Exercise Capacity in Pediatric Sickle Cell Anemia

Study Overview
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Brief Summary
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The purpose of this study is to use comprehensive exercise testing to examine causes of exercise limitation in children and young adults with sickle cell anemia.

Detailed Description
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Although the burden of sickle cell anemia (SCA) on affected individuals is significant, few studies have examined the influence of having SCA on such measures of physical function as exercise capacity. Moreover, the physiologic basis of poor physical functioning in children with SCA is unknown and has not been studied extensively. The purpose of this proposal is to use cardiopulmonary exercise testing (CPET) to gain a comprehensive understanding of exercise capacity, as a measure of physical function, in children and young adults with SCA. The specific aims of this project are to: 1) Measure peak oxygen consumption (VO2), the reference standard for exercise capacity, in children and young adults with SCA classified by primary pathophysiologic contributor to their decreased exercise capacity, and 2) Examine the acute inflammatory response, measured by an increase in soluble vascular cell adhesion molecule (sVCAM) activity, in subjects undergoing CPET. These aims will be performed in 60 subjects with SCA and 30 matched controls without SCA. In a secondary analysis, we will also study the impact of baseline exercise capacity and the inflammatory response to exercise on short and long-term disease related morbidity. This study is essential because it will address several areas of exercise capacity, including the physiologic contributors to exercise limitation that remain fundamental knowledge gaps in SCA.

Official Title
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The Physiologic Assessment of Exercise Capacity in Pediatric Sickle Cell Anemia

Conditions
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Sickle Cell Anemia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: age 10 to 21 years old; AND Hb SS or S-β0 thalassemia disease, confirmed by hemoglobin analysis Exclusion Criteria: inability to perform maximal testing due to physical limitation (e.g. stroke or avascular necrosis); OR history of exercise-induced syncope or arrhythmias. Subjects will wait at least 2 weeks following any vaso-occlusive pain episode and 12 weeks following any disease-related complication requiring transfusion support. Individuals on hydroxyurea will be eligible. A total of 30 controls without SCA or sickle cell trait will be matched for age, sex and race and recruited from the siblings, friends or relatives of subjects enrolled on this study

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Subjects with Sickle Cell Anemia<br>Subjects with Sickle Cell Anemia, 10-21 years of age | |
| Healthy controls<br>Healthy controls, 10 to 21 years of age | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| VO2 max on cardiopulmonary exercise test |  | Baseline | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Skin fold measurements to detemine percent body fat |  | Baseline | 
| All patient reported pain episodes |  | Every 2 months up to 2 years after baseline | 
| Change in secondary biomarkers in response to exercise test |  | Baseline (Pre-exercise) and Post-exercise | 
| Change in VCAM level in response to exercise testing |  | Baseline (pre-exercise) and Post-exercise | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Sickle cell anemia, Exercise testing, Cardiopulmonary disease