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
Corresponding Author
Gian Luca Forni
Centro della Microcitemia e delle Anemia Congenite – Ematologia, Ospedale Galliera, Genoa, Italy
Correspondence: Gian Luca Forni, Centro della Microcitemia e delle Anemie Congenite, Ospedale Galliera, Via Volta 6, 16128 Genoa, Italy.
E-mail: [email protected]
Search for more papers by this authorSilverio Perrotta
Department of Paediatrics and Orthopaedics, Second University of Naples, Naples, Italy
Search for more papers by this authorAndrea Giusti
Department of Gerontology and Muscloskeletal Sciences, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorGiovanni Quarta
Division of Haematology, Presidio Ospedaliero A. Perrino, Brindisi, Italy
Search for more papers by this authorLorella Pitrolo
UO Ematologia con Talassemia, Ospedali Villa Sofia-V.Cervello, Palermo, Italy
Search for more papers by this authorMaria Domenica Cappellini
Fondazione IRCCS ‘Ca Granda’, Ospedale Maggiore Policlinico, Università di Milano, Milan, Italy
Search for more papers by this authorDomenico Giuseppe D'Ascola
Centro Microcitemia, Ospedale ‘Bianchi Melacrino Morelli’, Reggio Calabria, Italy
Search for more papers by this authorCaterina Borgna Pignatti
Department of Clinical and Experimental Medicine, University of Ferrara, Arcispedale Sant'Anna, Ferrara, Italy
Search for more papers by this authorPaolo Rigano
UO Ematologia con Talassemia, Ospedali Villa Sofia-V.Cervello, Palermo, Italy
Search for more papers by this authorAldo Filosa
UOS Talassemia Pediatrica ed Emoglobinopatie Pediatriche, Azienda Ospedaliera ‘Antonio Cardarelli’, Naples, Italy
Search for more papers by this authorGiovanni Iolascon
Department of Paediatrics and Orthopaedics, Second University of Naples, Naples, Italy
Search for more papers by this authorBruno Nobili
Department of Paediatrics and Orthopaedics, Second University of Naples, Naples, Italy
Search for more papers by this authorMarina Baldini
Fondazione IRCCS ‘Ca Granda’, Ospedale Maggiore Policlinico, Università di Milano, Milan, Italy
Search for more papers by this authorAlessandra Rosa
Centro della Microcitemia e delle Anemia Congenite – Ematologia, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorValeria Pinto
Centro della Microcitemia e delle Anemia Congenite – Ematologia, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorErnesto Palummeri
Department of Gerontology and Muscloskeletal Sciences, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorCorresponding Author
Gian Luca Forni
Centro della Microcitemia e delle Anemia Congenite – Ematologia, Ospedale Galliera, Genoa, Italy
Correspondence: Gian Luca Forni, Centro della Microcitemia e delle Anemie Congenite, Ospedale Galliera, Via Volta 6, 16128 Genoa, Italy.
E-mail: [email protected]
Search for more papers by this authorSilverio Perrotta
Department of Paediatrics and Orthopaedics, Second University of Naples, Naples, Italy
Search for more papers by this authorAndrea Giusti
Department of Gerontology and Muscloskeletal Sciences, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorGiovanni Quarta
Division of Haematology, Presidio Ospedaliero A. Perrino, Brindisi, Italy
Search for more papers by this authorLorella Pitrolo
UO Ematologia con Talassemia, Ospedali Villa Sofia-V.Cervello, Palermo, Italy
Search for more papers by this authorMaria Domenica Cappellini
Fondazione IRCCS ‘Ca Granda’, Ospedale Maggiore Policlinico, Università di Milano, Milan, Italy
Search for more papers by this authorDomenico Giuseppe D'Ascola
Centro Microcitemia, Ospedale ‘Bianchi Melacrino Morelli’, Reggio Calabria, Italy
Search for more papers by this authorCaterina Borgna Pignatti
Department of Clinical and Experimental Medicine, University of Ferrara, Arcispedale Sant'Anna, Ferrara, Italy
Search for more papers by this authorPaolo Rigano
UO Ematologia con Talassemia, Ospedali Villa Sofia-V.Cervello, Palermo, Italy
Search for more papers by this authorAldo Filosa
UOS Talassemia Pediatrica ed Emoglobinopatie Pediatriche, Azienda Ospedaliera ‘Antonio Cardarelli’, Naples, Italy
Search for more papers by this authorGiovanni Iolascon
Department of Paediatrics and Orthopaedics, Second University of Naples, Naples, Italy
Search for more papers by this authorBruno Nobili
Department of Paediatrics and Orthopaedics, Second University of Naples, Naples, Italy
Search for more papers by this authorMarina Baldini
Fondazione IRCCS ‘Ca Granda’, Ospedale Maggiore Policlinico, Università di Milano, Milan, Italy
Search for more papers by this authorAlessandra Rosa
Centro della Microcitemia e delle Anemia Congenite – Ematologia, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorValeria Pinto
Centro della Microcitemia e delle Anemia Congenite – Ematologia, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorErnesto Palummeri
Department of Gerontology and Muscloskeletal Sciences, Ospedale Galliera, Genoa, Italy
Search for more papers by this authorSummary
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.)
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