Details

Autor(en) / Beteiligte

• Choi, Jung Yoon
• Hong, Che Ry
• Hong, Kyung Taek
• Kang, Hyoung Jin
• Kim, Seongkoo
• Lee, Jae Wook
• Jang, Pil Sang
• Chung, Nack-Gyun
• Cho, Bin
• Kim, Hyery
• Koh, Kyung-Nam
• Im, Ho Joon
• Seo, Jong Jin
• Hahn, Seung Min
• Han, Jung Woo
• Lyu, Chuhl Joo
• Yang, Eu Jeen
• Lim, Young Tak
• Yoo, Keon Hee
• Koo, Hong Hoe
• Kook, Hoon
• Jeon, In Sang
• Cho, Hana
• Shin, Hee Young

Titel

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

Ist Teil von

• Cancer Research and Treatment, 2021, 53(4), , pp.1184-1194

Ort / Verlag

Korea (South): Korean Cancer Association

Erscheinungsjahr

2021

Quelle

MEDLINE

Beschreibungen/Notizen

• 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. 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 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. 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.