Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis
Mucopolysaccharidosis type IVA (MPS IVA or Morquio A), a lysosomal storage disease with an autosomal recessive inherited pattern, is induced by <i>GALNS</i> gene mutations causing deficiency in N-acetylgalactosamine-6-sulfatase activity (GALNS; EC 3.1.6.4). Currently, intravenous (IV) en...
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MDPI AG
2022-08-01
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| Series: | Journal of Personalized Medicine |
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| Online Access: | https://www.mdpi.com/2075-4426/12/8/1338 |
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| author | Chung-Lin Lee Chih-Kuang Chuang Yu-Min Syu Huei-Ching Chiu Yuan-Rong Tu Yun-Ting Lo Ya-Hui Chang Hsiang-Yu Lin Shuan-Pei Lin |
| author_facet | Chung-Lin Lee Chih-Kuang Chuang Yu-Min Syu Huei-Ching Chiu Yuan-Rong Tu Yun-Ting Lo Ya-Hui Chang Hsiang-Yu Lin Shuan-Pei Lin |
| author_sort | Chung-Lin Lee |
| collection | DOAJ |
| description | Mucopolysaccharidosis type IVA (MPS IVA or Morquio A), a lysosomal storage disease with an autosomal recessive inherited pattern, is induced by <i>GALNS</i> gene mutations causing deficiency in N-acetylgalactosamine-6-sulfatase activity (GALNS; EC 3.1.6.4). Currently, intravenous (IV) enzyme replacement therapy (ERT) with elosulfase alfa is employed for treating MPS IVA patients. A systematic literature review was conducted to evaluate the efficacy and safety of IV elosulfase alfa for MPS IVA by searching the National Center for Biotechnology Information, U.S. National Library of Medicine National Institutes of Health (PubMed), Excerpta Medica dataBASE, and Cochrane Library databases, limited to clinical trials. Four cohort studies and two randomized controlled trials, with a total of 550 participants (327 on ERT treatment versus 223 on placebo treatment), satisfied the inclusion criteria. Pooled analysis of proportions and confidence intervals were also utilized to systematically review clinical cohort studies and trials. Per the pooled proportions analysis, the difference in means of urinary keratan sulfate (uKS), 6-min walk test, 3-min stair climb test, self-care MPS-Health Assessment Questionnaire, caregiver assistance and mobility, forced vital capacity, the first second of forced expiration, and maximal voluntary ventilation between the ERT and placebo treatment groups were −0.260, −0.102, −0.182, −0.360, −0.408, −0.587, −0.293, −0.311, and −0.213, respectively. Based on the currently available data, our meta-analysis showed that there is uKS, physical performance, quality of life, and respiratory function improvements with ERT in MPS IVA patients. It is optimal to start ERT after diagnosis. |
| first_indexed | 2024-03-09T04:14:35Z |
| format | Article |
| id | doaj.art-f6f61dbe87cc47cdb3580212d0ebbe29 |
| institution | Directory Open Access Journal |
| issn | 2075-4426 |
| language | English |
| last_indexed | 2024-03-09T04:14:35Z |
| publishDate | 2022-08-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Personalized Medicine |
| spelling | doaj.art-f6f61dbe87cc47cdb3580212d0ebbe292023-12-03T13:56:32ZengMDPI AGJournal of Personalized Medicine2075-44262022-08-01128133810.3390/jpm12081338Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-AnalysisChung-Lin Lee0Chih-Kuang Chuang1Yu-Min Syu2Huei-Ching Chiu3Yuan-Rong Tu4Yun-Ting Lo5Ya-Hui Chang6Hsiang-Yu Lin7Shuan-Pei Lin8Department of Pediatrics, MacKay Memorial Hospital, Taipei 10449, TaiwanDivision of Genetics and Metabolism, Department of Medical Research, MacKay Memorial Hospital, Taipei 10449, TaiwanDepartment of Pediatrics, MacKay Memorial Hospital, Taipei 10449, TaiwanDepartment of Pediatrics, MacKay Memorial Hospital, Taipei 10449, TaiwanDivision of Genetics and Metabolism, Department of Medical Research, MacKay Memorial Hospital, Taipei 10449, TaiwanDepartment of Rare Disease Center, MacKay Memorial Hospital, Taipei 10449, TaiwanDepartment of Pediatrics, MacKay Memorial Hospital, Taipei 10449, TaiwanDepartment of Pediatrics, MacKay Memorial Hospital, Taipei 10449, TaiwanDepartment of Pediatrics, MacKay Memorial Hospital, Taipei 10449, TaiwanMucopolysaccharidosis type IVA (MPS IVA or Morquio A), a lysosomal storage disease with an autosomal recessive inherited pattern, is induced by <i>GALNS</i> gene mutations causing deficiency in N-acetylgalactosamine-6-sulfatase activity (GALNS; EC 3.1.6.4). Currently, intravenous (IV) enzyme replacement therapy (ERT) with elosulfase alfa is employed for treating MPS IVA patients. A systematic literature review was conducted to evaluate the efficacy and safety of IV elosulfase alfa for MPS IVA by searching the National Center for Biotechnology Information, U.S. National Library of Medicine National Institutes of Health (PubMed), Excerpta Medica dataBASE, and Cochrane Library databases, limited to clinical trials. Four cohort studies and two randomized controlled trials, with a total of 550 participants (327 on ERT treatment versus 223 on placebo treatment), satisfied the inclusion criteria. Pooled analysis of proportions and confidence intervals were also utilized to systematically review clinical cohort studies and trials. Per the pooled proportions analysis, the difference in means of urinary keratan sulfate (uKS), 6-min walk test, 3-min stair climb test, self-care MPS-Health Assessment Questionnaire, caregiver assistance and mobility, forced vital capacity, the first second of forced expiration, and maximal voluntary ventilation between the ERT and placebo treatment groups were −0.260, −0.102, −0.182, −0.360, −0.408, −0.587, −0.293, −0.311, and −0.213, respectively. Based on the currently available data, our meta-analysis showed that there is uKS, physical performance, quality of life, and respiratory function improvements with ERT in MPS IVA patients. It is optimal to start ERT after diagnosis.https://www.mdpi.com/2075-4426/12/8/1338mucopolysaccharidosis type IVAMorquio Aenzyme replacement therapyelosulfase alfameta-analysis |
| spellingShingle | Chung-Lin Lee Chih-Kuang Chuang Yu-Min Syu Huei-Ching Chiu Yuan-Rong Tu Yun-Ting Lo Ya-Hui Chang Hsiang-Yu Lin Shuan-Pei Lin Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis Journal of Personalized Medicine mucopolysaccharidosis type IVA Morquio A enzyme replacement therapy elosulfase alfa meta-analysis |
| title | Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis |
| title_full | Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis |
| title_fullStr | Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis |
| title_full_unstemmed | Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis |
| title_short | Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis |
| title_sort | efficacy of intravenous elosulfase alfa for mucopolysaccharidosis type iva a systematic review and meta analysis |
| topic | mucopolysaccharidosis type IVA Morquio A enzyme replacement therapy elosulfase alfa meta-analysis |
| url | https://www.mdpi.com/2075-4426/12/8/1338 |
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