Meta AnalysisID 7847

黏多糖贮积症患者不同治疗策略的安全性、疗效和有效性结局:随机对照试验的系统评价和网状Meta分析

CRD42023470374

Safety, efficacy, and effectiveness outcomes of different treatment strategies in patients with mucopolysaccharidosis:a systematic review and network meta-analysis of randomized controlled trials

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Record Fields

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Meta Analysis Id
7847
Evidence Id
16405
Core Evidence Id
16405
Source Meta Analysis Id
7845
Herb2 Meta Analysis Id
HBMA007845
Crd Id
CRD42023470374
Title
Safety, efficacy and effectiveness outcomes of different treatment strategies in mucopolysaccharidosis patients: a systematic review and network meta-analysis of randomized control trials.
Review Question
Safety, efficacy, and effectiveness outcomes of different treatment strategies in patients with mucopolysaccharidosis:a systematic review and network meta-analysis of randomized controlled trials
Study Type Included
randomized controlled trials
Condition Being Studied
Mucopolysaccharidosis(MPS) is a lysosomal accumulation disease caused by defects in lysosomal hydrolases and inhibiting the degradation of acidic mucopolysaccharides (glucosaminoglycans), which leads to the accumulation of mucopolysaccharides in the body and results in a series of clinical symptoms. Patients with mucopolysaccharide storage syndrome have some clinical symptoms and imaging features due to excessive accumulation of mucopolysaccharides in tissues or organs such as bone and cartilage, which affects the normal development of these tissues or organs. Mucopolysaccharide storage syndrome is a congenital or primary metabolic disorder syndrome.
Participant
Patients with mucopolysaccharidosis that included in randomized studies.
Animal
Human Disease Modelled
Intervention
Enzyme replacement therapies or other disease treatment therapies.
Comparator Control
Placebo or no treatment.
Main Outcome
The primary efficacy endpoint variable were the 3MSC, urine GAG and 12MWT. Measures of effect Continuous variable and incidence rate.
Outcome Measure
Additional Outcome
Secondary efficacy endpoints included cognitive function and respiratory function. Measures of effect Continuous variable and incidence rate.
Study Method
Meta-analysis荟萃分析, Systematic review系统评价
Keyword
MeSH headings have not been applied to this record
Contact
Lingling Huang [email protected]
Organisational Affiliation
Southeast University
Funding Source
Research on Data Mining and Risk Prediction of Pharmacovigilance (Project ref 22/05/7).Funding provided by Shenzhen Institute of Pharmacovigilance and Risk Management Institute. Grant number(s) State the funder, grant or award number and the date of award
Other Selection Criteria
Final Publication
Same Topic Review
Published Protocol
Review Type
Language
English
Country
China
Review Stage
Review Ongoing
First Submission Date
2023-10-21
Registration Date
2023-11-01
Anticipated Start Date
2023-10-21
Anticipated Completion Date
2023-12-31
Title Cn
黏多糖贮积症患者不同治疗策略的安全性、疗效和有效性结局:随机对照试验的系统评价和网状Meta分析
Title En
Safety, efficacy and effectiveness outcomes of different treatment strategies in mucopolysaccharidosis patients: a systematic review and network meta-analysis of randomized control trials.
Bilingual Status
complete