title: A randomized controlled trial of oral phosphate binders in the treatment of pseudoxanthoma elasticum.
abstract: BACKGROUND
  Pseudoxanthoma elasticum (PXE) is a rare connective tissue disorder involving fragmentation and mineralization of elastic fibers predominantly in the skin, eyes, and cardiovascular system.
  
  
  OBJECTIVE
  The objective of this study was to assess the efficacy of sevelamer hydrochloride on the reversal of elastic fiber calcification and clinical lesions of PXE.
  
  
  METHODS
  This was a randomized, double-blind, placebo-controlled, two-part prospective study. In the first year, 40 patients with PXE were randomized to receive either sevelamer hydrochloride (800 mg by mouth three times daily) or placebo in a 1:1 ratio. In the second year, all patients received sevelamer hydrochloride (800 mg by mouth three times daily).
  
  
  RESULTS
  In the first year, the placebo and treatment groups' mean calcium scores decreased from 29.52 to 15.97 (41.93% mean improvement) and 27.48 to 16.75 (38.37% mean improvement), respectively. In the second year, the mean calcium scores decreased to 13.36 (53.94%) and 14.03 (51.35%) in these groups. The mean clinical score in the placebo group decreased from 6.25 to 6.05 at year 1 (2% improvement) whereas the mean clinical score in the sevelamer hydrochloride group decreased from 7.10 to 6.55 (7% improvement). In year 2, the scores in the original placebo and sevelamer hydrochloride groups decreased to 5.33 (14% improvement) and 5.72 (19% improvement), respectively.
  
  
  CONCLUSIONS
  Magnesium stearate in our placebo and active drugs may have played a confounding role in this study, contributing to the small differences observed in these two groups.
  
  
  CONCLUSIONS
  Sevelamer hydrochloride produced a reduction in both calcification levels and clinical scores; however, this difference was not statistically significant compared with placebo. Future clinical studies should examine the inhibitory role and potential therapeutic effect of magnesium in PXE.
authors.affiliation: Department of Dermatology, Mount Sinai School of Medicine, New York, New York.
authors.forename: Jane Y
authors.initials: JY
authors.lastname: Yoo
authors.affiliation: Department of Dermatology, Mount Sinai School of Medicine, New York, New York.
authors.forename: Robin R
authors.initials: RR
authors.lastname: Blum
authors.affiliation: Department of Dermatology, Mount Sinai School of Medicine, New York, New York.
authors.forename: Giselle K
authors.initials: GK
authors.lastname: Singer
authors.affiliation: Department of Dermatology, Mount Sinai School of Medicine, New York, New York.
authors.forename: Dana K
authors.initials: DK
authors.lastname: Stern
authors.affiliation: Department of Dermatopathology, Mount Sinai School of Medicine, New York, New York.
authors.forename: Patrick O
authors.initials: PO
authors.lastname: Emanuel
authors.affiliation: Department of Ophthalmology, Mount Sinai School of Medicine, New York, New York.
authors.forename: Wayne
authors.initials: W
authors.lastname: Fuchs
authors.affiliation: Department of Dermatology, Mount Sinai School of Medicine, New York, New York; Department of Dermatopathology, Mount Sinai School of Medicine, New York, New York.
authors.forename: Robert G
authors.initials: RG
authors.lastname: Phelps
authors.affiliation: PXE International, Washington, District of Columbia.
authors.forename: Sharon F
authors.initials: SF
authors.lastname: Terry
authors.affiliation: Department of Dermatology, Mount Sinai School of Medicine, New York, New York. Electronic address: lebwohl@aol.com.
authors.forename: Mark G
authors.initials: MG
authors.lastname: Lebwohl
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000704:Analysis of Variance; D001707:Biopsy, Needle; D002118:Calcium / Q000378:metabolism; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007150:Immunohistochemistry; D008297:Male; D008875:Middle Aged; D011073:Polyamines / Q000008:administration & dosage*; D011446:Prospective Studies; D011561:Pseudoxanthoma Elasticum / Q000188:drug therapy* / Q000473:pathology*; D012016:Reference Values; D018570:Risk Assessment; D000069603:Sevelamer; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D011073:Polyamines; D000069603:Sevelamer; D002118:Calcium