Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy

The Comparison of Pulmonary and Upper Extremity Functions Between Children With Duchenne Muscular Dystrophy and Healthy Peers

Although it is known that the functions of pulmonary and upper limb is affected in late stage of Duchenne Muscular Dystrophy (DMD) negatively, the investigators do not have clear information about its early stage. The aim of this study was to investigate the differences in pulmonary and upper limb functions between children with DMD in early stage and healthy peers.

Study Overview

• Status: Completed
• Conditions: Duchenne Muscular Dystrophy; Upper Limb Function; Respiratory Function Test
• Intervention / Treatment: Other: The Performance of Upper Limb Questionnaire (PUL)

• Study Type: Observational
• Enrollment (Actual): 47

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 8 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Probability Sample

Study Population

Fourty seven participants including 31 children with DMD and 16 healthy peers were included

Description

Inclusion Criteria:

children with DMD was;

• age between 5-10 years
• being on treatment with corticosteroids for more than 6 months
• being at Level I according to Brooke Upper and Lower Extremity Classification Systems

Healthy peers;

• age between 5-10 years
• not having any diagnosed disease

Exclusion Criteria:

• Children has undergo surgery and disease affect upper extremity or pulmonary function in last six months.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patient Group; Children whose ages between 5-10 years with Duchenne Muscular Dystrophy.	Other: The Performance of Upper Limb Questionnaire (PUL); Other Names: Pulmonary Function Test (PFT)
Healthy Group; Healthy peers	Other: The Performance of Upper Limb Questionnaire (PUL); Other Names: Pulmonary Function Test (PFT)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pulmonary Function Test (PFT)	The pulmonary function tests of the participants were evaluated with a spirometry. The child was asked to perform the spirometric tests for three times with maximum effort. The child was motivated before each attempt for stronger and longer expiration.The result of Pulmonary function Test were recorded.	5 minutes
The Performance Upper Limb (PUL)	Upper limb functions of participants were evaluated with The Performance of Upper Limb (PUL) scale. This scale, which was shown to be reliable in DMD, has 22 items in total. It consists of 3 different levels of function: high, mid, and distal. PUL also has four items determining timed performance.	15 minutes

Collaborators and Investigators

• Sponsor: Hacettepe University

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2017
• Primary Completion (Actual): July 31, 2017
• Study Completion (Actual): March 31, 2018

Study Registration Dates

• First Submitted: May 25, 2018
• First Submitted That Met QC Criteria: June 10, 2018
• First Posted (Actual): June 12, 2018

Study Record Updates

• Last Update Posted (Actual): June 12, 2018
• Last Update Submitted That Met QC Criteria: June 10, 2018
• Last Verified: June 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: GO 16/773

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

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