COSMIN database

Challenges of patient-reported outcome assessment in hemophilia care-a state of the art review

Bullinger, M., Globe, D., Wasserman, J., Young, N. L., and von Mackensen, S.
INTRODUCTION: One of the recent advances in assessing outcomes of medical care is the inclusion of the patient perspective. The term patient-reported outcome (PRO) is used to reflect the patient perceptions of disease and its consequences as well as of treatment and health-care provision. The development of PRO measures has advanced rapidly, and implementation in clinical research and practice is now underway. From an evaluation perspective, recommendations for the choice of PRO measures and an appraisal of the potential benefits of PRO data collection within specific health conditions are needed.

METHODS: Hemophilia is a rare and clinically well-defined health condition with established and cost-intensive treatment strategies, in which PRO assessment is increasingly recognized as important. For this reason, measurement of PROs in hemophilia focusing on health-related quality of life (HRQoL) and patient preferences were reviewed to identify appropriate measures, to make recommendations for their choice, and to critically examine their impact in international hemophilia research and practice.

RESULTS: Using literature searches and expert discussion strategies, generic and targeted measures for HRQoL and patient preferences in adults and children with hemophilia were screened, and 20 were reviewed on the basis of their psychometric properties and international availability. Only a few of the 20 measures have been used in clinical settings or research related to persons with hemophilia.

CONCLUSION: Consequently, an increased use of these measures is recommended to understand patient views on disease and treatment and to judge the impact of PROs for improvements in health care.
Value Health
1524-4733 (Electronic)
Publication year:
Symptom status:
Cognitive/mental state
Emotional state
Physical state
Functional status:
Physical functioning
Role functioning
Social functioning
General health perceptions / HRQoL:
Health-related quality of life
Adults (18-65)
Children (0-18)
Diseases of and symptoms related to the blood and blood-forming organs and certain disorders involving the immune mechanism
Diseases of and symptoms related to the musculoskeletal system and connective tissue
PRO / non-PRO:
Patient Reported Outcome
Type of measurement instrument:
1 - Questionnaires
AIMS2 - Arthritis Impact Measurement Scales 2   
CHO-KLAT - Canadian Haemophilia Outcomes–Kids Life Assessment Tool
CHQ - Child Health Questionnaire
EQ-5D - EuroQoL-5 Dimensions   
HAEMO-QOL - Hemophilia Quality of Life Questionnaire
HHS - Hemophilia Health States
HUI-2 - Health Utilities Index Mark 2
HUI-3 - Health Utilities Index-Mark 3
KIDSCREEN-10 Index Score - 10-item Screening for, and Promotion of, Health-Related Quality of Life in Children and Adolescents
KIDSCREEN-27 items - Health Related Quality of Life Questionnaire for Children and Young People and their Parents-27 items
KIDSCREEN - 52 items - Health Related Quality of Life Questionnaire for Children and Young People and their Parents-52 items
NHP - Nottingham Health Profile   
PedsQL 4.0 Generic Core Scales - Pediatric Quality of Life Inventory Generic Core Scales (versions: PedsQL 4.0 Generic Short Form; PedsQL Generic Core Scales version 4.0 TODDLER; PedsQL Generic Core Scales version 4.0 YOUNG CHILD; PedsQL Generic Core Scales version 4.0 CHILD; PedsQL Generic Core Scales version 4.0 TEENAGER)
Quality of life for young patients
SF-12 - 12-item Short Form Health Survey [alias: RAND-12]
SF-36 - 36-item Short Form Health Survey [alias: RAND-36][alias: Medical Outcomes Study (MOS) SF-36]]    |  | also see:
SF-6D - Short-Form 6D (derived from SF-36 or F-12)
SIP - Sickness Impact Profile   
TACQOL - Netherlands Organization for Applied Scientific Research Academic Medical Center-Child Quality of Life   
WHOQOL-100 - World Health Organization Quality Of Life Assessment Instrument 100 items
WHOQOL-BREF - World Health Organization Quality of Life questionnaire–Abbreviated Questionnaire [alias: WHO-QOL BREF]   
WOMAC - Western Ontario and McMaster Universities Osteoarthritis Index (Also I-WOMAC - Individualized WOMAC)