Brief Title
Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia
Official Title
Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia
Brief Summary
This study will use neuropsychological tests to look at nervous system side effects of
Cyclosporine (CsA) in patients with aplastic anemia. CsA is used as part of an
immunosuppressive regimen in treating severe aplastic anemia. The drug can produce nervous
system side effects, such as tremor and, less commonly, insomnia, anxiety, headache,
confusion or seizures. This study will look at effects of CsA on intellectual ability,
depression, anxiety, attention, concentration, memory, perception, coordination, and thought
processing in patients
Patients 15 years of age or older who have severe aplastic anemia may be eligible for this
study if they:
- are co-enrolled in a Clinical Center protocol in which they will receive CsA
- have not taken CsA for 6 months before enrolling in this study
Participants undergo neuropsychological testing. In addition, they provide blood samples and
their clinical data are reviewed for things that may influence the interpretation of findings
from the testing, such as results of blood tests, types of medications taken, number of
transfusions required, etc. The procedures are as follows:
Before first dose of cyclosporine:
- Patients are asked about prior problems with their nervous system, prior treatment for
their aplastic anemia (including transfusions), prior infections, and current
medications. They then complete the following sets of tests:
- Battery 1: A set of three tests that measure intellectual ability, level of depression
(if any) and level of anxiety (if any).
- Battery 2: A set of seven tests that measure changes in the central nervous system and
how these changes affect attention, concentration, memory, perception, coordination, and
thought processing.
- Patients provide a half teaspoon of blood for this study at the same time blood is
collected for their primary treatment protocol.
6 months and 12 months after starting cyclosporine
- Patients are asked about treatment for their aplastic anemia (including transfusions),
infections, and changes in medications that have occurred since they started taking
cyclosporine. They then repeat the set of tests in Battery 2.
- Patients provide a half teaspoon of blood for this study at the same time blood is
collected for their primary treatment protocol.
Detailed Description
Cyclosporine (CsA) is widely used in the treatment of aplastic anemia as well as to provide
immunosuppression after transplantation. CsA has a spectrum of neurologic and more subtle,
poorly investigated neuropsychological functional effects. In the NHLBI, a unique opportunity
exists to evaluate changes in neuropsychological function following CsA in a large cohort of
aplastic anemia patients accrued to NHLBI treatment protocols.
With increased success in treating blood diseases with immunosuppressive therapy or stem cell
transplantation that utilize CsA in the treatment regimen, patients are enjoying prolonged
survival during which quality of life becomes an increasingly relevant concern. We therefore
propose this natural history protocol designed to evaluate neuropsychological functioning
before, during, and after CsA administration in severe aplastic anemia patients.
The primary objective is to evaluate changes in attention, language, memory, spatial/motor
and executive function domains following CsA therapy using well established
neuropsychological tests including the Neuropsychological Assessment Battery (NAB) Screening
Module, Revised Hopkins Verbal Learning Test, Revised Brief Visual-spatial Memory Test,
Judgment of Line Orientation test, Grooved Pegboard, Trail Making Test and the two
performance subsets in Wechsler Adult Intelligence Scale-III. IQ test by Wechsler Abbreviated
Scale of Intelligence and depression/anxiety level will be screened once prior to formal
testing. Measures will also be correlated with clinical status, standard CsA side effects,
CsA drug levels and relevant biochemical lab values at each time point to control the testing
accuracy and explore potential correlation.
Study Type
Observational
Condition
Aplastic Anemia
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
7
Start Date
September 23, 2008
Completion Date
June 28, 2011
Eligibility Criteria
- INCULSION CRITERIA:
- Diagnosed with aplastic anemia.
- Co-enrolled on a Clinical Center CsA treatment protocol that prescribes CsA (included
but not limited to initial treatment cohort in both rabbit and horse ATG arm for the
aplastic anemia patients).
- Age greater than or equal to 15 years old.
EXCLUSION CRITERIA:
- Prior use of cyclosporine within 6 months to next line of treatment.
- History of learning disability (i.e. dyslexia).
- Unable to read and speak English (the neuropsychological testing tools are validated
for use in English speaking subjects only).
- Life expectancy less than six months or when clinical status prevents full performance
with testing.
- Either adult patients or guardians for the minor patient, unable to comprehend the
investigational nature of the study and provide informed consent.
- Inability or unwillingness to come to Clinical Center for the 6-month and 12-month
follow-up appointments.
Gender
All
Ages
15 Years - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00767650
Organization ID
080222
Secondary IDs
08-H-0222
Study Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study Sponsor
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Verification Date
June 28, 2011