Long-term safety in patients with recurrent ovarian cancer treated with niraparib versus placebo: Results from the phase III ENGOT-OV16/NOVA trial

Mansoor R. Mirza, B. Benigno, A. Dørum, S. Mahner, P. Bessette, I. Bover Barceló, D. Berton-Rigaud, J. A. Ledermann, B. J. Rimel, J. Herrstedt, S. Lau, A. du Bois, A. Casado Herráez, E. Kalbacher, J. Buscema, D. Lorusso, I. Vergote, T. Levy, P. Wang, F. A. de JongD. Gupta, U. A. Matulonis

Research output: Contribution to journalArticlepeer-review


Objective: Niraparib is a poly(ADP-ribose) polymerase (PARP) inhibitor approved for use in heavily pretreated patients and as maintenance treatment in patients with newly-diagnosed or recurrent ovarian cancer following a response to platinum-based chemotherapy. We present long-term safety data for niraparib from the ENGOT-OV16/NOVA trial. Methods: This multicenter, double-blind, randomized, controlled phase III trial evaluated the efficacy and safety of niraparib for the treatment of recurrent ovarian cancer. Patients were randomly assigned 2:1 to receive either once-daily niraparib 300 mg or placebo. Two independent cohorts were enrolled based on germline BRCA mutation status. The primary endpoint was progression-free survival, reported previously. Long-term safety data were from the most recent data cutoff (September 2017). Results: Overall, 367 patients received niraparib 300 mg once daily. Dose reductions due to TEAEs were highest in month 1 (34%) and declined every month thereafter. Incidence of any-grade and grade ≥ 3 hematologic and symptomatic TEAEs was also highest in month 1 and subsequently declined. Incidence of grade ≥ 3 thrombocytopenia decreased from 28% (month 1) to 9% and 5% (months 2 and 3, respectively), with protocol-directed dose interruptions and/or reductions. Acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) were reported in 2 and 6 niraparib-treated patients, respectively, and in 1 placebo patient each. Treatment discontinuations due to TEAEs were <5% in each month and time interval measured. Conclusion: These data demonstrate the importance of appropriate dose reduction according to toxicity criteria and support the safe long-term use of niraparib for maintenance treatment in patients with recurrent ovarian cancer. Trial registration: ClinicalTrials.gov identifier: NCT01847274.

Original languageEnglish
Pages (from-to)442-448
Number of pages7
JournalGynecologic Oncology
Issue number2
Publication statusPublished - Nov 2020


  • Gynecologic oncology
  • Long-term safety
  • Niraparib
  • Ovarian cancer
  • Poly(ADP ribose) polymerase inhibitor

ASJC Scopus subject areas

  • Oncology
  • Obstetrics and Gynaecology


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