Registry study of immune-related adverse events using electronic patient-reported outcome in patients with cancer receiving immune checkpoint inhibitors: protocol for a multicentre cohort study

Our plain-English summary of Registry study of immune-related adverse events using electronic patient-reported outcome in patients with cancer receiving immune checkpoint inhibitors: protocol for a multicentre cohort study, by T. Hirata, T. Kawaguchi, K. Azuma, et al (BMJ Open, 2023).

The study

• Authors: T. Hirata, T. Kawaguchi, K. Azuma, A. Torii, H. Usui, S. Kim, T. Hayama, D. Hirate, Y. Kawahara, Y. Kumihashi, T. Chisaka, T. Wako, A. Yoshimura, T. Miyaji, T. Yamaguchi
• Journal: BMJ Open
• Published: 2023;13(11):e073724
• Cited by: 2 articles, as counted by NIH iCite
• Read it: PubMed · Publisher · Free full text

Abstract

Introduction: The use of immune checkpoint inhibitors (ICIs) is rapidly expanding in cancer treatment. ICIs have a unique safety profile, characterised by immune-related adverse events (irAEs). The safety profile of ICIs lacks patient experience and perspectives. This study primarily aims to obtain a database for descriptive research on the status of irAEs using the Patient-Reported Outcomes version of the Common Terminology Criteria (PRO-CTCAE) in patients with gastrointestinal cancer, lung cancer and malignant pleural mesothelioma treated with regimens containing ICIs.

Methods And Analysis: This is an ongoing, multicentre, observational study in Japan. Eligible patients must be at least 20 years old and have been diagnosed with lung cancer, malignant pleural mesothelioma or gastrointestinal cancer and plan to use ICIs. Participants will install the electronic PRO (ePRO) application and report adverse events via ePRO using PRO-CTCAE once weekly for up to 48 weeks. A registry will be established using background information obtained from medical records. The sample size is determined by 1 year projection without using statistical methods. Statistical analyses will include point estimates and 95% CIs for the incidence of each adverse event by cancer type and regimen at each time point.

Ethics And Dissemination: This research will be conducted per the Declaration of Helsinki, the Ethical Guidelines for Life Science and Medical Research Involving Human Subjects issued by the Ministry of Education, Culture, Sports, Science and Technology and the Ministry of Health, Labor and Welfare, and the revised Personal Information Protection Law. The study protocol was approved by the Ethics Committee (approval ID T2021-0180) of Tokyo Medical University Hospital on 15 October 2021.

Registration Details: The study began enrolling patients in December 2021. The target enrolment is 260; as of October 2022, 141 have been enrolled, and the enrolment is scheduled to end on 30 June 2023.

Trial Registration Number: UMIN000046418.

© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

In plain English

This article is a study protocol. It describes a study that was still enrolling patients and reports no results. The authors planned an observational study at nine hospitals in Japan. It enrolls adults with lung cancer, malignant pleural mesothelioma or gastrointestinal cancer who plan to receive cancer drugs called immune checkpoint inhibitors. The study tracks the immune-related side effects of these drugs. Participants use a smartphone or tablet app to report side effects once a week for up to 48 weeks, using a patient-reported questionnaire called PRO-CTCAE. Background details come from medical records. The target enrolment was 260. As of October 2022, 141 people had been enrolled. The planned analysis is to estimate how often each side effect occurs at each time point, by cancer type and drug regimen. The study does not examine asbestos exposure. The authors stated limitations. The sample size was based on patient numbers at the sites rather than statistical methods. Only pre-selected symptoms were asked about. People without a suitable device, or with severe psychiatric illness or cognitive dysfunction affecting survey completion, were excluded, and the authors said most excluded patients are likely to be older adults.

We wrote this plain-English summary from the full article with the help of AI, and a second AI model checked each sentence against the article. It is not the authors' wording; the abstract and the article are their own account.

What the study did and found

• Design: Study protocol for an ongoing, multicentre, longitudinal, observational (registry) study; no results reported
• Who was studied: Patients aged at least 20 at nine hospitals in Japan with lung cancer, malignant pleural mesothelioma or gastrointestinal cancer who plan to use immune checkpoint inhibitors. Enrolment began December 2021. The target was 260, and 141 had been enrolled as of October 2022.
• Exposure: Treatment with regimens containing immune checkpoint inhibitors (ICIs); asbestos exposure is not studied
• The authors' conclusion: No conclusions yet, since this is a protocol. The authors stated that the study may provide information on side effects for future ICI treatment in clinical practice.
• Limitations the authors state:
  • The study does not include all cancer types for which ICIs can be given
  • Sample size was based on patient numbers at the participating sites, not statistical methods, which limits generalisability
  • Only pre-selected symptom items are asked about, so other symptoms cannot be captured
  • Patients without a suitable device, or with severe psychiatric illness or cognitive dysfunction that affects completing the survey, are excluded; the authors stated that most excluded patients are likely to be older adults

Topics

The National Library of Medicine indexes this study under subjects our site covers: Lung cancer, Mesothelioma.

Medical terms in this study, explained in our Lexicon: Immune-Related Adverse Events, checkpoint inhibitors, Cohort Study, Lung Cancer, Pleural Mesothelioma.

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