Improving real-world evaluation of patient- and physician-reported tolerability: niraparib for recurrent ovarian cancer (NiQoLe).

IF 3.4 Q2 ONCOLOGY JNCI Cancer Spectrum Pub Date : 2024-12-14 DOI:10.1093/jncics/pkae114

Florence Joly, Fernando Bazan, Delphine Garbay Decoopman, Yaelle Ouldbey, Philippe Follana, Élise Champeaux-Orange, Eric Legouffe, Pierre-Emmanuel Brachet, Dominique Spaeth, Pierre Combe, Anne-Claire Hardy-Bessard, Frédéric Selle, Julien Grenier, Coriolan Lebreton, Olfa Derbel, Elise Bonnet, Pierre Fournel, Yolanda Fernandez Diez, Valérie Delecroix, Sheik Emambux, Jérôme Alexandre, Thomas Grellety, Dominique Mille, Hubert Orfeuvre, Catherine Favier, Delphine Le Roux, Marie-Ange Mouret-Reynier, Stanislas Quesada, Jean-Emmanuel Kurtz

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Abstract

Background: Maintenance niraparib at an individualized starting dose (ISD) is established in platinum-sensitive recurrent ovarian cancer (PSROC). However, patients' perspectives on the burden of prolonged maintenance therapy have not been reported in prospective trials or routine practice.

Methods: In the real-life multicenter NiQoLe study, patients with PSROC received ISD maintenance niraparib. The primary objective was to describe physician-reported adverse events (AEs) leading to treatment modification during the first 3 months. Secondary endpoints included patient-reported outcomes (symptomatic AEs using PRO-CTCAE, self-reported fatigue and impact on daily activities/function using FACT-F) collected remotely weekly using a specifically designed electronic device.

Results: Most (80%) of 139 treated patients (median age 70 years) began niraparib at 200 mg/day. Median treatment duration was 5.7 (range 0.2-21.4) months. During the first 3 months, 86 patients (62%) required treatment modification (median 27 days to modification). Physician-reported grade ≥3 niraparib-related AEs occurred in 34 patients (24%); 68 patients (49%) had treatment modification for AEs, predominantly thrombocytopenia. The most frequent patient-reported AEs (PRO-CTCAE) were fatigue, insomnia, constipation, and dry mouth. Self-reported AEs were severe in 66% of patients. At baseline, 33% of patients reported severe fatigue (FACT-F), which generally persisted during niraparib. Physicians systematically underestimated major patient-reported symptoms.

Conclusions: In routine practice, dose modification was often required during the first 3 months despite individualized dosing. Physicians underestimated the burden of fatigue and symptomatic AEs. Digital self-reporting of AEs is feasible, provides patient-centered information complementing physician-reported AEs, and allows fuller appreciation of toxicity in real-world studies.

Clinical trial information: NCT03752216.

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改进患者和医生报告的耐受性真实世界评估：尼拉帕利治疗复发性卵巢癌 (NiQoLe)。

背景：在铂敏感的复发性卵巢癌（PSROC）中，个体化起始剂量（ISD）维持尼拉帕尼是建立的。然而，患者对长期维持治疗负担的看法尚未在前瞻性试验或常规实践中报道。方法：在现实生活中的多中心NiQoLe研究中，PSROC患者接受ISD维持性尼拉帕尼。主要目的是描述医生报告的导致前3个月治疗调整的不良事件（ae）。次要终点包括患者报告的结果（使用PRO-CTCAE的症状性ae，使用FACT-F的自我报告疲劳和对日常活动/功能的影响），每周使用专门设计的电子设备远程收集。结果：139名接受治疗的患者（中位年龄70岁）中，大多数（80%）开始使用200 mg/天的尼拉帕尼。中位治疗时间为5.7个月（0.2-21.4个月）。在前3个月，86名患者（62%）需要修改治疗方案（中位数为27天）。34例患者（24%）发生医生报告的≥3级尼拉帕尼相关不良事件；68例（49%）患者因不良反应而改变治疗方案，主要是血小板减少症。患者报告的ae （PRO-CTCAE）中最常见的是疲劳、失眠、便秘和口干。66%的患者自我报告的不良事件严重。在基线时，33%的患者报告严重疲劳（FACT-F），通常在尼拉帕尼期间持续存在。医生系统性地低估了患者报告的主要症状。结论：在常规实践中，尽管个体化给药，但前3个月往往需要调整剂量。医生低估了疲劳和症状性ae的负担。数字化的不良反应自我报告是可行的，提供了以患者为中心的信息，补充了医生报告的不良反应，并允许在现实世界的研究中更全面地评估毒性。临床试验信息：NCT03752216。

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