Abstract 3566
Background
Patients with rare cancers account for 25% of cancer-related deaths but have limited treatment options. As immunotherapy has potential applicability across cancer types, we conducted an open-label phase II trial of pembrolizumab in patients with advanced rare cancers.
Methods
Eligible patients who had progressed on standard therapies in the last 6 months were enrolled in 9 tumor-specific cohorts and in a 10th cohort for other histologies. Pembrolizumab 200 mg every 3 weeks was administered for 2 years or until disease progression or intolerable toxicity. Tumor imaging was performed every 9 weeks; response was assessed by RECIST v1.1 and irRECIST. Adverse events (AEs) were graded per NCI-CTCAE v4.03.
Results
A total of 127 patients with advanced rare cancer were treated. Median age = 56 years and median number of prior therapies = 2. At data cutoff date, the non-progression rate (NPR) at 27 weeks was 28%, ORR 14% (immune-related [ir] complete response [irCR] = 1 and partial response [irPR] = 14, 11 continuing therapy at data cut-off), and clinical benefit rate (CBR; irCR + irPR + ir stable disease ≥4 months) 38%. In the squamous cell carcinoma (SCC) of the skin cohort, NPR at 27 weeks was 36%, ORR 31% (5 of 16 evaluable; irCR = 1 and irPR = 4, 4 continuing therapy), and CBR 38%. In the adrenocortical carcinoma (ACC) cohort, NPR at 27 weeks was 31%, ORR 15% (2 of 13 evaluable; irPR = 2), and CBR 54%. In the carcinoma of unknown primary (CUP) cohort, NPR at 27 weeks was 33%, ORR 23% (3 of 13 evaluable; irPR = 3, all continuing therapy), and CBR 54%. In the paraganglioma-pheochromocytoma cohort, NPR at 27 weeks was 43% and CBR 75% (6 of 8 evaluable). A higher proportion of patients with baseline PD-L1 H-score >42.5 were alive and progression free at 27 weeks (47%, vs 20% for H-score ≤ 42.5; p = 0.02). During the study, 66 (52%) patients had drug-related AEs, 12 (9%) with grade ≥3 AEs. irAEs were observed in 20% of patients. The most common AEs were fatigue (n = 25) and rash (n = 17) and irAEs were hypothyroidism (n = 14), pneumonitis (n = 4) and hyperthyroidism (n = 4). There were 6 deaths during the study, 5 due to progressive disease and 1 due to acute kidney injury secondary to sepsis.
Conclusions
Toxicity with pembrolizumab was manageable and antitumor activity was observed in patients with SCC of skin, ACC, CUP, and paraganglioma-pheochromocytoma.
Clinical trial identification
NCT02721732.
Editorial acknowledgement
Legal entity responsible for the study
Aung Naing, MD.
Funding
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.
Disclosure
All authors have declared no conflicts of interest.
Resources from the same session
3117 - A modified Edmonton Symptom Assessment Scale for assessing symptoms in one day chemotherapy clinic
Presenter: Anjuleta Kampitsi
Session: Poster Display session 3
Resources:
Abstract
6058 - Level of physical activity and nutritional status in cancer patients with fatigue: an exploratory cross-sectional study
Presenter: Patrick Jahn
Session: Poster Display session 3
Resources:
Abstract
1980 - Catheter related necrotizing fascitiis in haematological patients. Case report and implications for nursing
Presenter: Arianna Rosich Soteras
Session: Poster Display session 3
Resources:
Abstract
3984 - Everyday life with Long-term Chemotherapy Induced Peripheral Neuropathy among Patient in Adjuvant Treatment for Colorectal Cancer – a Multi Methods Study
Presenter: Marlene Jensen
Session: Poster Display session 3
Resources:
Abstract
2202 - Scalp cooler is effective in reducing chemotherapy-induced alopecia among breast cancer patients : a single institution experience
Presenter: Emilia Gianotti
Session: Poster Display session 3
Resources:
Abstract
5942 - Nursing management of fatigue in cancer patients: mixed methods study
Presenter: Angela Tolotti
Session: Poster Display session 3
Resources:
Abstract
2930 - Awareness of Nursing Students about the Warning Signs of Cancer
Presenter: Hatice Yakar
Session: Poster Display session 3
Resources:
Abstract
2978 - Assessment of quality of life in patients with cancer and diabetes 2 in Northern Greece.
Presenter: STYLIANI MICHALOPOULOU
Session: Poster Display session 3
Resources:
Abstract
3400 - Radiation dose variables related to the causes of skin toxicities in women with breast cancer: a study proposal
Presenter: EULALIA PUJOL
Session: Poster Display session 3
Resources:
Abstract
2156 - How should the symptoms be managed after breast cancer surgery? An example of mobile app
Presenter: AYDANUR AYDIN
Session: Poster Display session 3
Resources:
Abstract