Prenatal Diagnosis and 10-Year Follow-up of Type-II Generalized Arterial Calcification of the Infancy

Daniele De Luca1,2, Daniela Laux3, Giulia Regiroli1,2

  • 1Division of Pediatrics and Neonatal Critical Care, APHP-Paris Saclay University, Paris, France.

Insights

Generalized arterial calcification of infancy (GACI) is a rare disorder. This study shows type II-GACI with ABCC6 mutations can be effectively managed with bisphosphonates, leading to normal long-term outcomes.

Area of Science:

  • Genetics
  • Pediatrics
  • Cardiology

Background:

  • Generalized arterial calcification of infancy (GACI) is an ultra-rare, life-threatening mineralization disorder.
  • Obstetricians and neonatologists must be aware of GACI's potential impact.

Purpose of the Study:

  • To describe twins with ATP binding cassette subfamily C member-6 (ABCC6) mutations.
  • To detail the multidisciplinary management and long-term (10-year) follow-up of a type II-GACI phenotype.

Main Methods:

  • Case study of twins with ABCC6 mutations.
  • Clinical observation and treatment of neonatal arterial hypertension.
  • Therapeutic intervention with bisphosphonates.
  • Long-term follow-up assessment.

Main Results:

  • One twin presented with type II-GACI, characterized by aortic calcifications and severe neonatal hypertension.
  • Bisphosphonate therapy led to calcification disappearance and hypertension resolution.
  • Ten-year follow-up revealed completely normal outcomes for the affected twin.

Conclusions:

  • Type II-GACI, associated with ABCC6 mutations, can be managed effectively with a multidisciplinary approach.
  • Early intervention with bisphosphonates can yield excellent long-term prognoses.
  • Type II-GACI phenotype may resemble pseudoxanthoma elasticum due to ABCC6 gene mutations.

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