Constructing a conceptual framework of patient-reported outcomes for metastatic hormone-refractory prostate cancer.
OBJECTIVE
A conceptual framework for patient-reported outcomes (PROs) is a structured representation of outcome concepts and issues. Our aim was to develop a conceptual framework of PROs for hormone-refractory prostate cancer (HRPC) to support measurement clarity.


METHODS
Relevant outcome issues were identified from review of recent clinical trials. This provided content for an interview with 15 metastatic HRPC patients and a survey of 10 practitioners. All participants were asked about the relevance and importance of 26 outcomes and were allowed to nominate new outcomes. Practitioners were also asked to determine which outcomes endorsed by patients were attributable to the disease (symptoms) versus treatment (side effects). Analyses of archived clinical trial data were used to verify and augment the interview and survey results.


RESULTS
Patients endorsed 11 concerns as relevant and important to HRPC including general pain, bone pain, urinary problems, fatigue, appetite loss, constipation, erectile dysfunction, peripheral neuropathy, diarrhea, PSA anxiety, and changes in self image. Practitioner judgments helped classify each concern into one of four categories, disease symptom, treatment side effect, both symptom and side effect, or psychological concern. Additionally, patients endorsed (and practitioners confirmed) the relevance and importance of several general domains of quality of life. Analyses of archived data confirmed the importance of these issues and suggested two additional concerns.


CONCLUSIONS
Findings were used to propose a conceptual framework of PROs for metastatic HRPC. Such frameworks can be used to help specify targets for assessment in clinical studies such as treatment trials.
Department of Health Sciences Research, Division of Health Care Policy & Research, Mayo Clinic, Rochester, MN, USA. eton.david@mayo.edu
David T
DT
Eton
Daniel H
DH
Shevrin
Jennifer
J
Beaumont
David
D
Victorson
David
D
Cella
D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D018931:Antineoplastic Agents, Hormonal / Q000627:therapeutic use*; D001007:Anxiety; D019540:Area Under Curve; D016001:Confidence Intervals; D003625:Data Collection; D019008:Drug Resistance, Neoplasm / Q000187:drug effects*; D006305:Health Status Indicators; D006801:Humans; D007407:Interviews as Topic; D008297:Male; D008875:Middle Aged; D008942:Mitoxantrone / Q000627:therapeutic use*; D009362:Neoplasm Metastasis; D017063:Outcome Assessment, Health Care / Q000379:methods; D010147:Pain Measurement; D011241:Prednisone / Q000627:therapeutic use*; D017430:Prostate-Specific Antigen; D011471:Prostatic Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000523:psychology; D011594:Psychometrics; D011788:Quality of Life / Q000523:psychology; D012649:Self Concept
D000970:Antineoplastic Agents; D018931:Antineoplastic Agents, Hormonal; D008942:Mitoxantrone; D017430:Prostate-Specific Antigen; D011241:Prednisone