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考来拉滨、依托泊苷和环磷酰胺联合治疗作为难治性急性白血病移植桥接的疗效有限:一项监测前瞻性研究的结果。

The combination of clofarabine, etoposide, and cyclophosphamide shows limited efficacy as a bridge to transplant for children with refractory acute leukemia: results of a monitored prospective study.

机构信息

Department of Pediatrics, Section of Pediatric Oncology, Hematology, Immunology and Nephrology, Skåne University Hospital, Lund, Sweden.

Division of Hematology and Transfusion Medicine, Department of Laboratory Medicine, University and Regional Laboratories, Lund, Sweden.

出版信息

Pediatr Hematol Oncol. 2021 Apr;38(3):216-226. doi: 10.1080/08880018.2020.1838012. Epub 2020 Nov 5.

Abstract

Clofarabine has been shown to effectively induce remission in children with refractory leukemia. We conducted a prospective trial (clinicval.trials.gov NCT01025778) to explore the use of clofarabine-based chemotherapy as a bridge-to-transplant approach. Children with refractory acute leukemia were enrolled to receive two induction courses of clofarabine, etoposide, and cyclophosphamide (CloEC). Responding patients were scheduled for T-cell depleted haploidentical hematopoietic stem cell transplantation (HSCT). The primary objective was to improve survival by achieving sufficient disease control to enable stem cell transplantation. Secondary objectives were to evaluate safety and toxicity. Seven children with active disease entered the study. Two children responded to induction courses and underwent transplantation. Five children did not respond to induction: one died in progression after the first course; two received off-protocol chemotherapy and were transplanted; and two succumbed to progressive leukemia. All transplanted children engrafted and no acute skin graft-versus-host disease > grade I was observed. One child is alive and well 7.5 years after the first CloEC course. One child developed fulminant adenovirus hepatitis and died in continuous complete remission 7 months after start of induction. Two children relapsed and died 6.5 and 7.5 months after enrollment. Infection was the most common toxicity. CloEC can induce responses in some patients with refractory acute leukemia but is highly immunosuppressive, resulting in substantial risk of life-threatening infections. In our study, haploidentical HSCT was feasible with sustained engraftment. No clinically significant organ toxicity was observed. Also, repeating CloEC probably does not increase the chance of achieving remission.

摘要

氯法拉滨已被证明能有效诱导难治性白血病患儿缓解。我们进行了一项前瞻性试验(clinicval.trials.gov NCT01025778),以探索使用基于氯法拉滨的化疗作为桥接移植的方法。招募难治性急性白血病患儿接受两个氯法拉滨、依托泊苷和环磷酰胺(CloEC)诱导疗程。有反应的患儿计划进行 T 细胞耗竭的半相合造血干细胞移植(HSCT)。主要目标是通过充分控制疾病以实现干细胞移植来提高生存率。次要目标是评估安全性和毒性。7 名患有活动性疾病的儿童入组该研究。2 名儿童对诱导疗程有反应并接受了移植。5 名儿童对诱导无反应:1 名在第一个疗程后进展死亡;2 名接受了方案外化疗并进行了移植;2 名死于进行性白血病。所有接受移植的儿童均植入,未观察到急性皮肤移植物抗宿主病>1 级。1 名儿童在接受第一个 CloEC 疗程 7.5 年后仍存活且情况良好。1 名儿童发生暴发性腺病毒肝炎,并在诱导开始后 7 个月死于持续完全缓解。2 名儿童复发并在入组后 6.5 和 7.5 个月死亡。感染是最常见的毒性。CloEC 可诱导部分难治性急性白血病患儿产生缓解,但具有高度免疫抑制性,导致危及生命的感染风险很大。在我们的研究中,半相合 HSCT 是可行的,且有持续植入。未观察到有临床意义的器官毒性。此外,重复 CloEC 可能不会增加获得缓解的机会。

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