Nebulised Hypertonic Saline in Children and Young People With Neuromuscular Disease and Cerebral Palsy

NCT03623698 | Completed Results

• 1 other identifier: 18IC4403
• Study Type: observational
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

Pneumonia, respiratory exacerbations, and chronic pulmonary infection are important causes of emergency admissions, hospitalisations and death in children with Neuromuscular disorders and Cerebral Palsy. Hence, there is a need for research on how to therapeutically aid airway clearance and decrease respiratory exacerbations. Studies have shown that nebulised Hypertonic Saline is well tolerated, reduces pulmonary exacerbations and improves lung function and Lung Clearance Index in patients with Cystic Fibrosis, and enhances mucociliary clearance in asthmatic patients. Nevertheless, to the investigators' knowledge, there is no available data concerning the use of nebulised Hypertonic Saline in the management of children with Neuromuscular disorders and Cerebral Palsy. This study aims to assess the effectiveness of nebulised Hypertonic Saline to decrease hospitalisations and courses of antibiotics in children with Neuromuscular disorders and Cerebral Palsy.

Conditions

Neuromuscular Diseases

Keywords

Hypertonic saline; nebulised; children; young people; adolescents; respiratory

Outcome Measures

Primary Outcomes (2)

• Change in Antibiotic Courses

  Treatments due to respiratory exacerbations

  Change from baseline (before treatment) and 12 months after treatment

• Number of Hospitalsations Due to Respiratory Exacerbations

  Number of respiratory exacerbations that required not planned hospitalisation

  Change from baseline (before treatment) and 12 months after treatment

Secondary Outcomes (13)

• Participant's Perception of Treatment

  At 12 months after starting treatment with hypertonic saline

• Parent's or Legal Guardian's Perception of Treatment

  At 12 months after starting treatment with hypertonic saline

• Score on the Ease of Airway Clearance Pictorial Analogue Scale From Children and Young Adults as Participants

  Change from baseline (before treatment) and 12 months after treatment

• Score on the Ease of Airway Clearance From Parents or Legal Guardians

  Change from baseline (before treatment) and 12 months after treatment

• Apnea Index (AI)

  Change from baseline (before treatment) and 12 months after treatment

Study Arms (2)

Before treatment

Children and young people with neuromuscular disease during the 12 months before being prescribed treatment with nebulised saline (0.9% - 7%)

After treatment

Children and young people with neuromuscular disease during the 12 months after being prescribed treatment with nebulised saline (0.9% - 7%)

Drug: Nebulised hypertonic saline

Interventions

Nebulised hypertonic saline DRUG

Nebulised hypertonic saline used for a period of at least 12 months

Also known as: 3% Sodium Chloride, 5% Sodium Chloride, 7% Sodium Chloride

After treatment

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children with Neuromuscular disease or Cerebral Palsy who have their care at the Royal Brompton Hospital.

You may qualify if:

• Children with Neuromuscular disease or Cerebral Palsy who have been on treatment with nebulised Hypertonic Saline for at least 12 months.

You may not qualify if:

• Children also diagnosed with cystic fibrosis.

Sponsors & Collaborators

• Imperial College London: lead
• Royal Brompton & Harefield NHS Foundation Trust: collaborator

Study Sites (1)

Royal Brompton Hospital

London, SW36NP, United Kingdom

Location

Related Publications (11)

• Allen J. Pulmonary complications of neuromuscular disease: a respiratory mechanics perspective. Paediatr Respir Rev. 2010 Mar;11(1):18-23. doi: 10.1016/j.prrv.2009.10.002. Epub 2009 Dec 2.

  PMID: 20113987 BACKGROUND

• Lo Mauro A, Aliverti A. Physiology of respiratory disturbances in muscular dystrophies. Breathe (Sheff). 2016 Dec;12(4):318-327. doi: 10.1183/20734735.012716.

  PMID: 28210319 BACKGROUND

• Boitano LJ. Management of airway clearance in neuromuscular disease. Respir Care. 2006 Aug;51(8):913-22; discussion 922-4.

  PMID: 16867201 BACKGROUND

• Gerdung CA, Tsang A, Yasseen AS 3rd, Armstrong K, McMillan HJ, Kovesi T. Association Between Chronic Aspiration and Chronic Airway Infection with Pseudomonas aeruginosa and Other Gram-Negative Bacteria in Children with Cerebral Palsy. Lung. 2016 Apr;194(2):307-14. doi: 10.1007/s00408-016-9856-5. Epub 2016 Feb 16.

  PMID: 26883134 BACKGROUND

• Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.

  PMID: 11751186 BACKGROUND

• Bell CF, Kurosky SK, Candrilli SD. Muscular dystrophy-related hospitalizations among male pediatric patients in the United States. Hosp Pract (1995). 2015;43(3):180-5. doi: 10.1080/21548331.2015.1033375. Epub 2015 Apr 1.

  PMID: 25833749 BACKGROUND

• Yuan JX, McGowan M, Hadjikoumi I, Pant B. Do children with neurological disabilities use more inpatient resources: an observational study. Emerg Themes Epidemiol. 2017 Apr 27;14:5. doi: 10.1186/s12982-017-0059-1. eCollection 2017.

  PMID: 28465710 BACKGROUND

• Elkins MR, Bye PT. Mechanisms and applications of hypertonic saline. J R Soc Med. 2011 Jul;104 Suppl 1(Suppl 1):S2-5. doi: 10.1258/jrsm.2011.s11101. No abstract available.

  PMID: 21719889 BACKGROUND

• Elkins MR, Robinson M, Rose BR, Harbour C, Moriarty CP, Marks GB, Belousova EG, Xuan W, Bye PT; National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med. 2006 Jan 19;354(3):229-40. doi: 10.1056/NEJMoa043900.

  PMID: 16421364 BACKGROUND

• Amin R, Subbarao P, Jabar A, Balkovec S, Jensen R, Kerrigan S, Gustafsson P, Ratjen F. Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function. Thorax. 2010 May;65(5):379-83. doi: 10.1136/thx.2009.125831.

  PMID: 20435858 BACKGROUND

• Galaz Souza N, Bush A, Tan HL. Exploratory study of the effectiveness of nebulised saline in children with neurodisability. Eur Respir J. 2021 Mar 18;57(3):2001407. doi: 10.1183/13993003.01407-2020. Print 2021 Mar.

  PMID: 33184120 DERIVED

MeSH Terms

Conditions

Neuromuscular Diseases

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Nervous System Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Limitations and Caveats

This was a retrospective study, with potential for bias from self-selection. Each patient was used as their own control. The use of questionnaires had the potential for recall bias. This study did not have adherence data.

Results Point of Contact

• Title: Mrs. Natalia Galaz Souza
• Organization: Imperial College London

natalia.galaz-souza16@imperial.ac.uk

07751842851

Study Officials

• Natalia Galaz Souza, Professional

  Imperial College London

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2018
• First Posted: August 9, 2018
• Study Start: June 15, 2018
• Primary Completion: August 14, 2019
• Study Completion: September 26, 2019
• Last Updated: September 16, 2020
• Results First Posted: September 16, 2020

Record last verified: 2020-08

Locations

GB (1)