Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease
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ClinicalTrials.gov Identifier: NCT01895998 |
Recruitment Status :
Completed
First Posted : July 11, 2013
Last Update Posted : December 3, 2014
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The proposed research study is a cross-sectional study enrolling young children with sickle cell disease between 5 and 12 years of age. They will be screened as outpatients for consent to perform pulmonary function testing (PFT) and echocardiography. In addition, the degree of bronchodilator response will be assessed at each session. To estimate presence of pulmonary hypertension, echocardiography will be performed at the time of PFT measures.
Study Design:
- Enroll children aged 5 to 12 years of age with sickle cell disease (HbSS, HbSC, HbS beta plus thalassemia, HbS beta zero thalassemia, and HbS OArab) who are established patients within the Duke Pediatric Sickle Cell Clinic.
- Perform a chart review of all enrolled subjects to obtain specific details regarding birth history, nutritional status (weight, height), family history, sickle cell genotype, parental smoking history, recent laboratory parameters, parental smoking history, any concurrent conditions (atopy, asthma, airway anomaly), history of sickle cell complications and prescribed medications.
- Perform spirometry and plethysmography with the administration of albuterol.
- Before or after completion the PFT session, the patient will have echocardiography in the PFT lab area
- Using medical record information, determine number of hospitalizations for any pulmonary symptoms indicative of acute chest syndrome (ACS) (dyspnea, fever, wheezing, hypoxia, cough, chest pain). In addition, we will track any respiratory or cardiac symptoms or therapies for each subject 6 years after enrollment up to age 18 years using the registry.
- As standard of care, refer any child identified as having lung disease or pulmonary hypertension to a pediatric pulmonologist and/or cardiologist for monitoring, treatment and ongoing care.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Sickle Cell Disease | Drug: Albuterol | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 5 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Diagnostic |
Official Title: | Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell Disease |
Study Start Date : | August 2013 |
Actual Primary Completion Date : | April 2014 |
Actual Study Completion Date : | April 2014 |

- Drug: Albuterol
This medication will be used as routine part of pulmonary function testing to assess for airway hyperreactivity. This is routinely done in a clinical capacity for pediatric patients with various pulmonary disease processes.
- Presence of obstructive or restrictive lung disease [ Time Frame: One testing session- approximately 3 hours total ]Obstructive lung disease will be defined as forced expiratory volume in 1 second (FEV1), or forced vital capacity (FVC) < 80% predicted based on normative data for age, sex, height, weight and race. Restrictive lung disease will be defined as total lung capacity (TLC) <80% predicted based on normative data for age, sex, height, weight and race.
- Presence of pulmonary hypertension [ Time Frame: one testing session- approximately 15 minutes ]Pulmonary hypertension will be defined as a tricuspid regurgitation (TR) jet gradient that predicts a pulmonary artery (PA) systolic pressure greater than half of the systemic systolic pressure.

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Ages Eligible for Study: | 5 Years to 12 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- children aged 5 to 12 years of age with sickle cell disease (HbSS, HbSC, HbS beta plus thalassemia, HbS beta zero thalassemia, and HbS OArab)
- established patients within the Duke Pediatric Sickle Cell Clinic.
- Subjects must have been full-term at birth
- any race or gender
Exclusion Criteria:
- significant chromosomal/congenital anomalies
- hemodynamically significant congenital heart disease (arrhythmia requiring medication, defects with chronic hypoxia, single ventricle physiology, heart failure)
- any child within 3 weeks of a respiratory tract infection, an asthma attack, an episode of ACS or of a vaso-occlusive or hemolytic crisis.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01895998
United States, North Carolina | |
Duke University Medical Center | |
Durham, North Carolina, United States, 27710 |
Principal Investigator: | Stacey Peterson-Carmichael, MD | Duke University |
Responsible Party: | Duke University |
ClinicalTrials.gov Identifier: | NCT01895998 |
Other Study ID Numbers: |
Pro00040933 |
First Posted: | July 11, 2013 Key Record Dates |
Last Update Posted: | December 3, 2014 |
Last Verified: | December 2014 |
sickle cell disease pulmonary hypertension airway hyperreactivity acute chest syndrome |
Lung Diseases Hypertension, Pulmonary Hypertension Anemia, Sickle Cell Vascular Diseases Cardiovascular Diseases Respiratory Tract Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Albuterol |
Bronchodilator Agents Autonomic Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Asthmatic Agents Respiratory System Agents Tocolytic Agents Reproductive Control Agents Adrenergic beta-2 Receptor Agonists Adrenergic beta-Agonists Adrenergic Agonists Adrenergic Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action |