Zoledronic acid for the treatment of osteoporosis in patients with beta-thalassemia: results from a single-center, randomized, placebo-controlled trial.

BACKGROUND AND OBJECTIVES

The aim of this study was to evaluate the effect of zoledronic acid (ZA) on thalassemia-induced osteoporosis.

DESIGN AND METHODS

We studied 66 thalassemia patients with osteoporosis, who were randomized to receive 4 mg ZA iv, every 6 months (23 patients; group A) or every 3 months (21 patients; group B), or to receive placebo every 3 months (22 patients; group C), for a period of 1 year. Bone mineral density (BMD) of the lumbar spine, femoral neck and wrist was determined before and 12 months after treatment. Pain scores and markers of bone resorption [C-telopeptide of collagen type-I (CTX), 5b-isoform of TRAP], bone formation [bone-alkaline phosphatase (bALP), osteocalcin (OC), C-telopeptide of procollagen type-I (CICP)], and osteoclast stimulators [sRANKL, osteoprotegerin (OPG), osteopontin] were also measured at baseline and before each treatment administration.

RESULTS

The values of CTX, bALP, CICP, sRANKL, and OPG were higher in the all patients than in the controls. Patients in group A showed no differences in BMD of all sites at 12 months, while they had reductions in bone pain, bALP, OC and OPG. Conversely patients in group B had a significant increase in their lumbar spine BMD, which was accompanied by dramatic reductions in bone pain, CTX, bALP, CICP, and OC. Patients in group C showed no alteration in BMD of any studied site or in bone pain, while they had an aggravation in bone resorption.

INTERPRETATION AND CONCLUSIONS

ZA, at a dose of 4 mg, iv, every 3 months is an effective treatment for increasing BMD and reducing bone resorption in thalassemia-induced osteoporosis.