Supplementtable 1. Characteristics of the included studies in the systematic review and the meta-analysis for renal function and left ventricular mass. NR= not reported, HT=hypertension, Agal alpha= agalsidase alpha, Agal beta= agalsidase beta, CRI: chronic renal insufficiency: creatinine >1.5 mg/dl, ACE: angiotensinI-converting enzyme inhibitors, ARB: angiotensin receptor blockers, eGFR= estimated GFR, MA: meta-analysis, SR: systematic review,Tx: transplantation.

GFR
First Author / SR&
MA / Gender (M/F) / Study design / Intervention / Exclusion criteria / N (patients) / Age in years (mean± SD or median (range) / Follow-up (years) / Use of ACE-ARB / Outcome
(mean slope in ml/min/1.73 m2 /yr ± SD or mean GFR at baseline vs. last GFR )unless specified otherwise
Branton, 2002(Branton et al, 2002) / SR / M / Retrospective, cohort / None / For the sub-analysis: patients with missing data / 39 CRI,
24 ESRD / 42 (19-54) (n=33) / 30 / Few (at least 5) / - 12.2 ± 8.1
from
CRI to ESRD (n=14)
Schiffmann,
2009 (Schiffmann et al, 2009) / SR & MA / M / Retrospective, cohort / None / Confounding co morbidity / 279 / 38.6 (5.0–73.0) / 5.6 (0.1–28.2) / eGFR ≥60 14%
eGFR 60 41% / Total: -2.93 (SD NR)
GFR ≥60: -3.0 (SE 0.1), n=117
GFR60: -6.8 (SE 1.5), n=28
SR & MA / F / Retrospective, cohort / None / Confounding co morbidity / 168 / 44.9 (10.3–77.1) / 5.6 (0.1–28.2) / eGFR ≥60 10%
eGFR 60 22% / Total: −1.02 (SD NR)
GFR ≥60: -0.9 (SE 0.9 ), n=42
GFR 60: -2.1 (SE 1.6 ), n=13
Wyatt,
2012(Wyatt et al, 2012) / SR / M / Pros-and retrospective analysis / No ERT, Agal alpha or beta / None / 120
(108 ERT) / 44.9± 14.5 / Time on ERT:
3.74± 2.66 / Number NR / –1.40 (SE 0.14 ) per year
SR / F / Pros-and retrospective analysis / No ERT, Agal alpha or beta / None / 169
(103 ERT) / 43.9± 15.1 / Time on ERT: 3.34±2.25 / Number NR / –0.69 (SE 0.09) per year
Eto, 2005 (Eto et al, 2005) / SR / M / Prospective / Agal beta / History of dialysis or renal transplantation / 13 / 26.6±5.5 / 20 weeks / NR / before ERT mean GFR 126.6±41.8 (ml/min), after ERT: 115.3±30.4 (ml/min) (ns)
Lubanda, 2009 (Lubanda et al, 2009) / SR / M / Prospective / Agal beta / Serum creatinine > 2.2 mg/dL, kidneyTx, dialysis, significant disease, co morbidity / 21 / 34±10 / 6 months/ 18 months (reduced dose) / n=0 at baseline, during follow-up n= 4/21 / Before ERT 92.5 ( 55-134), at week 24 : 93.1 (range NR), at week 96: 92.8 (range NR )
West, 2009 (West et al, 2009) / SR & MA / M / Prospective, pooled analysis / Agal alpha / <2 Creatinine measurements / 108 / 34.4±9.3 / 2±1 / 26/93 (30%) / -7.0 ± 32.9, Cases with hyperfiltration excluded
Schiffmann, 2007 (Schiffmann et al, 2007) / SR / M / Prospective / Agal alpha / ESRD / 11 / 41.4±8.4 / Agal-alpha/ eow: 2-4
Agal-alpha weekly:2 / Agal-alpha/ eow: 2/11
Agal-alpha weekly: 9/11 / Agal-alpha 0.2
mg/kg/eow
-8.0 ± 0.8
Agal-alpha0.2
mg/kg/w
-3.3 ±1.4
Whybra, 2009 (Whybra et al, 2009) / SR & MA / F / Prospective, cohort / Agal alpha / GFR>135; 3 nephropathies for which corticosteroids (in sub analysis ) / 36 / 47.0±17.9 / 4 / n=7 (19.4%) at start / before ERT mean GFR: 91.0 ± 29.7, after ERT:91.0 ± 25.6 (ns)
Rombach
, 2013(Rombach et al, 2013) / SR & MA / M / Prospective, cohort / Agal alpha or beta / < 6 months follow-up / 30 / 38.8±14.3 / 5.5 (0.51-10.0) / 20 (67%) / -3.4 (SE 0.2)
SR & MA / F / Prospective, cohort / Agal alpha or beta / < 6 months follow-up / 27 / 46.8±12.3 / 5.5 (0.5-7.1) / 23 (85%) / -0.8 (SE 0.3)
LVmass
First Author / SR/ MA / Gender / Study design / Intervention / Exclusion criteria / N / Age in years (mean± SD or median (range) / Follow-up (years) / Use of ACE-ARB) / Outcome
(mean slope g/m2.7 /year (SD)) unless specified otherwise
Kampmann, 2008(Kampmann et al, 2008a) / SR & MA / M / Prospective, cohort / None / HT/ antihypertensive medication / 39 / 31.3±11.9 / 4.5 (1.1-9.0) / 0% / Total:
+ 4.07±1.03
LVH +: +6.59±8.5
SR & MA / F / Prospective, cohort / None / HT/ antihypertensive drugs / 39 / 44.7±20.4 / 4.5 (1.1-9.0) / 0% / Total:
+ 2.31±0.81
LVH +: 3.77±7.7
Wyatt et al, 2012(Wyatt et al, 2012) / SR / M / Pros-and retrospective analysis / No ERT, Agal alpha or beta / None / 120
(108 ERT) / 44.9± 14.5 / Time on ERT:
3.74± 2.66 / NR / Untreated +1.52 g/m2/year *(corrected for gender)
*In case of ERT
>36 months
- 8.17 g/m2(p =0.05)
SR / F / Pros-and retrospective analysis / No ERT, Agal alpha or beta / None / 169
(103 ERT) / 43.9± 15.1 / Time on ERT: 3.34±2.25 / NR / Untreated +1.52 g/m2/year*(corrected for gender)
*In case of ERT
>36 months
- 8.17 g/m2(p =0.05)
Hughes, 2008(Hughes et al, 2008) / SR / M / RCT, double-blind / Agal alpha / pacemaker, contraindication for cardiac biopsy / 15 / >18 / 2.5 years (n=7), 2 years (n=8) / NR / -20.4± 27.2 g /6 months (n=7)
Motwani, 2012(Motwani et al, 2012) / SR / M / Retrospective analysis / Agal beta / None / 44 / 41±14 / 3 (2-5) / NR / before ERT mean LV mass 123±2 g/m2, after ERT 120±26 g/m2 (p<0.001)
SR / F / Retrospective analysis / Agal beta / None / 22 / 44±14 / 3 (2-5) / NR / before ERT, mean LV mass 101±22g/m2, after ERT 98±20 g/m2 (p<0.001)
Kampmann, 2009(Kampmann et al, 2009) / SR & MA / M / Retrospective, pooled analysis / Agal alpha / NR / 34 / LVH (n=9),
41.3±7.6
No LVH (n=25) 36.0±9.2 / 12 or 36 months / NR / LVH+:
12 months ERT: -11.8± 7.5 (n=6)
36 months ERT -3.2 ±8.2 (n=7)
LVH-:
12 months ERT: + 3.7± 5.8 (n=22)
36 months ERT +2.2 ±8.3 (n=23)
Whybra,2009(Whybra et al, 2009) / SR & MA / F / Prospective, cohort / Agal alpha / GFR>135; 3 patients with nephropathies for which corticosteroids / 36 / 47.0±17.9 / 4 / n=7 (19.4%) at start / LVH>85: -10 (SE 4.4)
LVH 60-85:-3.6 (SE 1.7)
LVH>48-60 :-1.8 (SE 0.4)
LVH <48:-1.1 (SE 0.7)
Rombach, 2013(Rombach et al, 2013) / SR & MA / M / Prospective, cohort / Agal alpha or beta / < 6 months follow-up / 27 / 37.1±13.0 / 5.5 (0.51-10.0) / 20 (67%) / Total:
+1.2 (SE 0.3)
LVH+:+1.5 ± 0.5
LVH-: +1.0 ± 0.3
SR & MA / F / Prospective, cohort / Agal alpha or beta / < 6 months follow-up / 26 / 46.6±12.3 / 5.5 (0.5-7.1) / 23 (85%) / Total:
(-0.3 SE 0.4)
LVH+:-0.4 ± 0.6
LVH-: -0.3 ± 0.5

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