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Factors for Fabry disease progression during enzyme replacement therapy

16 Aug 2017

Hypertension, cardiac fibrosis, renal dysfunction and proteinuria predict faster progression of Fabry disease (FD) while on enzyme replacement therapy (ERT), according to a recent study.

Investigators classified 293 treatment-naïve FD patients (median age at initiation 45.5 years; 55.6 male) into two according to FD phenotype: classical (n=203) and nonclassical (n=90). All patients subsequently received ERT with either agalsidase alfa or agalsidase beta for 9 months.

Over a median follow-up of 6.8 (0.8 to 15.4) years, 102 (34.8 percent) clinical events in patients on ERT were recorded. The resulting rate of events was 0.66 per 10 patient years at risk. Cardiac events, observed in 53 patients, were the most common. This was followed by stroke or transient ischaemic attacks (n=24), renal events (n=16) and death (n=9).

Cox regression analysis showed that a history of clinical (hazard ratio [HR], 2.37; 95 percent CI, 1.47 to 3.85; p<0.001) and renal (HR, 1.29; 1.81 to 7.21; p<0.001) events prior to ERT initiation, and hypertension (HR, 1.87; 1.18 to 2.97; p<0.01) significantly raised the risk of new clinical events.

Additionally, the risk of clinical events significantly increased by 19 percent (p<0.001) with every 10 mL/min/1.73m2-decrease in estimated glomerular filtration rate (eGFR) at baseline. Patients with proteinuria also experienced a significant decline in eGFR (p=0.005).

An eGFR of <60 mL/min/1.73m2 was also significantly associated with a higher risk of development of clinical events during ERT (HR, 3.58; 2.12 to 6.05; p<0.001).

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Most Read Articles
3 days ago
Combining the DASH (Dietary Approaches to Stop Hypertension) diet with low sodium intake reduces systolic blood pressure (SBP) in individuals with pre- and stage 1 hypertension, with progressively higher reductions at greater levels of baseline SBP, a recent study has shown.
Tristan Manalac, Yesterday
Major depressive disorder (MDD) appears to be more prevalent in females than in males, particularly in those who are divorced or widowed, a recent study from Singapore has found.
6 days ago
Cognitive deficits in bipolar disorder are neurodevelopmental rather than neurodegenerative in nature, a recent study has reported. Furthermore, cognitive impairment in bipolar patients appears to be stable, in the majority at least.
3 days ago
Tadalafil may not be effective for reducing the decline in ambulatory ability in boys with Duchenne muscular dystrophy (DMD), as shown in a recent study.