1. Effectiveness and Safety of Clofarabine Monotherapy or Combination Treatment in Relapsed/Refractory Childhood Acute Lymphoblastic Leukemia: A Pragmatic, Non-interventional Study in Korea
- Author
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Jung Woo Han, Jae Wook Lee, Hong Hoe Koo, Hana Cho, Che Ry Hong, Hyery Kim, Kyung-Nam Koh, Pil Sang Jang, Nack-Gyun Chung, Hoon Kook, Hee Young Shin, Bin Cho, Eu Jeen Yang, In Sang Jeon, Ho Joon Im, Keon Hee Yoo, Kyung Taek Hong, Chuhl Joo Lyu, Jung Yoon Choi, Young Tak Lim, Jong Jin Seo, Hyoung Jin Kang, Seongkoo Kim, and Seung Min Hahn
- Subjects
Adult ,Male ,0301 basic medicine ,Antimetabolites, Antineoplastic ,Cancer Research ,medicine.medical_specialty ,Adolescent ,Combination therapy ,Pediatric Cancer ,medicine.medical_treatment ,Hematopoietic stem cell transplantation ,Acute lymphoblastic leukemia ,Young Adult ,03 medical and health sciences ,0302 clinical medicine ,Refractory ,Internal medicine ,Republic of Korea ,medicine ,Humans ,Clofarabine ,Prospective Studies ,Child ,Adverse effect ,Childhood Acute Lymphoblastic Leukemia ,Salvage Therapy ,Leukemia ,business.industry ,Hematopoietic Stem Cell Transplantation ,Precursor Cell Lymphoblastic Leukemia-Lymphoma ,Prognosis ,Combined Modality Therapy ,Pediatric cancer ,Pediatric malignancy ,Survival Rate ,Regimen ,030104 developmental biology ,Oncology ,Drug Resistance, Neoplasm ,Child, Preschool ,030220 oncology & carcinogenesis ,Female ,Original Article ,Neoplasm Recurrence, Local ,business ,Follow-Up Studies ,medicine.drug - Abstract
Purpose Effectiveness and safety of clofarabine (one of the treatment mainstays in pediatric patients with relapsed/refractory acute lymphoblastic leukemia [ALL]) was assessed in Korean pediatric patients with ALL to facilitate conditional coverage with evidence development.Materials and Methods In this multicenter, prospective, observational study, patients receiving clofarabine as mono/combination therapy were followed up every 4-6 weeks for 6 months or until hematopoietic stem cell transplantation (HSCT). Response rates, survival outcomes, and adverse events were assessed.Results Sixty patients (2-26 years old; 65% B-cell ALL, received prior ≥ 2 regimen, 68.3% refractory to previous regimen) were enrolled and treated with at least one dose of clofarabine; of whom 26 (43.3%) completed 6 months of follow-up after the last dose of clofarabine. Fifty-eight patients (96.7%) received clofarabine combination therapy. Overall remission rate (complete remission [CR] or CR without platelet recovery [CRp]) was 45.0% (27/60; 95% confidence interval [CI], 32.4 to 57.6) and the overall response rate (CR, CRp, or partial remission [PR]) was 46.7% (28/60; 95% CI, 34.0 to 59.3), with 11 (18.3%), 16 (26.7%), and one (1.7%) patients achieving CR, CRp, and PR, respectively. The median time to remission was 5.1 weeks (95% CI, 4.7 to 6.1). Median duration of remission was 16.6 weeks (range, 2.0 to 167.6 weeks). Sixteen patients (26.7%) proceeded to HSCT. There were 24 deaths; 14 due to treatment-emergent adverse events.Conclusion Remission with clofarabine was observed in approximately half of the study patients who had overall expected safety profile; however, there was no favorable long-term survival outcome in this study.
- Published
- 2021
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