Department of Medical Oncology, Chris O'Brien Lifehouse, Sydney, New South Wales, Australia.
Faculty of Medicine and Health, University of Sydney, Sydney, New South Wales, Australia.
Cancer Rep (Hoboken). 2021 Apr;4(2):e1327. doi: 10.1002/cnr2.1327. Epub 2020 Dec 13.
Sarcomas represent 10%-15% of cancers in adolescent and young adult (AYA) patients, and survival for those with metastatic disease or relapse is poor. Immunotherapy with checkpoint inhibition has improved outcomes in multiple tumor types, but data in advanced sarcomas, particularly within the AYA population, are limited.
We aim to evaluate response and toxicity for AYA patients with sarcoma treated with pembrolizumab.
We retrospectively reviewed AYA patients with advanced bone and soft tissue sarcoma who received self-funded pembrolizumab between May 2015 and January 2019. Eighteen patients were identified. One patient with Ewing sarcoma had a sustained complete response to therapy. Two patients with alveolar soft part sarcoma received a clinical benefit from pembrolizumab: one had a radiological partial response with an excellent clinical response and one patient achieved stable disease. Four patients died of disease prior to first scheduled assessment and thus were not evaluable. The remaining eleven patients had progressive disease.
The role of immunotherapy in AYA sarcoma warrants further investigation. Biomarkers of response need to be further evaluated in order to guide patient selection.
肉瘤占青少年和年轻成人（AYA）患者癌症的 10%-15%，转移性疾病或复发患者的生存率较差。检查点抑制的免疫疗法已改善了多种肿瘤类型的预后，但在晚期肉瘤中，特别是在 AYA 人群中的数据有限。
我们旨在评估接受派姆单抗治疗的肉瘤 AYA 患者的反应和毒性。
我们回顾性地分析了 2015 年 5 月至 2019 年 1 月期间接受自费派姆单抗治疗的晚期骨和软组织肉瘤 AYA 患者。共确定了 18 名患者。1 名尤文肉瘤患者对治疗有持续的完全缓解。2 名腺泡软组织肉瘤患者从派姆单抗治疗中获得了临床获益：1 名患者有影像学部分缓解和极好的临床反应，1 名患者病情稳定。4 名患者在首次预定评估前因疾病死亡，因此无法评估。其余 11 名患者疾病进展。
免疫疗法在 AYA 肉瘤中的作用需要进一步研究。需要进一步评估反应的生物标志物，以指导患者选择。
