Modification of existing patient-reported outcome measures: qualitative development of the MD Anderson Symptom Inventory for malignant pleural mesothelioma (MDASI-MPM)

Our plain-English summary of Modification of existing patient-reported outcome measures: qualitative development of the MD Anderson Symptom Inventory for malignant pleural mesothelioma (MDASI-MPM), by L.A. Williams, M.S. Whisenant, T.R. Mendoza, et al (Quality of Life Research, 2018).

The study

• Authors: L.A. Williams, M.S. Whisenant, T.R. Mendoza, S. Haq, K.N. Keating, B. Cuffel, C.S. Cleeland
• Journal: Quality of Life Research
• Published: 2018;27(12):3229-3241
• Type of article: Review
• Cited by: 14 articles, as counted by NIH iCite
• Read it: PubMed · Publisher

Abstract

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.

In plain English

This was a qualitative study at MD Anderson Cancer Center in Houston. It adapted an existing symptom questionnaire, the MD Anderson Symptom Inventory (MDASI), for malignant pleural mesothelioma (MPM). It did not study asbestos exposure. The authors reviewed published questionnaires and interviewed 20 patients with advanced MPM. They also interviewed six patients, mostly women with gynecological cancer, who had taken an unnamed investigational drug in an earlier Phase I trial. The literature review and the 20 MPM interviews identified 31 MPM-related symptoms. The drug interviews identified six drug-specific symptoms. At diagnosis or recurrence, 75% of MPM patients mentioned shortness of breath, and 40% each mentioned pain, fatigue and distress. During standard treatment, pain (55%) and fatigue (45%) were mentioned most. A panel of doctors, other providers, patients and caregivers rated how relevant the symptoms were. Five MPM-specific items were added to the 13 core MDASI symptoms. Eye problems were to be added for a planned drug trial. The authors concluded that the questionnaire had content validity and was ready for psychometric testing. They noted that most participants were male and white, and that the drug group was small.

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: Qualitative interview study, combined with a literature review and an expert-panel rating, to develop a symptom questionnaire (an MPM module of the MDASI)
• Who was studied: Two groups at The University of Texas MD Anderson Cancer Center, Houston. The first was 20 adults with advanced malignant pleural mesothelioma (18 male, 2 female, all white, mean age 68.4 years). The second was 6 patients who had received the investigational drug in a previous Phase I trial: 1 with MPM, 2 with primary peritoneal cancer and 3 with ovarian cancer; 5 were women. These 6 came from 45 drug recipients, of whom 8 were alive and still seen at the center. One MPM patient was in both groups, giving 25 interviews in total. The expert panel had 19 members: 5 physicians, 5 other health care providers, 5 patients with MPM and 4 family caregivers.
• Exposure: No asbestos exposure was studied. The study looked at symptoms attributed to MPM itself, to standard treatments (chemotherapy, radiation, surgery) and to an unnamed investigational drug.
• Main results:
  • The literature review and the interviews with 20 MPM patients identified 31 MPM-related symptoms.
  • Among the 20 MPM patients, the disease symptoms mentioned by 20% or more at diagnosis or recurrence were shortness of breath (75%), pain (40%), fatigue (40%) and distress (40%).
  • Among the 20 MPM patients, the symptoms mentioned by 20% or more as occurring or worsening during standard treatment were pain (55%), fatigue (45%), shortness of breath (35%), nausea (30%), trouble remembering (25%), disturbed sleep (20%), lack of appetite (20%) and vomiting (20%).
  • The types of interference with daily life that patients described matched the six standard MDASI interference items: general activity, mood, work, walking, relations with others and enjoyment of life.
  • Five MPM-specific items and the 13 core MDASI symptoms were selected for a provisional MDASI-MPM. The five items were coughing, feeling of malaise, trouble with balance or falling, chest heaviness or tightness, and muscle weakness.
  • Interviews with the 6 patients who had received the investigational drug identified six drug-specific symptoms, three of which were mentioned by more than one patient.
  • Of the drug-related symptoms, pain and numbness or tingling (mentioned by three or more of the 6 drug recipients) are already core MDASI items. Eye problems, mentioned by three patients, were to be added for the planned Phase II trial.
• The authors' conclusion: The authors concluded that the MDASI-MPM has established content validity and that, with one added symptom item, it is ready for psychometric testing as fit-for-purpose for a clinical trial of an investigational agent.
• Limitations the authors state:
  • Most MPM participants were male and white, despite the authors' purposive sampling.
  • The group of patients who had received the investigational drug was small. Saturation was not reached, so drug-specific symptoms may have been missed.
  • Experts did not rate the symptoms linked to the investigational drug, because few experts were available.

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: Pleural Mesothelioma, Pleura, Clinical Trial.

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