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Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa.

Publication ,  Journal Article
Gospe, SM; Santiago-Turla, C; DeArmey, SM; Cummings, TJ; Kishnani, PS; Bhatti, MT
Published in: Cornea
July 2019

PURPOSE: To assess for ectopic ocular calcification in a series of patients with hypophosphatasia (HPP) treated with asfotase alfa, a recombinant tissue-nonspecific alkaline phosphatase. METHODS: This is a retrospective analysis of subjects enrolled at Duke University Medical Center in ENB-009-10 (ClinicalTrials.gov: NCT01163149), a randomized controlled trial of asfotase alfa in adolescents and adults with HPP. Seven patients between the ages of 45 and 66 years diagnosed with HPP based on clinical features and low serum alkaline phosphatase levels were enrolled at our site. Subjects were randomized to receive either daily subcutaneous injections of asfotase alfa or no treatment. After 24 weeks, during the open-label extension phase, all subjects received treatment for at least 4 years. All subjects underwent comprehensive eye examinations at baseline and at 24-week intervals throughout the study to assess for development of ocular calcifications. RESULTS: By week 120, all 7 subjects developed asymptomatic white refractile deposits in the interpalpebral perilimbal conjunctiva. Biopsy of the conjunctival lesions in 2 subjects revealed elastosis with subepithelial calcification. The lesions were nonprogressive and in 5 subjects exhibited some degree of regression. CONCLUSIONS: Asfotase alfa was invariably associated with development of mild focal conjunctival calcification, likely through disinhibition of hydroxyapatite crystal propagation. The calcifications were not symptomatic or vision-threatening and should not preclude enzyme replacement therapy for patients with this rare and often debilitating disease.

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Published In

Cornea

DOI

EISSN

1536-4798

Publication Date

July 2019

Volume

38

Issue

7

Start / End Page

896 / 900

Location

United States

Related Subject Headings

  • Young Adult
  • Recombinant Fusion Proteins
  • Ophthalmology & Optometry
  • Middle Aged
  • Male
  • Immunoglobulin G
  • Hypophosphatasia
  • Humans
  • Female
  • Enzyme Replacement Therapy
 

Citation

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Chicago
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Gospe, S. M., Santiago-Turla, C., DeArmey, S. M., Cummings, T. J., Kishnani, P. S., & Bhatti, M. T. (2019). Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa. Cornea, 38(7), 896–900. https://doi.org/10.1097/ICO.0000000000001947
Gospe, Sidney M., Cecilia Santiago-Turla, Stephanie M. DeArmey, Thomas J. Cummings, Priya S. Kishnani, and M Tariq Bhatti. “Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa.Cornea 38, no. 7 (July 2019): 896–900. https://doi.org/10.1097/ICO.0000000000001947.
Gospe SM, Santiago-Turla C, DeArmey SM, Cummings TJ, Kishnani PS, Bhatti MT. Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa. Cornea. 2019 Jul;38(7):896–900.
Gospe, Sidney M., et al. “Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa.Cornea, vol. 38, no. 7, July 2019, pp. 896–900. Pubmed, doi:10.1097/ICO.0000000000001947.
Gospe SM, Santiago-Turla C, DeArmey SM, Cummings TJ, Kishnani PS, Bhatti MT. Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa. Cornea. 2019 Jul;38(7):896–900.

Published In

Cornea

DOI

EISSN

1536-4798

Publication Date

July 2019

Volume

38

Issue

7

Start / End Page

896 / 900

Location

United States

Related Subject Headings

  • Young Adult
  • Recombinant Fusion Proteins
  • Ophthalmology & Optometry
  • Middle Aged
  • Male
  • Immunoglobulin G
  • Hypophosphatasia
  • Humans
  • Female
  • Enzyme Replacement Therapy