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

作者：L Haggström, Yeh Chen Lee, Maria-Pilar Barretina-Ginesta, Anna Jaeger, Linn Woelber, Hannah Woopen, Madeline Rhind, Jean-Sébastien Frenel, Aanum Aanum, Katharina Bischof, Marta Mongillo, Allison L. Brodsky, Jonathan Ledermann, Talayeh Ghezelayagh, I A Boere, Sabrina Kaiser, Constanza Maximiano, Ora Rosengarten, Tamar Safra, A. Pertejo, Sabrina Cecere, Isabelle Ray-Coquard, Stephanie Lheureux, Ros Glasspool, Marta Gil-Martin, L Tookman, Se Ik Kim, Suzana Mittelstadt, Miguel Corbellas Aparicio, Isabel Palacio, Cristina Martín-Lorente, Rebecca Kristeleit, Helena de la Cueva, Nelleke Ottevanger, María Quindós Varela, Hiroyuki Yoshida, T Link, Lydia Gaba, Gloria Marquina, Nikki Burdett, Paul Johannet, Juliane Reichenbach, Tjoung-Won Park-Simon, Philip H. Klecker, Antonio González-Martín, C Marth, Clemens Liebrich, Dzotsenidze Ll, Sabine Heublein, Lawrence Kasherman, Cristina Perez, María Masvidal, P. Guillén Sentís, Nikolaus de Gregorio, F Hemptenmacher, Felix Hilpert, Jalid Sehouli, Clare Scott, Katherine Mary Tucker, Jack Dong, Cristina Fortuño, Amanda B. Spurdle, M L Friedlander, Susan Ellard, Anna Tinker, Andrew Clamp, Iain McNeish, N J COLOMBO, Sandro Pignata, K Lindemann, Patricia Roxburgh, Jae-Weon Kim, Shannon Westin, Jonathan Berek, William Tew, Carol Aghajanian, Philipp Harter · 发表于：JAMA Oncology · 年份：2026 · DOI：10.1001/jamaoncol.2026.1924 · 研究领域：PARP inhibition in cancer therapy、Ovarian cancer diagnosis and treatment、Advanced Breast Cancer Therapies

Importance: A subset of patients with platinum-sensitive recurrent ovarian cancer (PS-ROC) treated with maintenance poly(adenosine diphosphate-ribose) polymerase (PARP) inhibitors have exceptional response. Although licensing recommends continuing PARP inhibitors until progression or unacceptable toxic effects, the optimal duration of PARP inhibitors, and the risks of late progression, myelodysplastic syndrome (MDS), or acute myeloid leukemia (AML) in patients with exceptional response are unknown. Objective: To determine the long-term outcomes of patients with PS-ROC who have exceptional response to PARP inhibitors, and to explore genotype-phenotype associations. Design, Setting, and Participants: This was an international, multicenter, retrospective cohort study of patients with exceptional response to PARP inhibitors, defined as patients with PS-ROC and progression-free survival (PFS) of 5 years or longer from PARP inhibitor commencement. The study was conducted across 41 sites in 14 countries from January 11, 2023, to November 10, 2025. Exposures: Treatment with PARP inhibitors. Main Outcomes and Measures: The primary end point was PFS, and secondary end points included overall survival, toxic effects, and dose reductions. Results: A total of 320 patients with exceptional response (mean [SD] age, 56.4 [9.4] years) were included, with a median follow-up of 6.8 years (95% CI, 6.6-7.0 years). The median (IQR) PARP inhibitor duration was 75.0 (64.0-91.0) months. Of patients w...