29 Cardiac outcomes in Fabry disease the age of 15 years onwards was used, because this study shows that cardiac events do not occur before the age of 15 (figure 2, table 2, supplemental table 2) and the event-free follow-up duration of especially the younger men with classical FD would impact unevenly on the event rate without this correction. Event-free survival was analyzed using the Kaplan-Meier (KM) method in which patients where stratified according to phenotype and sex. If less than 50% of the patients developed a specific event, the median age of onset for those patients that experienced the event was reported instead. Pair-wise comparison between FD patient groups was performed using a log-rank test, with Bonferroni correction. Next, these analysis were performed correcting for competing risks (CR) (e.g. non- cardiovascular death for the outcome of cardiovascular death, supplemental figures 2-6). Cox regression analyses were performed to assess the effect of phenotype and sex and the interaction between these variables on the occurrence of events. Since no events occurred in the subgroup of women with non-classical FD, we used Firth’s penalized Cox regression to obtain stable models. P values <0.008 (after Bonferroni correction) were considered statistically significant. SPSS (v.25) and R (version 3.6.1) were used for statistical analysis. Results 213 patients were included: 57 (27%) men and 98 (46%) women with classical FD, 26 (12%) men and 32 (15%) women with non-classical FD. Median age at last outpatient visit or death was 50 years (range 19-83). Patients characteristics are described in table 1. Figure 2 shows the occurrence of cardiac events for all patients in the 4 different groups. 2
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