Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial

Acute and chronic pulmonary complications with concomitant inflammatory response are a
leading cause of morbidity and mortality in children with sickle cell disease (SCD). Acute
chest syndrome (ACS), defined broadly as an increase in respiratory effort, fever and new
radiodensity on chest x-ray, is a major cause of death in children and adults with SCD.
There is a high rate of ACS in children between 1 and 4 years of age that is associated with
an asthma diagnosis, and children with ACS events before 4 years of age have a 50% rate of
being hospitalized for either ACS or pain within 1 year of admission. For children with SCD
that develop ACS, the investigators propose that the use of budesonide inhalation suspension
(BIS) will attenuate pulmonary inflammation after an ACS episode and will decrease future
vaso-occlusive pain and ACS episodes. Through a single-arm prospective feasibility trial and
in preparation for a limited-institution randomized trial, the investigators plan to test
the following primary hypothesis for a phase III definitive trial: In children with SCD
admitted to the hospital for an ACS episode between 1 and 4 years of age, low dose BIS for 6
months will result in a 50% reduction in the recurrent incidence rate of ACS or pain
requiring hospitalization. Through this trial, the investigators will determine the
acceptability of and adherence to BIS in the study population. The investigators will track
respiratory symptoms in cases versus controls over 6 months. Finally, the investigators will
explore the impact of BIS on biological correlates (sVCAM-1).