Clinical trial NCT01239901: Iron Mediated Vascular Disease in Sickle Cell Anemia Patients
Official title: Iron-mediated Vascular Disease in Sickle Cell Disease.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Keywords: sickle cell disease
sickle cell anemia
sickled cells
blood vessel
iron overload
Lead sponsor: Children's Hospital Los Angeles
Sponsor class: OTHER
First posted: 2010-11-15
Enrollment: 150
Sex: ALL
Minimum age (years): 13
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: CROSS_SECTIONAL
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.

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Liver iron concentration (LIC), pancreas R2*, and kidney R2*, measured by MRI
timeFrame: 15 min MRI done completed during one time study visit
Secondary outcomes:
measure: Vascular function.
timeFrame: Scheduled during one time study visit
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
Study officials:
affiliation: Children's Hospital Los Angeles
name: John C Wood, MD, PhD
role: PRINCIPAL_INVESTIGATOR
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