Cancer Res Treat.  2021 Oct;53(4):1184-1194. 10.4143/crt.2020.289.

Effectiveness and Safety of Clofarabine Monotherapy or Combination Treatment in Relapsed/Refractory Childhood Acute Lymphoblastic Leukemia: A Pragmatic, Non-interventional Study in Korea

• Choi JY
• Hong CR
• Hong KT
• Kang HJ
• Kim S
• Lee JW
• Jang PS
• Chung NG
• Cho B
• Kim H
• Koh KN
• Im HJ
• Seo JJ
• Hahn SM
• Han JW
• Lyu CJ
• Yang EJ
• Lim YT
• Yoo KH
• Koo HH
• Kook H
• Jeon IS
• Cho H
• Shin HY

• KMID: 2521593
• DOI: http://doi.org/10.4143/crt.2020.289

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.

Keyword

Acute lymphoblastic leukemia; Clofarabine; Leukemia; Pediatric malignancy; Pediatric cancer

Figure

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  Fig. 1 Patient disposition. HSCT, hematopoietic stem cell transplantation.

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  Fig. 2 Survival status with clofarabine use. Overall survival (A) and survival by HSCT (B). CI, confidence interval; HR, hazard ratio; HSCT, hematopoietic stem cell transplantation.

Reference

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