Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients

Respiratory system affects in mucopolysaccharidosis (MPS) disease. Respiratory system symptoms are seen in almost every MPS patients and respiratory failure is one of the most common causes of death in this population. The aim of the study was to evaluate respiratory muscle strength and endurance, cough effectiveness and functional capacity in MPS patients and to defined the factors that affected the respiratory problem in this.

Study Overview

• Status: Completed
• Conditions: Mucopolysaccharidoses
• Intervention / Treatment: Diagnostic test: functional capacity

Detailed Description

This study was participated MPS patients. Patients' pulmonary function test, functional capacity, respiratory muscle strength and endurance, peak cough flow was assessed and recorded.

Pulmonary function test was assessed with spirometry, functional capacity was evaluated with 3-minute step test and six minute walk test (MWT). Respiratory muscle strength was evaluated mouth pressure measurement (MIP- maximal inspiratory pressure and MEP- maximal expiratory pressure) and Sniff nasal inspiratory pressure (SNIP). Respiratory muscle endurance was assessed Powerbreathe. And cough effectiveness was evaluated peak cough flow.

• Study Type: Observational
• Enrollment (Actual): 24

Contacts and Locations

Study Locations

• Turkey
  • Ankara, Turkey
    • Hacettepe University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 30 years (Child, Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

People who diagnosed Mucopolysaccharidoses disease in Hacettepe University were recruited the study.

Description

Inclusion Criteria:

• People who diagnosed Mucopolysaccharidoses disease in Hacettepe University.
• Cooperation

Exclusion Criteria:

• Patients who were not walking ability.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
functional capacity	Six minute walk test: Distance walked in six minute was recorded	15 minutes
3 minute step test	number of completed step in 3 minute was recorded	5 minutes
Respiratory muscle strength assessment	Respiratory muscle strength was evaluated by measuring maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) and sniff nasal inspiratory pressure(SNIP). Also dynamic inspiratory muscle strength was evaluated Powerbreathe K5 device.	15 minutes
Respiratory muscle endurance	Pressure time unit: A constant load test was used to evaluate respiratory muscle endurance. Using a PowerBreathe device (HaB International Ltd., Southam, UK), the participants were asked to breathe maintaining a constant threshold (60% of MIP) until task failure, and the test duration was recorded. Results were obtained by multiplying the set pressure by the test duration	10 minutes
Pulmonary functions	FVC (Forced vital capacity) assessment via spirometry	15 minutes
Pulmonary functions	FEV 1(forced expiratory volume in first second) assessment via spirometry	15 minutes
Pulmonary functions	PEF (peak expiratory flow) assessment via spirometry	15 minutes
MVV (Maximal ventilatory assesssment)	Maximize ventilation by inhaling and exhaling as quickly and deeply as possible for 15 s via spirometry	5 minutes
cough effectiveness	cough effectiveness assessment was evaluated peak cough flow meter.	5 minutes

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
heart rate	difference pre and post test heart beat number	6 minutes
oxygen saturation	difference pre and post test percentage of oxygen saturation	6 minutes

Collaborators and Investigators

• Sponsor: Hacettepe University
• Investigators: Study Director: Deniz Dogru- Ersoz, Professor, Hacettepe University; Study Chair: Naciye Vardar- Yagli, PhD, Hacettepe University; Study Chair: Dilber Ademhan- Tural, MD, Hacettepe University; Study Chair: Nural Kiper, Professor, Hacettepe University; Study Chair: Ugur Ozcelik, Professor, Hacettepe University; Study Chair: Serap Sivri, Professor, Hacettepe University; Study Chair: Aysegul Tokatlı, Professor, Hacettepe University

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2019
• Primary Completion (Actual): February 1, 2020
• Study Completion (Actual): February 1, 2020

Study Registration Dates

• First Submitted: July 19, 2020
• First Submitted That Met QC Criteria: July 27, 2020
• First Posted (Actual): July 29, 2020

Study Record Updates

• Last Update Posted (Actual): July 29, 2020
• Last Update Submitted That Met QC Criteria: July 27, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Keywords: exercise test; Respiratory muscles
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mucopolysaccharidoses
• Other Study ID Numbers: GO 19/97

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No