Increased Risk of Myelodysplastic Syndrome and Acute Myeloid Leukemia in Ovarian Cancer Patients Treated with PARP Inhibitors

Researchers have found that ovarian cancer patients treated with first-line maintenance PARP inhibitors have a significantly higher risk of developing myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) compared to those treated with platinum-based chemotherapy without PARP inhibitors. The study, published in The Oncologist, analyzed data from 3,402 ovarian cancer patients treated with PARP inhibitors and 1,653 patients treated with platinum-based chemotherapy. The results showed that the overall incidence of MDS or AML was 1.9% in the PARP inhibitor group and 0.1% in the control group.

Key Takeaways:

  • The study found that ovarian cancer patients treated with first-line maintenance PARP inhibitors had a 2.46 times higher risk of developing MDS or AML compared to those treated with platinum-based chemotherapy.
  • The increased risk was observed in both the platinum-sensitive relapse and first-line treatment settings.
  • No significant differences were found across the types of PARP inhibitors used.
  • The study used a cohort of 3,402 ovarian cancer patients treated with first-line maintenance PARP inhibitors and 1,653 patients treated with platinum-based chemotherapy.
  • The cohorts were propensity score matched (PSM) 1:1 for age, race, bevacizumab treatment, and genetic susceptibility to neoplasms.
  • The hazard ratio (HR) was used to compare the incidence of MDS and AML between the matched cohorts.
  • The study was conducted using the TriNetX platform, which is a network of hospitals and research institutions that share data to improve patient outcomes.
  • The Department of Medical Oncology at IRCCS Istituto Romagnolo per lo Studio dei Tumori in Meldola, Italy, led the research study.
  • Additional researchers from the Department of Medical Oncology, including Alberto Farolfi, Nicola Gentili, Sara Testoni, Francesca Rusconi, Emilio Francesco Giunta, Nicole Brighi, Giorgia Gurioli, Daniela Montanari, Gema Hernandez Ibarburu, and Ugo De Giorgi, contributed to the study.

Statistics:

  • The overall incidence of MDS or AML was 1.9% in the PARP inhibitor group.
  • The overall incidence of MDS or AML was 0.1% in the control group.
  • The hazard ratio (HR) for MDS or AML was 2.46 (95% CI 1.27-4.75, p = 0.006) in the first-line treatment setting.
  • The hazard ratio (HR) for MDS or AML was 1.76 (95% CI 0.42-7.37, p = 0.432) in the platinum-sensitive relapse setting.

Sources:

  • NewsRx. Research from Department of Medical Oncology Reveals New Findings on Acute Myeloid Leukemia (Risk of Myelodysplastic Syndrome and Acute Myeloid Leukemia related to PARP inhibitor Maintenance Line in Real-world Ovarian Cancer Patients). OBGYN & Reproduction Week. August 25, 2025; p 865.
  • Risk of Myelodysplastic Syndrome and Acute Myeloid Leukemia related to PARP inhibitor Maintenance Line in Real-world Ovarian Cancer Patients. The Oncologist, 2025.
  • Chiara Casadei, Dept. of Medical Oncology, IRCCS Istituto Romagnolo per lo Studio dei Tumori (IRST) Dino Amadori, Meldola, Italy.
  • Oxford Univ Press, Great Clarendon St, Oxford OX2 6DP, England.