Examining Cognitive Function and Brain Abnormalities in Adults With Sickle Cell Disease - Pilot Intervention Study
Condition: Anemia, Sickle Cell
MeSH terms: Anemia, Sickle Cell
Study type: Interventional
Phase: Phase 1

Summary: 
      Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease
      that can cause intense pain episodes and may lead to organ failure. Preliminary studies have
      shown that adults with SCD may have brain abnormalities that contribute to problems with
      cognitive functioning, including attention and memory difficulties. This study will use brain
      magnetic resonance imaging (MRI) and neuropsychological testing to examine the differences in
      cognitive functioning in adults with SCD who are treated for anemia with monthly blood
      transfusions for 6 months versus adults with SCD who receive usual care for 6 months.
    

Detailed description: 
      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.
    

Intervention name(s): Monthly blood transfusion regimen; Usual care
Intervention type(s): Procedure; Behavioral
Intervention description(s): 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.; Participants will receive usual care for the treatment of SCD.
Other intervention name(s): Chronic transfusion therapy; Standard care alone, guided by disease symptoms.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Sickle Cell Disease; Sickle Cell Anemia; Hemoglobin SS; Hemoglobin SB0