Protocol for a mixed-methods modified Delphi study for the development of a core domain set to assess the health-related quality of life of patients with mycosis fungoides and Sézary syndrome in clinical trials

BMJ Open, vol. 16, pp. e103498

Abstract

INTRODUCTION: Cutaneous T cell lymphoma (CTCL) is a group of non-Hodgkin lymphomas that primarily affects the skin and can mimic inflammatory dermatoses. Unlike many skin diseases, CTCL can lead to disabling symptoms, and advanced CTCL can even be fatal. Early studies investigating health-related quality of life (HRQOL) in patients with mycosis fungoides (MF) and Sézary syndrome (SS), common subtypes of CTCL, demonstrated significant impairment across numerous domains. The aim of this current study is to develop a core domain set (CDS) to identify the essential aspects of MF/SS that influence HRQOL that should be measured in therapeutic clinical trials. In the future, this set of core concepts will be used to identify the best patient- reported outcome measure(s) (PROM) for HRQOL for MF/SS clinical research. METHODS AND ANALYSIS: Multiple strategies will be used to generate candidate concepts: systematic review of the literature, qualitative study and a survey study of healthcare providers. A Delphi consensus process including a comprehensive group of stakeholders (patients, caregivers/care partners, a multidisciplinary group of healthcare professionals, patient advocacy groups, pharmaceutical industry representatives, methodologists and government agencies) will be used to achieve consensus. Statistical corrections for multiple significance testing and false positive findings will be undertaken. ETHICS AND DISSEMINATION: The study was submitted for and received institutional review board approval at the University of Washington (IRB# STUDY00018890 and STUDY00019407). Informed consent will be obtained from all participants where necessary. We will disseminate our findings through peer-reviewed, open access publications and presentations at national/international conferences. We will provide a plain language summary in lay terms for patients and families to patient advocacy groups for distribution to their network. REGISTRATION DETAILS: The protocol is registered in the Core Outcome Measures in Effectiveness Trials (COMET) database.

Authors 13

1. Christina Asare

   University of Washington

   Affiliation as printed

   Department of Dermatology, University of Washington, Seattle, Washington, USA

2. Caroline Raymundo

   University of Washington

   Affiliation as printed

   University of Washington School of Medicine, Seattle, Washington, USA

3. Jennifer Chen

   University of Washington

   Affiliation as printed

   Department of Dermatology, University of Washington, Seattle, Washington, USA

4. Lourdes Maria Perez-Chada

   Harvard University · Mass General Brigham

   Affiliation as printed

   Department of Dermatology, Mass General Brigham, Boston, Massachusetts, USA

   Harvard Medical School, Boston, Massachusetts, USA

5. Marianne C. Tawa

   Dana-Farber Cancer Institute

   Affiliation as printed

   Dana-Farber Cancer Institute, Boston, Massachusetts, USA

6. Susan Thornton

   Cutaneous Lymphoma Foundation

   Affiliation as printed

   Cutaneous Lymphoma Foundation, Birmingham, Michigan, USA

7. Rosanne Ottevanger Aachen Department of Dermatology, Leiden University Medical Center

   Leiden University Medical Center

   Affiliation as printed

   Department of Dermatology, Leiden University Medical Center, Leiden, Netherlands

8. Julia Jane Scarisbrick

   University Hospitals Birmingham NHS Foundation Trust · University of Birmingham

   Affiliation as printed

   The University of Birmingham and Department of Dermatology, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK

9. E A Olsen

   Duke Medical Center

   Affiliation as printed

   Department of Dermatology, Duke University Medical Center, Durham, North Carolina, USA

10. Niloufer Khan

    City Of Hope National Medical Center · City of Hope

    Affiliation as printed

    Division of Lymphoma, Department of Hematology & Hematopoietic Cell Transplantation, City of Hope, Duarte, California, USA

11. Ellen J. Kim

    University of Pennsylvania

    Affiliation as printed

    Department of Dermatology, University of Pennsylvania, Philadelphia, Pennsylvania, USA

12. Michi M. Shinohara

    University of Washington

    Affiliation as printed

    Department of Dermatology, University of Washington, Seattle, Washington, USA

13. Cecilia A. Larocca corresponding

    Dana-Farber Cancer Institute · Mass General Brigham

    Affiliation as printed

    Dana-Farber Cancer Institute, Boston, Massachusetts, USA

    Department of Dermatology, Mass General Brigham, Boston, Massachusetts, USA