The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy

NCT ID: NCT05878418

Last Updated: 2024-09-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-10-11
• Study Completion Date: 2024-09-10

Brief Summary

Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation. The main problems are posture disorders, scoliosis, pelvic curvature, contracture, hip dislocation, foot and chest deformities. In this study, examining the effectiveness of trunk support used to alleviate the progression of scoliosis in children diagnosed with SMA Type I will contribute to the current literature.In addition to Individualized Trunk Exercises (ITE), Individualized Pulmonary Rehabilitation (IPR) and Chest Care (CC) Programme, the use of thoracolumbosacral spinal orthosis in Type I children will be used for the first time in our country and in the world literature. SMA. Our aim in the project is to examine the effectiveness of this treatment program on the motor functions, scoliosis Cobb angle, pelvic curvature and chest deformity of children with Type I SMA.The project is planned to be carried out with children diagnosed with Type I SMA who are followed up at Medipol Mega University Hospital Pediatric Chest Diseases Polyclinic.In evaluating the development of scoliosis as the primary outcome measure; Radiological evaluation (Cobb Angle) and examination of chest deformity; Lung X-ray (Basal Chest Wall Upper-Lower Ratio Measurement) will be used. As secondary outcome measures, the Children's Hospital of Philadelphia Infant Test for Neuromuscular Disorders and the Hammersmith Functional Motor Scale Expanded were used to assess motor functions and examine the level of motor development; In the World Health Organization Motor Development Scale body posture assessment; Supine Trunk Rotation Angle Test and Pelvic Curvature Test, pulse oximetry to assess oxygenation; In determining the level of satisfaction with orthosis use; Children/families' information will be questioned through the Quebec Assistive Technology User Satisfaction Evaluation Survey and Personal Information Form.The active control group will receive the ITE, IPR and CC program as a home program and once a week in the outpatient clinic for 8 weeks, 7 days a week, once a day, each session being 50-60 minutes. In the ITE-IPR-CC + spine orthosis group, in addition to the control group program, a thoracolumbosacral spine orthosis specially designed for the child will be used every day of the week and 8 hours a day for 8 weeks. Evaluations will be made at baseline and at week 8.

Detailed Description

With the increase in scoliosis and scoliosis-related symptoms after increased survival in patients with Type I SMA, the need for physiotherapy applications has become more important than ever (Trenkle, et al., 2021; Mercuri, et al., 2018). The presence of scoliosis significantly affects lung capacity, postural control, functionality and quality of life in patients with Type I SMA. For this reason, it is of great importance to treat these patients in a timely and effective manner. There is no consensus on the type of spinal orthosis and application protocol to be used in patients with SMA (Mercuri, et al., 2018). When the literature was examined, no study was found about the effect of spinal orthosis use on scoliosis and chest deformity in Type I SMA patients.

Conditions

Spinal Muscular Atrophy Type I; Scoliosis; Chest Deformities; Spinal Orthosis; Pulmonary Rehabilitation; Exercise

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

Trunk Exercises, Pulmonary Rehabilitation and Pulmonary Care Program + Spinal Orthosis Group

Trunk Exercises, Pulmonary Rehabilitation and Pulmonary Care Program + Toracolumbosacral Spinal Orthosis

Group Type: EXPERIMENTAL

Thoracolumbosacral Spinal Orthosis (TLSO)

Intervention Type: DEVICE

In the TE, PR, PC + spinal orthosis group, in addition to the control group program, a thoracolumbosacral spinal orthosis (TLSO) designed specifically for the child will be used for 8 hours a day for 8 weeks, every day of the week.

Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)

Intervention Type: OTHER

The active control group will receive a TE, PR and PC program in the outpatient clinic once a week for 8 weeks, 7 days a week, once a day, 50-60 minutes per session, for 8 weeks.

Trunk Exercises, Pulmonary Rehabilitation and Pulmonary Care Program Group

Trunk Exercises, Pulmonary Rehabilitation and Pulmonary Care Program

Group Type: ACTIVE_COMPARATOR

Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)

Intervention Type: OTHER

The active control group will receive a TE, PR and PC program in the outpatient clinic once a week for 8 weeks, 7 days a week, once a day, 50-60 minutes per session, for 8 weeks.

Interventions

Thoracolumbosacral Spinal Orthosis (TLSO)

In the TE, PR, PC + spinal orthosis group, in addition to the control group program, a thoracolumbosacral spinal orthosis (TLSO) designed specifically for the child will be used for 8 hours a day for 8 weeks, every day of the week.

Intervention Type: DEVICE

Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)

The active control group will receive a TE, PR and PC program in the outpatient clinic once a week for 8 weeks, 7 days a week, once a day, 50-60 minutes per session, for 8 weeks.

Intervention Type: OTHER

Other Intervention Names

Pulmonary Rehabilitation; Pulmonary Care; Trunk Exercise

Eligibility Criteria

Inclusion Criteria

• 2-6 years old,
• Clinically and genetically diagnosed as Type I SMA,
• Having scoliosis (20-40 Cobb angle),
• Children who have not had any previous spinal surgery,
• Children who have received or continue to receive their medical treatments (nusinersen and onasemnogene abeparvovec).

Exclusion Criteria

• Having acute respiratory failure and/or serious airway infections,
• 24-hour mechanical ventilation dependency,
• Continuing medical treatment in intensive care,
• Having other orthopedic and neurological problems,
• Children of parents who did not agree to participate in the study,
• Children whose chest expansion and/or oxygenation are at risk of being affected and who cannot tolerate the orthosis,
• Children and their parents who do not use the thoracolumbosacral spinal orthosis in accordance with the study protocol and do not comply with trunk exercises, pulmonary rehabilitation and pulmonary care practices.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Medipol University

OTHER

Sponsor Role: lead

Responsible Party

Emre DANSUK

MSc, Physiotherapist

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Emre Dansuk, MSc

Role: PRINCIPAL_INVESTIGATOR

Medipol University

Locations

Istanbul Medipol University

Istanbul, , Turkey (Türkiye)

Countries

Turkey (Türkiye)

References

Dansuk E, Van Den Berg ANT, Ata G, Yikilmaz SK, Oktem S. The effect of thoracolumbosacral orthosis on scoliosis progression and chest deformity in children with type 1 spinal muscular atrophy: A randomized controlled trial. PLoS One. 2025 Sep 15;20(9):e0323341. doi: 10.1371/journal.pone.0323341. eCollection 2025.

Reference Type: DERIVED

PMID: 40953000 (View on PubMed)

Other Identifiers

E-10840098-772.02-200

Identifier Type: -

Identifier Source: org_study_id