Bone mineral density changes in patients on drug therapy for spinal muscular atrophy

IF 2.7 4区医学 Q2 CLINICAL NEUROLOGY

Neuromuscular Disorders Pub Date : 2025-02-05 DOI:10.1016/j.nmd.2025.105300

Abdelrahman Osman , Halley Wasserman , Paul S. Horn , Eileen Broomall

引用次数: 0

Abstract

Patients with Spinal Muscular Atrophy (SMA) are at higher risk of diminished bone health due to decreased ambulation and mobility. With the advent of new FDA-approved therapies, we aimed to analyze the effects of SMA therapy on bone mineral density (BMD) using dual energy X-ray absorptiometry (DXA) scan data of 27 patients diagnosed with SMA Type 1, 2, or 3. Patients were divided into those with DXA scans both before and after treatment (older cohort), and patients with first DXA post-treatment (cohort treated in infancy). In patients with DXA both before and after treatment, SMA drug therapy did not normalize (Z-score > -2) bone density in anyone with pre-established low bone mineral density. Patients with first DXA post-treatment initiation had a higher rate of normal bone mineral density compared to older cohort. Fractures were also tracked; frequency of fractures after therapy decreased from 52 % of patients to 14 % (p = 0.0584).

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脊髓性肌萎缩症药物治疗患者骨密度的变化

脊髓性肌萎缩症（SMA）患者由于行走和活动能力下降，骨骼健康下降的风险较高。随着fda批准的新疗法的出现，我们旨在使用双能x线吸收仪（DXA）扫描数据分析SMA治疗对27例诊断为1、2或3型SMA患者骨密度（BMD）的影响。患者分为治疗前后均进行DXA扫描的患者（老年队列）和治疗后首次进行DXA扫描的患者（婴儿期治疗的队列）。在DXA患者治疗前后，SMA药物治疗均未恢复正常(Z-score >；-2)骨密度预先确定的低骨密度。与老年队列相比，治疗后首次DXA患者的正常骨密度率更高。骨折也被跟踪；治疗后骨折发生率从52%下降到14% （p = 0.0584）。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Neuromuscular Disorders 医学-临床神经学

CiteScore

4.60

自引率

3.60%

发文量

543

审稿时长

53 days

期刊介绍： This international, multidisciplinary journal covers all aspects of neuromuscular disorders in childhood and adult life (including the muscular dystrophies, spinal muscular atrophies, hereditary neuropathies, congenital myopathies, myasthenias, myotonic syndromes, metabolic myopathies and inflammatory myopathies). The Editors welcome original articles from all areas of the field: • Clinical aspects, such as new clinical entities, case studies of interest, treatment, management and rehabilitation (including biomechanics, orthotic design and surgery). • Basic scientific studies of relevance to the clinical syndromes, including advances in the fields of molecular biology and genetics. • Studies of animal models relevant to the human diseases. The journal is aimed at a wide range of clinicians, pathologists, associated paramedical professionals and clinical and basic scientists with an interest in the study of neuromuscular disorders.