Iron Mediated Vascular Disease in Sickle Cell Anemia Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01239901
Recruitment Status : Active, not recruiting
First Posted : November 15, 2010
Last Update Posted : March 28, 2017
Information provided by (Responsible Party):
John C. Wood, Children's Hospital Los Angeles

Brief Summary:
The purpose of this research study is to determine the frequency and severity of iron overload in patients with Sickle Cell Anemia and its relationship to blood vessel function. The investigators hypothesize that intermittent transfusions that these patients receive during hospitalizations produces significant iron overload and impairs blood vessel relaxation.

Condition or disease
Sickle Cell Disease

Detailed Description:
Patients with sickle cell anemia often require blood transfusion as part of the treatment for their disease. Each teaspoon of transfused blood contains about 5 mg of iron and the levels of iron in sickle cell patients increase rapidly with each transfusion. While iron is necessary for many bodily functions, too much iron damages blood vessels, liver, hormone producing glands (pancreas, pituitary and thyroid) and the heart. It is important to know how iron damages blood vessels because most of the problems experienced by sickle cell anemia patients (stroke, kidney failure, pulmonary hypertension, heart disease) result from blood vessel damage. In this trial, iron in the liver, pancreas, and kidney will be measured noninvasively by MRI while vascular function will be measured by ultrasound and tissue Doppler. Patients will be recruited primarily from the greater Los Angeles area, although patients from greater distances will be allowed to participate.

Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Case-Only
Time Perspective: Cross-Sectional
Official Title: Iron-mediated Vascular Disease in Sickle Cell Disease.
Study Start Date : December 2009
Estimated Primary Completion Date : October 2018
Estimated Study Completion Date : October 2018

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Liver iron concentration (LIC), pancreas R2*, and kidney R2*, measured by MRI [ Time Frame: 15 min MRI done completed during one time study visit ]
    Liver iron concentration (LIC) will be used as a surrogate for total body iron, pancreatic iron represents a surrogate for extravascular iron deposition, and renal iron as a surrogate for chronic intravascular hemolysis. In addition, labile plasma iron will be measured in blood plasma.

Secondary Outcome Measures :
  1. Vascular function. [ Time Frame: Scheduled during one time study visit ]
    Measurements will be done through several procedures: Ultrasound of artery in upper arm, Ultrasound of artery in neck, and blood tests of ascorbate and hydrobiopterins.

Biospecimen Retention:   Samples With DNA
Red Blood Cells (RBCs), Peripheral Blood Mononuclear Cells (PBMCs), Plasma, Serum, and Urine

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Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Our large and well-established sickle cell Center has strong ties to the SCD community in a large metropolitan area (Los Angeles), placing us in an ideal position to rapidly accrue sufficient subjects to study the prevalence of iron overload and determine the relation between directly measured iron loading, vascular responsiveness and clinically relevant biomarkers of vascular function.

Inclusion Criteria:

  • Age > 13 years
  • Documented diagnosis of sickle cell anemia (SS, SC, Sß0, Sß+)
  • Transfused no more than 8 times in a year.

Exclusion Criteria:

  • Cardiac pacemaker, implantable neurostimulator or other MRI incompatible device.
  • History of extreme claustrophobia in MRI machine or other reason for inability to do MRI without sedation.
  • Inability to be positioned on the MRI table for sufficient time to complete the MRI exams.
  • Any medical or psychological condition that, in the opinion of the local investigator, would make it unsafe or ill-advised for the subject to participate.
  • Currently not receiving chronic transfusion therapy, defined as greater than 8 transfusions per year.

Information from the National Library of Medicine

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 identifier (NCT number): NCT01239901

United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Sponsors and Collaborators
Children's Hospital Los Angeles
Principal Investigator: John C Wood, MD, PhD Children's Hospital Los Angeles


Responsible Party: John C. Wood, Associate Professor of Pediatrics - Division of Cardiology, Children's Hospital Los Angeles Identifier: NCT01239901     History of Changes
Other Study ID Numbers: 1RC1HL099412-01 ( U.S. NIH Grant/Contract )
First Posted: November 15, 2010    Key Record Dates
Last Update Posted: March 28, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by John C. Wood, Children's Hospital Los Angeles:
sickle cell disease
sickle cell anemia
sickled cells
blood vessel
iron overload

Additional relevant MeSH terms:
Anemia, Sickle Cell
Vascular Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Cardiovascular Diseases