Neridronate improves bone mineral density and reduces back pain in β-thalassaemia patients with osteoporosis: results from a phase 2, randomized, parallel-arm, open-label study.

Abstract

Neridronate is a third generation bisphosphonate with established efficacy in metabolic bone disease. In this randomized, open-label study, 118 adults with β-thalassaemia and bone mineral density (BMD) Z scores ≤-2·0 were randomized 1:1-500 mg calcium with 400 international unis (iu) vitamin D daily or 500 mg calcium with 400 iu vitamin D daily plus neridronate 100 mg intravenously every 90 d. Significant increases in BMD at the lumbar spine and total hip were noted in the neridronate group at 6 and 12 months from baseline (P < 0·001), and values were significantly higher than the control group at both time intervals. Neridronate also significantly decreased serum bone alkaline phosphatase and C-telopeptide of collagen type 1 levels from as early as 3 months (P = 0·04 and P < 0·001, respectively), reaching significantly lower values at 12 months compared with the control group (P < 0·05). Reductions in back pain and analgesic use were also evident, starting 3 months from commencing treatment. Treatment was well tolerated by all patients. In this largest randomized trial in thalassaemia-induced osteoporosis to date, neridronate was safe and effective in reducing bone resorption and increasing BMD. The associated reduction in back pain and improved quality of life will encourage adherence to therapy. (Clinicaltrials.gov identifier NCT01140321.).

• Authors: Gian Luca Forni, Silverio Perrotta, Andrea Giusti, Giovanni Quarta, Lorella Pitrolo, Maria Domenica Cappellini, Domenico Giuseppe D'Ascola, Caterina Borgna Pignatti, Paolo Rigano, Aldo Filosa, Giovanni Iolascon, Bruno Nobili, Marina Baldini, Alessandra Rosa, Valeria Pinto, Ernesto Palummeri
• Journal: British journal of haematology (Br J Haematol) Vol. 158 Issue 2 Pg. 274-282 (Jul 2012) ISSN: 1365-2141 [Electronic] England
• PMID: 22571408 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: © 2012 Blackwell Publishing Ltd.

Chemical References

• Biomarkers
• Bone Density Conservation Agents
• Collagen Type I
• Diphosphonates
• Peptides
• collagen type I trimeric cross-linked peptide
• 6-amino-1-hydroxyhexane-1,1-diphosphonate
• Alkaline Phosphatase

Topics

• Adolescent
• Adult
• Alkaline Phosphatase (blood)
• Back Pain (etiology, prevention & control)
• Biomarkers (blood)
• Bone Density (drug effects)
• Bone Density Conservation Agents (adverse effects, therapeutic use)
• Bone Remodeling (drug effects)
• Collagen Type I (blood)
• Diphosphonates (adverse effects, therapeutic use)
• Female
• Femur Neck (physiopathology)
• Hip Joint (physiopathology)
• Humans
• Lumbar Vertebrae (physiopathology)
• Male
• Middle Aged
• Osteoporosis (drug therapy, etiology, physiopathology)
• Peptides (blood)
• Quality of Life
• Treatment Outcome
• Young Adult
• beta-Thalassemia (complications)