Clinical trial NCT04491747: Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients
Official title: Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Mucopolysaccharidoses
Keywords: Respiratory muscles
exercise test
Lead sponsor: Hacettepe University
Sponsor class: OTHER
First posted: 2020-07-29
Enrollment: 24
Sex: ALL
Minimum age (years): 7
Maximum age (years): 30
Standard ages: CHILD
ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: People who diagnosed Mucopolysaccharidoses disease in Hacettepe University were recruited the study.

Brief summary: 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.

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.

Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

* Patients who were not walking ability.

Primary outcomes:
measure: functional capacity
timeFrame: 15 minutes
measure: 3 minute step test
timeFrame: 5 minutes
measure: Respiratory muscle strength assessment
timeFrame: 15 minutes
measure: Respiratory muscle endurance
timeFrame: 10 minutes
measure: Pulmonary functions
timeFrame: 15 minutes
measure: Pulmonary functions
timeFrame: 15 minutes
measure: Pulmonary functions
timeFrame: 15 minutes
measure: MVV (Maximal ventilatory assesssment)
timeFrame: 5 minutes
measure: cough effectiveness
timeFrame: 5 minutes
Secondary outcomes:
measure: heart rate
timeFrame: 6 minutes
measure: oxygen saturation
timeFrame: 6 minutes
Other outcomes:
Locations:
city: Ankara
country: Turkey
facility: Hacettepe University
Study officials:
affiliation: Hacettepe University
name: Deniz Dogru- Ersoz, Professor
role: STUDY_DIRECTOR
affiliation: Hacettepe University
name: Naciye Vardar- Yagli, PhD
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Dilber Ademhan- Tural, MD
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Nural Kiper, Professor
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Ugur Ozcelik, Professor
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Ebru Yalcin, Professor
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Deniz Inal-Ince, Professor
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Melda Saglam, PhD
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Serap Sivri, Professor
role: STUDY_CHAIR
affiliation: Hacettepe University
name: Aysegul Tokatlı, Professor
role: STUDY_CHAIR
References: