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Table 4 Study predictive relationships reported

From: Targeted literature review exploring the predictive value of estimated glomerular filtration rate and left ventricular mass index as indicators of clinical events in Fabry disease

Study

Method of analysis

Results

OR

HR

Spinelli et al. [9]

Univariate Cox analysis with predictor variable thresholds established by ROC curve analysis

NR

Predictors of events*:

LVMI (g/h2.7) > 54.3: 1.022 (1.008–1.036), p = 0.002

eGFR ≤ 69: 0.978 (0.961–0.996), p = 0.016

Graziani et al. [6, 15]

Univariate and multivariate Cox regressions

NR

Predictors of events*:

eGFR: 0.97 (0.95–0.99), p 0.008

LVMI (g/m2) (univariate): 1.01 (1.00–1.01) p < 0.001

LVMI (g/m2) (multivariate): 1.01 (1.00–1.03) p = 0.03

Hanneman et al. [8, 16]

Univariate and multivariate Cox regressions

NR

HR for 5 g/m2 increase in LVMI

1.1 (1.1–1.2), p < 0.001 (univariate)

1.1 (1.04–1.2), p < 0.001 (multivariate)

Feriozzi et al. [12, 18]

Multivariate Cox regression analysis

NR

HR for cardiovascular event

LVMI (g/h2.7) at baseline (abnormal vs normal*) 1.57 (1.21–2.05) p < 0.001

eGFR at baseline (abnormal vs normal) 1.33 (1.04–1.70) p = 0.021

HR for renal event

LVMI (g/h2.7) at baseline (abnormal* vs normal) 1.90 (0.94–3.85) p = 0.074

eGFR at baseline (abnormal* vs normal) 5.88 (2.73–12.68) p < 0.001

* Abnormal LVMI defined as > 50 g/h2.7 in males and > 48 g/m2.7 in females; abnormal eGFR defined as < 90 mL/min/1.73 m2

Lenders et al. [10]

Cox proportional hazards model

NR

Patients without endpoints had an average eGFR at baseline of 102.6 (± 26.6) compared to 71.3 (± 28.1)

HR for endpoint-free survival of on ERT patients with an eGFR =  < 75 mL/min/1.73 m2: 4.77 (1.93–11.81), p < 0.001

HR for cardiovascular endpoints after ERT initiation endpoint-free survival for patients with an eGFR ≤ 75 mL/min/1.73 m2: 3.59 (1.15–11.18); p = 0.03

Siegenthaler et al. [13, 17]

Univariate and multivariate Cox regressions

NR

HR for occurrence of primary endpoint per SD increase in LVMMI: Crude (2.14, CI 1.52–3.01, p < 0.001), Model 1 (1.65, CI 1.06–2.58, p = 0.03), Model 2 (1.67, CI 1.04–2.73, p = 0.03), Model 3 (1.67, CI 1.04–2.73, p = 0.03)

HR for occurrence of primary endpoint per SD increase in eGFR: Crude (0.42, CI 0.28–0.61, p < 0.001), Model 1 (0.45, CI 0.27–0.74, p = 0.002), Model 2 (0.42, CI 0.24–0.74, p = 0.003), Model 3 (0.45, CI 0.25–0.83, p = 0.01)

HR for occurrence of primary endpoint for patients with eGFR < 90 mL/min/1.73 m2 at baseline, Crude (6.38 CI 1.91–21.33 p = 0.003) Model 1 (4.3 CI 1.06–17.52 p = 0.04) Model 2 (4.88 CI 1.12–21.24) Model 3 (4.41 CI 0.97–20.06)

HR for occurrence of primary endpoint for patients with LVMMI > 107 g/m2 at baseline, Crude (4.28, CI 1.58–11.56 p = 0.004), Model 1 (1.79, CI 0.55–5.82 p = 0.37), Model 2 (1.83, CI 0.56–5.97 p = 0.32), Model 3 (1.75 CI 0.54–5.69 p = 0.35)

Higher LVMMI and lower eGFR at baseline associated with a greater risk of developing an adverse clinical event (HR 2.21 [1.43–3.42], p < 0.001 for LVMMI, HR 0.41 [0.27–0.62], p < 0.001 for eGFR) (per SD increase in LVMMI and SD decrease in eGFR)—Excluding atrial fibrillation

Crude model contained only LVMMI/eGFR as predictor, Model 1 included age and gender, Model 2: Model 1 + transplant status, dialysis status and Model 3: Model 2 + hypertension status and baseline LVMMI/eGFR

Patel et al. [11]

Univariate logistic regression models, eGFR not considered in multivariate model as not selected during stepwise selection

OR of experiencing a CV event for patients with low eGFR (< 60 mL/min/1.73 m2)

Men OR: 2.33 (1.22–4.45), p < 0.05

Women OR: 3.85 (1.78–8.32), p < 0.0001

NR

Arends et al. [5, 14]

Univariate and multivariate Cox regressions

NR

HRs of the additional potential prognostic variables on the clinical event rate, adjusted for age at start of ERT, sex and phenotype

Mixed-effect models

eGFR (per −10 mL/min/1.73 m2) HR: 1.19 (1.11–1.27), p < 0.001

eGFR < 60 mL/min/1.73 m2 HR: 3.58 (2.21–6.05), p < 0.001

LVMI (per 10 g/m2.7) HR: 1.25 (1.08–1.45), p < 0.01

Multivariate

eGFR (per −10 mL/min/1.73 m2) HR: 1.12 (1.03–1.22), p < 0.01

LVMI (per 10 g/m2.7) HR: 1.16 (0.99–1.36), p > 0.05

Multivariate analysis with eGFR as dichotomous variable

eGFR (< 60 mL/min/1.73 m2) 2.66, CI 1.45–4.90, p = 0.002

LVMI (per 10 g/m2.7) 1.19, CI 1.02–1.38, p = 0.028

Multivariate analysis, excluding renal events

eGFR (per -10 mL/min/1.73 m2) 1.01, CI 0.93–1.10, p = 0.78

LVMI (per 10 g/m2.7) 1.19, CI 1.02–1.38, p = 0.03

  1. Key: CI, confidence interval; CV, cardiovascular; eGFR, estimated glomerular filtration rate; ERT, enzyme replacement therapy; HR, hazard ratio; LVMI, left ventricular mass index; LVMMI, left ventricular myocardial mass index; NR, not reported; OR, odds ratio; ROC, receiver operating characteristic; SD, standard deviation
  2. *All events described in Table 2
  3. ‘increase’ is based on this papers’ authors interpretation of the study results and conclusions, rather than the author of said paper’s results table