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Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning

Significant improvements in hematopoietic stem cell transplantation (HSCT) with haploidentical family donors (HFD) have confirmed its therapeutic role in severe aplastic anemia (SAA) and led to the evolution of treatment algorithms. However, the optimal conditioning regimen for HFD-HSCT remains unde...

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Autores principales: Yamei, Wu, Rongmu, Luo, Yongbin, Cao, Yingjian, Si, Xiaohong, Li, Xiaomei, Zhang, Pei, Yan, Zhenlan, Du, Haitao, Wang, Jing, Wang, Bojing, Wang, Xiaoxiong, Wu, Wanming, Da
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Impact Journals LLC 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5663557/
https://www.ncbi.nlm.nih.gov/pubmed/29137385
http://dx.doi.org/10.18632/oncotarget.19745
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author Yamei, Wu
Rongmu, Luo
Yongbin, Cao
Yingjian, Si
Xiaohong, Li
Xiaomei, Zhang
Pei, Yan
Zhenlan, Du
Haitao, Wang
Jing, Wang
Bojing, Wang
Xiaoxiong, Wu
Wanming, Da
author_facet Yamei, Wu
Rongmu, Luo
Yongbin, Cao
Yingjian, Si
Xiaohong, Li
Xiaomei, Zhang
Pei, Yan
Zhenlan, Du
Haitao, Wang
Jing, Wang
Bojing, Wang
Xiaoxiong, Wu
Wanming, Da
author_sort Yamei, Wu
collection PubMed
description Significant improvements in hematopoietic stem cell transplantation (HSCT) with haploidentical family donors (HFD) have confirmed its therapeutic role in severe aplastic anemia (SAA) and led to the evolution of treatment algorithms. However, the optimal conditioning regimen for HFD-HSCT remains undefined, especially the dosage of cyclophosphamide (Cy). A total of 77 patients with SAA from two research centers, who received HFD-HSCT with reduced-intensity fludarabine + cyclophosphamide + thymoglobulin ± busulfan conditioning regimen plus third-party cells infusion were included in this study, of which 67 pairs had 4-5 loci mismatched. We were particularly interested in whether the dosage of Cy significantly impacted graft failure (GF) and overall survival (OS). All patients showed sustained hematopoietic engraftment without any increase in severe aGVHD and transplantation-related mortality (TRM). The incidences of grade II-IV aGVHD, grade III-IV aGVHD and extensive cGVHD were 18%, 10% and 7%, respectively. The probabilities of 1-year and 5-year OS were 93.1% and 87.9%, respectively. Furthermore, patient age <15 years, MNC cells >8×10(8)/kg and donor age <45 years were associated with better survival (P=0.043, P=0.023, and P=0.037, respectively) and engraftment (P=0.019, P=0.008, and P=0.001, respectively). Our findings indicated that SAA patients lack MSD benefited the most if HFD-HSCT was performed with reduced-intensity fludarabine-based conditioning regimen. Improved outcomes with HFD-HSCT may lead to a salvaged therapy and an expanded direct role for SAA in the future.
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spelling pubmed-56635572017-11-13 Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning Yamei, Wu Rongmu, Luo Yongbin, Cao Yingjian, Si Xiaohong, Li Xiaomei, Zhang Pei, Yan Zhenlan, Du Haitao, Wang Jing, Wang Bojing, Wang Xiaoxiong, Wu Wanming, Da Oncotarget Research Paper Significant improvements in hematopoietic stem cell transplantation (HSCT) with haploidentical family donors (HFD) have confirmed its therapeutic role in severe aplastic anemia (SAA) and led to the evolution of treatment algorithms. However, the optimal conditioning regimen for HFD-HSCT remains undefined, especially the dosage of cyclophosphamide (Cy). A total of 77 patients with SAA from two research centers, who received HFD-HSCT with reduced-intensity fludarabine + cyclophosphamide + thymoglobulin ± busulfan conditioning regimen plus third-party cells infusion were included in this study, of which 67 pairs had 4-5 loci mismatched. We were particularly interested in whether the dosage of Cy significantly impacted graft failure (GF) and overall survival (OS). All patients showed sustained hematopoietic engraftment without any increase in severe aGVHD and transplantation-related mortality (TRM). The incidences of grade II-IV aGVHD, grade III-IV aGVHD and extensive cGVHD were 18%, 10% and 7%, respectively. The probabilities of 1-year and 5-year OS were 93.1% and 87.9%, respectively. Furthermore, patient age <15 years, MNC cells >8×10(8)/kg and donor age <45 years were associated with better survival (P=0.043, P=0.023, and P=0.037, respectively) and engraftment (P=0.019, P=0.008, and P=0.001, respectively). Our findings indicated that SAA patients lack MSD benefited the most if HFD-HSCT was performed with reduced-intensity fludarabine-based conditioning regimen. Improved outcomes with HFD-HSCT may lead to a salvaged therapy and an expanded direct role for SAA in the future. Impact Journals LLC 2017-07-31 /pmc/articles/PMC5663557/ /pubmed/29137385 http://dx.doi.org/10.18632/oncotarget.19745 Text en Copyright: © 2017 Yamei et al. http://creativecommons.org/licenses/by/3.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License 3.0 (http://creativecommons.org/licenses/by/3.0/) (CC BY 3.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Research Paper
Yamei, Wu
Rongmu, Luo
Yongbin, Cao
Yingjian, Si
Xiaohong, Li
Xiaomei, Zhang
Pei, Yan
Zhenlan, Du
Haitao, Wang
Jing, Wang
Bojing, Wang
Xiaoxiong, Wu
Wanming, Da
Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning
title Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning
title_full Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning
title_fullStr Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning
title_full_unstemmed Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning
title_short Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning
title_sort improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning
topic Research Paper
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5663557/
https://www.ncbi.nlm.nih.gov/pubmed/29137385
http://dx.doi.org/10.18632/oncotarget.19745
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