Efficacy and Safety of Teriparatide in Beta-Thalassemia Major Associated Osteoporosis: A Real-Life Experience

Abstract

Osteoporosis represents a relevant cause of morbidity in adult Thalassemia Major (TM) population. Antiresorptive drugs such as bisphosphonates were demonstrated effective in preventing bone loss. Teriparatide (TP) is an anabolic agent approved for osteoporosis management in the general population, but its use has been very limited in TM patients so far. We evaluated TP efficacy and safety in TM-associated osteoporosis in real-life clinical practice. Retrospective evaluation of 11 TM patients (6 males, 5 females; mean age = 45 ± 4.38 years) with severe osteoporosis and multiple fractures under TP treatment. Mean TP treatment duration was 19 ± 7 months. TP withdrawal was due to poor compliance and side effects (fever and osteo-muscular pain) in two and three patients, respectively. After 12 and 24 months, BMD significantly increased at lumbar (+ 19% and 22%) and femoral sites (+ 13% and 13%). Osteocalcin and cross-laps levels increased after 12 and 24 months (+ 225 and + 54.2%; + 159 and 141%, respectively). No new fractures were detected during TP treatment. Baseline VAS score values (3 ± 3) did not significantly change after 12 and 24 months (3 ± 3 and 2 ± 3, respectively). Five out of eleven patients developed side effects. TP might be an effective treatment for TM-associated osteoporosis since it improves BMD, especially at the lumbar spine, and prevents fragility fractures. TM patients may have a higher frequency of side effects, especially muscle and bone pain under TP treatment, as compared to no TM population. Further studies are needed.

Beta‐thalassaemia major: Prevalence, risk factors and clinical consequences of hypercalciuria

“…Liver stiffness measurement by transient elastography was evaluated by FibroScan (Echosens, Paris, France) 34 . Magnetic resonance imaging was employed to evaluate cardiac and liver iron overload by measuring the T2* signal, and liver iron content (LIC), as previously described 35 . Endocrinopathies were diagnosed according to current guidelines 2…”

“…34 Magnetic resonance imaging was employed to evaluate cardiac and liver iron overload by measuring the T2* signal, and liver iron content (LIC), as previously described. 35 Endocrinopathies were diagnosed according to current guidelines. 2 Statistical analysis was performed by means of Fisher's exact test for qualitative measures, by a t-test for parametrically distributed quantitative measures, and by the Mann-Whitney test for non-parametrically distributed quantitative measures.…”

New Entity—Thalassemic Endocrine Disease: Major Beta-Thalassemia and Endocrine Involvement

“…We identified a single study from 2022 on teriparatide (real life experience), as bone anabolic agent for severe OP, in 11 adults with TBD (male/female ratio of 6/5, mean age of 45 ± 4.38 y). The therapy was followed for 19 ± 7 months; the main causes of withdrawal were side effects (5/11) and poor compliance to therapy; the results showed significant increase in BMD, respective of serum osteocalcin, and cross-laps [ 109 ]. The drug seems promising in TBD; however, the level of statistical evidence is low and its administration is restricted to specific countries protocols.…”

The role of opportunistic quantitative computed tomography in the evaluation of bone disease and risk of fracture in thalassemia major

“…Osteoporosis is generally defined by low bone mass and microarchitectural deterioration of bone tissue, leading to an increase in bone fragility and the risk of fracture. The characteristics of thalassemia‐associated osteoporosis are, however, unique compared to those of the more typical idiopathic osteoporosis observed in the general community as a result of several factors contributing to the complex pathophysiology and management of this condition 2–5 …”

“…The characteristics of thalassemia-associated osteoporosis are, however, unique compared to those of the more typical idiopathic osteoporosis observed in the general community as a result of several factors contributing to the complex pathophysiology and management of this condition. [2][3][4][5] Measurement of areal bone mineral density (BMD) by dualenergy X-ray absorptiometry (DXA) of the spine and hip represents the cornerstone for osteoporosis diagnosis and therapy monitoring. 6 However, numerous drawbacks for the use of DXA have been observed in thalassaemic subjects, with several open issues related to their young age, the presence of bone deformities and degenerative changes, and the reduced skeletal size due to the failure to reach peak bone mass.…”