title: Treatment of type 1 Gaucher's disease affecting bone with aminohydroxypropylidene bisphosphonate (pamidronate).
abstract: Two patients with Type 1 (adult) Gaucher's disease and major skeletal involvement with multiple fractures have been treated with the second generation bisphosphonate pamidronate for extensive periods. There was evidence of an immediate reduction in bone resorption, with increased calcium absorption (delayed in Patient 1), improved calcium balance and maintained or improved bone density indices in the axial and peripheral skeleton. There was no evidence of immediate relapse on treatment cessation. No toxic effects of pamidronate treatment were identified and subjective skeletal pain diminished in both patients. Histomorphometry of transiliac bone biopsies obtained before the start of treatment, after double in vivo tetracycline labelling, represents one of the earliest reports of the quantitative findings in iliac bone invaded by Gaucher cells.
authors.affiliation: Bone Disease Research Group, MRC Clinical Research Centre, Harrow, UK.
authors.forename: L
authors.initials: L
authors.lastname: Ostlere
authors.forename: T
authors.initials: T
authors.lastname: Warner
authors.forename: P J
authors.initials: PJ
authors.lastname: Meunier
authors.forename: P
authors.initials: P
authors.lastname: Hulme
authors.forename: R
authors.initials: R
authors.lastname: Hesp
authors.forename: R W
authors.initials: RW
authors.lastname: Watts
authors.forename: J
authors.initials: J
authors.lastname: Reeve
mesh_terms: D000328:Adult; D015519:Bone Density / Q000187:drug effects; D001847:Bone Diseases / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D001853:Bone Marrow / Q000473:pathology; D001842:Bone and Bones / Q000473:pathology; D002118:Calcium / Q000378:metabolism; D004164:Diphosphonates / Q000627:therapeutic use*; D005260:Female; D005776:Gaucher Disease / Q000150:complications / Q000188:drug therapy* / Q000473:pathology; D006801:Humans; D008297:Male; D000077268:Pamidronate
chemical_list: D004164:Diphosphonates; D000077268:Pamidronate; D002118:Calcium