Open AccessPF787 THE EFFICACY OF ALENDRONATE FOR THE TREATMENT OF THALASSEMIA‐ASSOCIATED OSTEOPOROSIS: A RANDOMIZED CONTROLLED TRIAL
Author(s) -
Piriyakhuntorn P.,
Tantiworawit A.,
Phimphilai M.,
Srichairatanakool S.,
Teeyasoontra W.,
Rattanathammethee T.,
ChaiAdisaksopha C.,
Rattarittamrong E.,
Norasetthada L.,
Charoenkwan P.
Publication year - 2019
Publication title -
hemasphere
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.677
H-Index - 11
ISSN - 2572-9241
DOI - 10.1097/01.hs9.0000561432.83737.6c
Subject(s) - medicine , homogeneous , ejection fraction , thalassemia , multislice , cardiology , randomized controlled trial , cardiac function curve , nuclear medicine , heart failure , physics , thermodynamics
Background: With adequate blood transfusion and iron chelation, thalassemia patients have longer life expectancy and experience long‐term metabolic complications including osteoporosis, fractures and bone pain. Alendronate, which is an oral bisphosphonate, is currently used for the treatment in various types of osteoporosis. However, the efficacy for the treatment of thalassemia‐associated osteoporosis remains unclear. Aims: To evaluate the efficacy of alendronate for the treatment of osteoporosis in thalassemia patients. Methods: In this randomized controlled trial, patients were included if there were males (18–50 years) or premenopausal females with low bone mineral density (BMD) (Z‐score < ‐2.0 SD) or positive vertebral deformities from vertebral fracture analysis (VFA). Stratified randomization was performed according to sex and transfusion status. Patients were 1:1 allocated to receive once weekly alendronate 70 mg orally or placebo for a total duration of 12 months. Markers of bone resorption (C‐terminal crosslinking telopeptide of type I collagen; CTX), bone formation (Procollagen type I N‐terminal propeptide; P1NP) and pain score were measured at baseline, 6 months and 12 months. The primary endpoints were changes of bone markers. Results: Of the 140 thalassemia patients who underwent screening, 28 patients were assigned to receive alendronate and 24 patients to receive placebo. At 6 months follow‐up, patients receiving alendronate had a significant reduction of serum CTX as compared to baseline with a mean difference of −0.215 ng/ml (95%CI −0.084 to −0.345, p = 0.003) (Figure 1A). Likewise, serum P1NP level showed a significant reduction at 6 months as compared to baseline in alendronate group with a mean difference of −37.14 ng/ml (95%CI −14.82 to −59.45, p = 0.002) (Figure 1B). These changes were consistent in both transfusion‐dependent and non‐transfusion‐dependent patients. There was no significant change of bone markers in the placebo group (Figure 1A, 1B). Back pain was significantly reduced among patients receiving alendronate at 6 months when compared to baseline (p = 0.027) but not in those receiving placebo. Side effects were rarely found and led to a discontinuation of study drug in 1 patient (grade 3 fatigue). Summary/Conclusion: Alendronate 70 mg oral once weekly is effective in reduction of bone resorption measured by serum bone markers and back pain in thalassemia patients with osteoporosis.