OBJECTIVES:

A review of the literature was performed to evaluate whether agalsidase alpha and agalsidase beta are effective and safe for the treatment of Fabry disease.

METHODS:

A review of the literature was conducted through a search in the Medline, Embase, Lilacs, Center for Reviews and Dissemination (CRD) and Cochrane databases, without restrictions. Manually, publications of the National Institute for Health and Care Excellence (NICE) and national and international guidelines have been consulted. Quality of evidence was assessed by GRADE (Grading of Recommendations Assessment, Development and Evaluation). Were estimated the costs of treatments.

RESULTS:

The search strategy recovered 2,337 articles and 10 were selected for evaluation. A Cochrane review of 2017 was used as the basis. Outcomes evaluated: renal complications, agalsidase alfa 15.3% [95% CI: 0.048, 0.303; p = 0.0005]; agalsidase beta 6% [95% CI 0.04, 0.07; and untreated patients 21.4% [95% CI: 0.1522; 0.2835 p <0.0001]; cardiovascular complications, agalsidase alfa 28% [95% CI: 0.07, 0.55; p <0.0001]; agalsidase beta 7% [95% CI 0.05, 0.08;]; and untreated patients 26.2% [95% CI: 0.149, 0.394; p <0.0001]; and for cerebrovascular complications, agalsidase alfa 11.1% [95% CI 0.058, 0.179; p = 0.0024]; agalsidase beta 3.5% [95% CI: 0.024, 0.046 p = 0.4209]; and untreated patients 18.3% [95% CI: 0.129; 0.245; p <0.0001]. All-cause mortality rate was higher in untreated patients (10.8%), followed by agalsidase alfa (9%) and agalsidase beta (4.4%), there was no significant difference. The treatment of an adult (70 Kg) for a month will cost about USD 8,109.00 for agalsidase alfa and about USD 10,243.00 for agalsidase beta. The quality of evidence was assessed as very low.

CONCLUSIONS:

The balance between the limited quality of evidence and the benefits demonstrated was favorable to both technologies. The differences in effect favored agalsidase beta. The cost of agalsidase alfa was about 21% lower.