Combined Levothyroxine and Propylthiouracil Treatment in Children with Monocarboxylate Transporter 8 Deficiency: A Multicenter Case Series of 12 Patients
Authors' conclusion
Combined treatment with PTU and LT4 normalized serum T3, fT4, and TT4 in patients with MCT8 deficiency.
Study context
- Population
- Male children with MCT8 deficiency whose parents/guardians consented to propylthiouracil (PTU) and levothyroxine (LT4) treatmentall male
- Participants
- 12 children
- Exposure
- Combined PTU and LT4, last encounter vs baseline
- PTU + LT4n = 12
- Outcome
- Treatment safety and outcomes: biochemical, metabolic and anthropometric parameters
- Follow-up
- Baseline and last encounter
Methodological detailsretrospective data collection January 2008 to June 24, 2024
- Retrospective data collection January 2008 to June 24, 2024
Main finding
As reported in the abstract
Thyroid markers · last encounter vs baseline
Total triiodothyronine (TT3) reduced by PTU at an average dose of 6.8 mg/kg/day
25%
Free T4 (fT4) increased from baseline by LT4 at an average dose of 4.3 µg/kg/day
40%
Observational data: associations, not effects.
Further findings
Metabolic and physical parameters · baseline vs last encounter
- Increase in weight (z-score)
- six of eight patients with data
- No statistical differences overall
Safety
Liver function and granulocyte numbers · throughout observation
- No adverse effects on liver function or granulocyte numbers
Original publication
Combined Levothyroxine and Propylthiouracil Treatment in Children with Monocarboxylate Transporter 8 Deficiency: A Multicenter Case Series of 12 Patients. Thyroid. Published 30 September 2024. doi:10.
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