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April 22, 2026Clinical Pediatric EndocrinologyOpen Access

Perinatal hypophosphatasia refractory to asfotase alfa with neutralizing antibodies that affected bone mineralization: a case report

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Authors

YKYusuke KamodaNKNanako KawataEUEmina Ubukata

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Overview

Case report reveals NAb effects on bone mineralization in perinatal hypophosphatasia, suggesting individualized ERT dosing.

Key Points

  • This report aims to highlight the challenges in treating severe perinatal hypophosphatasia with asfotase alfa due to neutralizing antibodies.
  • Administered enzyme replacement therapy asfotase alfa starting at 2 months and adjusted dose at 3 years.
  • Monitored rickets resolution and recurrence alongside neutralizing antibody status.
  • Initially resolved rickets by 7 months, but recurrence was noted by 18 months linked to NAb presence.
  • Increased AA dose led to nearly complete resolution of rickets with no NAbs detected at 6 years.

Cite This Study

Kamoda et al. (2026) studied this question.

synapsesocial.com/papers/69e864866e0dea528dde94edhttps://doi.org/10.1297/cpe.2026-0008
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