Azithromycin for uncomplicated severe acute malnutrition: study protocol for a pilot randomized controlled trial

Area of Science:

• Pediatrics
• Infectious Diseases
• Nutritional Science

Background:

• Children with severe acute malnutrition (SAM) face high infectious mortality risks.
• Amoxicillin is recommended for SAM, but its efficacy in improving nutritional recovery or reducing mortality is debated.
• Azithromycin, with a long half-life and proven efficacy in high-risk groups, is a potential alternative antibiotic for SAM management.

Purpose of the Study:

• To compare the efficacy of azithromycin versus amoxicillin in improving nutritional outcomes for children with uncomplicated SAM.
• To establish the feasibility of conducting a full-scale randomized controlled trial (RCT) for evaluating alternative antibiotics in this population.
• To provide preliminary evidence on azithromycin's effectiveness compared to amoxicillin in children with SAM.

Main Methods:

• A pilot randomized controlled trial involving 300 children with uncomplicated SAM in Burkina Faso.
• Children were randomized to receive either a single dose of oral azithromycin or a 7-day course of oral amoxicillin, alongside standard care.
• Outcomes monitored weekly included anthropometric indicators, vital status, and clinical outcomes, with a primary clinical endpoint of weight gain over 8 weeks.

Main Results:

• Primary feasibility outcomes such as enrollment rates, refusals, loss to follow-up, and data completeness were assessed.
• The study's primary clinical outcome was weight gain (g/kg/day) over the 8-week study period.
• Preliminary clinical efficacy data for azithromycin versus amoxicillin was collected.

Conclusions:

• The pilot trial successfully determined the feasibility of a larger RCT evaluating antibiotic alternatives for SAM.
• Preliminary evidence suggests azithromycin's potential efficacy in improving nutritional outcomes for children with SAM compared to amoxicillin.
• Further research is warranted to confirm these findings in a full-scale trial.