Use of Romosozumab for Osteoporosis in β-Thalassemia Major

Thalassemia bone disease is a common and severe complication of β-thalassemia major (TM), resulting from marrow expansion, hormonal deficiencies including hypogonadism, iron toxicity and increased bone turnover. These factors contribute to reduced bone mineral density (BMD) and increased susceptibility to minimal trauma fracture (MTF). Optimising transfusions and iron chelation therapy, management of hormonal deficiencies and antiresorptive therapies are the cornerstone of management. We report the use of romosozumab in 2 patients with TM and severe osteoporosis. This is the first case series describing the efficacy of romosozumab in the treatment of osteoporosis in patients with TM. After completion of 12 months of monthly romosozumab, dual-energy X-ray absorptiometry scan (DXA) demonstrated significant improvements in BMD despite previous prolonged exposure to anti-resorptive therapy in both subjects. This represents a promising treatment option in patients with TM and osteoporosis.