Clinical trial NCT01558076: Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia
Official title: Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Anemia
Keywords: Sickle cell anemia
exercise testing
cardiopulmonary disease
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2012-03-20
Enrollment: 35
Sex: ALL
Minimum age (years): 8
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Sickle cell anemia patients followed at Children's Memorial Hospital. Healthy controls without sickle cell anemia or sickle cell trait are recruited through flyers posted in Children's Memorial Hospital.

Brief summary: The purpose of this study is to use comprehensive exercise testing to examine longitudinal changes in exercise capacity over a 2 year period in children and young adults with sickle cell anemia.

Detailed description: 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 longitudinal changes in exercise capacity, and the effect of poor exercise capacity on quality of life 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) compare results of this exercise test with the results of previously completed exercise tests to determine longitudinal changes in exercise capacity. These aims will be performed in 60 subjects with SCA and 30 matched controls who participated in a previous study, IRB# 2009-13659; "The Physiologic Assessment of Exercise Capacity in Pediatric Sickle Cell Anemia". In a secondary analysis, we will also study the participants quality of life using a set of validated questionnaires. This study is essential because it will address several areas of exercise capacity, including the longterm effects of physiologic contributors to exercise limitation, that remain fundamental knowledge gaps in SCA.

Eligibility criteria: Inclusion Criteria:

1. age 8 to 21 years old; AND
2. Hb SS or S-β0 thalassemia disease, confirmed by hemoglobin analysis; AND
3. Previously participated in ClinicalTrials.gov ID: NCT01527799

Exclusion Criteria:

1. inability to perform maximal testing due to physical limitation (e.g. stroke or avascular necrosis); OR
2. 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

Primary outcomes:
measure: VO2 max on cardiopulmonary exercise test
timeFrame: Baseline
Secondary outcomes:
measure: Quality of life questionnaires
timeFrame: Baseline
measure: Echocardiogram
timeFrame: Baseline
measure: Dual Energy X-ray Absorbtiometry
timeFrame: Baseline
measure: Pulmonary Function Test
timeFrame: Baseline
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Robert I Liem, MD MS
role: PRINCIPAL_INVESTIGATOR
References: