A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease
Status:
Withdrawn
Trial end date:
2017-12-31
Target enrollment:
Participant gender:
Summary
Acute chest syndrome (ACS), a lung complication in sickle cell disease (SCD), is the second
most common cause of hospitalization and leading cause of death in SCD. ACS is associated
with airway inflammation, and a major cause is pulmonary infection from atypical organisms.
To date, there are no drugs available to reduce inflammation and risk of recurrent ACS.
Macrolides are a group of antibiotics that exert immunomodulatory and anti-inflammatory
actions both in vitro and in vivo. In addition, macrolides reduce bacterial burden in the
airway of atypical organisms, all of which play an important role in the pathophysiology of
ACS. Numerous studies have evaluated macrolide prophylaxis in conditions associated with lung
inflammation, such as cystic fibrosis, asthma, bronchiectasis etc., and high quality evidence
have found macrolides to be beneficial as a disease modifying agent that leads to improvement
in airway inflammation, reduced pulmonary exacerbations and improved lung function. The
investigators hypothesize that azithromycin prophylaxis is well tolerated and has the
potential to reduce inflammation and improve lung outcome in children with SCD with a history
of ACS. A prospective, single arm, open label feasibility study of azithromycin prophylaxis
will be performed in children with SCD with a history ACS with the specific aim to examine
the feasibility, safety and tolerability of azithromycin prophylaxis administration in
participants with SCD , and to examine whether azithromycin prophylaxis has the potential to
improve lung outcome. In addition, this study will determine whether azithromycin prophylaxis
reduces inflammation in participants with SCD with a history of ACS.