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rickets type-1; pseudo-vitamin D deficiency

Pathology: 1) inadequate endogenous synthesis of 1,25(OH)2 vit D3 a) 1-alpha-hydroxylation deficiency b) 25-hydroxylation deficiency 2) reduced tubular resorption of phosphate 3) hyperchloremic acidosis may occur Genetics: - autosomal recessive - associated with defects in CYP27B1 Clinical manifestations: - rickets begins < 2 years of age - muscle weakness Laboratory: 1) serum chemistries a) serum Ca+2 is low (hyocalcemia) b) parathyroid hormone (PTH) is elevated c) alkaline phosphatase is elevated d) phosphorous is variable e) 1,25(OH)2vit-D3 is variable f) hyperchloremia may occur 2) urine chemistries a) Ca+2 is diminished b) aminoaciduria may occur c) urinary cAMP increases normally in response to PTH infusion Management: 1) calcitriol (observe for hypercalcemia) 2) calcium 0.5-2.0 g QD

General

hereditary rickets

Database Correlations

OMIM correlations

References

  1. OMIM :accession 264700
  2. Harrison's Principles of Internal Medicine, 13th ed. Isselbacher et al (eds), McGraw-Hill Inc. NY, 1994, pg 1328