Cancer Research and Treatment

Original Articles

Pediatric cancer

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 Choi, 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; Cancer Res Treat. 2021;53(4):1184-1194. Published online January 4, 2021; DOI: https://doi.org/10.4143/crt.2020.289

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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.

Citations

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Revolutionizing cancer treatment: ROS-induced apoptosis via nanoformulated alkaloids
Swathi Putta, Santhosh Kumar Chinnaiyan, Ramadevi Korni, Venkata Radha Gadela
Journal of Drug Delivery Science and Technology.2025; 104: 106556. CrossRef
Clofarabine

Reactions Weekly.2023; 1958(1): 155. CrossRef
Patient-Level Meta-analysis of Clofarabine in Acute Lymphoblastic Leukemia
Sima Jeha, Hiroaki Goto, André Baruchel, Emmanuelle Boëlle-Le Corfec, Christine Geffriaud-Ricouard, Rob Pieters, Hee Young Shin
Advances in Therapy.2023; 40(12): 5447. CrossRef
Novel Treatments for Pediatric Relapsed or Refractory Acute B-Cell Lineage Lymphoblastic Leukemia: Precision Medicine Era
Shang Mengxuan, Zhou Fen, Jin Runming
Frontiers in Pediatrics.2022;[Epub] CrossRef

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Extra-cranial Malignant Rhabdoid Tumor in Children: A Single Institute Experience: Che Ry Hong, Hyoung Jin Kang, Hee Young Ju, Ji Won Lee, Hyery Kim, Sung-Hye Park, Il Han Kim, Kyung Duk Park, Hee Young Shin; Cancer Res Treat. 2015;47(4):889-896. Published online January 2, 2015; DOI: https://doi.org/10.4143/crt.2013.176

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Purpose
Malignant rhabdoid tumor (MRT) is a rare and highly aggressive tumor that affects young children. Due to its extreme rarity, most of the available data are based on retrospective case series. To add to the current knowledge of this disease, we reviewed the patients treated for extra-cranial MRT in our institute. Materials and Methods A retrospective medical record review was conducted on children treated for pathologically confirmed extra-cranial MRT at Seoul National University Children’s Hospital between January 2003 and May 2013. Results Eleven patients (7 boys, 4 girls) were diagnosed with extra-cranial MRT at a median age of 9 months old. INI1 staining was important in the pathological confirmation. Six patients (55%) had renal MRT and five (45%) had soft tissue MRT. Five patients (45%) had metastases at diagnosis. All patients underwent chemotherapy, eight patients (73%) underwent surgery, six patients (55%) received therapeutic radiotherapy, and four patients (36%) underwent high dose chemotherapy with autologous stem cell rescue (HDCT/ASCR) with melphalan, etoposide, and carboplatin. Five patients (45%) died of disease following progression (n=3) or relapse (n=2), however, there was no treatment related mortality. The overall survival of the cohort was 53.0% and the event-free survival was 54.5% with a median follow-up duration of 17.8 months (range, 2.3 to 112.3 months). Conclusion Extra-cranial MRT is still a highly aggressive tumor in young children. However, the improved survival of our cohort is promising and HDCT/ASCR with melphalan, etoposide, and carboplatin may be a promising treatment option.

Citations

Citations to this article as recorded by

Extracranial malignant rhabdoid tumors in children: high mortality even with the help of an aggressive clinical approach
Siqi Xie, Yuanyuan Fang, Yingying Yang, Lan Liu, Jianxi Bai, Sheng Lin, Bing Zhang, Yifan Fang
European Journal of Pediatrics.2023; 183(2): 557. CrossRef
Infantile Extracranial Rhabdoid Tumor of the Scalp
Sura Al Rawabdeh, Deifallah Alsharari, Hayat Khasawneh, Ola M. Al Waqfi, Qamar Yaser Malabeh, Hiathem Abu Alhaija, Raed Mohammad Aljubour, Hamzeh M. Alkhawaldeh, Pablo Fernandez Pe as
Case Reports in Medicine.2021; 2021: 1. CrossRef
Pharmacokinetics of high-dose carboplatin in children undergoing high-dose chemotherapy and autologous stem cell transplantation with BSA-based dosing
Che Ry Hong, Hyoung Jin Kang, Seol Ju Moon, Jaeseong Oh, Kyung Taek Hong, Jung Yoon Choi, Kyung-Sang Yu, Hee Young Shin
Bone Marrow Transplantation.2020; 55(1): 137. CrossRef
Successful Treatment of Extra‐Renal Noncerebral Rhabdoid Tumors with VIDE
Naoko Yasui, Akihiko Yoshida, Eisuke Kobayashi, Fumihiko Nakatani, Hiroshi Kawamoto
Pediatric Blood & Cancer.2016; 63(2): 352. CrossRef

14,593 View
149 Download
5 Web of Science
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