Aspirin Prophylaxis in Sickle Cell Disease
Condition: Sickle Cell Disease
MeSH terms: Anemia, Sickle Cell
Study type: Interventional
Phase: Phase 1

Summary: 
      Neurologic complications secondary to cerebrovascular damage are prevalent in children with
      sickle cell disease. These patients experience both clinically overt cerebrovascular
      accidents and "silent infarctions" demonstrated by magnetic resonance imaging (MRI). They are
      also at risk for neurocognitive abnormalities.We hypothesize that daily, low-dose aspirin
      therapy will safely diminish the incidence and progression of cognitive deficits as well as
      the predisposition to overt and silent stroke in children with homozygous sickle cell disease
      (Hgb SS) or hemoglobin S Beta Zero Thalassemia (Hgb SB-0 Thal). In order to optimize the
      design of a future trial to test this hypothesis, we propose a pilot study to test the safety
      and tolerability of aspirin in young children with sickle cell disease.
    

Detailed description: 
      The trial's primary objective is to evaluate the safety and tolerability of daily low-dose
      aspirin in children with sickle cell disease. The secondary objectives are to assess (1) The
      feasibility of recruiting children with Hgb SS and Hgb S Beta-0 Thalassemia to an aspirin
      trial, (2) The level of compliance with aspirin administration in the proposed patient
      population, (3) The most useful assessments in a battery of age-appropriate neurocognitive
      tests, (4) The feasibility of magnetic resonance imaging (MRI) and magnetic resonance
      angiography (MRA) studies and the utility of classification systems for use in group
      comparisons, (5) Preliminary data regarding trends in transcranial Doppler (TCD) ultrasound
      velocities over time and the validity of using trends for group comparisons, (6) Preliminary
      data regarding the effect of aspirin therapy on the incidence of cognitive deficit, imaging
      changes, overt stroke, painful crises, and acute chest syndrome. Subjects will include
      children between the ages of 2 and 7.99 years with documented Hgb SS or Hgb S Beta-0
      Thalassemia who are followed at Golisano Children's Hospital at Strong and the University of
      Miami. All subjects will receive daily aspirin (about 2.5 - 5.1 mg/kg daily). Subjects will
      receive therapy for 12 months. There will be careful laboratory and clinical monitoring every
      3-6 months and more frequently if needed. Pre and post treatment clinical complications,
      neurocognitive testing, MRI, MRA, and TCD studies will be assessed.
    

Intervention name(s): aspirin
Intervention type(s): Drug
Intervention description(s): 81 mg flavored chewable tablets. Subjects between the ages of 2.0 and 4.99 years will receive half of an 81 mg aspirin tablet each day. Those older than 5.0 years will receive a daily 81 mg aspirin tablet. The subject will receive the study drug for a period of 12 months.
Other intervention name(s): Acetylsalicyclic Acid; ASA
Intervention MeSH terms: Aspirin
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: sickle cell disease; hemoglobin SS disease; hemoglobin S Beta-0 Thalassemia; silent infarction in sickle cell disease; overt stroke in sickle cell disease; aspirin; transcranial Doppler ultrasound; neurocognitive testing