Table 3.
Clinical studies assessing the impact of ERT on PROs in MPS patients
| Reference | MPS type | Treatment | Comparator | N | Mean age (years)a | Study duration | PRO instrument |
|---|---|---|---|---|---|---|---|
| [33] | I 83 % Hurler-Scheie, 13 % Scheie |
iv laronidase (0.58 mg/kg/week) |
Placebo | 45 | 15.6 | 26 weeks | (C)HAQ |
| [23] | I | iv laronidase (0.58 mg/kg/week) |
/ | 45 | 15.7 | 3.5 year (extension of [66] |
(C)HAQ |
| [52] | I | iv laronidase (0.58 mg/kg/week) |
/ | 5 | 12.0 | 6 year | Modified MPS HAQ |
| [53] | I Scheie, Hurler- Scheie |
iv laronidase (0.58 mg/kg/week) |
/ | 7 | 16.3 | 52–208 weeks | MPS HAQ |
| [55] | II | iv idursulfase (0.5 mg/kg/week) |
/ | 94 | 14.5 | 2 years extension | (C)HAQ |
| [32, 45] | IVA | iv elosulfase alfa (2.0 mg/kg every other week or weekly) |
Placebo | 176 | 15.3 and 13.1 | 24 weeks | MPS HAQ |
| [29] | IVA | iv elosulfase alfa (2.0 or 4.0 mg/kg/week) |
/ | 25 | 13.7 | 27 weeks | APPT |
| [24] | VI | iv galsulfase (1.0 or 2.0 mg/kg/week) |
/ | 5 | 11.0 | 48 weeks | (C)HAQ |
| [25] | VI | iv galsulfase (1.0 mg/kg/week) |
/ | 10 | 12.7 | 48 weeks | (C)HAQ |
| [26] | VI | iv galsulfase (1.0 mg/kg/week) |
Placebo | 39 | 13.7 | 24 weeks | Joint pain and stiffness, physical energy level |
| [56] | VI | iv galsulfase (1.0 mg/kg/week) |
/ | 9 | NA | 2 years | (C)HAQ |
| [5] | VI | iv galsulfase (1.0 mg/kg/week) |
/ | 55 | 12.0 | 6.8 ± 2.2 years | (C)HAQ |
| [44] | VI | iv galsulfase (1.0 mg/kg/week) |
/ | 8 | 6.8 | 1.0–4.5 years | TAPQOL/TACQOLb |
aMean age at baseline from all patients or from ERT group
bThe TAPQOL was completed by the parents of four patients <6 years, TACQOL was completed by seven parents of patients ≥6 years
NA not available