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九例接受氟达拉滨-白消安-环磷酰胺预处理的儿童幼年粒单细胞白血病患者的脐带血移植的临床结果。

Clinical outcome of cord blood transplantation for nine children with juvenile myelomonocytic leukemia receiving fludarabine-busulfan-cyclophosphamide-based conditioning.

机构信息

Division of Life Sciences and Medicine, Department of Geriatrics, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China.

Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China.

出版信息

Pediatr Transplant. 2022 Mar;26(2):e14181. doi: 10.1111/petr.14181. Epub 2021 Nov 8.

Abstract

BACKGROUND

Juvenile myelomonocytic leukemia (JMML) is a rare hematological malignancy in young children and can only be cured through the allogeneic stem cell transplantation.

PROCEDURE

We have retrospectively analyzed the outcomes of nine children with JMML after unrelated cord blood transplantation (UCBT).

RESULTS

Eight patients who have received a myeloablative conditioning regimen of fludarabine (FLU), busulfan (BU), and cyclophosphamide (CY) have gotten engraftment. None of the nine patients has relapsed following initial UCBT. Six patients are still alive and in complete remission after UCBT with a median observation time of 43 months (range: 10-80 months).

CONCLUSIONS

This study shows that UCBT with FLU-BU-CY conditioning regimen can represent a suitable option for children with JMML.

摘要

背景

幼年髓单核细胞白血病(JMML)是一种罕见的儿童血液系统恶性肿瘤,只能通过异基因造血干细胞移植治愈。

方法

我们回顾性分析了 9 例 JMML 患儿接受非亲缘脐带血移植(UCBT)后的结果。

结果

8 例接受氟达拉滨(FLU)、白消安(BU)和环磷酰胺(CY)预处理的患者均获得了植入。9 例患者在初始 UCBT 后均无复发。6 例患者在接受 UCBT 后仍存活并处于完全缓解状态,中位观察时间为 43 个月(范围:10-80 个月)。

结论

本研究表明,FLU-BU-CY 预处理的 UCBT 可为 JMML 患儿提供一种合适的选择。

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