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Use of Oxandrolone to Promote Growth in Neonates following Surgery for Complex Congenital Heart Disease: An
Phillip T Burch1, Michael G Spigarelli2,3, Linda M Lambert1
1Divisions of Cardiothoracic Surgery, University of Utah and Primary Children's Hospital, Salt Lake City, UT, USA.
Insights
Oxandrolone therapy is safe for neonates after complex congenital heart disease surgery. The medium-chain triglyceride (MCT) oil preparation showed the most promising results for weight gain in infants.
Area of Science:
- Pediatric Cardiology
- Neonatal Surgery
- Pharmacology
Background:
- Malnutrition and poor weight gain are common complications in neonates post-complex congenital heart disease (CHD) repair, increasing morbidity and mortality.
- Oxandrolone, an anabolic steroid, has demonstrated efficacy in promoting weight gain in older children with growth failure.
- The safety and efficacy of oxandrolone in neonates undergoing complex CHD surgery remain to be established.
Purpose of the Study:
- To assess the feasibility and safety of oxandrolone therapy in neonates following surgical repair of complex CHD.
- To evaluate the efficacy of oxandrolone in improving weight gain in this vulnerable population.
- To compare different dosage and preparation protocols for oxandrolone administration.
Main Methods:
- A prospective pilot trial involving neonates with RACHS-1 score >3 undergoing complex CHD surgery.
- Three cohorts received open-label oxandrolone (0.1 mg/kg/day aqueous, 0.2 mg/kg/day aqueous, or 0.1 mg/kg/day in MCT oil) for 28 days.
- A control cohort received no oxandrolone; anthropometric measurements, physical examinations for virilization, safety labs, and adverse events were monitored.
Main Results:
- Fifteen neonates received oxandrolone therapy; no virilization, safety lab changes, or serious adverse events were observed.
- The decline in weight-for-age z-score was significantly different across cohorts (P=.012).
- The 0.1 mg/kg/day MCT oil preparation group exhibited the least decline in weight-for-age z-score during the study period.
Conclusions:
- Oxandrolone therapy appears safe for neonates post-complex CHD surgery at the studied doses.
- The MCT oil preparation demonstrated potentially better bioavailability and a possible growth benefit.
- Further research is warranted to optimize dosing and confirm the efficacy of oxandrolone in this population.
Objective:
Malnutrition and poor weight gain, common in neonates following repair of complex congenital heart disease (CHD), are associated with increased morbidity and mortality. Oxandrolone, an anabolic steroid, improves weight gain in older children at high-risk for growth failure. We sought to determine feasibility, safety, and efficacy of oxandrolone therapy in neonates following surgery for complex CHD.
Design:
Neonates with RACHS-1 score >3 were eligible to receive open-label oxandrolone for 28 days in this prospective pilot trial. There were 3 cohorts of 5 subjects receiving oxandrolone therapy under 3 specified dosage and preparation protocols: 0.1 mg/kg/day aqueous solution, 0.2 mg/kg/day aqueous solution, and 0.1 mg/kg/day preparation in medium chain triglyceride (MCT) oil. Age- and diagnosis-matched neonates who underwent surgery, but received no oxandrolone, served as a control cohort. Anthropometric measurements, physical examination for virilization, safety labs, and adverse events were monitored.
Results:
Of 25 eligible patients, 15 consented (60%, 13/15 with Norwood procedure). There was no evidence of virilization, no changes in safety labs, and no serious adverse events related to oxandrolone among subjects receiving therapy. No subject met criteria for termination of study drug. There was a significant difference in change in weight-for-age z-score among the four cohorts, with subjects receiving 0.1 mg/kg/day in MCT oil having the lowest decline during the study period (-1.8 ± 0.5 for controls, -1.7 ± 0.4 for 0.1 mg/kg/day aqueous, -1.0 ± 0.4 for 0.2 mg/kg/day aqueous, and -0.6 ± 0.7 for 0.1 mg/kg/day MCT oil, P = .012).
Conclusions:
Oxandrolone therapy at the doses studied appears safe in neonates after surgery for complex CHD. The decline in weight-for-age z-score was lowest in those receiving the MCT oil preparation suggesting better bioavailability of this preparation and a potential growth benefit with oxandrolone therapy. Further investigation is needed to define optimal dosing and assess efficacy.
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