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Sunderland Repository records the research produced by the University of Sunderland including practice-based research and theses.

Phase 2 study of pembrolizumab in patients with advanced rare cancers

Naing, Aung, Meric-Bernstam, Funda, Stephen, Bettzy, Karp, Daniel D, Hajjar, Joud, Rodon Ahnert, Jordi, Piha-Paul, Sarina A, Colen, Rivka R, Jimenez, Camilo, Raghav, Kanwal P, Ferrarotto, Renata, Tu, Shi-Ming, Campbell, Matthew, Wang, Linghua, Sabir, Sarjeel H, Tapia, Coya, Bernatchez, Chantale, Frumovitz, Michael, Tannir, Nizar, Ravi, Vinod, Khan, Saria, Painter, Jeane M, Abonofal, Abulrahman, Gong, Jing, Alshawa, Anas, McQuinn, Lacey M, Xu, Mingxuan, Ahmed, Sara, Subbiah, Vivek, Hong, David S, Pant, Shubham, Yap, Timothy A, Tsimberidou, Apostolia M, Dumbrava, Ecaterina E Ileana, Janku, Filip, Fu, Siqing, Simon, Richard M, Hess, Kenneth R, Varadhachary, Gauri R and Amir Habra, Mouhammed (2020) Phase 2 study of pembrolizumab in patients with advanced rare cancers. Journal for ImmunoTherapy of Cancer, 8 (1). e000347. ISSN 2051-1426

Item Type: Article


Background Patients with advanced rare cancers have poor prognosis and few treatment options. As immunotherapy is effective across multiple cancer types, we aimed to assess pembrolizumab (programmed cell death 1 (PD-1) inhibitor) in patients with advanced rare cancers.

Methods In this open-label, phase 2 trial, patients with advanced rare cancers whose tumors had progressed on standard therapies, if available, within the previous 6 months were enrolled in nine tumor-specific cohorts and a 10th cohort for other rare histologies. Pembrolizumab 200 mg was administered intravenously every 21 days. The primary endpoint was non-progression rate (NPR) at 27 weeks; secondary endpoints were safety and tolerability, objective response rate (ORR), and clinical benefit rate (CBR).

Results A total of 127 patients treated between August 15, 2016 and July 27, 2018 were included in this analysis. At the time of data cut-off, the NPR at 27 weeks was 28% (95% CI, 19% to 37%). A confirmed objective response (OR) was seen in 15 of 110 (14%) evaluable patients (complete response in one and partial response in 14). CBR, defined as the percentage of patients with an OR or stable disease ≥4 months, was 38% (n=42). Treatment was ongoing in 11 of 15 patients with OR at last follow-up. In the cohort with squamous cell carcinoma (SCC) of the skin, the NPR at 27 weeks was 36%, ORR 31%, and CBR 38%. In patients with adrenocortical carcinoma (ACC), NPR at 27 weeks was 31%, ORR 15%, and CBR 54%. In the patients with carcinoma of unknown primary (CUP), NPR at 27 weeks was 33%, ORR 23%, and CBR 54%. In the paraganglioma–pheochromocytoma cohort, NPR at 27 weeks was 43%, ORR 0%, and CBR 75%. Treatment-related adverse events (TRAEs) occurred in 66 of 127 (52%) patients, and 12 (9%) had grade ≥3 TRAEs. The most common TRAEs were fatigue (n=25) and rash (n=17). There were six deaths, all of which were unrelated to the study drug.

Conclusions The favorable toxicity profile and antitumor activity seen in patients with SCC of skin, ACC, CUP, and paraganglioma–pheochromocytoma supports further evaluation of pembrolizumab in this patient population.

Trial registration number NCT02721732

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Depositing User: Matthew Campbell


Item ID: 13129
Identification Number:
ISSN: 2051-1426
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ORCID for Matthew Campbell: ORCID iD

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Date Deposited: 05 Feb 2021 10:41
Last Modified: 05 Feb 2021 10:45


Author: Matthew Campbell ORCID iD
Author: Aung Naing
Author: Funda Meric-Bernstam
Author: Bettzy Stephen
Author: Daniel D Karp
Author: Joud Hajjar
Author: Jordi Rodon Ahnert
Author: Sarina A Piha-Paul
Author: Rivka R Colen
Author: Camilo Jimenez
Author: Kanwal P Raghav
Author: Renata Ferrarotto
Author: Shi-Ming Tu
Author: Linghua Wang
Author: Sarjeel H Sabir
Author: Coya Tapia
Author: Chantale Bernatchez
Author: Michael Frumovitz
Author: Nizar Tannir
Author: Vinod Ravi
Author: Saria Khan
Author: Jeane M Painter
Author: Abulrahman Abonofal
Author: Jing Gong
Author: Anas Alshawa
Author: Lacey M McQuinn
Author: Mingxuan Xu
Author: Sara Ahmed
Author: Vivek Subbiah
Author: David S Hong
Author: Shubham Pant
Author: Timothy A Yap
Author: Apostolia M Tsimberidou
Author: Ecaterina E Ileana Dumbrava
Author: Filip Janku
Author: Siqing Fu
Author: Richard M Simon
Author: Kenneth R Hess
Author: Gauri R Varadhachary
Author: Mouhammed Amir Habra

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Faculty of Health Sciences and Wellbeing


Sciences > Biomedical Sciences

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