Table 3.
Main Studies Reporting Data on the Effectiveness of Agalsidase Alfa Replacement Therapy on Pain
| Author | Type of Study | Duration | Population | Renal Outcome |
|---|---|---|---|---|
| Schiffmann R et al22 | Randomized double-masked placebo-controlled | 1 year | 26 male patients | Significant reduction in pain and pain-related quality of life in the treated group |
| Schiffmann R et al75 | Open-label extension study | 3 years | 26 male patients | On the 10 patients who crossed from placebo to ERT, there was a significant improvement in pain. In addition, warm and cold thresholds, as well as sweating improved |
| Beck M et al17 | FOS retrospective study | 2 years | 314 patients (203 males, 111 females) | Significant improvements in walking ability, general activity, regular work and social relationships. There was also a substantial improvement in pain. |
| Ries et al76 | Prospective open-label study | 6 months | 24 children (19 males,5 females) | No significant improvement in pain severity |
| Schiffmann R et al77 | Prospective long-term open-label | 3.5 years | 17 children (16 males, 1 female) who completed the 6-month trial of Ries et al 2006 | Significant reduction in pain severity |
| Whybra C et al51 | Prospective, single-centre, open-label | 4 years | 36 adult female ERT-naïve patients | Significant improvement of the “pain at its worst” section of BPI |
| Hughes DA et al36 | FOS retrospective study | 4 years | 250 patients (172 males, 78 females) aged >18 years | No significant reduction of pain in both males and females after 4 years |
| Ramaswami U et al54 | FOS retrospective study | 2 years | 98 children (64 boys, 34 girls) aged <18 years | Reductions in pain prevalence and pain crises were observed in the patients showing pain attacks or chronic pain at baseline |
| Hughes DA et al78 | Randomized double-masked placebo-controlled study | 14 weeks | 18 patients | Using more frequent doses of Agalsidase alfa: 0.1 mg/kg/week, 0.2 mg/kg/week. No significant differences were found in pain scores. |
| Vedder A. et al79 | Prospective randomized open-label | 2 years | 34 adult patients who received either agalsidase alfa or beta 0.2mg/kg | No difference in pain scores between groups |
| Hoffmann B. et al Clin J Pain.74 | FOS retrospective analysis | 3 years | 24 months: 81 adult patients (50 males, 31 females).36 months: 62 patients (41 males, 21 females) | Significant improvement in the severity of pain both at 24 and 36 months |