Outcomes of Phentermine-Topiramate in Children Living with Duchenne Muscular Dystrophy
Authors' conclusion
Treatment with PHEN/TPM in a small group of children with DMD and obesity was associated with clinically relevant reductions in measures of obesity and was generally well-tolerated with no significant adverse cardiac effects observed during extended follow-up. These findings support the potential use of PHEN/TPM as a weight management option in carefully selected individuals with DMD, though larger prospective studies are warranted.
Study context
- Population
- Children with genetically diagnosed duchenne muscular dystrophy (DMD) and obesity prescribed PHEN/TPM, Children's Hospital Colorado, 2015–2025; aged 9.9–16.9 years, baseline BMI 29.5–39.7 kg/m²treated ≥6 months
- Participants
- 6 children
- Outcome
- Change in obesity measures
- Follow-up
- First year of therapy
Methodological detailssingle-centre, case series
- Single-centre
- Case series
Main finding
As reported in the abstract
Median BMI decrease (IQR −5.5 to −2.6)
3.75 kg/m²
Median BMI SD decrease (IQR −0.99 to −0.31)
0.57
Median weight SD decrease (IQR −1.17 to −0.59)
0.73
Observational data: associations, not effects.
Further findings
Median annualized changes over full treatment course · 0.6-4.2 years
- BMI (IQR −2.93 to 0.17)
- −1.73 kg/m²
- BMI SD (IQR −0.44 to −0.15)
- −0.31
- Weight SD (IQR −0.70 to −0.11)
- −0.30
Longitudinal trends
- Significant nonlinear trends for BMI and BMI SD, with nadir effect by year two
Safety
Cardiac parameters
- Initial transient HR increase, median at 0.5 years
- +13.5 bpm
- No significant longitudinal changes in HR, systolic and diastolic BP, or LV ejection fraction
Adverse events
- Kidney stones
- 2 individuals
- Other adverse events mild to moderate
Original publication
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