elosulfase alfa treatment in morquio a patients in iran: a before and after study

Background: morquio a, an autosomal recessive lysosomal storage disease, is caused by a defect in the enzyme n-acetylgalactosamine-6-sulfatase. this leads to the accumulation of the glycosaminoglycans chondroitin-6-sulfate (c6s) and keratan sulfate(ks), resulting in various skeletal manifestations, multisystemic impairments, and significant morbidities.objectives: this study aimed to evaluate the impact of the addition of elosulfase alfa to the hospital protocol on treating iranianpediatricians with morquio a syndrome.methods: a before and after study was conducted on ten patients with morquio a syndrome diagnosis from 2019 to 2020. elosulfasealfa was prescribed with the standard dose of 2 mg/kg/weekly iv infusion for 54 weeks. then, growth indices, quality of life,and cardiopulmonary data were collected by research assistants using a pre-designed check. data were entered in spss version 23.quantitative variables were compared between the two periods using the student’s t-test, and qualitative variables were comparedusing the 2 test or fisher’s exact test.results: ten pediatricians with mps iv were included, seven of whom were female, with the mean age of 5.82.3 years. the meantimefor walking 6m(p = 0.005), standing (p = 0.005), the stair climb test (p = 0.007), and quality of life (p = 0.015) had significantstatistical difference before and after treatment by elosulfase alfa.conclusions: the addition of elosulfase alfa in patients with morquio a syndrome was associated with a significant improvementoutcome in the ‘after’ treatment period.