Section of Endocrinology, Geriatrics & Internal Medicine, Dept of Medical Sciences, University of Ferrara, Via Fossato di Mortara 64/B, 44121, Ferrara, Italy.
Unit of Thalassaemia and Haemoglobinopathies Day Hospital, Regional HUB Centre, Department of Medicine, Azienda Ospedaliero Universitaria S. Anna, Cona, Ferrara, Italy.
Calcif Tissue Int. 2022 Jul;111(1):56-65. doi: 10.1007/s00223-022-00963-3. Epub 2022 Mar 4.
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.
骨质疏松症是成人重型地中海贫血（TM）患者发病和致残的重要原因。抗吸收药物，如双磷酸盐，已被证明可有效预防骨质流失。特立帕肽（TP）是一种用于治疗骨质疏松症的合成代谢药物，已在普通人群中得到批准，但迄今为止，在 TM 患者中的使用非常有限。我们评估了 TP 在真实临床实践中治疗 TM 相关骨质疏松症的疗效和安全性。回顾性评估 11 例 TM 患者（6 名男性，5 名女性；平均年龄 45±4.38 岁）在接受 TP 治疗时有严重骨质疏松症和多处骨折。TP 的平均治疗时间为 19±7 个月。由于两名和三名患者分别出现依从性差和副作用（发热和肌肉骨骼疼痛），TP 被停用。12 个月和 24 个月后，腰椎（+19%和 22%）和股骨部位（+13%和 13%）的 BMD 显著增加。12 个月和 24 个月后，骨钙素和交联胶原水平分别增加了 225%和 54.2%（+159%和 141%）。在 TP 治疗期间没有发现新的骨折。TP 治疗 12 个月和 24 个月后，VAS 评分（3±3）没有明显变化（3±3 和 2±3）。11 例患者中有 5 例出现副作用。TP 可能是治疗 TM 相关骨质疏松症的有效方法，因为它可以提高 BMD，特别是腰椎的 BMD，并且可以预防脆性骨折。与非 TM 人群相比，TM 患者在接受 TP 治疗时可能会出现更高频率的副作用，尤其是肌肉和骨骼疼痛。需要进一步的研究。
