Examining Cognitive Function and Brain Abnormalities in Adults With Sickle Cell Disease - Pilot Intervention Study
|Anemia, Sickle Cell||Procedure: Monthly blood transfusion regimen Behavioral: Usual care||Phase 1|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: No masking
Primary Purpose: Treatment
|Official Title:||Neuropsychological Dysfunction and Neuroimaging Abnormalities in Neurologically Intact Adults With Sickle Cell Disease - A Pilot Intervention Study|
- Cognitive function [ Time Frame: Measured at Month 6 ]
|Study Start Date:||December 2004|
|Study Completion Date:||December 2010|
|Primary Completion Date:||December 2010 (Final data collection date for primary outcome measure)|
Participants will receive monthly blood transfusions.
Procedure: Monthly blood transfusion regimen
Participants will receive blood transfusions at 3- to 4-week intervals for 6 months for the treatment of SCD-related anemia; the total number of transfusions that participants will receive will vary between six and eight.
Other Name: Chronic transfusion therapy
Active Comparator: 2
Participants will receive usual care.
Behavioral: Usual care
Participants will receive usual care for the treatment of SCD.
Other Name: Standard care alone, guided by disease symptoms.
SCD is an inherited blood disorder. Symptoms include anemia, infections, organ damage, and intense episodes of pain, which are called "sickle cell crises." In the past, SCD was considered a fatal disease, and many people with SCD died at a young age. Due to advances in medical care, people with SCD are now living longer lives; however, they often experience a deterioration in quality of life due to progressive organ failure. Past research has suggested that children with SCD commonly have frontal lobe dysfunction syndrome, which is a brain disorder that can affect cognitive functioning in areas such as attention, concentration, information processing, and decision making. Often times, however, neurocognitive and brain disorders are not diagnosed or treated in people with SCD. In preliminary brain imaging studies, at least half of adult participants with SCD had visible cognitive dysfunction, while participants without SCD rarely had visible changes in the brain. Brain dysfunction may be one of the most important and least-studied problems affecting adults with SCD.
Most people with SCD have anemia, or low levels of red blood cells, which are the cells that carry oxygen to the body's tissues, especially the brain. Research has shown that in people with anemia who do not have SCD, memory and attention problems have decreased after receiving treatment for anemia. The purpose of this study is to determine whether people with SCD who receive monthly blood transfusions to treat their anemia experience greater cognitive functioning than adults with SCD who receive usual care.
The first phase of this study was an observational study that enrolled adults with SCD and a control group of healthy adults who did not have SCD. Study procedures included questionnaires, neuropsychological testing, and MRI testing. At the end of the first phase, participants were asked if they were willing to take part in a second phase of the overall study in the future. Enrollment into the first phase ended in February 2008.
This current pilot study is the second phase of the overall study. In this study, participants will begin by completing questionnaires, a medical history review, a physical exam, a neurological exam, and a blood collection. Women will provide a urine sample for a pregnancy test. An MRI and neuropsychological testing will also occur. Participants will then be randomly assigned to receive either blood transfusions or usual care for 6 months. Participants assigned to blood transfusions will receive the transfusions every 3 to 4 weeks for 6 months. Before each transfusion, participants will undergo blood collection and a review of medical history and medication history. Participants assigned to usual care will receive a telephone call from study researchers at Months 1, 2, 4, and 5, at which time medical and medication history will be reviewed. At study visits at Months 3 and 6, these participants will also undergo a blood collection. At Month 6, all participants will complete health and quality of life questionnaires, neuropsychological testing, and an MRI.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00850018
|United States, California|
|Children's Hospital & Research Center at Oakland|
|Oakland, California, United States, 94609|
|United States, District of Columbia|
|Washington, District of Columbia, United States, 20001|
|United States, Florida|
|Memorial Cancer Institute|
|Hollywood, Florida, United States, 33021|
|University of Miami Miller School of Medicine|
|Miami, Florida, United States, 33136|
|United States, Georgia|
|Medical College of Georgia|
|Augusta, Georgia, United States, 30912|
|United States, Maryland|
|Baltimore, Maryland, United States, 94117|
|United States, Massachusetts|
|Boston Medical Center|
|Boston, Massachusetts, United States, 02118|
|United States, Michigan|
|Karmanos Cancer Institute at Wayne State University|
|Detroit, Michigan, United States, 48201|
|United States, Missouri|
|St. Louis, Missouri, United States, 63110|
|United States, North Carolina|
|University of North Carolina at Chapel Hill|
|Chapel Hill, North Carolina, United States, 27514|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27710|
|United States, Ohio|
|Cincinnati Children's Hospital|
|Cincinnati, Ohio, United States, 45229-3039|
|University of Cincinnati Medical Center|
|Cincinnati, Ohio, United States, 45267|
|United States, Texas|
|University of Texas Medical Branch|
|Galveston, Texas, United States, 77555|
|Principal Investigator:||Elliott Vichinsky, MD||Children's Hospital & Research Center Oakland|