INCORPORATING PATIENT INPUT IN SELECTING PATIENT REPORTED OUTCOMES INSTRUMENTS FOR CLINICAL STUDIES IN MULTIPLE MYELOMA

Author(s)

Fleming S1, Eremenco S2, Gleeson S3, Brooks A3, Chiou C4
1Janssen, Raritan, NJ, USA, 2Critical Path Institute, Tucson, AZ, USA, 3Evidera, Bethesda, MD, USA, 4Janssen, Singapore, Singapore

OBJECTIVES:  To conduct a literature review and concept elicitation interviews generating supportive evidence for multiple myeloma (MM) clinical trial PRO selection incorporating direct patient disease experience. METHODS:  A provisional conceptual model was developed from a literature review (2005‑2015) of studies which included signs, symptoms, and functional impacts of MM. Concept mapping was against EORTC QLQ-C30, EORTC QLQ-MY20, FACT-MM, FACT-Anemia, FACIT Fatigue Scale, and BFI. A qualitative, cross-sectional study confirmed key symptoms and impacts from the literature review. Concept elicitation telephone interviews were conducted with 10 US patients (4 sites) using a semi-structured discussion guide. Thematic content analysis on interview transcripts was performed using qualitative software (ATLAS.ti). Results were incorporated into the concept mapping exercise and the conceptual model was revised. RESULTS:  In 44 articles, 21 symptoms/signs and 23 functional impacts were identified. 13/44 studies used EORTC QLQ-C30 for symptom and functional impacts. Interview sample included newly diagnosed (5), relapsed refractory (RR; 4), and maintenance (1) non-Hispanic White patients (mean 65 years; range 52-78). Patients endorsed all 23 impacts and 22 symptoms, 16 were in the literature and 6 were new. 90% endorsed fatigue, pain; 70% endorsed bone pain, back pain, pain elsewhere, hematologic abnormalities. EORTC QLQ-C30 covered 10 symptoms/17 impacts; EORTC QLQ-MY20 an additional 7 symptoms/4 impacts. FACT-MM included comparable symptoms, and 19 impacts but no activities of daily living (ADLs or IADLs) unlike EORTC-QLQ-C30. FACT-An and FACIT Fatigue covered anemia and fatigue-related symptoms and impacts more than other instruments including BFI. CONCLUSIONS:  EORTC QLQ-C30 generally supported the conceptual model with enhanced coverage with MY20. Endorsement of fatigue by newly diagnosed and RRMM patients indicates that this is not treatment-related alone. This population could benefit from FACT-An or FACIT Fatigue Scale in future trials to provide better coverage. Quantitative research will further enable PRO selection to fit the purpose of clinical studies.

Conference/Value in Health Info

2017-05, ISPOR 2017, Boston, MA, USA

Value in Health, Vol. 20, No. 5 (May 2017)

Code

PSY94

Topic

Patient-Centered Research

Topic Subcategory

Patient-reported Outcomes & Quality of Life Outcomes

Disease

Systemic Disorders/Conditions

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