TY - JOUR
T1 - Modification of existing patient-reported outcome measures
T2 - qualitative development of the MD Anderson Symptom Inventory for malignant pleural mesothelioma (MDASI-MPM)
AU - Williams, Loretta A.
AU - Whisenant, Meagan S.
AU - Mendoza, Tito R.
AU - Haq, Shireen
AU - Keating, Karen N.
AU - Cuffel, Brian
AU - Cleeland, Charles S.
N1 - Funding Information:
Jeanie F. Woodruff, BS, ELS provided editorial support. The study was sponsored by funding from Bayer HealthCare. The MD Anderson Symptom Inventory is copyrighted and licensed by The University of Texas MD Anderson Cancer Center and Charles S. Cleeland. Karen Keating and Brian Cuffel are employees of Bayer HealthCare, the sponsor of the study. The authors report no other conflicts of interest.
Publisher Copyright:
© 2018, Springer Nature Switzerland AG.
PY - 2018/12/1
Y1 - 2018/12/1
N2 - Purpose: Malignant pleural mesothelioma (MPM) is an aggressive cancer of the lung pleura. The MD Anderson Symptom Inventory (MDASI) is a patient-reported outcome (PRO) measure of symptom burden, the combined impact of disease-related and treatment-related symptoms on functioning. Validated PRO measures may require modification for use in specific study populations. We sought to modify the MDASI for patients with MPM and create a fit-for-purpose symptom-burden measure for use in a clinical trial, according to US Food and Drug Administration guidance on PRO utilization to support labeling claims. Methods: A literature review for MPM symptoms was conducted. Patients with MPM were qualitatively interviewed about experiences of disease and treatment. Descriptive analysis identified symptoms and interference with functioning to define MPM-related symptom burden. An expert panel rated the relevance of identified symptoms to patients with MPM. Patients who received the investigational drug in a previous Phase I study were interviewed for drug-specific symptoms. Results: Literature review and interviews of 20 patients identified 31 MPM-related symptoms. A conceptual model of MPM-related symptom burden was developed. After expert-panel relevance review, five MPM-specific items and the 13 core MDASI symptoms met criteria for inclusion in a provisional MDASI-MPM for psychometric testing. Interviews with six patients identified six drug-specific symptoms; three were mentioned by multiple patients. Of these three, one was not in the core MDASI. Conclusions: The MDASI-MPM has established content validity and, with the addition of one symptom item, is ready for psychometric testing as fit-for-purpose for a clinical trial of an investigational agent.
AB - Purpose: Malignant pleural mesothelioma (MPM) is an aggressive cancer of the lung pleura. The MD Anderson Symptom Inventory (MDASI) is a patient-reported outcome (PRO) measure of symptom burden, the combined impact of disease-related and treatment-related symptoms on functioning. Validated PRO measures may require modification for use in specific study populations. We sought to modify the MDASI for patients with MPM and create a fit-for-purpose symptom-burden measure for use in a clinical trial, according to US Food and Drug Administration guidance on PRO utilization to support labeling claims. Methods: A literature review for MPM symptoms was conducted. Patients with MPM were qualitatively interviewed about experiences of disease and treatment. Descriptive analysis identified symptoms and interference with functioning to define MPM-related symptom burden. An expert panel rated the relevance of identified symptoms to patients with MPM. Patients who received the investigational drug in a previous Phase I study were interviewed for drug-specific symptoms. Results: Literature review and interviews of 20 patients identified 31 MPM-related symptoms. A conceptual model of MPM-related symptom burden was developed. After expert-panel relevance review, five MPM-specific items and the 13 core MDASI symptoms met criteria for inclusion in a provisional MDASI-MPM for psychometric testing. Interviews with six patients identified six drug-specific symptoms; three were mentioned by multiple patients. Of these three, one was not in the core MDASI. Conclusions: The MDASI-MPM has established content validity and, with the addition of one symptom item, is ready for psychometric testing as fit-for-purpose for a clinical trial of an investigational agent.
KW - Content domain
KW - MD Anderson Symptom Inventory
KW - Mesothelioma
KW - Patient-reported outcomes
KW - Qualitative
KW - Symptom burden
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U2 - 10.1007/s11136-018-1982-5
DO - 10.1007/s11136-018-1982-5
M3 - Article
C2 - 30187393
AN - SCOPUS:85053425576
SN - 0962-9343
VL - 27
SP - 3229
EP - 3241
JO - Quality of Life Research
JF - Quality of Life Research
IS - 12
ER -