Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II study

Pituitary carcinoma is an aggressive tumor characterized by metastatic spread beyond the sellar region. Symptoms can be debilitating due to hormonal excess and survival is poor. Pituitary carcinomas recur despite conventional multimodality treatments. Given the recent advances in the use of immune c...

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Main Authors: Siqing Fu, Aung Naing, Filip Janku, Mingxuan Xu, Anas Alshawa, Nazanin Majd, Steven G Waguespack, Marta Penas-Prado, Carlos Kamiya-Matsuoka, Shaan M Raza, Ian E McCutcheon
Format: Article
Language:English
Published: BMJ Publishing Group 2020-10-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/8/2/e001532.full
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author Siqing Fu
Aung Naing
Filip Janku
Mingxuan Xu
Anas Alshawa
Nazanin Majd
Steven G Waguespack
Marta Penas-Prado
Carlos Kamiya-Matsuoka
Shaan M Raza
Ian E McCutcheon
author_facet Siqing Fu
Aung Naing
Filip Janku
Mingxuan Xu
Anas Alshawa
Nazanin Majd
Steven G Waguespack
Marta Penas-Prado
Carlos Kamiya-Matsuoka
Shaan M Raza
Ian E McCutcheon
author_sort Siqing Fu
collection DOAJ
description Pituitary carcinoma is an aggressive tumor characterized by metastatic spread beyond the sellar region. Symptoms can be debilitating due to hormonal excess and survival is poor. Pituitary carcinomas recur despite conventional multimodality treatments. Given the recent advances in the use of immune checkpoint inhibitors (CPIs) to treat various solid cancers, there has been interest in exploring the role of immunotherapy for treating aggressive, refractory pituitary tumors. We treated 4 patients with pituitary carcinoma with pembrolizumab as part of a phase II clinical trial. Two patients (patients 1 and 2) with functioning corticotroph pituitary carcinomas (refractory to surgery, radiotherapy and chemotherapy) had partial radiographic (60% and 32% per Immune-Related Response Evaluation Criteria In Solid Tumors, respectively) and hormonal responses. Patient 1’s response continues 42 months after initiation of pembrolizumab and his tumor tissue obtained after treatment with temozolomide demonstrated a hypermutator phenotype with MSH2 and MSH6 gene mutations. Patient 2’s tumor after exposure to temozolomide was not sampled, but prior somatic mutational testing was negative. One patient with a non-functioning corticotroph tumor (patient 3) had a best response of stable disease for 4 months. One patient with a prolactin-secreting carcinoma (patient 4) had progressive disease. The latter 2 patients’ tumors did not demonstrate a hypermutator phenotype after treatment with temozolomide. Programmed death-ligand 1 staining was negative in all tumors. We report 2 cases of corticotroph pituitary carcinoma responsive to pembrolizumab after prior exposure to alkylating agents. The role of CPIs in treating patients with pituitary carcinoma, the relationship between tumor subtype and response to immunotherapy and mechanisms of hypermutation in this orphan disease require further study.Trial registration number: NCT02721732.
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spelling doaj-art-e142b33dba1045f1b1ac27cad95671c12025-08-20T02:13:22ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262020-10-018210.1136/jitc-2020-001532Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II studySiqing Fu0Aung Naing1Filip Janku2Mingxuan Xu3Anas Alshawa4Nazanin Majd5Steven G Waguespack6Marta Penas-Prado7Carlos Kamiya-Matsuoka8Shaan M Raza9Ian E McCutcheon10Aff2 0000 0001 2291 4776grid.240145.6Department of Investigational Cancer TherapeuticsThe University of Texas MD Anderson Cancer Center 1515 Holcombe Blvd 77030 Houston TX USA14 Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA5 Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USAInvestigational Cancer Therapeutics, University of Texas MD Anderson Cancer Center, Houston, Texas, USAInvestigational Cancer Therapeutics, University of Texas MD Anderson Cancer Center, Houston, Texas, USA1 Department of Neuro-Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA2 Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA1 Department of Neuro-Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA1 Department of Neuro-Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA4 Department of Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA4 Department of Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, Texas, USAPituitary carcinoma is an aggressive tumor characterized by metastatic spread beyond the sellar region. Symptoms can be debilitating due to hormonal excess and survival is poor. Pituitary carcinomas recur despite conventional multimodality treatments. Given the recent advances in the use of immune checkpoint inhibitors (CPIs) to treat various solid cancers, there has been interest in exploring the role of immunotherapy for treating aggressive, refractory pituitary tumors. We treated 4 patients with pituitary carcinoma with pembrolizumab as part of a phase II clinical trial. Two patients (patients 1 and 2) with functioning corticotroph pituitary carcinomas (refractory to surgery, radiotherapy and chemotherapy) had partial radiographic (60% and 32% per Immune-Related Response Evaluation Criteria In Solid Tumors, respectively) and hormonal responses. Patient 1’s response continues 42 months after initiation of pembrolizumab and his tumor tissue obtained after treatment with temozolomide demonstrated a hypermutator phenotype with MSH2 and MSH6 gene mutations. Patient 2’s tumor after exposure to temozolomide was not sampled, but prior somatic mutational testing was negative. One patient with a non-functioning corticotroph tumor (patient 3) had a best response of stable disease for 4 months. One patient with a prolactin-secreting carcinoma (patient 4) had progressive disease. The latter 2 patients’ tumors did not demonstrate a hypermutator phenotype after treatment with temozolomide. Programmed death-ligand 1 staining was negative in all tumors. We report 2 cases of corticotroph pituitary carcinoma responsive to pembrolizumab after prior exposure to alkylating agents. The role of CPIs in treating patients with pituitary carcinoma, the relationship between tumor subtype and response to immunotherapy and mechanisms of hypermutation in this orphan disease require further study.Trial registration number: NCT02721732.https://jitc.bmj.com/content/8/2/e001532.full
spellingShingle Siqing Fu
Aung Naing
Filip Janku
Mingxuan Xu
Anas Alshawa
Nazanin Majd
Steven G Waguespack
Marta Penas-Prado
Carlos Kamiya-Matsuoka
Shaan M Raza
Ian E McCutcheon
Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II study
Journal for ImmunoTherapy of Cancer
title Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II study
title_full Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II study
title_fullStr Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II study
title_full_unstemmed Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II study
title_short Efficacy of pembrolizumab in patients with pituitary carcinoma: report of four cases from a phase II study
title_sort efficacy of pembrolizumab in patients with pituitary carcinoma report of four cases from a phase ii study
url https://jitc.bmj.com/content/8/2/e001532.full
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