Table 1.
Author (year) |
Uni/ Multi-Center |
Follow-Up | N | Included Both Genders | Included Classic and Late-Onset | Renal Biomarker |
Other Biomarkers |
---|---|---|---|---|---|---|---|
Feldt-Rasmussen, et al. (2020) [18] | Multicenter (10 countries) * |
30-months | 48 | Yes | Yes | eGFR proteinuria |
-cardiac function -stroke/TIA -α-GalA -Lyso-Gb3 |
Müntze et al. (2019) [21] | Unicenter (Germany) |
12-months | 14 | Yes | Yes | eGFR proteinuria |
-cardiac function - α-GalA -Lyso-Gb3 |
Lenders et al. (2022) [19] | Multicenter (Germany) |
24-months | 54 | Yes | Yes | eGFR proteinuria |
-LVMi -stroke/TIA -MSSI -DS3 -Lyso-Gb3 |
Mauer et al. (2017) [23] | Unicenter (United States) |
6-months | 8 | Not (only males) |
Not (only classic) |
eGFR proteinuria renal histology |
-Lyso-Gb3 |
Germain et al. (2016) [22] | Multicenter (11 countries) ** |
12-months | 50 | Yes | Yes | eGFR proteinuria renal histology |
-cardiac function - α-GalA -Lyso-Gb3 -GSRS -SF-36 -PS-SF |
Riccio et al. (2020) [20] | Unicenter (Italy) |
12-months | 7 | Not (only males) |
Yes | eGFR proteinuria |
-cardiac function -neurologic changes -MSSI -SF-36 |
Abbreviations: eGFR: estimated glomerular filtration rate; α-GalA: α-galactosidase A; LVMi: left ventricular mass index; TIA: transient ischaemic attack; MSSI: Mainz Severity Score Index; DS3: Disease Severity Scoring System; GSRS: Gastrointestinal Symptom Rating Scale; SF-36: the Medical Outcomes Study 36-Item Short-Form Health Survey version 2; PS-SF: pain-severity component of the Brief Pain Inventory–Short Form. References: * Australia, Austria, Belgium, Brazil, Denmark, France, Italy, Japan, the United Kingdom, and the United States; ** France, the United Kingdom, Australia, Canada, Brazil, the United States, Italy, Turkey, Argentina, Denmark, Egypt, Spain.