Bisphosphonate treatment of bone fibrous dysplasia in McCune-Albright syndrome.
One of the main features of McCune-Albright syndrome is bone fibrous dysplasia (BFD) often associated with severe clinical outcomes, such as bone pain, bone deformities and pathological fractures. Medical treatment with bisphosphonates started 15 years ago. Recent trials in pediatric patients with BFD have shown encouraging results. We evaluated long-term efficacy and safety of pamidronate treatment of BFD in children and adolescents with MAS. The drug was administered at 4 month-1 year intervals according to alkaline phosphatase levels. The study included 14 patients (10 females and 4 males between the ages of 5.3 and 18.7 years) with moderate or severe BFD. Follow up lasted 1.9-9 years. Bone pain, fractures, deformities, and bone turnover markers were evaluated before every therapeutic course. The study shows the beneficial effects of long-term bisphosponate treatment on BFD lesions leading to reduced fracture rate and bone pain, and radiological evidence of long bone lesion healing.
Lala
Roberto
R
Pediatric Endocrinology, Regina Margherita Children's Hospital, Turin, Italy. info@malattie-rare.org
Matarazzo
Patrizia
P
Andreo
Maria
M
Marzari
Daniela
D
Bellone
Jaele
J
Corrias
Andrea
A
de Sanctis
Carlo
C
D000293:Adolescent; D000469:Alkaline Phosphatase / Q000378:metabolism; D050071:Bone Density Conservation Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004164:Diphosphonates / Q000627:therapeutic use*; D005260:Female; D005359:Fibrous Dysplasia, Polyostotic / Q000150:complications / Q000188:drug therapy* / Q000201:enzymology; D005500:Follow-Up Studies; D005598:Fractures, Spontaneous / Q000188:drug therapy / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D008297:Male; D010146:Pain / Q000188:drug therapy / Q000517:prevention & control; D000077268:Pamidronate; D018709:Statistics, Nonparametric; D016896:Treatment Outcome
D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D000469:Alkaline Phosphatase; D000077268:Pamidronate