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Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study.
Biology of Blood and Marrow Transplantation ( IF 5.609 ) Pub Date : 2019-07-15 , DOI: 10.1016/j.bbmt.2019.07.011
Adriana Balduzzi 1 , Jean-Hugues Dalle 2 , Jacek Wachowiak 3 , Isaac Yaniv 4 , Akif Yesilipek 5 , Petr Sedlacek 6 , Marc Bierings 7 , Marianne Ifversen 8 , Sabina Sufliarska 9 , Krzysztof Kalwak 10 , Arjan Lankester 11 , Jacek Toporski 12 , Lucia Di Maio 1 , Evgenia Glogova 13 , Ulrike Poetschger 13 , Christina Peters 13
Affiliation  

Eligibility criteria for hematopoietic stem cell transplantation (HSCT) in acute lymphoblastic leukemia (ALL) vary according to disease characteristics, response to treatment, and type of available donor. As the risk profile of the patient worsens, a wider degree of HLA mismatching is considered acceptable. A total of 138 children and adolescents who underwent HSCT from HLA-identical sibling donors (MSDs) and 210 who underwent HSCT from matched donors (MDs) (median age, 9 years; 68% male) in 10 countries were enrolled in the International-BFM ALL SCT 2007 prospective study to assess the impact of donor type in HSCT for pediatric ALL. The 4-year event-free survival (65 ± 5% vs 61 ± 4%; P = .287), overall survival (72 ± 4% versus 68 ± 4%; P = .235), cumulative incidence of relapse (24 ± 4% versus 25 ± 3%; P = .658) and nonrelapse mortality (10 ± 3% versus 14 ± 3%; P = .212) were not significantly different between MSD and MD graft recipients. The risk of extensive chronic (cGVHD) was lower in MD graft recipients than in MSD graft recipients (hazard ratio [HR], .38; P = .002), and the risks of severe acute GVHD (aGVHD) and cGVHD were higher in peripheral blood stem cell graft recipients than in bone marrow graft recipients (HR, 2.06; P = .026). Compared with the absence of aGVHD, grade I-II aGVHD was associated with a lower risk of graft failure (HR, .63; P = .042) and grade III-IV aGVHD was associated with a higher risk of graft failure (HR, 1.85; P = .020) and nonleukemic death (HR, 8.76; P < .0001), despite a lower risk of relapse (HR, .32; P = .021). Compared with the absence of cGVHD, extensive cGVHD was associated with a higher risk of nonleukemic death (HR, 8.12; P < .0001). Because the outcomes of transplantation from a matched donor were not inferior to those of transplantation from an HLA-identical sibling, eligibility criteria for transplantation might be reviewed in pediatric ALL and possibly in other malignancies as well. Bone marrow should be the preferred stem cell source, and the addition of MTX should be considered in MSD graft recipients.

中文翻译:

匹配供体与HLA同胞的急性淋巴细胞白血病儿童和青少年的移植:结果可比吗?国际BFM ALL SCT 2007研究的结果。

急性淋巴细胞白血病(ALL)中造血干细胞移植(HSCT)的资格标准因疾病特征,对治疗的反应以及可用供体的类型而异。随着患者风险状况的恶化,更广泛程度的HLA失配被认为是可以接受的。在10个国家/地区中,共有138名儿童和青少年从HLA相同的同胞捐赠者(MSD)接受了HSCT,而210名从配对捐赠者(MD)接受了HSCT(中位年龄为9岁;男性为68%)。 BFM ALL SCT 2007前瞻性研究评估了供体类型在HSCT中对儿科ALL的影响。4年无事件生存率(65±5%vs 61±4%; P = .287),总生存期(72±4%vs 68±4%; P = .235),累积复发率(24 ±4%与25±3%; P =。658)和非复发死亡率(10±3%比14±3%; P = .212)在MSD和MD移植接受者之间无显着差异。MD移植接受者的广泛性慢性(cGVHD)风险低于MSD移植接受者(风险比[HR] ,. 38; P = .002),而重症急性GVHD(aGVHD)和cGVHD的风险更高。外周血干细胞移植受者比骨髓移植受者高(HR,2.06; P = .026)。与没有aGVHD的情况相比,I-II级aGVHD的移植失败风险较低(HR,.63; P = .042),而III-IV级aGVHD的移植失败的风险较高(HR,尽管复发风险较低(1.85; P = .020)和非白血病死亡(HR,8.76; P <.0001),但复发风险较低(HR,.32; P = .021)。与没有cGVHD的情况相比,广泛的cGVHD与非白血病死亡风险更高相关(HR,8.12; P <.0001)。由于匹配供体的移植结果不逊于HLA同胞的移植结果,因此可在儿科ALL以及其他恶性肿瘤中审查移植的资格标准。骨髓应该是首选的干细胞来源,MSD移植接受者应考虑添加MTX。
更新日期:2019-07-15
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