Assessing disease experience across the life span for individuals with osteogenesis imperfecta: challenges and opportunities for patient-reported outcomes (PROs) measurement: a pilot study

• DOI: 10.1186/s13023-019-1004-x
• Publication Date: 2019-01-29T07:04:07Z
• Authors: Laura L. Tosi; Marianne K. Floor; Christina M. Dollar; Austin P. Gillies; Tracy S. Hart; David D. Cuthbertson; V. Reid Sutton; Jeffrey P. Krischer
• Keywords: Disease management; Patient-reported outcome

Patient reported outcome (PRO) information is crucial for establishing better patient-provider communication, improving shared decision-making between clinicians and patients, assessing patient responses to therapeutic interventions, increasing satisfaction with care. We used the Brittle Bones Disease Consortium (BBDC) Contact Registry People OI, managed by Rare Clinical Research Network (RDCRN) (1) evaluate construct validity of Patient-Reported Outcome Measurement Information System® (PROMIS®) record important components disease experience among individuals OI; (2) explore feasibility using a registry recruit OI report on health status. Our long-term goal enhance communication management findings back community, especially those who do not have access major clinical centers. demonstrated PROMIS instruments in OI. results confirm that scores from most domains differ significantly general US population: worse symptom burden functioning. found no excessive floor or ceiling effects. study demonstrates BBDC can be participants online status surveys. However, there are numerous challenges must addressed: lack self-knowledge type, under-representation men, limited ethnic diversity, imperfect questionnaire completion rates. pilot contact respondents community obtain analyzable data regarding experience. Because population avoid effects, assess response interventions Future directions will include development validation an OI-specific patient-based classification system aggregates persons similar characteristics risks complications identify treatment needs; integrating these PRO tools into routine care research studies.