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Cancer Research and Treatment > Accepted Articles
doi: https://doi.org/10.4143/crt.2020.289    [Accepted]
Effectiveness and Safety of Clofarabine Monotherapy or Combination Treatment in Relapsed/Refractory Childhood Acute Lymphoblastic Leukemia: A Pragmatic, Non-interventional Study in Korea
Jung Yoon Choi1, Che Ry Hong1, Kyung Taek Hong1, Hyoung Jin Kang1, Seongkoo Kim2, Jae-Wook Lee2, Pil Sang Jang2, Nack-Gyun Chung2, Bin Cho2, Hyery Kim3, Kyung-Nam Koh3, Ho Joon Im3, Jong Jin Seo3, Seung Min Hahn4, Jung Woo Han4, Chuhl Joo Lyu4, Eu Jeen Yang5, Young Tak Lim5, Keon Hee Yoo6, Hong Hoe Koo6, Hoon Kook7, In Sang Jeon8, Hana Cho9, Hee Young Shin1
1Department of Pediatrics, Seoul National University Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea
2Department of Pediatrics, College of Medicine, The Catholic University of Korea, Seoul, Korea
3Division of Pediatric Hematology/Oncology, Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea
4Division of Pediatric Hematology and Oncology, Department of Pediatrics, Yonsei University College of Medicine, Yonsei University Health System, Seoul, Korea
5Department of Pediatrics, Pusan National University Children’s Hospital, Pusan National University School of Medicine, Yangsan, Korea
6Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea
7Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea
8Department of Pediatrics, Gachon University, Gil Medical Center, Incheon, Korea
9Employee of Sanofi Aventis, Korea
Correspondence  Hee Young Shin ,Tel: 82-2-2072-2971, Fax: 82-2-3675-0999, Email: hyshin@snu.ac.kr
Received: April 7, 2020;  Accepted: January 2, 2021.  Published online: January 4, 2021.
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.
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 six 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 1 (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). Sixteen patients (26.7%) proceeded to HSCT. There were 24 deaths; 14 due to treatment-emergent adverse events.
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.
Key words: Acute lymphoblastic leukemia, Clofarabine, Leukemia, Pediatric malignancy, Pediatric cancer
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