Table 1.
Overview of selected PRO instruments.
| Age Group | Type of Measures* | Name of questionnaire | Instrument | Dimension/subscales | N.°of Items** | Way of administration | Reliability | Validity in Haemophilia Examined | N° of languages | Use in haemophilia Research |
|---|---|---|---|---|---|---|---|---|---|---|
| Children | HRQoL | Haemo-QoL | Haemophilia -specific QoL Questionnaire for children patients and parents | 8-12 (physical health, feeling, view of yourself, family, friends, perceived support, others, sports and school, dealing with haemophilia, treatment, future, relationships, global health) | I: 21 for children aged 4-7 II: 64 for children aged 8-12 III: 77 for adolescents aged 13-16 | Self/Proxy | α = 0.85-0.91 (for the different age group versions) | yes | 40 | Epidemiological study (describing the quality of life of patients with reference to other chronic conditions), Clinical trials (to evaluate the potential benefits of different treatment regimes), Quality assurance (identifying the quality of care perceived by patients, e.g., in Haemophilia Comprehensive Care Centres (HCCC), Health-economic studies (assessing costs and benefits of haemophilia treatment with regard to economic indicators) and Routine treatment |
| Adults | Haem-A-QoL | Disease-specific questionnaire for adults patients | 10 (physical health, feelings, view of yourself, sport and leisure, work and school, dealing with haemophilia, treatment, future, family planning, partnership and sexuality) | 46 | Self | α = 0.74-0.88 | yes | 32 | ||
| Medtap (Haemo-QoL-A) | Haemophilia-specific QoL questionnaire for adults | 4 (day-to-day activities, mood and feelings, work or school life, family life -social life, haemophilia treatment) | 41 | Self | α = 0.75-0.95 | yes | 20 | |||
| TS | Hemo-SatA | Haemophilia Treatment satisfaction Question- naire | 6 (Ease and convenience, Efficacy Burden, Specialist/nurses, Centre/Hospital, General satisfaction with your treatment | 34 | Self | α = 0.71-0.95 | yes | 24 | To evaluate patients' therapy and experience, in follow-up analysis | |
| Functioning | HEP-Test Q | Subjective assessment questionnaire of the effects of physical functioning in adult with haemophilia | 5 (physical status, mobility strength-coordination, endurance, body perception) | 25 + 1 | Self | α = 0.70-0.90 | yes | 3 | Evaluation of patients' daily activities or functioning, Follow-up analysis, Rehabilitation Programs |