VO2max & HRQoL in Children With Sickle Cell Disease

Children Clinical Trial

— VO2drépano  

Official title:

Aerobic Physical Fitness and Health-related Quality of Life in Children With Sickle Cell Disease.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05995743
• Other study ID #: RECHMPL22_0192
• Status: Completed
• Start date: November 1, 2021
• Est. completion date: November 1, 2022

Study information

• Verified date: August 2023
• Source: University Hospital, Montpellier
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Sickle cell disease is the most common inherited genetic disorder, accounting for 300,000 births worldwide per year. It is caused by an autosomal recessive mutation of the β-globin gene, responsible for an abnormal hemoglobin, the main protein in red blood cells, responsible for transporting oxygen from the lungs to the tissues. The abnormal hemoglobin, known as "Sickle" or S, deforms the red blood cell, causing chronic hemolytic anemia, organ damage (heart, spleen, etc.) and vaso-occlusive crises. Therapeutic progress and specialised patient follow-up have considerably improved the vital and functional prognosis of children and adolescents with sickle cell disease. Physical fitness, measured during a cardiorespiratory exercise test (CPET), is used to determine maximal oxygen uptake (VO2max). Patients with sickle cell disease have a multifactorial limitation of exercise tolerance, which may affect their physical fitness. Authors have shown that VO2max is impaired in children and adolescents with sickle cell disease, independently of their baseline hemoglobin level. Yet VO2max is a key determinant of health-related quality of life (HRQoL) in patients being monitored for a chronic disease. In the past, our team has contributed to the assessment of HRQoL in several groups of pediatric patients suffering from chronic disease (congenital heart disease, PAH). To date, the link between impaired physical fitness and HRQoL has not been demonstrated in sickle cell children. The pathophysiological determinants of reduced physical capacity and exercise tolerance in sickle cell patients have also not been fully elucidated. Studying these factors will enable us to propose appropriate treatment in the future, with the aim of improving physical fitness and HRQoL in children and adolescents with sickle cell disease.

Description:

This prospective case-control study included sickle cell children and healthy controls from 6 to 17 years old. Patients refuse the use of medical data will be excluded.

After description of the study sample, we will first compare the VO2max Z-score between cases and controls. We will correlate the VO2max to PedsQL self- and proxy-related and Ricci and Gagnon scores (use of the coefficient of correlation rhô). Then we will compare others CPET parameters and will determine associated factors of VO2max with others resting data : hematological, respiratory, cardiologic, anthropometric, educational (use of the coefficient of correlation rhô, and multivariate linear regression model).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 72
• Est. completion date: November 1, 2022
• Est. primary completion date: November 1, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 17 Years

Eligibility

Sickle cell children

Inclusion Criteria:

• Child from 6 to 17 years old, with a confirmed diagnosis of sickle cell disease (i.e., homozygous HbS/S or heterozygous HbS/C mutations), during their routine follow-up, having performed :

• a hematology consultation : physical examination, blood test

• a cardiology consultation : electrocardiogram, transthoracic echocardiography

• a respiratory plethysmography

• a CPET and to fill in the study questionnaires.

Exclusion Criteria:

• Parents' refusal to use medical data.

Healthy children

Inclusion Criteria:

• Child from 6 to 17 years old having performed a cardio-respiratory exercise test for chest pain, dyspnea on exertion, heart murmur and whose results do not find:

• Congenital heart disease (normal echocardiography and ECG)

• Respiratory disease (normal FEV1 and FVC)

• Child having performed a maximal cardio-respiratory stress exercise until exhaustion.

Exclusion Criteria:

• Child taking long-term drug treatment

• Child with chronic disease

• Parents' refusal to use medical data.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Children
• Sickle Cell

Locations

Country	Name	City	State
France	Pediatric and Congenital Cardiology Department, Arnaud De Villeneuve University Hospital	Montpellier	Occitanie

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Montpellier

Country where clinical trial is conducted

France, 

References & Publications (24)

Abassi H, Gavotto A, Picot MC, Bertet H, Matecki S, Guillaumont S, Moniotte S, Auquier P, Moreau J, Amedro P. Impaired pulmonary function and its association with clinical outcomes, exercise capacity and quality of life in children with congenital heart disease. Int J Cardiol. 2019 Jun 15;285:86-92. doi: 10.1016/j.ijcard.2019.02.069. Epub 2019 Mar 1. — View Citation

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Amedro P, Gavotto A, Guillaumont S, Bertet H, Vincenti M, De La Villeon G, Bredy C, Acar P, Ovaert C, Picot MC, Matecki S. Cardiopulmonary fitness in children with congenital heart diseases versus healthy children. Heart. 2018 Jun;104(12):1026-1036. doi: 10.1136/heartjnl-2017-312339. Epub 2017 Nov 23. — View Citation

Amedro P, Gavotto A, Legendre A, Lavastre K, Bredy C, De La Villeon G, Matecki S, Vandenberghe D, Ladeveze M, Bajolle F, Bosser G, Bouvaist H, Brosset P, Cohen L, Cohen S, Corone S, Dauphin C, Dulac Y, Hascoet S, Iriart X, Ladouceur M, Mace L, Neagu OA, Ovaert C, Picot MC, Poirette L, Sidney F, Soullier C, Thambo JB, Combes N, Bonnet D, Guillaumont S. Impact of a centre and home-based cardiac rehabilitation program on the quality of life of teenagers and young adults with congenital heart disease: The QUALI-REHAB study rationale, design and methods. Int J Cardiol. 2019 May 15;283:112-118. doi: 10.1016/j.ijcard.2018.12.050. Epub 2018 Dec 20. — View Citation

Amedro P, Huguet H, Macioce V, Dorka R, Auer A, Guillaumont S, Auquier P, Abassi H, Picot MC. Psychometric validation of the French self and proxy versions of the PedsQL 4.0 generic health-related quality of life questionnaire for 8-12 year-old children. Health Qual Life Outcomes. 2021 Mar 4;19(1):75. doi: 10.1186/s12955-021-01714-y. — View Citation

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Gavotto A, Mura T, Rhodes J, Yin SM, Hager A, Hock J, Guillaumont S, Vincenti M, De La Villeon G, Requirand A, Picot MC, Huguet H, Souilla L, Moreau J, Matecki S, Amedro P. Reference values of aerobic fitness in the contemporary paediatric population. Eur J Prev Cardiol. 2023 Jul 12;30(9):820-829. doi: 10.1093/eurjpc/zwad054. — View Citation

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* Note: There are 24 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Difference between aerobic fitness evaluated by VO2max, expressed in Z-score between cases and controls		1 day
Secondary	Correlation between aerobic fitness and quality of life in cases group	To analyze this, we will correlate the VO2max Z-score and the assessment of health-related quality of life (measured by PedQL 4.0).	1 day
Secondary	Correlation between aerobic fitness and physical activity level, in cases group	To analyze this, we will correlate the VO2max Z-score and the assessment of physical activity level (measured by Ricci and Gagnon questionnaire).	1 day
Secondary	Correlation between aerobic fitness and educational level (knowledge about disease), in cases group	To analyze this, we will correlate the VO2max Z-score and the assessment of educational level (local disease knowledge questionnaire).	1 day
Secondary	Correlation between aerobic fitness and VO2max limiting factors in cases group: demographic, genetic mutation, clinical (comorbidities, respiratory function, cardiac function, anemia, CPET data)	To analyze this, we will correlate the VO2max Z-score and demographic data, genetic mutation, spirometry, plethysmography, trans-thoracic echocardiography, hemoglobin level, other CPET data.	1 day