改善患者和医生报告的耐受性的真实世界评估：尼拉帕利用于复发性卵巢癌（NiQoLe）。
Improving real-world evaluation of patient- and physician-reported tolerability: niraparib for recurrent ovarian cancer (NiQoLe).
作者信息
Joly Florence, Bazan Fernando, Garbay Delphine, Ouldbey Yaelle, Follana Philippe, Champeaux-Orange Élise, Legouffe Eric, Brachet Pierre-Emmanuel, Spaeth Dominique, Combe Pierre, Hardy-Bessard Anne-Claire, Selle Frédéric, Grenier Julien, Lebreton Coriolan, Derbel Olfa, Bonnet Elise, Fournel Pierre, Fernandez Diez Yolanda, Delecroix Valérie, Emambux Sheik, Alexandre Jérôme, Grellety Thomas, Mille Dominique, Orfeuvre Hubert, Favier Catherine, Le Roux Delphine, Mouret-Reynier Marie-Ange, Quesada Stanislas, Kurtz Jean-Emmanuel
机构信息
Department of Medical Oncology, Centre François Baclesse, University Unicaen, Caen, France.
Department of Oncology, CHRU Besançon-Hôpital Jean Minjoz, Besançon, France.
出版信息
JNCI Cancer Spectr. 2025 Jan 3;9(1). doi: 10.1093/jncics/pkae114.
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, niraparib 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.
背景
在铂敏感复发性卵巢癌（PSROC）中，采用个体化起始剂量（ISD）维持使用尼拉帕利已获认可。然而，前瞻性试验或常规实践中尚未报道患者对延长维持治疗负担的看法。
方法
在真实生活多中心NiQoLe研究中，PSROC患者接受ISD维持使用尼拉帕利。主要目标是描述医生报告的在最初3个月内导致治疗调整的不良事件（AE）。次要终点包括使用专门设计的电子设备每周远程收集的患者报告结局（使用PRO-CTCAE的症状性AE、自我报告的疲劳以及使用FACT-F对日常活动/功能的影响）。
结果
139例接受治疗的患者（中位年龄 = 70岁）中，大多数（80%）开始使用尼拉帕利的剂量为每日200毫克。中位治疗持续时间为5.7（范围 = 0.2 - 21.4）个月。在最初3个月内，86例患者（62%）需要治疗调整（调整的中位时间 = 27天）。医生报告的≥3级尼拉帕利相关AE发生在34例患者（24%）中；68例患者（49%）因AE进行了治疗调整，主要是血小板减少症。患者报告的最常见AE（PRO-CTCAE）是疲劳、失眠、便秘和口干。66%的患者自我报告的AE较为严重。基线时，33%的患者报告有严重疲劳（FACT-F），在使用尼拉帕利期间通常持续存在。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，尽管采用了个体化给药，但在最初3个月内仍经常需要调整尼拉帕利剂量。医生低估了疲劳和症状性AE的负担。AE的数字自我报告是可行的，可提供以患者为中心的信息，补充医生报告的AE，并有助于在真实世界研究中更全面地了解毒性。
临床试验信息
NCT03752216。
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