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Efficacy and Safety of Miglustat in the Treatment of Gm2 Gangliosidosis: A Systematic Review Publisher Pubmed



Mansouri V1 ; Tavasoli AR2, 3 ; Khodarahmi M4 ; Dakkali MS5 ; Daneshfar S6 ; Ashrafi MR3, 7 ; Heidari M3, 8 ; Hosseinpour S9 ; Sharifianjazi F10 ; Bemanalizadeh M3, 11
Authors
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Authors Affiliations
  1. 1. Gene Therapy Research Center, Digestive Diseases Research Institute, Shariati Hospital, Tehran University of Medical Sciences, Tehran, Iran
  2. 2. Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, PA, United States
  3. 3. Pediatric Neurology Division, Department of Pediatrics, Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran
  4. 4. Bahar Medical Imaging Center, Karaj, Iran
  5. 5. School of Medicine, Zahedan University of Medical Sciences, Zahedan, Iran
  6. 6. Faculty of Medicine, Islamic Azad University, Tabriz Branch, Tabriz, Iran
  7. 7. Pediatric Cell and Gene Therapy Research Center (PCGTRC), Tehran University of Medical Sciences, Tehran, Iran
  8. 8. Pediatric Neurology Division, Myelin Disorders Clinic, Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran
  9. 9. Division of Pediatric Neurology, Department of Pediatrics, Vali-e-Asr Hospital, Imam Khomeini Hospital Complex, Tehran University of Medical Sciences, Tehran, Iran
  10. 10. School of Science and Technology, University of Georgia, Tbilisi, Georgia
  11. 11. Child Growth and Development Research Center, Research Institute for Primordial Prevention of Non-Communicable Disease, Isfahan University of Medical Sciences, Isfahan, Iran

Source: European Journal of Neurology Published:2023


Abstract

Background: Since the results of previous studies regarding the safety and efficacy of miglustat in GM2 gangliosidosis (GM2g) were inconsistent, we aimed to assess miglustat therapy in GM2g patients. Methods: This study followed the latest version of PRISMA. We included the observational or interventional studies reporting GM2g patients under miglustat therapy by searching PubMed, Web of Science, and Scopus. Data extracted included the natural history of individual patient data, as well as the safety and efficacy of miglustat in GM2g patients. The quality assessment was performed using the Joanna Briggs Institute Critical Appraisal checklist. Results: A total of 1023 records were identified and reduced to 621 after removing duplicates. After screening and applying the eligibility criteria, 10 articles and 2 abstracts met the inclusion criteria. Overall, the studies represented 54 patients with GM2g under treatment with miglustat and 22 patients with GM2g in the control group. Among patients with available data, 14 and 54 have been diagnosed with Sandhoff disease and Tay-Sachs disease, respectively. Patients included in this review consisted of 23 infantile, 4 late-infantile, 18 juvenile, and 31 adult-onset GM2g. Conclusions: Although miglustat should not be considered a definite treatment for GM2g, it appears that patients, particularly those with infantile or late-infantile GM2g, could benefit from miglustat therapy to some extent. We also make some suggestions regarding future studies presenting their findings in a standard format to facilitate pooling the available data in such rare diseases for a more comprehensive conclusion. © 2023 European Academy of Neurology.
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