Table 2.
Questionnaire completion rate
| Pathfinder™5 (N=68) | Pathfinder™2 (N=184)b | |||||||
|---|---|---|---|---|---|---|---|---|
| Age group | 0–3 years | 4–7 years | 8–11 years | 13–16 years | ≥17 years | |||
| No. of patients enrolled in study | 20 | 23 | 25 | 16 | 168 | |||
| Number of patients with completed PRO scores n (%) | ||||||||
| Respondent | Parents | Children | Parents | Children | Parents | Children | Parents | Adults |
| Haemo-QoL | Haem-A-QoL | |||||||
| Baseline | – | 15 (62.2) | 21 (91.3) | 22 (88.0) | 22 (88.0) | 16 (100) | 16 (100) | 166 (98.8) |
| End of main phase | – | 13 (56.5) | 14 (60.9) | 21 (84.0) | 21 (84.0) | 10 (62.5) | 9 (56.3) | 165 (98.2) |
| Change in scorea | – | 7 (30.4) | 14 (60.9) | 21 (84.0) | 21 (84.0) | 10 (62.5) | 9 (56.3) | 163 (97.0) |
| Hemo-SatP | Hemo-SatA | |||||||
| Baseline | 19 (95.0) | – | 20 (87.0) | – | 22 (88.0) | – | 10 (62.5) | 167 (99.4) |
| End of main phase | 16 (80.0) | – | 18 (78.3) | – | 21 (84.0) | – | 9 (56.3) | 163 (97.0) |
| Change in scoresa | 16 (80.0) | – | 17 (73.9) | – | 21 (84.0) | – | 4 (25.0) | 162 (96.4) |
Notes:
Some patients had a missing baseline score or end of main phase score, therefore a change in score was not able to be computed for these patients.
Two patients who were 12 years old in pathfinder™ 2 were excluded from the analyses as they completed the Haemo-QoL II at baseline and Haemo-QoL III at the end of the main phase and a change of score cannot be computed. The Hemo-Sat questionnaire is an instrument designed specifically to assess TS in patients with hemophilia. The Hemo-SatA consists of 34 items covering six domains. The Hemo-SatP (a version for parents of children with hemophilia who are aged <17 years) it measures the satisfaction with their child’s treatment and includes the same items and domains as the Hemo-SatA, but with one additional item in the “ease and convenience” domain.
Abbreviations: PRO, patient-reported outcomes; QoL, quality-of-life.