Table 4.
Effect of ERT in patients with FD
| Organ | Background/Setting/Measures | Effect on Pain | Reference |
|---|---|---|---|
| Peripheral nervous system | |||
| Agalsidase-α | Randomized, placebo-controlled study, 26 males treated for 6 mo, BPI (worst) changed by −1.9 (P=0.02) | Reduction of BPI (worst) | 20 |
| Retrospective FOS analysis, 25 patients treated for up to 2 yr, BPI (average) reported to decrease | Reduction of BPI (average) | 42 | |
| Retrospective FOS analysis, 53 patients treated for up to 5 yr, BPI (average) changed by −1.2±2.7 (n=53, P=0.002), BPI (worst) changed by −1.3±3.5 (n=49, P=0.01) | Reduction of BPI (average and worst) | 56 | |
| Single-center prospective open-label study, 36 females treated for 4 yr, BPI (worst) changed after 12 mo from 4.6±2.9 to 3.3±2.9 (P=0.001) and remained stable | Reduction of BPI (worst) | 57 | |
| Retrospective FOS analysis, 20 (16 males) patients treated for up to 2 yr, BPI (average, now, worst) decreased after the second year (all P<0.05) | Reduction of BPI (average, now, worst) | 69 | |
| Retrospective FOS analysis, 62 patients treated for 3 yr, BPI (average, least, worst) decreased (P<0.05) | Reduction of BPI (average, least, worst) | 70 | |
| Agalsidase-β | Single-center prospective open-label study, 22 male patients treated for up to 23 mo, total symptom score for neuropathic pain changed from 1.76±1.97 to 0.83±1.53 (P=0.04), quantitative sensory testing revealed improvements for VDT (P<0.05) and HP (P<0.01) | Reduction of pain, improvement of nerve function | 71 |
BPI, brief pain inventory; FOS, Fabry outcome survey; VDT, thresholds of vibration; HP, heat-pain.