This international, multicenter, retrospective cohort study (41 sites, 14 countries) evaluated long-term outcomes — including PFS, overall survival, toxic effects, and MDS/AML risk — in 320 patients with platinum-sensitive recurrent ovarian cancer (PS-ROC) who had an exceptional response (PFS ≥5 years) to maintenance PARP inhibitors, with a median follow-up of 6.8 years.
At 10 years, PFS was 78.7% overall; patients who stopped PARP inhibitors without progression (n=85) had a 10-year PFS of 90.1% vs 72.5% for those who continued. Late-onset MDS/AML occurred in only 5 patients (1.6%). Exceptional responders were enriched for BRCA1 RING domain and BRCA2 DNA-binding domain variants.
- Retrospective design introduces selection bias; patients who discontinued may differ systematically from those who continued. - Full text was inaccessible, so granular subgroup data, OS figures, and dose-reduction details could not be verified beyond the abstract. - Genotype-phenotype associations are exploratory and require prospective validation.
For ovarian cancer patients with ≥5 years of disease control on a PARP inhibitor, discontinuation does not appear to compromise long-term outcomes and may be a reasonable option to discuss — particularly given the low but real MDS/AML risk (1.6%) with prolonged use. BRCA variant location (RING or DNA-binding domains) may help identify patients most likely to achieve functional cure.
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