Liu Joyce, Matulonis Ursula A
Dana-Farber Cancer Institute, Boston, Massachusetts.
Clin Adv Hematol Oncol. 2025 Mar-Apr;23(2):100-110.
Poly(adenosine diphosphate-ribose) polymerase (PARP) inhibitors (PARPis) were first granted US Food and Drug Administration (FDA) approval for ovarian cancer. Trials have focused on high-grade serous histology, in which BRCA mutations and homologous recombination deficiency (HRD) are most common. The initial clinical trials of PARPis were performed in patients with heavily pretreated recurrent BRCA-mutated (BRCAm) ovarian cancer. Since then, concerns over possible reductions in overall survival with long-term PARPi treatment in recurrent disease have led to the withdrawal of most FDA approvals in this setting, and the use of PARPis has moved to the maintenance setting in newly diagnosed advanced ovarian cancer, in which trials have demonstrated significant progression-free survival benefits and trends for overall survival benefit with certain PARPis in patients who have BRCA mutations. Additionally, the risks of secondary acute myeloid leukemia and myelodysplastic syndrome are lower in the newly diagnosed setting than in the recurrent setting, potentially because of a predefined duration of PARPi treatment and/or less prior exposure to chemotherapy. Currently, several PARPis are FDA-approved in ovarian cancer: (1) olaparib (BRCAm), niraparib (BRCAm and BRCA wild-type [BRCAwt]), and olaparib/bevacizumab (BRCAm and BRCAwt/HRD) as maintenance therapy after platinum in newly diagnosed advanced disease; and (2) olaparib, niraparib, and rucaparib for recurrent BRCAm platinum-sensitive disease. This review discusses PARPi data in the newly diagnosed and recurrent settings, how current FDA approvals have evolved, and PARPi combination data.
聚(腺苷二磷酸 - 核糖)聚合酶(PARP)抑制剂(PARPis)首次获得美国食品药品监督管理局(FDA)批准用于卵巢癌。试验主要集中在高级别浆液性组织学类型,其中BRCA突变和同源重组缺陷(HRD)最为常见。PARPis的初始临床试验是在经过大量预处理的复发性BRCA突变(BRCAm)卵巢癌患者中进行的。从那时起,由于担心复发性疾病长期使用PARPi治疗可能会降低总生存期,导致FDA在这种情况下撤回了大多数批准,PARPis的使用已转向新诊断的晚期卵巢癌的维持治疗,在该领域的试验表明,对于有BRCA突变的患者,某些PARPis可显著延长无进展生存期,并显示出总生存期受益的趋势。此外,新诊断情况下继发性急性髓系白血病和骨髓增生异常综合征的风险低于复发情况,这可能是由于PARPi治疗的预定持续时间和/或先前较少接触化疗。目前,几种PARPis已获得FDA批准用于卵巢癌:(1)奥拉帕利(用于BRCAm)、尼拉帕利(用于BRCAm和BRCA野生型[BRCAwt])以及奥拉帕利/贝伐单抗(用于BRCAm和BRCAwt/HRD),作为新诊断晚期疾病铂类治疗后的维持治疗;(2)奥拉帕利、尼拉帕利和鲁卡帕利用于复发性BRCAm铂敏感疾病。本综述讨论了新诊断和复发情况下的PARPi数据、当前FDA批准的演变过程以及PARPi联合用药数据。