The efficacy of niraparib in pediatric recurrent PFA-type ependymoma.

Objective To investigate the efficacy and safety of the targeted drug poly ADP-ribose polymerase (PARP) inhibitor niraparib combined with low-dose chemotherapy drugs cisplatin and etoposide in the treatment of pediatric recurrent PFA-type ependymoma. Methods A total of 12 pediatric patients with recurrent PFA - type ependymoma, treated in Children's Hospital of Fudan University from January 2022 to January 2024, were included in the study. All patients received a treatment regimen combining niraparib with low-dose cisplatin and etoposide chemotherapy. The objective response rate (ORR) of the tumor was statistically evaluated to assess treatment efficacy, and patient tolerability and safety indicators, including liver and kidney function, cardiac function, nausea and vomiting, fatigue, bone marrow suppression, gastrointestinal reactions, and infections, were recorded. Results Among 12 pediatric patients, 10 cases showed a tumor reduction of ≥ 30%, in which 2 cases showed a tumor reduction of 100%, while one case showed no change in tumor size, and one case showed tumor progression. The overall ORR was 10/12. Adverse reactions included 5 cases of severe vomiting, primarily occurring during chemotherapy period, and 8 cases of varying degrees of bone marrow suppression, all of which were alleviated after symptomatic treatment. Conclusions Niraparib combined with low-dose chemotherapy drugs shows good efficacy and safety in the treatment of pediatric recurrent PFA-type ependymoma. [ABSTRACT FROM AUTHOR]