Romosozumab for the treatment of thalassemia-associated osteoporosis in two female patients.

Osteoporosis is a major complication of thalassemia bone disease, leading to increased fracture risk and impaired quality of life. Anti-resorptive agents, such as bisphosphonates and denosumab, have shown efficacy in randomized trials, but data are scarce, regarding the role of anabolic agents in severe cases with fragility fractures. We report 2 cases with severe osteoporosis associated to thalassemia with multiple vertebral and non-vertebral fractures, despite long term-treatment with anti-resorptive agents. Both patients were initiated on subcutaneous romosozumab (210 mg once monthly) for 12 mo. At 12 mo, BMD showed marked improvement at the LS by 6.8%-15.4%, TH by 14.6%, and FN by 8.7%-24.6%. Bone turnover markers changed in line with clinical trials of postmenopausal osteoporosis. No adverse events occurred during the treatment period. Following completion of romosozumab therapy, each patient received a single infusion of zoledronate to consolidate the bone mass gained. Over 24 mo follow-up, no new fragility fractures were observed. Romosozumab can be an effective and safe anabolic option for patients with thalassemia and high fracture risk. While anti-resorptive therapy remains the standard of care, romosozumab may provide additional benefit in thalassemia patients with severe osteoporosis and increased fracture risk.