Table 5.

Drivers and barriers associated with early treatment initiation in FD that achieved consensus in PREDICT-FD

Drivers
1. Male sex, young age, and clinical findings, such as severe pain and signs/symptoms of organ involvement, are key drivers of early initiation of treatment (4.8)
2. Improving clinical outcomes and preventing disease progression are key drivers of early initiation of FD-specific treatment (4.6)
3. A family history of FD, especially if severe or with major organ involvement or premature death, is a key driver of early initiation of treatment (4.4)

Barriers
1. A lack of biomarkers predicting which patients will progress and which will respond to treatment is a key barrier to early initiation of treatment (4.1)
2. Misdiagnosis is a key barrier to early initiation of treatment (3.9)

Data in parentheses are the mean of agreement scores awarded by 21 panel members based on a 5-point pivoted Likert scale (1 = strongly disagree; 3 = neither agree nor disagree; 5 = strongly agree). The criterion for consensus was an agreement rating of at least 4 awarded by more than 67% of those who voted

Bold indicates the consensus statements describing the possible impact of PREDICT-FD

FD, Fabry disease; PREDICT-FD, PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease