xml: <clinical_study>
    <!-- This xml conforms to an XML Schema at:
      https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
    <required_header>
      <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
      <link_text>Link to the current ClinicalTrials.gov record.</link_text>
      <url>https://clinicaltrials.gov/ct2/show/NCT05995743</url>
    </required_header>
    <id_info>
      <org_study_id>RECHMPL22_0192</org_study_id>
      <nct_id>NCT05995743</nct_id>
    </id_info>
    <brief_title>VO2max &amp; HRQoL in Children With Sickle Cell Disease</brief_title>
    <acronym>VO2drépano</acronym>
    <official_title>Aerobic Physical Fitness and Health-related Quality of Life in Children With Sickle Cell Disease.</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>University Hospital, Montpellier</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>University Hospital, Montpellier</source>
    <oversight_info>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        Sickle cell disease is the most common inherited genetic disorder, accounting for 300,000&#xD;
        births worldwide per year. It is caused by an autosomal recessive mutation of the β-globin&#xD;
        gene, responsible for an abnormal hemoglobin, the main protein in red blood cells,&#xD;
        responsible for transporting oxygen from the lungs to the tissues. The abnormal hemoglobin,&#xD;
        known as &quot;Sickle&quot; or S, deforms the red blood cell, causing chronic hemolytic anemia, organ&#xD;
        damage (heart, spleen, etc.) and vaso-occlusive crises. Therapeutic progress and specialised&#xD;
        patient follow-up have considerably improved the vital and functional prognosis of children&#xD;
        and adolescents with sickle cell disease. Physical fitness, measured during a&#xD;
        cardiorespiratory exercise test (CPET), is used to determine maximal oxygen uptake (VO2max).&#xD;
        Patients with sickle cell disease have a multifactorial limitation of exercise tolerance,&#xD;
        which may affect their physical fitness. Authors have shown that VO2max is impaired in&#xD;
        children and adolescents with sickle cell disease, independently of their baseline hemoglobin&#xD;
        level. Yet VO2max is a key determinant of health-related quality of life (HRQoL) in patients&#xD;
        being monitored for a chronic disease. In the past, our team has contributed to the&#xD;
        assessment of HRQoL in several groups of pediatric patients suffering from chronic disease&#xD;
        (congenital heart disease, PAH). To date, the link between impaired physical fitness and&#xD;
        HRQoL has not been demonstrated in sickle cell children. The pathophysiological determinants&#xD;
        of reduced physical capacity and exercise tolerance in sickle cell patients have also not&#xD;
        been fully elucidated. Studying these factors will enable us to propose appropriate treatment&#xD;
        in the future, with the aim of improving physical fitness and HRQoL in children and&#xD;
        adolescents with sickle cell disease.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        This prospective case-control study included sickle cell children and healthy controls from 6&#xD;
        to 17 years old. Patients refuse the use of medical data will be excluded.&#xD;
  &#xD;
        After description of the study sample, we will first compare the VO2max Z-score between cases&#xD;
        and controls. We will correlate the VO2max to PedsQL self- and proxy-related and Ricci and&#xD;
        Gagnon scores (use of the coefficient of correlation rhô). Then we will compare others CPET&#xD;
        parameters and will determine associated factors of VO2max with others resting data :&#xD;
        hematological, respiratory, cardiologic, anthropometric, educational (use of the coefficient&#xD;
        of correlation rhô, and multivariate linear regression model).&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Completed</overall_status>
    <start_date type="Actual">November 1, 2021</start_date>
    <completion_date type="Actual">November 1, 2022</completion_date>
    <primary_completion_date type="Actual">November 1, 2022</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Case-Control</observational_model>
      <time_perspective>Cross-Sectional</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>Difference between aerobic fitness evaluated by VO2max, expressed in Z-score between cases and controls</measure>
      <time_frame>1 day</time_frame>
    </primary_outcome>
    <secondary_outcome>
      <measure>Correlation between aerobic fitness and quality of life in cases group</measure>
      <time_frame>1 day</time_frame>
      <description>To analyze this, we will correlate the VO2max Z-score and the assessment of health-related quality of life (measured by PedQL 4.0).</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Correlation between aerobic fitness and physical activity level, in cases group</measure>
      <time_frame>1 day</time_frame>
      <description>To analyze this, we will correlate the VO2max Z-score and the assessment of physical activity level (measured by Ricci and Gagnon questionnaire).</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Correlation between aerobic fitness and educational level (knowledge about disease), in cases group</measure>
      <time_frame>1 day</time_frame>
      <description>To analyze this, we will correlate the VO2max Z-score and the assessment of educational level (local disease knowledge questionnaire).</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Correlation between aerobic fitness and VO2max limiting factors in cases group: demographic, genetic mutation, clinical (comorbidities, respiratory function, cardiac function, anemia, CPET data)</measure>
      <time_frame>1 day</time_frame>
      <description>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.</description>
    </secondary_outcome>
    <number_of_groups>2</number_of_groups>
    <enrollment type="Actual">72</enrollment>
    <condition>Sickle Cell</condition>
    <condition>Children</condition>
    <arm_group>
      <arm_group_label>Cases: Children with sickle cell disease</arm_group_label>
      <description>Children aged 6 to 17 years with a confirmed diagnosis of sickle cell disease (i.e., homozygous HbS/S or heterozygous HbS/C mutations)</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Controls: Healthy children referred for a non-severe functional symptom linked to exercise</arm_group_label>
      <description>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.</description>
    </arm_group>
    <eligibility>
      <study_pop>
        <textblock>
          Sickle cell children cohort consisted of children with a genetic diagnosis of sickle cell&#xD;
          disease (HbS/S or HbS/C), followed at the Department of Pediatrics of Montpellier&#xD;
          University Hospital, a tertiary care academic institution in southern France.&#xD;
  &#xD;
          Healthy children cohort consisted of children referred to a pediatric cardiologist for a&#xD;
          nonsevere functional symptom related to exercise (murmur, palpitation, chest pain, and&#xD;
          dyspnoea) or for a medical sports certificate. These children needed to have a completely&#xD;
          normal check-up, including physical examination, ECG, and echocardiography. Children with&#xD;
          any chronic disease, medical condition or medical treatment, and those requiring any&#xD;
          further specialized medical consultation were not eligible.&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Sickle cell children&#xD;
  &#xD;
          Inclusion Criteria:&#xD;
  &#xD;
            -  Child from 6 to 17 years old, with a confirmed diagnosis of sickle cell disease (i.e.,&#xD;
               homozygous HbS/S or heterozygous HbS/C mutations), during their routine follow-up,&#xD;
               having performed :&#xD;
  &#xD;
            -  a hematology consultation : physical examination, blood test&#xD;
  &#xD;
            -  a cardiology consultation : electrocardiogram, transthoracic echocardiography&#xD;
  &#xD;
            -  a respiratory plethysmography&#xD;
  &#xD;
            -  a CPET and to fill in the study questionnaires.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Parents' refusal to use medical data.&#xD;
  &#xD;
          Healthy children&#xD;
  &#xD;
          Inclusion Criteria:&#xD;
  &#xD;
            -  Child from 6 to 17 years old having performed a cardio-respiratory exercise test for&#xD;
               chest pain, dyspnea on exertion, heart murmur and whose results do not find:&#xD;
  &#xD;
            -  Congenital heart disease (normal echocardiography and ECG)&#xD;
  &#xD;
            -  Respiratory disease (normal FEV1 and FVC)&#xD;
  &#xD;
            -  Child having performed a maximal cardio-respiratory stress exercise until exhaustion.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Child taking long-term drug treatment&#xD;
  &#xD;
            -  Child with chronic disease&#xD;
  &#xD;
            -  Parents' refusal to use medical data.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>6 Years</minimum_age>
      <maximum_age>17 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Corentin Laurent-Lacroix, Resident</last_name>
      <role>Principal Investigator</role>
      <affiliation>Montpellier University Hospital</affiliation>
    </overall_official>
    <location>
      <facility>
        <name>Pediatric and Congenital Cardiology Department, Arnaud De Villeneuve University Hospital</name>
        <address>
          <city>Montpellier</city>
          <state>Occitanie</state>
          <zip>34295</zip>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location_countries>
      <country>France</country>
    </location_countries>
    <reference>
      <citation>Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865. No abstract available.</citation>
      <PMID>28423290</PMID>
    </reference>
    <reference>
      <citation>Ferraresi M, Panzieri DL, Leoni S, Cappellini MD, Kattamis A, Motta I. Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease. Eur J Pediatr. 2023 Jun;182(6):2509-2519. doi: 10.1007/s00431-023-04900-w. Epub 2023 Mar 31.</citation>
      <PMID>36997768</PMID>
    </reference>
    <reference>
      <citation>Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.</citation>
      <PMID>21131035</PMID>
    </reference>
    <reference>
      <citation>Connes P, Machado R, Hue O, Reid H. Exercise limitation, exercise testing and exercise recommendations in sickle cell anemia. Clin Hemorheol Microcirc. 2011;49(1-4):151-63. doi: 10.3233/CH-2011-1465.</citation>
      <PMID>22214686</PMID>
    </reference>
    <reference>
      <citation>Panepinto JA, O'Mahar KM, DeBaun MR, Loberiza FR, Scott JP. Health-related quality of life in children with sickle cell disease: child and parent perception. Br J Haematol. 2005 Aug;130(3):437-44. doi: 10.1111/j.1365-2141.2005.05622.x.</citation>
      <PMID>16042695</PMID>
    </reference>
    <reference>
      <citation>Liem RI, Reddy M, Pelligra SA, Savant AP, Fernhall B, Rodeghier M, Thompson AA. Reduced fitness and abnormal cardiopulmonary responses to maximal exercise testing in children and young adults with sickle cell anemia. Physiol Rep. 2015 Apr;3(4):e12338. doi: 10.14814/phy2.12338. Erratum In: Physiol Rep. 2016 Jan;4(1). pii: e12680. doi: 10.14814/phy2.12680.</citation>
      <PMID>25847915</PMID>
    </reference>
    <reference>
      <citation>Callahan LA, Woods KF, Mensah GA, Ramsey LT, Barbeau P, Gutin B. Cardiopulmonary responses to exercise in women with sickle cell anemia. Am J Respir Crit Care Med. 2002 May 1;165(9):1309-16. doi: 10.1164/rccm.2002036.</citation>
      <PMID>11991885</PMID>
    </reference>
    <reference>
      <citation>Woodson RD. Hemoglobin concentration and exercise capacity. Am Rev Respir Dis. 1984 Feb;129(2 Pt 2):S72-5. doi: 10.1164/arrd.1984.129.2P2.S72. No abstract available.</citation>
      <PMID>6696347</PMID>
    </reference>
    <reference>
      <citation>Pianosi P, D'Souza SJ, Esseltine DW, Charge TD, Coates AL. Ventilation and gas exchange during exercise in sickle cell anemia. Am Rev Respir Dis. 1991 Feb;143(2):226-30. doi: 10.1164/ajrccm/143.2.226.</citation>
      <PMID>1990932</PMID>
    </reference>
    <reference>
      <citation>Klings ES, Wyszynski DF, Nolan VG, Steinberg MH. Abnormal pulmonary function in adults with sickle cell anemia. Am J Respir Crit Care Med. 2006 Jun 1;173(11):1264-9. doi: 10.1164/rccm.200601-125OC. Epub 2006 Mar 23.</citation>
      <PMID>16556694</PMID>
    </reference>
    <reference>
      <citation>Olorunyomi OO, Liem RI, Hsu LL. Motivators and Barriers to Physical Activity among Youth with Sickle Cell Disease: Brief Review. Children (Basel). 2022 Apr 17;9(4):572. doi: 10.3390/children9040572.</citation>
      <PMID>35455616</PMID>
    </reference>
    <reference>
      <citation>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.</citation>
      <PMID>29170358</PMID>
    </reference>
    <reference>
      <citation>American Thoracic Society; American College of Chest Physicians. ATS/ACCP Statement on cardiopulmonary exercise testing. Am J Respir Crit Care Med. 2003 Jan 15;167(2):211-77. doi: 10.1164/rccm.167.2.211. No abstract available. Erratum In: Am J Respir Crit Care Med. 2003 May 15;1451-2.</citation>
      <PMID>12524257</PMID>
    </reference>
    <reference>
      <citation>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.</citation>
      <PMID>30857849</PMID>
    </reference>
    <reference>
      <citation>Amedro P, Basquin A, Gressin V, Clerson P, Jais X, Thambo JB, Guerin P, Cohen S, Bonnet D. Health-related quality of life of patients with pulmonary arterial hypertension associated with CHD: the multicentre cross-sectional ACHILLE study. Cardiol Young. 2016 Oct;26(7):1250-9. doi: 10.1017/S1047951116000056. Epub 2016 Mar 16.</citation>
      <PMID>26980152</PMID>
    </reference>
    <reference>
      <citation>Das BB, Sobczyk W, Bertolone S, Raj A. Cardiopulmonary stress testing in children with sickle cell disease who are on long-term erythrocytapheresis. J Pediatr Hematol Oncol. 2008 May;30(5):373-7. doi: 10.1097/MPH.0b013e318165b298.</citation>
      <PMID>18458572</PMID>
    </reference>
    <reference>
      <citation>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.</citation>
      <PMID>36809338</PMID>
    </reference>
    <reference>
      <citation>Takken T, Blank AC, Hulzebos EH, van Brussel M, Groen WG, Helders PJ. Cardiopulmonary exercise testing in congenital heart disease: equipment and test protocols. Neth Heart J. 2009 Sep;17(9):339-44. doi: 10.1007/BF03086280.</citation>
      <PMID>19949476</PMID>
    </reference>
    <reference>
      <citation>Varni JW, Burwinkle TM, Seid M, Skarr D. The PedsQL 4.0 as a pediatric population health measure: feasibility, reliability, and validity. Ambul Pediatr. 2003 Nov-Dec;3(6):329-41. doi: 10.1367/1539-4409(2003)0032.0.co;2.</citation>
      <PMID>14616041</PMID>
    </reference>
    <reference>
      <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.</citation>
      <PMID>33663527</PMID>
    </reference>
    <reference>
      <citation>Vuillemin A, Denis G, Guillemin F, Jeandel C. [A review of evaluation questionnaires for physical activity]. Rev Epidemiol Sante Publique. 1998 Feb;46(1):49-55. French.</citation>
      <PMID>9533234</PMID>
    </reference>
    <reference>
      <citation>Pianosi P, D'Souza SJ, Charge TD, Beland MJ, Esseltine DW, Coates AL. Cardiac output and oxygen delivery during exercise in sickle cell anemia. Am Rev Respir Dis. 1991 Feb;143(2):231-5. doi: 10.1164/ajrccm/143.2.231.</citation>
      <PMID>1990933</PMID>
    </reference>
    <reference>
      <citation>Powell AW, Alsaied T, Niss O, Fleck RJ, Malik P, Quinn CT, Mays WA, Taylor MD, Chin C. Abnormal submaximal cardiopulmonary exercise parameters predict impaired peak exercise performance in sickle cell anemia patients. Pediatr Blood Cancer. 2019 Jun;66(6):e27703. doi: 10.1002/pbc.27703. Epub 2019 Mar 7.</citation>
      <PMID>30848046</PMID>
    </reference>
    <reference>
      <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.</citation>
      <PMID>30616811</PMID>
    </reference>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 9, 2023</study_first_submitted>
    <study_first_submitted_qc>August 9, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 16, 2023</study_first_posted>
    <last_update_submitted>August 16, 2023</last_update_submitted>
    <last_update_submitted_qc>August 16, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 21, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Cardiopulmonary exercise test</keyword>
    <keyword>VO2max</keyword>
    <keyword>Sickle cell disease</keyword>
    <keyword>Child</keyword>
    <keyword>Heath-related quality of life</keyword>
    <keyword>Cardiopulmonary rehabilitation</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Anemia, Sickle Cell</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: download date: ClinicalTrials.gov processed this data on September 20, 2023
  link text: Link to the current ClinicalTrials.gov record.
  url: https://clinicaltrials.gov/ct2/show/NCT05995743
  org study id: RECHMPL22_0192
  nct id: NCT05995743
  lead sponsor: 
  is fda regulated drug: No
  is fda regulated device: No
  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.
  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).
  observational model: Case-Control
  time perspective: Cross-Sectional
  measure: Difference between aerobic fitness evaluated by VO2max, expressed in Z-score between cases and controls
  time frame: 1 day
  measure: Correlation between aerobic fitness and quality of life in cases group
  time frame: 1 day
  description: To analyze this, we will correlate the VO2max Z-score and the assessment of health-related quality of life (measured by PedQL 4.0).
  measure: Correlation between aerobic fitness and physical activity level, in cases group
  time frame: 1 day
  description: To analyze this, we will correlate the VO2max Z-score and the assessment of physical activity level (measured by Ricci and Gagnon questionnaire).
  measure: Correlation between aerobic fitness and educational level (knowledge about disease), in cases group
  time frame: 1 day
  description: To analyze this, we will correlate the VO2max Z-score and the assessment of educational level (local disease knowledge questionnaire).
  measure: 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
  description: 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.
  arm group label: Cases: Children with sickle cell disease
  description: Children aged 6 to 17 years with a confirmed diagnosis of sickle cell disease (i.e., homozygous HbS/S or heterozygous HbS/C mutations)
  arm group label: Controls: Healthy children referred for a non-severe functional symptom linked to exercise
  description: 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.
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: 6 Years
  maximum age: 17 Years
  healthy volunteers: No
  last name: Corentin Laurent-Lacroix, Resident
  role: Principal Investigator
  affiliation: Montpellier University Hospital
  facility: 
  country: France
  citation: Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865. No abstract available.
  PMID: 28423290
  citation: Ferraresi M, Panzieri DL, Leoni S, Cappellini MD, Kattamis A, Motta I. Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease. Eur J Pediatr. 2023 Jun;182(6):2509-2519. doi: 10.1007/s00431-023-04900-w. Epub 2023 Mar 31.
  PMID: 36997768
  citation: Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010 Dec 11;376(9757):2018-31. doi: 10.1016/S0140-6736(10)61029-X. Epub 2010 Dec 3.
  PMID: 21131035
  citation: Connes P, Machado R, Hue O, Reid H. Exercise limitation, exercise testing and exercise recommendations in sickle cell anemia. Clin Hemorheol Microcirc. 2011;49(1-4):151-63. doi: 10.3233/CH-2011-1465.
  PMID: 22214686
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  responsible party type: Sponsor
  mesh term: Anemia, Sickle Cell
  sharing ipd: No