Endocrine Abstracts

Introduction: Osteopenia/osteoporosis in young adults with β-thalassemia major (BTM) is a prominent cause of morbidity despite adequate transfusion and iron chelation therapy. The reported frequency of osteoporosis, even in well treated TM patients varies from 13.6% to 50%, with an additional 45% affected by osteopenia.

Case report: We present the case of a 36-years-old male patient with BTM treated for 1 year with teriparatide for osteoporosis. Our patient was diagnosed with BTM at 6 months of age and he was treated with chronic blood transfusions since that moment associated intermittently with chelation therapy due to limited availability and lack of compliance of the patient. From his medical history we mention splenectomy at the age of 3, iron overload-related liver disease, chronic hepatitis C, cardiomegaly (hemosiderosis), femural dyaphysis fracture (1992), hypogonadotropic hypogonadism, secondary diabetes mellitus (2008), and hyperparathyroidism (2009). For his endocrine complications he received substitutive treatment with testosterone undecanoate, calcium, vitamin D and insulin. In January 2009 he was diagnosed with severe osteoporosis with multiple vertebral fractures, he had vertebroplasty performed at T11 and T12; alendronate treatment was initiated until November 2013 when a new vertebral fracture occurred at L5. In this context, the alendronate treatment was considered suboptimal/ineffective; therefore, therapy with rPTH was initiated. After 1 year of therapy with teriparatide his DXA measured lumbar spine BMD significantly improved (9.8% increment) without any signs of a new fracture.

Conclusions: The osteoporosis in BTM has a complex mechanism; both increased bone resorption and decreased formation being involved. Probably the extent of contribution of the two mechanisms varies in each patient. Although the bisphosphonates are the most used treatment for osteoporosis in BTM, our case report suggests that, at least in some patients, decreased bone formation is the predominant mechanism and teriparatidum could be the most appropriate therapy.

Disclosure: This paper is partly supported by the Sectorial Operational Programme Human Resources Development (SOPHRD), financed by the European Social Fund and the Romanian Government under the contract number POSDRU 141531.