Table 3 Patient characteristics of included studies

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)

Graziani et al. [6, 15]

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)

Hanneman et al. [8, 16]

N/A

90

Mean: 44 (SD ± 15)

NR

Mean: 78 (SD ± 32)

22% on ERT at baseline

32% initiated after follow-up

43

Feriozzi et al. [12, 18]

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

Siegenthaler et al. [13, 17]

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

Arends et al. [5, 14]

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)