Lomax A J, Beith J, Bhadri V, Boyer M, Grimison P, Horvath L G, Kao S, Tattersall M, Thomas D, McNeil C
Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.
Department of Medical Oncology, Royal Prince Alfred Hospital, Sydney, New South Wales, Australia.
Intern Med J. 2016 Dec;46(12):1392-1398. doi: 10.1111/imj.13232.
Immunotherapy agents show anti-cancer activity in several solid cancers. Efficacy in non-melanoma solid tumours for non-approved indications is unknown.
To evaluate patient and disease characteristics, rate and duration of response, and toxicity of self-funded pembrolizumab in patients with non-melanoma solid cancers.
Retrospective review describing outcomes and toxicity of self-funded pembrolizumab in patients with non-melanoma solid cancers treated at Chris O'Brien Lifehouse.
From April 2015 to December 2015, 21 patients received or were planned to receive self-funded pembrolizumab. The median age was 50 years (16-76), 28 and 10% had an Eastern Cooperative Oncology Group performance status of 2, and 3-4 respectively. Sixty-two percent received at least two to four lines of prior drug treatment. Median follow-up was 3.0 months (range, 0.4-9.6). Fourteen (67%) patients requested pembrolizumab. Pembrolizumab was clinician offered for 7 (33%) patients. Patients who requested pembrolizumab had worse outcomes. Three patients died before receiving pembrolizumab. Of the 18 patients that received at least one dose, a partial response was observed in 3 (17%). Progressive disease occurred in 83%. Four patients received only one cycle of pembrolizumab and died after a median of 27 days (range 13-43). Immune-related adverse events of any grade occurred in 33%. No grade 3-4 events were observed.
Pembrolizumab was well tolerated. Meaningful responses were observed in 17% of treated patients. Response continues after 5-6.5 months follow-up in 11% and >8 months of follow-up for the other responding patient. Financial impact to the patient can be substantial. Outcomes for 33% were poor with three patients dying prior to receiving therapy and four dying within weeks of receiving one dose. This highlights issues regarding the careful selection of patients, futility of anti-cancer therapy at the end-of-life and patients' perceived benefit of receiving this therapy.
免疫治疗药物在多种实体癌中显示出抗癌活性。其在非黑色素瘤实体瘤中用于未获批适应症的疗效尚不清楚。
评估自费使用帕博利珠单抗治疗非黑色素瘤实体癌患者的患者及疾病特征、缓解率和缓解持续时间以及毒性。
回顾性分析描述了在克里斯·奥布赖恩生命之屋接受治疗的非黑色素瘤实体癌患者自费使用帕博利珠单抗的疗效及毒性。
2015年4月至2015年12月，21例患者接受或计划接受自费帕博利珠单抗治疗。中位年龄为50岁（16 - 76岁），东部肿瘤协作组体能状态评分为2分、3 - 4分的患者分别占28%和10%。62%的患者此前至少接受过两到四线药物治疗。中位随访时间为3.0个月（范围0.4 - 9.6个月）。14例（67%）患者主动要求使用帕博利珠单抗。7例（33%）患者由临床医生建议使用帕博利珠单抗。主动要求使用帕博利珠单抗的患者预后较差。3例患者在接受帕博利珠单抗治疗前死亡。在18例至少接受一剂治疗的患者中，3例（17%）观察到部分缓解。疾病进展的患者占83%。4例患者仅接受了一个周期的帕博利珠单抗治疗，中位27天（范围13 - 43天）后死亡。33%的患者发生了任何级别的免疫相关不良事件。未观察到3 - 4级事件。
帕博利珠单抗耐受性良好。17%的接受治疗患者观察到有意义的缓解。11%的患者在随访5 - 6.5个月后仍有缓解，另一名有反应的患者随访超过8个月后仍有缓解。对患者的经济影响可能很大。33%的患者预后较差，3例患者在接受治疗前死亡，4例在接受一剂治疗后的数周内死亡。这突出了关于患者的谨慎选择、临终时抗癌治疗的无效性以及患者对接受该治疗的感知益处等问题。
