Table 9.

Targeted Treatment of Hypophosphatasia

Targeted TreatmentDosageConsiderations
Asfotase alfa ERT 1, 2Asfotase alfa ERT is given as a subcutaneous injection.
  • Infants: total dose ≤9 mg/kg per week
  • Adolescents & adults: total dose of 6 mg/kg per week
  • The most common regimens are a 1 mg/kg injection 6x per week or a 2 mg/kg injection 3x per week.
  • In absence of ERT, calcitonin & steroids could be attempted short term; efficacy is limited. 3

ERT = enzyme replacement therapy

1.

The treatment duration and long-term effects of ERT with asfotase alfa remain unknown for perinatal and infantile hypophosphatasia. In theory, ERT would be less effective once endochondral bone formation is complete after the epiphyses fuse.

2.

Clinical trials in adults are limited to those with documented childhood disease, and in theory the treatment has occurred after endochondral bone formation is complete (remodeling phase). Biochemical and limited functional improvement can be documented, but treatment end points, duration, and long-term effects are unknown for adult hypophosphatasia.

3.

From: Hypophosphatasia

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