SCCRIP: Sickle Cell Research and Intervention Program

Description

The purpose of this research study is to collect data on sickle cell disease participants from birth to end of life. With these data, researchers will better understand problems caused by sickle cell disease both in children and in adults. They will also look at long term effects of current treatments, and use these data as a building block for future research.

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.

All secondary aim objectives will be evaluated every five years.

• To determine the incidence, prevalence, and severity of SCD complications and adverse health conditions within the SCD cohort during five stages of development: the newborn period (birth to 6 months), the infant/pre-school stage (ages 6 months to 6 years), the early school stage (ages 6 to 12 years), the adolescent stage (ages 12 to 18 years), and adulthood (ages 18 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.

Eligibility

Inclusion Criteria

• Participant has a diagnosis of Sickle Cell Disease of any genotype.

Exclusion Criteria

• Participant does not have a diagnosis of Sickle Cell Disease of any genotype.

Principal Investigator

Jane Hankins, MD