Sickle Cell Disease
Conditions
Keywords
Sickle Cell Disease, Cardiac magnetic resonance imaging, Coronary Disease, Pulmonary Hypertension
Brief summary
In this research study, we are using heart imaging exams and blood testing, in order to gain an improved understanding of the pulmonary (lung) hypertension and cardiovascular (heart) complications that often occur in sickle cell patients. Information gathered from the healthy volunteers that participate in this study will be compared to information from the sickle cell patients in this study in order to help further our understanding.
Detailed description
Cardiac magnetic resonance (CMR) has gained increasing clinical application in cardiopulmonary diseases. Due to its 3-dimensional nature, CMR is considered the gold-standard for quantifying left and right ventricular systolic function and size. Additionally, its high tissue contrast allows for a detailed characterization of myocardial tissue. Specifically, the use of techniques such as late gadolinium enhancement can be used to detect the presence of tiny amounts of myocardial scar. Other techniques have been shown to correlate strongly with myocardial iron content. Just as importantly, CMR perfusion imaging can accurately quantify myocardial blood flow and can provide tremendous insight into the function of the microcirculation. CMR's high spatial and temporal resolution, its 3-dimensional approach, its ability to characterize the tissue, and its ability to evaluate the micro- and macro-circulation make it a comprehensive technique for the evaluation of heart disease. Recently, one CMR study has already shown the presence of cardiac microvascular disease in a subset of adult sickle cell disease (SCD) patients in the absence of infarcted myocardium, myocardial iron overload, or coronary artery disease, increasing the evidence for the contribution of left heart disease to pulmonary hypertension (PH) development in these patients; unfortunately, strong conclusions could not be made because the study was underpowered. Thus, this proposal will leverage the advantages offered by CMR to better characterize and detect the PH and cardiopulmonary subphenotypes in the SCD patient population.
Interventions
PROCEDUREMRI, Transthoracic Echocardiography, tonometry, EKG
Unless contraindicated, subjects will receive Regadenoson and Gadolinium contrast agent during the Cardiac magnetic resonance. The tonometer, EKG, and echo are non-invasive procedures.
Sponsors
University of Chicago
Study design
Observational model
CASE_CONTROL
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* Patients must be 18+ * Patients who were diagnosed with SCD confirmed by high-pressure liquid chromatography or hemoglobin electrophoresis will be eligible for the study * Only patients in stable condition will be included * Patients receiving transfusions will not be excluded
Exclusion criteria
* Patients with vaso-occlusive crises or an episode of acute chest syndrome within the previous four weeks (after 4 weeks have passed, the patients may be re-evaluated for eligibility) * Patients with high degree heart block; active, hemodynamically significant, ventricular arrhythmias; unstable coronary syndromes; history of myocardial infarction within 1 month of the study. * Contraindications to gadolinium-enhanced magnetic resonance examination such as severe claustrophobia, Pacemaker, defibrillators, cerebral aneurysm clips, or neurostimulator. * Pregnancy * Patients with sinus node dysfunction
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| MRI Parameter - Systemic Diastolic Blood Pressure, mm Hg | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - LVEDVi, mL/cm2 (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - LVESVi, mL/cm2 - (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - LV Mass Index, g/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - RVEDVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - RVESVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - LAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - RAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - LVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - RVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | Parameter at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Late Gadolinium Enhancement, Performed Via Visual Inspection, Normally None Should be Present | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Myocardial T2-star, ms, Performed Using Decay Curves (Normal >20ms) | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Hepatic T2-star, ms, Performed Using Decay Curves, Normal >18ms | Parameter at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Myocardial Perfusion Reserve Index, Measured Using Upslope Technique. Control Subjects Available for Normal Ranges | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Diastolic Dysfunction, Determined According to American Society of Echocardiography Guidelines | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Lateral E/e', Measured Using Doppler Echo. Controls Available as Normal Ranges | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Augmentation Pressure, See Controls for Normal Ranges | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Augmentation Index, See Control Subjects for Normal Ranges | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
| MRI Parameter - Systemic Systolic Blood Pressure | Parameter measured at baseline. | Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Genome-Wide Gene Expression and Targeted Genetic Polymorphisms in SCD Patients Linked to a Quantitative Noninvasive-based PH Phenotype. | median follow up 3 years | To detect genome-wide gene expression and targeted genetic polymorphisms in SCD patients linked to a quantitative noninvasive-based PH phenotype. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Subjects With Sickle Cell Disease (SCD) 38 clinically stable black patients with Sickle Cell Disease (SCD) (including individuals with hemoglobin SS, SC, and β-thalassemia demonstrated by high-performance liquid chromatographic separation or gel electrophoresis).
MRI, Transthoracic Echocardiography, tonometry, EKG: Unless contraindicated, subjects will receive Regadenoson and Gadolinium contrast agent during the Cardiac magnetic resonance. The tonometer, EKG, and echo are non-invasive procedures. | 38 |
| Healthy Volunteers 13 healthy control subjects were frequency matched to patients with SCD on age, sex, and race.
MRI, Transthoracic Echocardiography, tonometry, EKG: Unless contraindicated, subjects will receive Regadenoson and Gadolinium contrast agent during the Cardiac magnetic resonance. The tonometer, EKG, and echo are non-invasive procedures. | 13 |
| Total | 51 |
Baseline characteristics
| Characteristic | Subjects With Sickle Cell Disease (SCD) | Healthy Volunteers | Total |
|---|---|---|---|
| Age, Customized Age | 32 years | 25 years | 29 years |
| Body Surface Area | 2.0 m^2 | 1.8 m^2 | 2.0 m^2 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 38 Participants | 13 Participants | 51 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Female | 21 Participants | 8 Participants | 29 Participants |
| Sex: Female, Male Male | 17 Participants | 5 Participants | 22 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 3 / 38 | 0 / 13 |
| other Total, other adverse events | 0 / 38 | 0 / 13 |
| serious Total, serious adverse events | 0 / 38 | 0 / 13 |
Outcome results
Primary
MRI Parameter - Augmentation Index, See Control Subjects for Normal Ranges
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Augmentation Index, See Control Subjects for Normal Ranges | 23.6 index | Standard Deviation 16.2 |
| Healthy Volunteers | MRI Parameter - Augmentation Index, See Control Subjects for Normal Ranges | 12.5 index | Standard Deviation 20.4 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.13t-test, 2 sided
Primary
MRI Parameter - Augmentation Pressure, See Controls for Normal Ranges
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Augmentation Pressure, See Controls for Normal Ranges | 9.0 percentage of the pulse pressure |
| Healthy Volunteers | MRI Parameter - Augmentation Pressure, See Controls for Normal Ranges | 2.0 percentage of the pulse pressure |
Comparison: Primary outcome was presented at baseline as median (interquartile range) based upon the Shapiro-Wilks test of normality, and then analyzed with a Wilcoxon (Mann-Whitney) test.p-value: 0.14Wilcoxon (Mann-Whitney)
Primary
MRI Parameter - Diastolic Dysfunction, Determined According to American Society of Echocardiography Guidelines
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
Population: The measure was unable to be measured for some patients with Sickle Cell Disease.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Diastolic Dysfunction, Determined According to American Society of Echocardiography Guidelines | 10 Participants |
| Healthy Volunteers | MRI Parameter - Diastolic Dysfunction, Determined According to American Society of Echocardiography Guidelines | 1 Participants |
Comparison: Primary outcome was presented was as frequencies and percentages, and then analyzed with a Fisher exact test.p-value: 0.25Fisher Exact
Primary
MRI Parameter - Hepatic T2-star, ms, Performed Using Decay Curves, Normal >18ms
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter at baseline.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Hepatic T2-star, ms, Performed Using Decay Curves, Normal >18ms | 10 ms |
| Healthy Volunteers | MRI Parameter - Hepatic T2-star, ms, Performed Using Decay Curves, Normal >18ms | 30 ms |
Comparison: Primary outcome was presented at baseline as median (interquartile range) based upon the Shapiro-Wilks test of normality, and then analyzed with a Wilcoxon (Mann-Whitney) test.p-value: 0.0038Wilcoxon (Mann-Whitney)
Primary
MRI Parameter - LAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - LAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 64.8 mL/cm2 | Standard Deviation 16.2 |
| Healthy Volunteers | MRI Parameter - LAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 41.1 mL/cm2 | Standard Deviation 9.1 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.0039t-test, 2 sided
Primary
MRI Parameter - Late Gadolinium Enhancement, Performed Via Visual Inspection, Normally None Should be Present
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
Population: Some parameters were unable to be collected from patients with Sickle Cell Disease.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Late Gadolinium Enhancement, Performed Via Visual Inspection, Normally None Should be Present | 8 Participants |
| Healthy Volunteers | MRI Parameter - Late Gadolinium Enhancement, Performed Via Visual Inspection, Normally None Should be Present | 0 Participants |
Comparison: Primary outcome was presented as frequencies and percentages, and then analyzed with a Fisher exact test.p-value: 0.08Fisher Exact
Primary
MRI Parameter - Lateral E/e', Measured Using Doppler Echo. Controls Available as Normal Ranges
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Lateral E/e', Measured Using Doppler Echo. Controls Available as Normal Ranges | 7.2 ratio |
| Healthy Volunteers | MRI Parameter - Lateral E/e', Measured Using Doppler Echo. Controls Available as Normal Ranges | 6.0 ratio |
Comparison: Primary outcome was presented at baseline as median (interquartile range) based upon the Shapiro-Wilks test of normality, and then analyzed with a Wilcoxon (Mann-Whitney) test.p-value: 0.0288Wilcoxon (Mann-Whitney)
Primary
MRI Parameter - LVEDVi, mL/cm2 (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - LVEDVi, mL/cm2 (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 124.0 mL/cm2 | Standard Deviation 26.8 |
| Healthy Volunteers | MRI Parameter - LVEDVi, mL/cm2 (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 78.7 mL/cm2 | Standard Deviation 11.9 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.0039t-test, 2 sided
Primary
MRI Parameter - LVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - LVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 58 percentage of ejection fraction |
| Healthy Volunteers | MRI Parameter - LVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 64 percentage of ejection fraction |
Comparison: Primary outcome was presented at baseline as median (interquartile range) based upon the Shapiro-Wilks test of normality, and then analyzed with a Wilcoxon (Mann-Whitney) test.p-value: 0.1Wilcoxon (Mann-Whitney)
Primary
MRI Parameter - LVESVi, mL/cm2 - (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - LVESVi, mL/cm2 - (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 47 mL/cm2 |
| Healthy Volunteers | MRI Parameter - LVESVi, mL/cm2 - (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 31 mL/cm2 |
Comparison: Primary outcome was presented at baseline as median (interquartile range) based upon the Shapiro-Wilks test of normality, and then analyzed with a Wilcoxon (Mann-Whitney test).p-value: <0.0001Wilcoxon (Mann-Whitney)
Primary
MRI Parameter - LV Mass Index, g/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - LV Mass Index, g/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 77.2 g/cm2 | Standard Deviation 19.2 |
| Healthy Volunteers | MRI Parameter - LV Mass Index, g/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 51.6 g/cm2 | Standard Deviation 13.6 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.0137t-test, 2 sided
Primary
MRI Parameter - Myocardial Perfusion Reserve Index, Measured Using Upslope Technique. Control Subjects Available for Normal Ranges
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Myocardial Perfusion Reserve Index, Measured Using Upslope Technique. Control Subjects Available for Normal Ranges | 1.4 index | Standard Deviation 0.3 |
| Healthy Volunteers | MRI Parameter - Myocardial Perfusion Reserve Index, Measured Using Upslope Technique. Control Subjects Available for Normal Ranges | 1.87 index | Standard Deviation 0.37 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.034t-test, 2 sided
Primary
MRI Parameter - Myocardial T2-star, ms, Performed Using Decay Curves (Normal >20ms)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Myocardial T2-star, ms, Performed Using Decay Curves (Normal >20ms) | 41.6 ms | Standard Deviation 13.4 |
| Healthy Volunteers | MRI Parameter - Myocardial T2-star, ms, Performed Using Decay Curves (Normal >20ms) | 38.4 ms | Standard Deviation 14.4 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.91t-test, 2 sided
Primary
MRI Parameter - RAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - RAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 76 mL/cm2 |
| Healthy Volunteers | MRI Parameter - RAi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 52 mL/cm2 |
Comparison: Primary outcome was presented at baseline as median (interquartile range) based upon the Shapiro-Wilks test of normality, and then analyzed with a Wilcoxon (Mann-Whitney) test.p-value: 0.0011Wilcoxon (Mann-Whitney)
Primary
MRI Parameter - RVEDVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - RVEDVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 126.4 mL/cm2 | Standard Deviation 27.7 |
| Healthy Volunteers | MRI Parameter - RVEDVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 83.0 mL/cm2 | Standard Deviation 13.7 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.0039t-test, 2 sided
Primary
MRI Parameter - RVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - RVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 56.1 percentage of ejection fraction | Standard Deviation 6.4 |
| Healthy Volunteers | MRI Parameter - RVEF, %, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 55.4 percentage of ejection fraction | Standard Deviation 2.7 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.48t-test, 2 sided
Primary
MRI Parameter - RVESVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges)
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - RVESVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 56.3 mL/cm2 | Standard Deviation 17.1 |
| Healthy Volunteers | MRI Parameter - RVESVi, mL/cm2, (Measured Using Method of Disks, Controls Serve as Normal Ranges) | 37.8 mL/cm2 | Standard Deviation 7.2 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.16t-test, 2 sided
Primary
MRI Parameter - Systemic Diastolic Blood Pressure, mm Hg
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Systemic Diastolic Blood Pressure, mm Hg | 68.3 mmHg | Standard Deviation 18.1 |
| Healthy Volunteers | MRI Parameter - Systemic Diastolic Blood Pressure, mm Hg | 74.9 mmHg | Standard Deviation 16.3 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.3t-test, 2 sided
Primary
MRI Parameter - Systemic Systolic Blood Pressure
Comprehensively and quantitatively characterized the cardiopulmonary complications of SCD and gained an improved understanding of the pathophysiology of pulmonary hypertension and diastolic dysfunction in patients with Sickle Cell Disease.
Time frame: Parameter measured at baseline.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Subjects With Sickle Cell Disease (SCD) | MRI Parameter - Systemic Systolic Blood Pressure | 122.3 mmHg | Standard Deviation 21.7 |
| Healthy Volunteers | MRI Parameter - Systemic Systolic Blood Pressure | 132 mmHg | Standard Deviation 17.6 |
Comparison: Primary outcome was presented at baseline as mean +/- standard deviation based upon the Shapiro-Wilks test of normality, and then analyzed with a Student t-test.p-value: 0.81t-test, 2 sided
Secondary
Genome-Wide Gene Expression and Targeted Genetic Polymorphisms in SCD Patients Linked to a Quantitative Noninvasive-based PH Phenotype.
To detect genome-wide gene expression and targeted genetic polymorphisms in SCD patients linked to a quantitative noninvasive-based PH phenotype.
Time frame: median follow up 3 years
Population: All efforts were taken to gather all possible data but none were obtained for this Outcome Measure.