Long-Term Outcomes in Patients With Recurrent Ovarian Cancer and Exceptional Response to PARP Inhibitors

Long-term outcomes and genotype-phenotype correlations in exceptional responders to PARP inhibitors in platinum-sensitive recurrent ovarian cancer

This international retrospective cohort (320 patients, 41 sites, 14 countries) characterizes exceptional responders to PARP inhibitors (progression-free survival ≥5 years) in platinum-sensitive recurrent ovarian cancer. Progression-free survival rates at 7.5 and 10 years were 88.8% and 78.7%, including in patients who discontinued treatment without progression. The risk of late myelodysplastic syndrome / acute myeloid leukemia was low (1.6%). Exceptional responders were enriched for variants in the BRCA1 RING domain and the BRCA2 DNA-binding domain, suggesting functional cure may be possible.

Synthesis written by Geno'X. For the full original abstract, please refer to the source publication.

Reassuring data for counseling long-term responders: low leukemogenic risk and the possibility of stopping treatment without immediate relapse. The enrichment in domain-specific variants (BRCA1 RING, BRCA2 DNA-binding) aligns with emerging literature linking the mutated domain to PARP response — a stratification lead to confirm prospectively. The retrospective design and lack of a comparator arm limit the scope.