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Table 1 Clinical characteristics of men exposed to agalsidase

From: Deep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRY

  Ab-positive Ab-negative n* p-value
N 18 27 45
Age visit (y.) 43.7 [32.8–49.2] 46.3 [41.4–59.6] 45 0.12 £
Age diag (y.) 28.8 [20.1–41.5] 41.1 [28.1–53.7] 45 0.09 £
Agalsidase exposure A/B/AB 4/8/6 9/11/7 45 0.70
Current migalastat 1 1 45 1 $
Cumul. Exp. (y.) 10.6 [3.3–12.2] 4.3 [3.2–7.0] 45 0.22 £
MTP/Missense 10/8 3/21 42d 0.006 $
RRA (%)b 0.38 [0.25–1.0] 1.10 [0.95–1.1] 43 0.0003 £
LysoGb3c (ng/ml) 25.4 [18.9–48.8] 10.2 [3.1–17.1] 33 0.0005 £
Mainz score total 23.5 [14.0–32.5] 22.0 [15.5–27] 45 0.74 £
Mainz cardiovascular score 2.5 [0.0–9.8] 9.0 [2.5–12.5] 45 0.14 £
Mainz renal score 8.0 [0–18] 0.0 [0.0–8.0] 45 0.22 £
Mainz neurological score 5.5 [2.3–8.8] 5.0 [1.5–8.0] 45 0.65 £
Mainz general score 4.5 [2.5–6.8] 4.0 [1.5–6.5] 45 0.33 £
Dialysis or kidney transplant 6 1 45 0.012 $
Classic/ Non classic phenotype 17/1 12/15 45 0.0005 $
  1. n* number of patients included in the analysis; median [IQR], RRAb patients under migalastat were excluded from the analysis, LysoGb3c available plasma of patients under agalsidase only, patients under migalastat were excluded from the analysis, MTPs mutations leading to a truncated protein (deletion, frameshift or non-sense mutations), MSSI Mainz severity score index, IQR interquartile range, RRA relative residual activity
  2. dthe genotype was unavailable for 3 patients
  3. £Mann-Whitney test; $Fischer’s exact test
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