Elosulfase Alfa Treatment in Morquio A Patients in Iran: A Before and After Study
| Author | Mohammadreza Alaei | en |
| Author | Marjan Shakiba | en |
| Author | Hedyeh Saneifard | en |
| Author | Ghamartaj Khanbabaee | en |
| Author | Mohammadreza Khalilian | en |
| Author | Asieh Mosallanejad | en |
| Author | Saleheh Tajalli | en |
| Author | Mojtaba Lotfi | en |
| Orcid | Hedyeh Saneifard [0000-0002-7926-0598] | en |
| Orcid | Asieh Mosallanejad [0000-0001-7926-8218] | en |
| Orcid | Saleheh Tajalli [0000-0002-2045-6430] | en |
| Issued Date | 2021-12-31 | en |
| Abstract | Background: Morquio A, an autosomal recessive lysosomal storage disease, is caused by a defect in the enzyme N-acetyl-galactosamine-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 Iranian pediatricians with Morquio A syndrome. Methods: A before and after study was conducted on ten patients with Morquio A syndrome diagnosis from 2019 to 2020. Elosulfase alfa 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 compared using the χ2 test or Fisher's exact test. Results: Ten pediatricians with MPS IV were included, seven of whom were female, with the mean age of 5.8 ± 2.3 years. The meantime for walking 6 m (P = 0.005), standing (P = 0.005), the stair climb test (P = 0.007), and quality of life (P = 0.015) had significant statistical 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 improvement outcome in the ‘after’ treatment period. | en |
| DOI | https://doi.org/10.5812/ijp.111562 | en |
| Keyword | Morquio Syndrome | en |
| Keyword | MPS IV | en |
| Keyword | Morquio A | en |
| Keyword | Elosulfase Alfa | en |
| Keyword | Pediatric | en |
| Keyword | Iran | en |
| Publisher | Brieflands | en |
| Title | Elosulfase Alfa Treatment in Morquio A Patients in Iran: A Before and After Study | en |
| Type | Research Article | en |
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