Background: Nivolumab, an anti–programmed cell death 1 immuno-oncology therapy, is approved as an adjuvant treatment for patients with completely resected stage III or stage IV melanoma. PRESERV MEL (Prospective and REtrospective Study of nivolumab thERapy in adjuVant MELanoma) is a real-world observational study evaluating the effectiveness and safety of adjuvant nivolumab in patients with completely resected stage III or stage IV melanoma in clinical practice in Belgium and Luxembourg. Methods: Patients were enrolled prospectively and retrospectively during a 2-year period (January 2019–January 2021), and will be followed for 5 years. The results reported here are for the second interim analysis (cutoff date 31 December 2021). The index date was the date of first administration of adjuvant nivolumab. Patients received nivolumab for up to 12 months per label. Outcomes included relapse-free survival (RFS), adverse events (AEs)/treatment-related AEs (TRAEs), and health-related quality of life (HRQoL; assessed in prospectively enrolled patients using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30), Functional Assessment of Cancer Therapy—Melanoma (FACT-M), and EQ-5D-3L instruments). HRQoL was evaluated at group level (mean change in scores from baseline based on minimally important differences) and individual patient level (percentage of patients with clinically important scores based on threshold of clinical importance). Outcomes were analyzed descriptively. Results: The study enrolled 152 patients (125 prospective, 27 retrospective) at 15 hospitals in Belgium and Luxembourg. Minimum potential follow-up at time of analysis was 11.4 months. Median age was 60 years (range 29–85), and 53% of patients were male. At 12 and 18 months, the RFS rates were 74.7% (95% confidence interval (CI): 66.9–80.9) and 68.4% (95% CI: 60.0–75.5), respectively. Median RFS was not reached. Grade 3 or 4 TRAEs were reported in 14% of patients. AEs led to treatment discontinuation in 23% of patients. Deaths occurred in 3% of patients and were not related to treatment. Questionnaire completion rates for HRQoL were high at baseline (90–94%) and at 24 months (78–81%). In the group-level analysis for HRQoL, mean changes in scores from baseline remained stable and did not exceed prespecified thresholds for minimally important differences during and after treatment, except for a clinically meaningful improvement in FACT-M surgery subscale scores. In the individual patient-level analysis for EORTC QLQ-C30 subscales, the percentages of patients who reported clinically relevant scores for fatigue and cognitive impairment increased during treatment (at 9 months) compared with baseline. After treatment cessation (at 18 months), the percentage of patients who reported clinically relevant scores for fatigue decreased. However, the percentages of patients who reported clinically relevant scores for emotional, cognitive, and social impairment increased at 18 months compared with during treatment. Most patients with emotional impairment at 9 and 18 months did not experience disease recurrence (91% and 89%, respectively). Conclusions: These results confirm the real-world effectiveness and safety of nivolumab as an adjuvant treatment for patients with completely resected stage III or stage IV melanoma. Cancer-specific, disease-specific, and generic HRQoL were maintained during and after treatment. The percentage of patients reporting emotional and cognitive impairment increased after treatment cessation, emphasizing the need for further investigation and tailored supportive care in these patients.
背景：纳武利尤单抗是一种抗程序性细胞死亡1的免疫肿瘤疗法，已被批准作为完全切除的III期或IV期黑色素瘤患者的辅助治疗。PRESERV MEL（纳武利尤单抗辅助治疗黑色素瘤的前瞻性与回顾性研究）是一项真实世界观察性研究，旨在评估比利时和卢森堡临床实践中，纳武利尤单抗辅助治疗完全切除的III期或IV期黑色素瘤患者的有效性和安全性。 方法：患者在两年期间（2019年1月至2021年1月）前瞻性和回顾性地入组，并将接受为期5年的随访。此处报告的结果为第二次中期分析（截止日期2021年12月31日）。索引日期为首次接受辅助纳武利尤单抗治疗的日期。患者根据药品说明书接受纳武利尤单抗治疗，最长可达12个月。研究结局包括无复发生存期（RFS）、不良事件（AEs）/治疗相关不良事件（TRAEs）以及与健康相关的生活质量（HRQoL；在前瞻性入组的患者中使用欧洲癌症研究与治疗组织生活质量问卷C30（EORTC QLQ-C30）、癌症治疗功能评估-黑色素瘤（FACT-M）和EQ-5D-3L量表进行评估）。HRQoL在组水平（基于最小重要差异的评分相对于基线的平均变化）和个体患者水平（基于临床重要性阈值的具有临床重要性评分的患者百分比）进行评估。结局采用描述性分析。 结果：研究在比利时和卢森堡的15家医院共入组152名患者（125名前瞻性，27名回顾性）。分析时的最短潜在随访时间为11.4个月。中位年龄为60岁（范围29-85岁），53%的患者为男性。在12个月和18个月时，RFS率分别为74.7%（95%置信区间（CI）：66.9-80.9）和68.4%（95% CI：60.0-75.5）。中位RFS尚未达到。14%的患者报告了3级或4级TRAEs。23%的患者因AEs导致治疗中止。3%的患者发生死亡，均与治疗无关。HRQoL问卷在基线（90-94%）和24个月时（78-81%）的完成率很高。在HRQoL的组水平分析中，评分相对于基线的平均变化保持稳定，并且在治疗期间和治疗后未超过预设的最小重要差异阈值，但FACT-M手术分量表评分有临床意义的改善除外。在EORTC QLQ-C30分量表的个体患者水平分析中，与基线相比，报告疲劳和认知障碍具有临床相关性评分的患者百分比在治疗期间（9个月时）有所增加。治疗停止后（18个月时），报告疲劳具有临床相关性评分的患者百分比下降。然而，与治疗期间相比，报告情绪、认知和社会功能损害具有临床相关性评分的患者百分比在18个月时有所增加。在9个月和18个月时出现情绪障碍的大多数患者并未经历疾病复发（分别为91%和89%）。 结论：这些结果证实了纳武利尤单抗作为完全切除的III期或IV期黑色素瘤患者辅助治疗在真实世界中的有效性和安全性。癌症特异性、疾病特异性和通用性HRQoL在治疗期间和治疗后均得以维持。治疗停止后报告情绪和认知障碍的患者百分比增加，强调了对这些患者进行进一步调查和个体化支持性护理的必要性。
肿瘤(癌症)患者之家