Effectiveness and Safety of Clofarabine Monotherapy or Combination Treatment in Relapsed/Refractory Childhood Acute Lymphoblastic Leukemia: A Pragmatic, Non-interventional Study in Korea
- Author:
Jung Yoon CHOI
1
;
Che Ry HONG
;
Kyung Taek HONG
;
Hyoung Jin KANG
;
Seongkoo KIM
;
Jae Wook LEE
;
Pil Sang JANG
;
Nack-Gyun CHUNG
;
Bin CHO
;
Hyery KIM
;
Kyung-Nam KOH
;
Ho Joon IM
;
Jong Jin SEO
;
Seung Min HAHN
;
Jung Woo HAN
;
Chuhl Joo LYU
;
Eu Jeen YANG
;
Young Tak LIM
;
Keon Hee YOO
;
Hong Hoe KOO
;
Hoon KOOK
;
In Sang JEON
;
Hana CHO
;
Hee Young SHIN
Author Information
- Publication Type:Original Article
- From:Cancer Research and Treatment 2021;53(4):1184-1194
- CountryRepublic of Korea
- Language:English
-
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.
