The St. Jude Pediatric SCD Program has developed a comprehensive plan of care that spans
the ages of 0 to 25, and provides the structure for screening and monitoring disease
progression and complications in infancy, childhood, and young adulthood. From age 0 to
18, SCD patients are followed at St. Jude Children's Research Hospital. At age 18, their
care is typically transferred to either the Methodist Adult Comprehensive Sickle Cell
Disease Center in Memphis, TN, or the Regional One Health, Diggs-Kraus Sickle Cell Center
in Memphis, TN, where they are routinely followed from age 18 to 25 years. After age 25,
participants will be followed and invited to return to St. Jude every 6 years for study
related tests until participants elect to come off study or until death.
St. Jude Children's Research Hospital, the Methodist Adult Comprehensive Sickle Cell
Disease Center and the Regional One Health Diggs-Kraus Sickle Cell Center, in Memphis, TN
serve as enrolling centers for the SCCRIP protocol. Two St. Jude Affiliate locations will
also be sites of enrollment for this protocol for patients age 0 to 18 years. These
include St. Jude Affiliate sites located in: Peoria, Illinois and Charlotte, North
Carolina. Approximately 300 additional participants are expected to be enrolled from
these affiliate sites. This protocol will collect data on SCD participants from birth to
end of life.
The SCD plan of care provides the specific sequence of laboratory and imaging studies
that are performed according to the patient's age and expected course of illness. The
following health outcomes are systematically monitored in patients with SCD: hematologic
indices, pulmonary function, cardiac function, renal function, cognitive function,
cerebral vasculopathy, vitamin D deficiency and bone health, parvovirus B19 immune
status, ophthalmologic status, and splenic function. These tests are used to direct the
patient's clinical management and initiate therapies when necessary.
Participants will be administered a developmental evaluation at approximately 1 year of
age. The evaluation will utilize standardized performance-based measures as well as
parent rating scales lasting approximately 2 hours. Domains assessed will include
cognitive, motor, language, and adaptive development.
A Neuropsychological Screener may be completed with participants within the Young Adult
and Adult cohorts, every 6 years. The screening will include a series of verbal and
nonverbal problem-solving activities, pencil & paper tasks, and will include a
computerized component.
Quality of Life evaluations (Pediatric Quality of Life Inventory (PedsQL™) will be
offered.
In this study, the results of these tests will be collected and entered into the study
database, providing longitudinal data that will inform health outcomes research regarding
SCD and how the course is altered by disease-modifying therapy, in addition to
facilitating future interventional projects.
Primary Objectives:
To establish a longitudinal clinical cohort of patients with sickle cell disease
(SCD) to serve as a research resource to facilitate evaluation of health outcomes in
SCD from pediatric care into adulthood.
To facilitate the collection of biological samples from patients with SCD to be used
in future studies investigating genetic and epigenetic contributions to disease
severity, response to treatment, and morbidity and mortality.
Secondary Objectives:
To determine the incidence, prevalence, and severity of SCD complications and
adverse health conditions within the SCD cohort during five stages of development
and adulthood: the newborn period (birth to 5.9 months), the infant/pre-school stage
(ages 6 months to 5.9 years), the early school stage (ages 6 to 11.9 years), the
adolescent stage (ages 12 to 17.9 years), young adulthood (ages 18 to 24.9 years)
and mature adulthood (ages 25 and above).
To identify and evaluate risk factors for premature mortality and long-term
morbidity in patients with SCD, including those related to disease-modifying
therapies, end-organ damage, genetics, neurocognitive deficits, psychosocial
factors, and behavioral causes.
To investigate the long-term effects of hydroxyurea and other therapies on
preservation of organ function, growth and development, and frequency and severity
of disease complications, and their long-term medical, neurocognitive, and
psychosocial toxicities.
To determine the functional aspects of the Transition to Adult Care Program within a
clinical research cohort by evaluating disease specific health literacy and
readiness in relation to healthcare utilization during adult care.
To explore the long-term alterations of prolonged antibiotic exposure on the
microbial community composition among people living with SCD through the collection
of swabs as guided by the Human Microbiome Project (HMP) Manual of Procedures.
Other Pre-Specified Objective:
- Define the drug-exposure to clinical response relationship of HU therapy in children
with SCD.