Exercise Capacity, Physical Activity Level and Quality of Life in Children With Primary Immunodeficiency

NCT ID: NCT05999422

Last Updated: 2023-08-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

52 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-08-01

Study Completion Date

2024-10-31

Brief Summary

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This study aims to compare the maximal exercise capacity, functional exercise capacity, respiratory functions, physical activity level, quality of life, respiratory muscle strength and endurance, peripheral muscle strength, muscle oxygenation and dyspnea in children with primary immunodeficiency (PID) and healthy individuals. The study was designed as a cross-sectional study on 26 patients diagnosed with primary immunodeficiency and 26 age- and sex-matched healthy individuals. Differences between both groups will be evaluated statistically. The results of this study aim to reveal how primary immunodeficiency patients are affected by factors such as respiratory functions, physical activity level and quality of life

Detailed Description

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Primary immune deficiencies (PIDs) are a heterogeneous group of diseases resulting from various abnormalities that affect the development, differentiation, and/or function of cells and components of the immune system. PIDs are clinically characterized by increased susceptibility to infections, autoimmune diseases, auto-inflammatory disorders, allergies, bone marrow failure, and/or malignancies. Although individually rare, the total number of individuals with PIDs constitutes a significant health burden. The prevalence and distribution of PIDs vary among populations, with an estimated prevalence of 1/1,000 to 1/5,000. The specific prevalence of PIDs in Turkey is not well known due to the lack of extensive studies and a national registry system. However, a study encompassing two centers in Turkey reported a prevalence of 30.5/100,000. Currently, approximately 500 PID diseases have been identified, with selective immunoglobulin A (IgA) deficiency being the most common and common variable immunodeficiency (CVID) being the most common symptomatic PID.

Pulmonary complications are highly prevalent among PID patients and significantly contribute to morbidity and mortality. Recurrent respiratory tract infections often serve as the initial warning sign in some PIDs and are a leading cause of mortality in adult PID patients. The presence of two or more pneumonia episodes per year is considered one of the ten warning signs of PID. Acute and chronic infections primarily constitute respiratory system diseases, while non-infectious respiratory complications include asthma, bronchiectasis, bronchiolitis obliterans, interstitial lung disease, granulomatous lung disease, and malignancies. These diseases significantly impact the quality of life of PID patients, limit their working abilities, and restrict their physical and social activities. Health-related quality of life in PID patients is also significantly affected by delays in the diagnosis and treatment of infections.

As survival from infections improves, non-infectious pulmonary complications become more common in PID patients. Particularly in PIDs characterized by antibody deficiencies, permanent lung damage is observed in 20-40% of patients.

Physical activity levels are also affected in PID patients, but there are only survey studies on this topic. Therefore, it is expected that respiratory muscle strength, as well as respiratory muscle endurance, functional exercise capacity, and peripheral muscle strength, would be affected in these patients. There is no research available on maximal exercise capacity, functional exercise capacity, respiratory muscle strength and endurance, and peripheral muscle strength.

The aim of this study is to compare the maximal exercise capacity, functional exercise capacity, respiratory functions, physical activity level, quality of life, respiratory muscle strength and endurance, peripheral muscle strength, muscle oxygenation and dyspnea in patients with primary immune deficiencies with healthy individuals.

Conditions

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Primary Immunodeficiency Diseases

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Patients with primary immunodeficiency

Maximal exercise capacity measured with Cardiopulmonary exercise testing, functional exercise capacity with six minute walk test, pulmonary function using spirometry, respiratory muscle strength using mouth pressure device, peripheral muscle strength using hand held dynamometer, muscle oxygenation using Moxy monitor, respiratory muscle endurance using incremental threshold loading test, life quality using "Pediatric Quality of Life Inventory™ 4.0 (PedsQL™ 4.0)" (Turkish version).

No interventions assigned to this group

Healthy control

Maximal exercise capacity measured with Cardiopulmonary exercise testing, functional exercise capacity with six minute walk test, pulmonary function using spirometry, respiratory muscle strength using mouth pressure device, peripheral muscle strength using hand held dynamometer, muscle oxygenation using Moxy monitor, respiratory muscle endurance using incremental threshold loading test, life quality using "Pediatric Quality of Life Inventory™ 4.0 (PedsQL™ 4.0)" (Turkish version).

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

Patients:

* Individuals aged 6-18 years, diagnosed with primary immune deficiency disease and receiving standard medical treatment, will be included in the study.

Healthy controls:

* Individuals between the ages of 6 and 18 without a known chronic disease will be included.

Exclusion Criteria

Patients:

* Patients who are uncooperative, have orthopedic or neurological disorders that will affect their exercise capacity, and have pneumonia or any acute infection during the evaluation will be excluded from the study.

Healthy controls:

* Those with a known chronic disease, uncooperative and orthopedic or neurological disorders that will affect their exercise capacity will not be included.
Minimum Eligible Age

6 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Gazi University

OTHER

Sponsor Role lead

Responsible Party

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Meral Boşnak Güçlü

Study director, PT, PhD, Prof. Dr. Faculty of Health Sciences, Department of Physiotherapy and Rehabilitation, Head of Cardiopulmonary Rehabilitation Clinic

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Meral Boşnak Güçlü, Prof.Dr.

Role: STUDY_DIRECTOR

Gazi University

Riad Bejta, B.sc PT

Role: STUDY_CHAIR

Gazi University

Caner Aytekin, Assoc. Prof

Role: PRINCIPAL_INVESTIGATOR

MH. Dr. Sami Ulus Children's Health and Diseases Training and Research Hospital

Locations

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Gazi University, Faculty of Health Sciences, Department of Physiotherapy and Rehabilitation, Cardiopulmonary Rehabilitation Unit

Ankara, Çankaya, Turkey (Türkiye)

Site Status RECRUITING

Countries

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Turkey (Türkiye)

Central Contacts

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Meral Boşnak Güçlü, Prof.Dr.

Role: CONTACT

+903122162647

Riad Bejta, B.sc PT

Role: CONTACT

+905466559427

Facility Contacts

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Meral BOŞNAK GÜÇLÜ, Prof.Dr.

Role: primary

+903122162647

Riad Bejta, B.sc PT

Role: backup

+905466559427

References

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Reference Type BACKGROUND
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Kilic SS, Ozel M, Hafizoglu D, Karaca NE, Aksu G, Kutukculer N. The prevalences [correction] and patient characteristics of primary immunodeficiency diseases in Turkey--two centers study. J Clin Immunol. 2013 Jan;33(1):74-83. doi: 10.1007/s10875-012-9763-3. Epub 2012 Sep 15.

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Hampson FA, Chandra A, Screaton NJ, Condliffe A, Kumararatne DS, Exley AR, Babar JL. Respiratory disease in common variable immunodeficiency and other primary immunodeficiency disorders. Clin Radiol. 2012 Jun;67(6):587-95. doi: 10.1016/j.crad.2011.10.028. Epub 2012 Jan 9.

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PMID: 22226567 (View on PubMed)

Verma N, Grimbacher B, Hurst JR. Lung disease in primary antibody deficiency. Lancet Respir Med. 2015 Aug;3(8):651-60. doi: 10.1016/S2213-2600(15)00202-7. Epub 2015 Jul 15.

Reference Type BACKGROUND
PMID: 26188881 (View on PubMed)

Modell V, Orange JS, Quinn J, Modell F. Global report on primary immunodeficiencies: 2018 update from the Jeffrey Modell Centers Network on disease classification, regional trends, treatment modalities, and physician reported outcomes. Immunol Res. 2018 Jun;66(3):367-380. doi: 10.1007/s12026-018-8996-5.

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Quinti I, Di Pietro C, Martini H, Pesce AM, Lombardi F, Baumghartner M, Colantuono S, Milito C, Tabolli S. Health related quality of life in common variable immunodeficiency. Yonsei Med J. 2012 May;53(3):603-10. doi: 10.3349/ymj.2012.53.3.603.

Reference Type BACKGROUND
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Routes J, Costa-Carvalho BT, Grimbacher B, Paris K, Ochs HD, Filipovich A, Hintermeyer M, de Melo KM, Workman S, Ito D, Ye X, Bonnet P, Li-McLeod J. Health-Related Quality of Life and Health Resource Utilization in Patients with Primary Immunodeficiency Disease Prior to and Following 12 Months of Immunoglobulin G Treatment. J Clin Immunol. 2016 Jul;36(5):450-61. doi: 10.1007/s10875-016-0279-0. Epub 2016 Apr 18.

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Urschel S, Kayikci L, Wintergerst U, Notheis G, Jansson A, Belohradsky BH. Common variable immunodeficiency disorders in children: delayed diagnosis despite typical clinical presentation. J Pediatr. 2009 Jun;154(6):888-94. doi: 10.1016/j.jpeds.2008.12.020. Epub 2009 Feb 23.

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Azizi G, Abolhassani H, Asgardoon MH, Alinia T, Yazdani R, Mohammadi J, Rezaei N, Ochs HD, Aghamohammadi A. Autoimmunity in common variable immunodeficiency: epidemiology, pathophysiology and management. Expert Rev Clin Immunol. 2017 Feb;13(2):101-115. doi: 10.1080/1744666X.2016.1224664. Epub 2016 Sep 16.

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Manson D, Reid B, Dalal I, Roifman CM. Clinical utility of high-resolution pulmonary computed tomography in children with antibody deficiency disorders. Pediatr Radiol. 1997 Oct;27(10):794-8. doi: 10.1007/s002470050235.

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Sowers KL, Litwin BA, Lee ACW, Galantino MLA. Exercise Perception and Behaviors in Individuals Living with Primary Immunodeficiency Disease. J Clin Immunol. 2018 Feb;38(2):174-184. doi: 10.1007/s10875-017-0472-9. Epub 2018 Jan 6.

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Other Identifiers

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Gazi University 55

Identifier Type: -

Identifier Source: org_study_id

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