Stem-Cell Transplantation in Children With Acute Lymphoblastic Leukemia: A Prospective International Multicenter Trial Comparing Sibling Donors With Matched Unrelated Donors—The ALL-SCT-BFM-2003 Trial

Abstract

Outcome among high-risk pediatric patients with ALL after hematopoietic stem-cell transplantation was not affected by donor type. Standardized myeloablative conditioning produced a low incidence of treatment-related mortality and effective control of leukemia.

Improved outcome of children transplanted for high-risk leukemia by using a new strategy of cyclosporine-based GVHD prophylaxis

“…DFS usually reported in children transplanted for high-risk leukemia from sibling or unrelated donors vary between 50 and 70%. 25,[27][28][29][30][31][32][33]40 Weiss et al 37 report a similar DFS (84%) using CsA alone as GVHD prophylaxis but in patients transplanted only from MSDs. 37 They also used CsA while targeting a lower and narrower TBC range (80-130 ng/mL) compared with those usually recommended (100-200 ng/mL).…”

“…The incidence of severe aGvHD reported in most studies on transplanted children for leukemia is generally much higher (8-19%), although GvHD prophylaxis regimens also include short courses of MTX or MMF. [27][28][29][30][31][32][33] However, observed values of CsA TBC are almost never reported, making difficult any comparison of CsA monitoring. The incidence of chronic GvHD reported in previous similar cohorts varies between 14 and 46%, 31,34 with a majority between 20 and 30%.…”

“…The incidence of chronic GvHD reported in previous similar cohorts varies between 14 and 46%, 31,34 with a majority between 20 and 30%. 30,32,[33][34][35][36][37] The incidence of chronic GvHD we report is lower (8.2%). The incidence of GvHD was not different between matched siblings or unrelated HSCT in our cohort, despite targeting similar CsA TBC.…”

Risk assessment of relapse by lineage-specific monitoring of chimerism in children undergoing allogeneic stem cell transplantation for acute lymphoblastic leukemia

“…The present study was an ancillary research project of the international multicenter ALL-SCT-BFM 2003 trial, 14 and was per- [15][16][17] and included children in first, second or subsequent complete morphological remission of ALL. Of 41 documented relapses in the entire cohort of patients studied, 28 occurred only in the bone marrow, eight were extramedullary, and five combined.…”

Selection of donor of allogeneic hematopoietic stem cell transplantation

“…Внедрение методов молеку-лярно-биологического HLA-типирования доноров, позволяющее определить их совместимость с реципи-ентом до уровня не только генного, но и аллельного соответствия, практически нивелировало различия в отдаленных результатах алло-ТГСК от родственного и неродственного донора. Так, при совпадении пары донор-реципиент 9-10/10 HLA-аллелей (локусы HLA-A, HLA-B, HLA-C, HLA-DRB1, HLA-DQB1), установленном методом молекулярно-биологического HLA-типирования с высоким разрешением доноров, ОВ детей с ОЛЛ во 2-й и 3-й ремиссиях составила 78 и 67 % соответственно [2]. При этом показано, что ОВ пациентов после алло-ТГСК от полностью совмести-мых (10/10) неродственных доноров, активированных в национальных регистрах, достоверно превышает ОВ после трансплантации от неродственных аллогенных до-норов из международных регистров (р = 0,047) (рис.…”