Ann Hematol (2006) 85: 605–609 DOI 10.1007/s00277-006-0136-y ORIGINAL ARTICLE Zaher K. Otrock . Sami T. Azar . Wael A. Shamseddeen . Dany Habr . Adlette Inati . Suzane Koussa . Rami A. R. Mahfouz . Ali T. Taher Intravenous zoledronic acid treatment in thalassemia-induced osteoporosis: results of a phase II clinical trial Received: 8 March 2006 / Accepted: 22 April 2006 / Published online: 8 July 2006 # Springer-Verlag 2006 Abstract Osteoporosis is an important cause of morbidity in beta-thalassemia patients. Bisphosphonates have been recently used for the treatment of osteoporosis in beta- thalassemia. This study is a prospective quasi-experimental study to assess the efficacy and safety of zoledronic acid in thalassemics with osteoporosis. Eighteen thalassemia patients with osteoporosis were given zoledronic acid 4 mg intravenously every 3 months over a period of 12 months. The efficacy of treatment was assessed by measuring bone mineral density (BMD) at the lumbar spine, femoral neck, and hip at baseline, 6, and 12 months. Z-score was used to measure the BMD. Other medical assessments included markers of bone formation and resorption (bone alkaline phosphatase (BAP), osteocalcin (OC), and urinary deoxypyridinoline), and the assessment of pain score, analgesic score, and performance score. Ten thalassemic osteoporotic patients were followed up only with serial BMDs as controls. Both groups had no significant difference with respect to age, gender, and baseline BMD. Patients taking zoledronic acid had a significant increase in their lumbar spine, femoral neck, trochanter, and total hip BMD measurements over the 12-month period. Patients in the control group did not have any significant change in BMD measurements. There was a significant change in the levels of OC and BAP over the 12-month follow-up period. There was also a significant decrease in the number of painful sites experienced by the patients. Treatment of thalassemic osteoporotic patients with zoledronic acid is very effective in increasing BMD at the lumbar spine and hip and in reducing pain; it is also well-tolerated. Keywords Clinical trial . Osteoporosis . Thalassemia . Zoledronic acid Introduction Thalassemia is a hereditary disease caused by defective globin synthesis resulting in abnormal as well as decreased quantity of globin chains. The life expectancy of beta- thalassemia patients has markedly improved over the last years as a result of regular blood transfusions and compliance with tight iron chelation therapies [1]. How- ever, beta-thalassemia patients still suffer from many complications of their chronic disease, including bone involvement. Bone disease in thalassemia is manifested by diffuse bone pain, spinal deformities like scoliosis, nerve com- pression, and various degrees of osteopenia, osteoporosis, and spontaneous fractures [2]. In fact, osteopenia and/or osteoporosis are major causes of morbidity in these patients who are surviving longer as a result of improved treatment [3]. The etiology of bone disease in thalassemia appears to be multifactorial and is still under investigation. Several factors may affect bone metabolism and turnover in beta- thalassemia patients, such as bone marrow expansion due to increased erythropoiesis, endocrine complications, iron overload and desferrioxamine chelation therapy, vitamins Z. K. Otrock . S. T. Azar . W. A. Shamseddeen . A. T. Taher Department of Internal Medicine, American University of Beirut-Medical Center, P.O. Box 113-0236, Riad El-Solh St., 1107–2020, Beirut, Lebanon D. Habr Novartis Pharma Services, Beirut, Lebanon A. Inati . S. Koussa . A. T. Taher Chronic Care Center, Hazmieh, Lebanon R. A. R. Mahfouz Department of Pathology and Laboratory Medicine, American University of Beirut-Medical Center, Beirut, Lebanon A. T. Taher (*) Hematology–Oncology Division, Department of Internal Medicine, American University of Beirut-Medical Center, P.O. Box 113-0236 Beirut, Lebanon e-mail: ataher@aub.edu.lb Tel.: +961-3-755669 Fax: +961-1-370814