Haematological Disorders Paediatrics
Conditions
Interventions
Hydroxyurea
Sponsors
Vanderbilt University Medical Centre
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) diagnosis of HbSS or HbSB0 confirmed by electrophoresis 2) informed consent from the parent/legal guardian and assent from patient 3) two TCD flow velocity readings of >200 cm/sec and 220 cm/sec; typically the repeat TCD is performed on the same day so treatment can start immediately 4) age between 5 and 12 years (assessment can take place up until the 13th birthday), which includes the peak age of onset of strokes in SCA, ~ 6 yo 5) ability to swallow the hydroxyurea capsule.
Exclusion criteria
Exclusion criteria: 1) prior stroke or TIA by history, or concern for moderate or severe neurological deficit based on a positive validated "10 questions" screening 2) other significant organ system dysfunction or other contraindication to hydroxyurea 3) Children who are already on therapy with either blood transfusion or hydroxyurea therapy 4) significant cytopenias (absolute neutrophil count (ANC) 9 g/dl], renal insufficiency (creatinine > 0.8 mg/dl) 5) other significant organ system dysfunction, or other contraindication to hydroxyurea therapy 6) history of seizures or diagnosis of epilepsy, and 7) metal in the body that would make MRI unsafe.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| (the primary outcome measure): The Nigerian radiologist's review panel (n=3) and the Nigerian psychiatrist/neurologist (n=1) will be blinded to the clinical history. As in the SPRING Trial cases, all potential strokes will be adjudicated in a masked fashion. We will use the WHO definition of a stroke28. The core radiology team has already been formally certified to detect strokes based on the MRI in children with sickle cell anemia and will follow the methodology previously established in the SIT trial study group. The Nigerian team clinical staff were trained to identify stroke using the Pediatric NIH Stroke Scale (PedNIHSS), a validated, standardized neurological examination assessing and quantifying the severity of stroke in children. Outcomes. The primary outcome will be an incident stroke or TIA. In addition to careful instructions reporting stroke symptoms to the hospital, parents will be asked about stroke symptoms, and children will have a neurological exam every 6 months at clinic visits. The pediatric NIH stroke scale30 and a gait exam have been used to detect strokes. Capacity building for Aim 1: Creating a coordinating data center for the SPRING-2 in Kano, Nigeria. A major goal of our capacity-building effort is transferring the skills required to become a data coordinating center and perform a DSMB. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary outcome measure will be hospitalization for any cause. As with SPRING, all hospital admissions will be centrally adjudicated without knowing the treatment assignment. Each hospitalization and unscheduled outpatient visit will be adjudicated for the primary reason for admission in the following hierarchal and mutually exclusive categories: stroke, acute chest syndrome, pain, or fever (temperature > 38°C), with each event assigned only one primary reason. The acute vaso-occlusive event will be defined as either pain or acute chest syndrome. Malaria will be diagnosed as per the World Health Organization and local study site clinical practice. | — |
Countries
Nigeria
Contacts
Public ContactBilya Musa
Trials Manager
Outcome results
None listed