Jansen Yanina Jeanne Leona, Seremet Teofila, Neyns Bart
Department of Medical Oncology, Universitair Ziekenhuis Brussel, Vrije Universiteit Brussel, Brussels, Belgium.
Dermatology, CHUV Lausanne university hospital, Lausanne, Switzerland.
Rare Tumors. 2020 Nov 11;12:2036361320971983. doi: 10.1177/2036361320971983. eCollection 2020.
Uveal melanoma is a rare disease. Up to 50% of the patients will develop metastases for which the treatment options are limited. No randomized controlled data for the treatment of uveal melanoma patients are available. In this study we describe the clinical course of nine uveal melanoma patients included in the pembrolizumab expanded access program (EAP) in Belgium. Nine uveal melanoma patients were treated in the EAP with 2mg/kg pembrolizumab every 3 weeks. Patients received pembrolizumab as first or second-line treatment. Baseline characteristics and tumor responses were prospectively collected. During a median follow-up of 40 weeks, the estimated median PFS was 18 weeks (95% CI 0.7-35) and median OS was 46 weeks (95% CI 33-59%). Four patients had stable disease (SD) for more than 20 weeks (PFS of 21, 22, and 27 weeks respectively) and 1 patient presented with SD for 119 weeks. No objective responses according to irRC were observed. One grade 3 hepatitis occurred which was reversible with the administration of high doses oral corticosteroids. Even though treatment with pembrolizumab is well tolerated, clinical benefit is disappointing. Nevertheless long-term diseases control can be achieved in selected cases.
葡萄膜黑色素瘤是一种罕见疾病。高达50%的患者会发生转移，而针对转移的治疗选择有限。目前尚无关于葡萄膜黑色素瘤患者治疗的随机对照数据。在本研究中，我们描述了比利时帕博利珠单抗扩大准入计划（EAP）中纳入的9例葡萄膜黑色素瘤患者的临床病程。9例葡萄膜黑色素瘤患者在EAP中接受每3周2mg/kg帕博利珠单抗治疗。患者接受帕博利珠单抗作为一线或二线治疗。前瞻性收集了基线特征和肿瘤反应。在中位随访40周期间，估计中位无进展生存期（PFS）为18周（95%置信区间0.7 - 35），中位总生存期（OS）为46周（95%置信区间33 - 59%）。4例患者疾病稳定（SD）超过20周（分别为21、22和27周的PFS），1例患者疾病稳定达119周。根据irRC未观察到客观反应。发生了1例3级肝炎，通过给予高剂量口服糖皮质激素可逆转。尽管帕博利珠单抗治疗耐受性良好，但临床获益令人失望。然而，在部分病例中可实现长期疾病控制。
