The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork problems and if they have any side effects.
The study will assess any thinking or attention problems participants may have both before taking this drug and after. Additionally, the study will assess the decision-making process of the caregiver that may influence using this drug or not.
Primary Objective:
• Assess the feasibility, acceptability, and adherence to MPH treatment in children with SCD and EF deficits.
Secondary Objective:
• Evaluate neurobehavioral and safety outcomes following MPH treatment.
Exploratory Objective:
• Evaluate decision-making and determinants influencing methylphenidate utilization among caregivers.
Diagnosed with SCD
Enrolled on the institutional protocol: Sickle Cell Clinical Research Intervention Program (SCCRIP)
Between the ages of 8.0 and 17.9 years
NIH Toolbox Flanker
Primary language other than English
Score below the 2nd percentile on the Wechsler Abbreviated Scale of Intelligence (WASI)-2 intelligence quotient (IQ) test (ever)
Uncontrolled seizures (seizure within the past 6 months)
Cardiomyopathy or known congenital structural cardiac defects
Single group
Extended-Release Methylphenidate
DrugParticipants will receive a weight-based dose of extended-release methylphenidate (0.6 mg/kg/day), rounded to either 10 mg or 20 mg, taken orally once daily for 4 weeks.
Assess feasibility of methylphenidate
Feasibility is measured by the participation rate (i.e., ratio of those who agree to participate to those approached).
Assess acceptability of methylphenidate
Acceptability is captured by parent- and self-report ratings on the Acceptability of Intervention Measure (AIM).
Assess adherence to methylphenidate
Adherence is measured through weekly pill counts. The primary adherence outcome is the ratio of the number of pills taken to those dispersed.
Behavior Assessment System for Children, 3rd Edition (BASC-3), Parent Report
Attention, Hyperactivity, and Depression scales are calculated. The investigators will determine the amount and pattern of missing data. The relative change (defined as (post - pre)/pre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.
Behavior Rating Inventory of Executive Function, 2nd Edition (BRIEF-2), Parent Report
Global Executive Composite Scale is calculated. The investigator will determine the amount and pattern of missing data for neurobehavioral outcomes. The relative change (defined as (post - pre)/pre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.
Pediatric Quality of Life Inventory (PedsQL) Sickle Cell Disease modules, Parent Report
The investigator will determine the amount and pattern of missing data for neurobehavioral outcomes. The relative change (defined as (post - pre)/pre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.
Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue modules, Parent Report
The investigator will determine the amount and pattern of missing data for neurobehavioral outcomes. The relative change (defined as (post - pre)/pre)) will be calculated for each outcome and rank these changes to determine sensitivity of the measures. The goal of measuring neurobehavioral outcomes in this pilot trial is to determine how feasible the measures are for the target population and their relative sensitivity to the effects of methylphenidate treatment.
Evaluate decision-making and determinants influencing methylphenidate utilization among parents
Semi-structured interviews will be conducted. The interviews will be transcribed and qualitatively coded.
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