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Teriparatide in Two Patients With Mucopolysaccharidosis Type IVB
Mark Wijnen1,2, Evert F S van Velsen2, J Gert-Jan Milhous3
1Department of Internal Medicine, Center for Lysosomal and Metabolic Diseases Erasmus Medical Center Rotterdam the Netherlands.
JIMD Reports
|April 16, 2026
Summary
Teriparatide treatment in mucopolysaccharidosis type IVB patients showed bone mineral density gains but led to serious cardiac issues, including aortic stenosis and outflow tract obstruction. Cautious use is recommended.
Area of Science:
- Biochemistry
- Genetics
- Medicine
Background:
- Mucopolysaccharidosis Type IV (MPS IV) is a genetic disorder causing skeletal dysplasia due to impaired glycosaminoglycan degradation.
- Low bone mineral density (BMD) and cardiovascular issues are key features of MPS IV.
- MPS IV is classified into Types A and B based on specific enzyme deficiencies.
Purpose of the Study:
- To evaluate the efficacy of teriparatide in improving BMD in adult MPS IVB patients.
- To investigate potential adverse effects of teriparatide, particularly cardiac complications, in MPS IVB patients.
Main Methods:
- Case report of two adult patients with MPS IVB.
- Treatment with teriparatide for low BMD.
- Monitoring of BMD and cardiac function throughout the treatment period.
Main Results:
- Teriparatide treatment led to significant BMD gain in one patient over 2 years, enabling fracture repair.
- The second patient showed stable BMD after 6 months of teriparatide.
- Both patients developed serious cardiac complications (aortic stenosis, worsening outflow tract obstruction) during teriparatide therapy, requiring invasive cardiac interventions.
Conclusions:
- Teriparatide may improve BMD in MPS IVB patients.
- A potential link between teriparatide and the onset or worsening of cardiac problems in MPS IVB patients warrants further investigation.
- Cautious use of teriparatide is advised in MPS IVB patients due to the risk of cardiac adverse events.
