Clinical trial NCT02187445: Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Official title: Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Sickle Cell Disease
Asthma
Acute Chest Syndrome
Keywords: Sickle Cell Disease
Budesonide inhalation suspension
Acute Chest Syndrome
Lead sponsor: Vanderbilt University Medical Center
Sponsor class: OTHER
Collaborators: name: Emory University
name: Children's National Research Institute
First posted: 2014-07-11
Enrollment: 36
Sex: ALL
Minimum age (years): 1
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: 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).


Eligibility criteria: Inclusion Criteria:

* 1) confirmed diagnosis of sickle cell disease (SCD)
* 2) age between 1 and 4 years (must have reached 1st but not yet 4th birthday)
* 3) a prior diagnosis of ACS, defined as acute respiratory illness with a new radiodensity on CXR, and one of the following: fever (temperature \> 38.50C), decrease in oxygen saturation more than 3% from baseline, or increase in respiratory rate above baseline

Exclusion Criteria:

* 1) patients already taking inhaled corticosteroids
* 2) those receiving blood transfusions for elevated TCD or strokes
* 3) presents over 2 weeks after discharge from hospital following initial ACS episode.

Participants may be on hydroxyurea and participate in this trial.

Primary outcomes:
measure: The acceptability of budesonide inhalation suspension
timeFrame: 6 Months
Secondary outcomes:
measure: The impact of BIS on biological correlates of inflammation.
timeFrame: 12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks)
Other outcomes:
measure: Quality of life for guardians of children with sickle cell disease and ACS
timeFrame: 0 weeks, 12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks)
measure: Respiratory symptoms
timeFrame: 6 months, monthly
Locations:
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
Study officials:
affiliation: Vanderbilt University Medical Center
name: Michael R DeBaun, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: