Study | Treatment given | N | Mean age | Baselines | % Treatment-naïve | Follow-up (months) | |
---|---|---|---|---|---|---|---|
eGFR | LVMI | ||||||
Spinelli et al. [9] | N/A | 96 | Mean: 41.8 (SD ± 14.7) | Mean: 93.2 (SD ± 31.9) | Mean: 48.5 (SD ± 19.7) | 17.6% naïve 4.2% with renal transplants 16.6% treated with ERT before baseline assessment 61.4% initiated ERT during follow-up | 63 (IQR 37–85) |
N/A | 45 | Mean: 52 (SD ± 16) | Mean: 87.6 (SD ± 31.1) | Mean: 135.2 (SD ± 78) | 22.3% naïve 32 patients (71.1%) were on ERT, 24 (53.3%) with agalsidase alfa, and 7 (15.5%) with agalsidase beta One patient (2.2%) was receiving treatment with migalastat 6.6% previous kidney transplant | 51.2 (SD ± 11.4) | |
N/A | 90 | Mean: 44 (SD ± 15) | NR | Mean: 78 (SD ± 32) | 22% on ERT at baseline 32% initiated after follow-up | 43 | |
Agalsidase alfa | 560 (LVMI outcomes) 1093 (renal outcomes) | Age at agalsidase alfa initiation LVMI group: Mean 4.8 (SD ± 14.6) Renal group: Mean 44.1 (SD ± 14.8) | LVMI group: Mean 92.7 (SD ± 25.8) eGFR group: Mean 94.1 (SD ± 27.8) | LVMI group: Mean 54.6 (SD ± 20.4) eGFR group: Mean 54.1 (SD ± 20.5) | 0%, all treated with agalsidase alfa | NR | |
Lenders et al. [10] | Agalsidase alfa or beta | 54 | Mean: 44.5 (SD ± 14.7) | Mean: 87.1 (SD ± 31.5) | NR | All patients naïve at baseline | 71 |
Agalsidase alfa or beta | 104 | Mean: 45 (SD ± 16) | NR | Median: 89 (IQR 64–127) | ACE inhibitor or ARB in 17 (16%) of patients at baseline, increasing to 34 (32%) at follow-up 48 (46%) patients were treated with agalsidase alfa and nine with agalsidase beta throughout the follow-up time | 103 (range 59–155) | |
Patel et al. [11] | NR | 2,869 | Mean: 48.5 (SD ± 11.71) | Mean: 91 (SD ± 39.54) | NR | All patients naïve at baseline/data obtained before ERT initiated | NR |
Agalsidase alfa or beta | 293 | Median: 45 (range: 18–79) | Median: 91 (range: 5–140) | Median: 51 (range: 16–149) | All patients naïve at baseline | 82 (range 9.6–185) |