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硼替佐米联合门诊低毒方案治疗儿科和 AYA 患者复发/难治性急性淋巴细胞白血病:单中心墨西哥经验。

Outpatient low toxic regimen with bortezomib in relapsed/refractory acute lymphoblastic leukemia in pediatrics and AYA patients: Single-center Mexican experience.

机构信息

Hematology Service, Facultad de Medicina y Hospital Universitario "Dr. José Eleuterio González", Universidad Autónoma de Nuevo León, Monterrey, Nuevo León, México.

出版信息

Pediatr Blood Cancer. 2020 May;67(5):e28241. doi: 10.1002/pbc.28241. Epub 2020 Mar 11.

Abstract

Relapsed or refractory acute lymphoblastic leukemia represents a major challenge in low- and middle-income countries where new therapies are not easily accessible. Combinations of cost-effective drugs should be considered as a bridge for hematopoietic stem cell transplantation. We retrospectively analyzed pediatric and adolescent and young adult patients who received reinduction with a protocol based on l-asparaginase, doxorubicin, vincristine, dexamethasone, and bortezomib (BZ). Fifteen patients were included. Total complete response (CR) was achieved by nine of 15 patients (60%); five patients achieved CR with negative minimal residual disease, two achieved complete morphological response (CR), and two complete morphological response without platelet recovery. Eleven patients (73%) were not hospitalized and 10 (66%) did not require any blood component transfusions. There were no cases of serious toxicity or mortality. Nine patients (60%) underwent transplant. Five-year overall survival was 40%. This BZ-based protocol is effective and safe when administered as an outpatient regimen and feasible in a low resource setting.

摘要

复发或难治性急性淋巴细胞白血病是中低收入国家面临的重大挑战,因为这些国家难以获得新的治疗方法。在可以进行造血干细胞移植之前,应考虑使用具有成本效益的药物组合作为桥梁。我们回顾性分析了接受基于左旋门冬酰胺酶、多柔比星、长春新碱、地塞米松和硼替佐米(BZ)的方案进行再诱导的儿科和青少年及年轻成人患者。共纳入 15 例患者。15 例患者中,9 例(60%)达到完全缓解(CR);5 例患者微小残留病灶阴性达 CR,2 例达到完全形态学缓解,2 例完全形态学缓解但血小板未恢复。11 例(73%)患者未住院,10 例(66%)患者无需任何血液成分输注。无严重毒性或死亡病例。9 例(60%)患者接受了移植。5 年总生存率为 40%。该 BZ 为基础的方案作为门诊方案使用时有效且安全,在资源有限的环境中是可行的。

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