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 BMD and increased susceptibility to minimal trauma fracture (MTF). Optimizing 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 mo of monthly romosozumab, DXA scan demonstrated significant improvements in BMD despite previous prolonged exposure to antiresorptive therapy in both subjects. This represents a promising treatment option in patients with TM and osteoporosis.