The Efficacy and Safety of Apatinib in Refractory/Relapse Advanced Pediatric Solid Tumor: A Retrospective Study
Received 21 April 2020
Accepted for publication 2 July 2020
Published 22 July 2020 Volume 2020:12 Pages 6177—6185
Checked for plagiarism Yes
Review by Single-blind
Peer reviewer comments 2
Editor who approved publication: Professor Bilikere Dwarakanath
Feifei Sun,1,2,* Suying Lu,1,2,* Zijun Zhen,1,2 Jia Zhu,1,2 Juan Wang,1,2 Junting Huang,1,2 Yu Zhang,1,3 Hui Li,1,4 Ruiqing Cai,1,2 Meiling Liu,1,2 Liuhong Wu,1,2 Xiaofei Sun,1,2 Yizhuo Zhang1,2
1State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-Sen University Cancer Center, Guangzhou, People’s Republic of China; 2Department of Pediatric Oncology, Sun Yat-Sen University Cancer Center, Guangzhou, People’s Republic of China; 3Department of Pathology, Sun Yat-Sen University Cancer Center, Guangzhou, People’s Republic of China; 4Department of Imaging, Sun Yat-Sen University Cancer Center, Guangzhou, People’s Republic of China
*These authors contributed equally to this work
Correspondence: Xiaofei Sun; Yizhuo Zhang Email email@example.com; firstname.lastname@example.org
Background: The prognosis of recurrent or refractory advanced childhood solid tumor patients is very poor and new therapeutic strategies are in urgent need. This study aimed to determine the efficacy and safety of apatinib in pediatric refractory/relapse advanced solid tumor patients.
Patients and Methods: The study retrospectively reviewed recurrent or refractory advanced pediatric solid tumor patients who were treated with apatinib, an oral small-molecule tyrosine kinase inhibitor (TKI) that targets vascular endothelial growth factor receptor-2 (VEGFR2), at the Sun Yat-sen University Cancer Center (China) from January 2016 to March 2019.
Results: Fifty-six patients were included in the safety evaluation and 49 patients were included in the efficacy evaluation. The objective responses rate (ORR) was 26.5% (95% CI 15– 41): 0 CR (complete response) and 13 PR (partial response). Disease control rate (DCR) (CR+PR+SD) was 79.6% (95% CI 65– 90). The median progression-free survival (PFS) was 4.0 months (95% CI 2.6– 5.4). There was no significant difference for ORR or PFS between the A (apatinib monotherapy), A+MT (apatinib combined with oral metronomic therapy) and A+SC (apatinib combined with salvage combination chemotherapy) group (p> 0.05). The most common grade 3 or 4 adverse events were neutropenia (9[16.1%]), thrombocytopenia (8[14.3%]), hand-foot syndrome (3[5.4%]), hypertension (3[5.4%]), anaemia (3[5.4%]) and mucositis (2[3.6%]). Hypertension was the most serious adverse event and one death that occurred was considered as drug-related.
Conclusion: Apatinib showed promising clinical activity in heavily treated recurrent or refractory advanced childhood solid tumor patients. However, it is necessary to pay special attention to monitoring blood pressure when using apatinib in children. Prospective randomized controlled clinical trial is warranted.
Keywords: apatinib, angiogenesis, cancer, pediatric, vascular endothelial growth factor receptor
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