Long-Term Effects of Pamidronate in Thalassemic Patients with Severe Bone Mineral Density Deficits

Osteoporosis is a common complication in thalassemia major (TM). Our previous study demonstrated severe bone mineral density (BMD) deficits at spine and hip in 62 and 35% of TM patients. This study assessed the effects of different treatments (calcium, vitamin D and bisphosphonate) on patients' BMD, which was measured at baseline and after 3-year treatments by dual energy X-ray absorptiometry (DEXA). Twenty-one untreated patients, 11 patients on calcium/vitamin D and seven patients on additional pamidronate, were recruited. They were comparable for gender (p = 0.630) and serum ferritin levels (p = 0.412). The median BMD Z-scores at lumbar spine and left hip improved only in patients with standard plus pamidronate treatments (baseline: −3.01 and −3.05, end-of-study: −2.12 and −2.09; p = 0.018 and 0.028, respectively). In contrast, BMD Z-scores at hip worsened in untreated patients (p = 0.034). In conclusion, long-term improvement in BMD in TM patients was observed with bisphosphonate but not calcium and vitamin D treatment.

[1]  I. Holm,et al.  Bone Disease in Thalassemia: A Frequent and Still Unresolved Problem , 2008, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[2]  E. Terpos,et al.  Continuous improvement of bone mineral density two years post zoledronic acid discontinuation in patients with thalassemia-induced osteoporosis: long-term follow-up of a randomized, placebo-controlled trial , 2008, Haematologica.

[3]  A. Gaudio,et al.  Bisphosphonates in the treatment of thalassemia-associated osteoporosis , 2008, Journal of endocrinological investigation.

[4]  R. Baron,et al.  Ethical guidelines for publication of research , 1989, Calcified Tissue International.

[5]  M. Athanassiou-Metaxa,et al.  Effect of zoledronic acid on markers of bone turnover and mineral density in osteoporotic patients with beta-thalassaemia , 2006, Annals of Hematology.

[6]  D. Bowden,et al.  A Randomized, Double-Blind, Placebo-Controlled Trial of Intravenous Zoledronic Acid in the Treatment of Thalassemia-Associated Osteopenia , 2006, Calcified Tissue International.

[7]  E. Neufeld,et al.  Prevalence of fractures among the Thalassemia syndromes in North America. , 2006, Bone.

[8]  E. Terpos,et al.  Zoledronic acid for the treatment of osteoporosis in patients with beta-thalassemia: results from a single-center, randomized, placebo-controlled trial. , 2006, Haematologica.

[9]  V. Lee,et al.  Bone mineral density in children with thalassaemia major: determining factors and effects of bone marrow transplantation , 2005, Bone Marrow Transplantation.

[10]  J. Baron,et al.  Overdiagnosis of osteoporosis in children due to misinterpretation of dual-energy x-ray absorptiometry (DEXA). , 2004, The Journal of pediatrics.

[11]  E. Terpos,et al.  Pamidronate is an effective treatment for osteoporosis in patients with beta‐thalassaemia , 2003, British journal of haematology.

[12]  N. Frisina,et al.  Bisphosphonates in the Treatment of Thalassemia-Induced Osteoporosis , 2002, Osteoporosis International.

[13]  Chi-kong Li,et al.  Patterns of bone diseases in transfusion-dependent homozygous thalassaemia major: predominance of osteoporosis and desferrioxamine-induced bone dysplasia , 2002, Pediatric Radiology.

[14]  Prescott,et al.  High prevalence of low bone mass in thalassaemia major , 1998, British journal of haematology.

[15]  K. M. Chan,et al.  Determinants of axial and peripheral bone mass in Chinese adolescents , 1998, Archives of disease in childhood.

[16]  M. Angastiniotis,et al.  Bone pain in thalassaemia: assessment of DEXA and MRI findings. , 1998, Journal of pediatric endocrinology & metabolism : JPEM.

[17]  A. Hoffbrand,et al.  Genetic and acquired predisposing factors and treatment of osteoporosis in thalassaemia major. , 1998, Journal of pediatric endocrinology & metabolism : JPEM.

[18]  S. Maio,et al.  Longitudinal monitoring of bone mineral density in thalassemic patients. Genetic structure and osteoporosis , 1997, Acta paediatrica.

[19]  P. Delmas,et al.  Measurement of bone mineral content of the lumbar spine by dual energy x-ray absorptiometry in normal children: correlations with growth parameters. , 1990, The Journal of clinical endocrinology and metabolism.

[20]  R. Girot,et al.  Bone disease in children with homozygous β-thalassemia , 1990 .