSCT 后接受治疗。中位随访 56 个月后,总生存率(OS)为 51%(95%可信区间,38-63),无白血病生存率(LFS)为 47%(95%可信区间,34-59),移植相关死亡率(TRM)为 17%(95%可信区间,10-30),复发率(RI)为 36%(95%可信区间,26-50)。在单因素和多因素分析中,首次完全缓解时进行移植、女性和诊断时白细胞计数较低与更好的 LFS 相关。与具有 p190 转录本的患者相比,具有 p210 转录本的患者预后较差。我们的系列研究证实了 HSCT 在消除 Ph+ ALL 中的作用。一旦获得形态缓解,建议尽早进行 HSCT。

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