Research Papers:

Salvaged allogeneic hematopoietic stem cell transplantation for pediatric chemotherapy refractory acute leukemia

Jingbo Wang _, Lei Yuan, Haoyu Cheng, Xinhong Fei, Yumin Yin, Jiangying Gu, Song Xue, Junbao He, Fan Yang, Xiaocan Wang, Yixin Yang and Weijie Zhang

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Oncotarget. 2018; 9:3143-3159. https://doi.org/10.18632/oncotarget.22809

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Jingbo Wang1,*, Lei Yuan1,*, Haoyu Cheng1, Xinhong Fei1, Yumin Yin1, Jiangying Gu1, Song Xue1, Junbao He1, Fan Yang1, Xiaocan Wang1, Yixin Yang1 and Weijie Zhang1

1Department of Hematology, China Aerospace Central Hospital, Beijing, China

*These authors contributed equally to this work

Correspondence to:

Jingbo Wang, email: [email protected]

Keywords: hematopoietic stem cell transplantation; pediatric; acute leukemia; primary refractory; relapsed refractory

Received: September 15, 2017     Accepted: October 27, 2017     Published: December 01, 2017


There is an ongoing debate concerning the performance of salvaged allogeneic hematopoietic stem cell transplantation (allo-HSCT) in pediatric patients with acute refractory leukemia, in whom the prognosis is quite dismal. Few studies have ever been conducted on this subject. This may be partly due to missed opportunities by majority of the patients in such situations. To investigate the feasibility, evaluate the efficiency, and identify the prognostic factors of allo-HSCT in this sub-setting, the authors performed a single institution-based retrospective analysis. A total of 44 patients, of whom 28 had acute myeloid leukemia (AML), 13 had acute lymphocytic leukemia (ALL), and 3 had mixed phenotype leukemia (MPL), were enrolled in this study. With a median follow-up of 19 months, the estimated 2-year overall survival (OS) and progression free survival (PFS) were 34.3% (95% CI, 17.9–51.4%) and 33.6% (95% CI, 18.0–50.1%), respectively. The estimated 2-year incidence rates of relapse and non-relapse mortality (NRM) were 43.8% (95% CI 26.4–60.0%) and 19.6% (95% CI 9.1–32.9%), respectively. The estimated 100-day cumulative incidence of acute graft versus host disease (aGvHD) was 43.6% (95% CI 28.7–57.5%), and the 1-year cumulative incidence of chronic GvHD (cGvHD) was 45.5% (95% CI 30.5–59.3%). Compared with the previous studies, the multivariate analysis in this study additionally identified that female donors and cGvHD were associated with lower relapse and better PFS and OS. Male recipients, age younger than 10 years, a diagnosis of ALL, and the intermediate-adverse cytogenetic risk group were associated with increased relapse. On the contrary, extramedullary disease (EMD) and aGvHD were only linked to worse PFS. These data suggested that although only one-third of the patients would obtain PFS over 2 years, salvaged allo-HSCT is still the most reliable and best therapeutic strategy for refractory pediatric acute leukemia. If probable, choosing a female donor, better management of aGvHD, and induction of cGvHD promotes patient survival.

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