Two Different Treatment Modalities in Patients With Spinal Muscular Atrophy

Summary

Spinal Muscular Atrophy (SMA) is a severe neuromuscular disorder characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive muscle atrophy and weakness, particularly in proximal and axial muscles. SMA causes respiratory muscle weakness, recurrent infections, and nocturnal hypoventilation, contributing significantly to morbidity and mortality. Children with SMA often display respiratory and trunk muscle weakness compared to healthy controls. Our project aims to investigate the impact of pulmonary rehabilitation, including inspiratory muscle training, along with trunk control exercises in children with SMA. The study will include 40 SMA patients aged 5-18, with maximum inspiratory capacity below 60 centimeters of water (cmH2O), and the ability to sit unsupported for more than 5 seconds. The participants will be randomly assigned to two groups: Pulmonary Rehabilitation Group (Group 1, n=20) and Trunk Control Training Group (Group 2, n=20). Group 1 will undergo breathing exercises and inspiratory muscle training (IMT), involving diaphragmatic, pursed-lip, and segmental breathing. IMT will be administered with a portable device, starting at appropriate resistance and consisting of 10 cycles, 10 minutes each, once a day, with designated rest intervals. Also applied by calculating 30% of the maximal inspiratory pressure (MIP). During weekly clinic visits, the MIP value will be recalculated and the current threshold pressure value will be determined. In Group 2, alongside pulmonary rehabilitation, children will engage in trunk control exercises, progressively increasing in difficulty, focusing on pelvic control, proximal stabilization, and strengthening trunk and gluteal muscles. All interventions will be performed in front of a mirror. At the end of the 8-week intervention, MIP and Maximal Expiratory Pressure (MEP) will be used to measure respiratory muscle performance, spirometry will be used to monitor lung volume changes, and Peak Cough Flow will be used to evaluate the effectiveness of cough. The Trunk Control Measurement Scale, the Revised Upper Extremity Module, and the Children's Quality of Life Scale will assess trunk control, upper extremity functions, and quality of life, respectively. The Hammersmith Functional Motor Scale will assess gross motor functions and the Zarit Caregiver Burden Scale will inquire about familial factors affecting the child.

Conditions

• Muscular Atrophy, Spinal

Interventions

OTHER

Pulmonary Rehabilitation

Pulmonary rehabilitation consists of diaphragmatic, pursed-lip, and segmental breathing techniques, and also included inspiratory muscle training (will adjust 30% of maximal inspiratory pressure value)

OTHER

Trunk Control Training

Trunk Control Exercises will be based on the neurodevelopmental process. Exercises will progressively include activities such as stretching, turning, etc. while sitting on different surfaces (firm/soft).

Sponsors & Collaborators

• The Scientific and Technological Research Council of Turkey

  collaborator OTHER

• Istanbul Medipol University Hospital

  lead OTHER

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

5 Years

Max Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2024-02-21

Primary Completion

2024-12-29

Completion

2024-12-29

Countries

• Turkey (Türkiye)

Study Locations