TABLE 1.
Patient‐reported outcome measures used in patients with autosomal inherited bleeding disorders
| Name of instrument | Type | Aim | Dimensions/ domains | Items and scoring | Studies (n), references | Psychometric qualities a |
|---|---|---|---|---|---|---|
| B‐IPQ | Generic | To assess the cognitive and emotional representations of illness | Consequences, timeline, personal control, treatment control, identity, concern, understanding, emotional response, and causal factors | Nine items rated on a linear scale from 0 to 10; higher scores indicate negative illness perception expect for the dimensions personal control, treatment control and understanding | 1 23 | |
| CES‐D | Generic | To assess severity of depressive symptoms | Depression | 20 items rated on 5‐point Likert scale; higher scores indicate greater symptoms | 1 34 | |
| CHQ‐CF87 | Generic | To assess HRQoL in children | General health perceptions, physical functioning, role/social physical functioning, bodily pain, role/social emotional functioning, role/social behavioural functioning, parent impact‐time, parent impact‐emotional, self‐esteem, mental health, behaviour, family activities, family cohesion, and change in health | 87 items rated on a 4‐ to 6‐point Likert scale; higher scores indicate better or more positive health states | 1 24 | |
| CHQ‐ PF50 | Generic | To assess caregiver's perceptions of their child's (ages 5–18) HRQoL | General health perceptions, physical functioning, role/social physical functioning, bodily pain, role/social emotional functioning, role/social behavioural functioning, parent impact‐time, parent impact‐emotional, self‐esteem, mental health, behaviour, family activities, family cohesion, and change in health | 50 items rated on a 4‐ to 6‐point Likert scale; higher scores indicate better or more positive health states | 1 24 | |
| D‐AIMS2affect | Disease‐ specific | To measure changes in global health, pain, mobility and social function in patients with arthritis | Mobility, physical activity, dexterity, household activity, social activities, activities of daily living, pain, depression, anxiety, arm function, social support, and work | 77 items; higher scores indicate better function | 1 26 | |
| Haemo‐QOL | Disease‐specific | To assess HRQoL in children with haemophilia | Physical health, feeling, view of yourself, family, friends, other people, nursery school/kindergarten, treatment, perceived support, coping, relationships, and future | 21, 64 or 77 items on a 5‐point Likert scale; higher scores indicate more impairment and poorer quality of life | 1 42 | |
| HUI23S4.15Q | Generic | To assess health status and HRQoL |
Sensation, mobility, self‐care, emotion, cognition, and pain (HUI2). Vision, hearing, speech, ambulation, dexterity, emotion, cognition, and pain (HUI3) |
14 items with different levels (from 3 to 6); higher scores indicate better HRQoL | 1 38 | |
| HUI3 | Generic | To assess health status | Vision, hearing, speech, ambulation, dexterity, emotion, cognition and pain | Eight items with different levels (5 or 6); higher score indicate better HRQoL | 1 38 , 39 | |
| HAL | Disease‐specific | To determine how bleeding episodes and progressive joint impairments impact self‐perceived functional abilities | Upper extremity activities, basic lower extremity activities, and complex lower extremity activities | 42 items across 7 domains rated on a 6‐point Likert scale; higher scores indicate greater functional status | 2 26 , 28 |
Construct validity HAL/HJHS : rs = ‐.71 HAL/SF‐36 physical functioning: rs = .65 HAL/SF‐36 physical component summary: rs = .62 HAL/IPAQ : rs = .69 |
| IPAQ | Generic | To assess the level of community participation | Autonomy indoors, autonomy outdoors, family roles, social relationships, paid work and education | 39 items rated on a 4‐point Likert scale; higher scores indicate greater restriction in participation or greater perception of problems in participation | 1 26 | |
| ITQOL | Generic | To assess caregiver's perceptions of their child's (ages 0–5) HRQoL | Physical functioning, growth and development, bodily pain, temperament and moods, general behaviour, getting along, general health perceptions, parental impact‐emotional, parental impact‐time, family cohesion, and change in health | 97 items rated on a 4‐ to 6‐ point Likert scale; higher scores indicate better HRQoL | 1 24 | |
| Kids’ ITP Tool | Disease‐specific | To assess HRQoL in children with ITP | No separate domains | 26 items on a 6‐point Likert scale: higher scores indicate greater impact of disease and poorer quality of life | 1 33 |
The Kids’ ITP Tool is not a valuable outcome measure for children with platelet disorders. The majority of the questions scored ‘not applicable’ or ‘never/rarely applicable’ |
| MPQ | Generic | To assess the different qualities of the subjective pain experience | The sensory, affective and evaluative aspects of pain | 20 items rated on a 5‐point Likert scale; higher scores indicate higher levels of pain | 1 26 | |
| SF‐36 | Generic | To assess the physical component and the mental component of HRQoL | Physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health | 36 items; higher scores indicate lower levels of disability | 7 22 , 23 , 25 , 27 , 28 , 40 , 41 | |
| VAS | Generic | To assess joint pain | Pain | Symptoms are recorded with a handwritten mark placed at one point along the length of a 10‐cm line; higher scores indicate higher levels of pain | 1 26 |
Abbreviations: B‐IPQ, Brief Illness Perception Questionnaire; CES‐D, Centre for Epidemiological Studies Depression Scale; CHQ‐CF87, Child Health Questionnaire Child Form 87; CHQ‐PF50, Child Health Questionnaire Parent Form 50; D‐AIMS2affect, Dutch Arthritis Impact Measurement Scales‐2, Haemo‐QOL, Haemophilia Quality of Life Questionnaire for Children; HAL, Health Utilities Index Mark 2; HUI23S4.15Q, Health Utilities Index Mark 2/3 15‐item Questionnaire; HUI3, Health Utilities Index Mark 3; HUI2, Haemophilia Activity List score; HJHS, Haemophilia Joint Health Score; HRQoL, Health‐Related Quality of Life; ITP, immune thrombocytopenia; IPAQ, Impact on Participation and Autonomy Questionnaire; IYQOL, Infant and Toddler Quality of Life Questionnaire; IPFD, Inherited platelet function disorders; MPQ, McGill Pain Questionnaire, QoL; Quality of Life, SF‐36; Short‐Form 36, VAS; Visual Analog Scale.