VO2max & HRQoL in Children With Sickle Cell Disease (VO2drépano)

August 16, 2023 updated by: University Hospital, Montpellier

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

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.

Study Overview

Status

Completed

Conditions

Detailed 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).

Study Type

Observational

Enrollment (Actual)

72

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
    • Occitanie
      • Montpellier, Occitanie, France, 34295
        • Pediatric and Congenital Cardiology Department, Arnaud De Villeneuve University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Sickle cell children cohort consisted of children with a genetic diagnosis of sickle cell disease (HbS/S or HbS/C), followed at the Department of Pediatrics of Montpellier University Hospital, a tertiary care academic institution in southern France.

Healthy children cohort consisted of children referred to a pediatric cardiologist for a nonsevere functional symptom related to exercise (murmur, palpitation, chest pain, and dyspnoea) or for a medical sports certificate. These children needed to have a completely normal check-up, including physical examination, ECG, and echocardiography. Children with any chronic disease, medical condition or medical treatment, and those requiring any further specialized medical consultation were not eligible.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Cases: Children with sickle cell disease
Children aged 6 to 17 years with a confirmed diagnosis of sickle cell disease (i.e., homozygous HbS/S or heterozygous HbS/C mutations)
Controls: Healthy children referred for a non-severe functional symptom linked to exercise
Children aged 6 to 17 years with a completely normal check-up, including physical examination, ECG, echocardiography, and spirometry. Children with any chronic disease, medical condition, or medical treatment and those requiring any further specialized medical consultation were not eligible.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Difference between aerobic fitness evaluated by VO2max, expressed in Z-score between cases and controls
Time Frame: 1 day
1 day

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Correlation between aerobic fitness and quality of life in cases group
Time Frame: 1 day
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
Correlation between aerobic fitness and physical activity level, in cases group
Time Frame: 1 day
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
Correlation between aerobic fitness and educational level (knowledge about disease), in cases group
Time Frame: 1 day
To analyze this, we will correlate the VO2max Z-score and the assessment of educational level (local disease knowledge questionnaire).
1 day
Correlation between aerobic fitness and VO2max limiting factors in cases group: demographic, genetic mutation, clinical (comorbidities, respiratory function, cardiac function, anemia, CPET data)
Time Frame: 1 day
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Montpellier

Investigators

  • Principal Investigator: Corentin Laurent-Lacroix, Resident, Montpellier University Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2021

Primary Completion (Actual)

November 1, 2022

Study Completion (Actual)

November 1, 2022

Study Registration Dates

First Submitted

August 9, 2023

First Submitted That Met QC Criteria

August 9, 2023

First Posted (Actual)

August 16, 2023

Study Record Updates

Last Update Posted (Actual)

August 21, 2023

Last Update Submitted That Met QC Criteria

August 16, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RECHMPL22_0192

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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