Global epidemiology of mucopolysaccharidosis type III (Sanfilippo syndrome): an updated systematic review and meta-analysis

Author:

Kong Weijing1,Wu Shanshan23,Zhang Jing1,Lu Cheng4,Ding Yingxue1,Meng Yan5ORCID

Affiliation:

1. Department of Pediatrics , Beijing Friendship Hospital, Capital Medical University , Beijing , China

2. Department of Clinical Epidemiology and EBM , Beijing Friendship Hospital, Capital Medical University , Beijing , China

3. Clinical Research Center for Digestive Diseases , Beijing Friendship Hospital, Capital Medical University , Beijing , China

4. Beijing Hong Jian Medical Device Company , Beijing , China

5. Department of Pediatrics , Chinese PLA General Hospital , Beijing , China

Abstract

Abstract Objectives Mucopolysaccharidosis III, an autosomal recessive lysosomal storage disorder, is characterized by progressive mental retardation and behavioral problems. Meta-analysis of global mucopolysaccharidosis III epidemiology, which serves as a fundamental reference for public health decision-making, was not available prior to this study. To provide a systematic review and meta-analysis of birth prevalence of mucopolysaccharidosis III in multiple countries. Methods MEDLINE and EMBASE databases were searched for original research articles on the epidemiology of mucopolysaccharidosis III from inception until 1st July, 2020. A checklist adapted from STROBE (STrengthening the Reporting of OBservational studies in Epidemiology) was used to assess the quality of all studies involved. Meta-analysis, adopting a random effects logistic model, was performed to estimate pooled birth prevalence of mucopolysaccharidosis III and its subtypes. Results Twenty-five studies screened out of 1,826 records were included for data extraction. The pooled global mucopolysaccharidosis III birth prevalence was 0.76 cases (95% CI: 0.57–0.96) per 100,000 live births. The pooled global birth prevalence of mucopolysaccharidosis III subtypes (A, B, and C) was 0.52 cases (95% CI: 0.33–0.72), 0.21 cases (95% CI: 0.12–0.30) and 0.01 cases (95% CI: 0.005–0.02) per 100,000 live births, respectively. Conclusions Based on the global population size (7.8 billion) and the life span of patients, there would be 12–19 thousand mucopolysaccharidosis III patients worldwide. To our knowledge, this is the first comprehensive systematic review that presented quantitative data fundamental for evidence-based public health decision-making by evaluating global epidemiology of mucopolysaccharidosis III.

Publisher

Walter de Gruyter GmbH

Subject

Endocrinology,Endocrinology, Diabetes and Metabolism,Pediatrics, Perinatology, and Child Health

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