QualityMetric Incorporated, LLC, Johnston, RI, USA.
Akcea Therapeutics, Cambridge, MA, USA.
BMC Neurol. 2021 Feb 12;21(1):70. doi: 10.1186/s12883-021-02094-y.
We aimed to compare neuropathic progression rate between hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and other peripheral neuropathies, including diabetic peripheral neuropathy (DPN) and Charcot-Marie-Tooth disease (CMT).
Literature searches identified studies reporting neuropathic progression, measured by Neuropathy Impairment Score (NIS) or NIS-Lower Limbs (NIS-LL). Our study also included unpublished data from a clinical registry of patients who were diagnosed with different peripheral neuropathies and seen at the Oregon Health & Science University (OHSU) during 2016-2020. Meta-analysis and meta-regression models examined and compared annual progression rates, calculated from extracted data, between studies of ATTRv-PN and other peripheral neuropathies.
Data were synthesized from 15 studies in which NIS and/or NIS-LL total scores were assessed at least twice, with ≥12 weeks between assessments, among untreated patients with ATTRv-PN or other peripheral neuropathies. Meta-analysis models yielded that the annual progression rate in NIS total scores was significantly different from zero for studies in ATTRv-PN and CMT (11.77 and 1.41; both P < 0.001), but not DPN (- 1.96; P = 0.147). Meta-regression models showed significantly faster annual progression in studies in ATTRv-PN, which statistically exceeded that in other peripheral neuropathies by 12.45 points/year for NIS, and 6.96 for NIS-LL (both P < 0.001).
Peripheral nervous function deteriorates more rapidly in patients with ATTRv-PN than for other peripheral neuropathies. These findings may improve understanding of the natural history of neuropathy in ATTRv-PN, facilitate early diagnosis, and guide the development of assessment tools and therapies specifically targeting neuropathic progression in this debilitating disease.
我们旨在比较遗传性转甲状腺素蛋白淀粉样变伴多发性神经病（ATTRv-PN）与其他周围神经病（包括糖尿病周围神经病[DPN]和 Charcot-Marie-Tooth 病[CMT]）的神经病变进展率。
文献检索确定了报告神经病变进展的研究，这些研究通过神经病变损伤评分（NIS）或 NIS 下肢（NIS-LL）进行测量。我们的研究还包括来自俄勒冈健康与科学大学（OHSU） 2016-2020 年期间诊断为不同周围神经病患者的临床登记处的未发表数据。荟萃分析和荟萃回归模型检查并比较了从提取数据计算得出的 ATTRv-PN 和其他周围神经病研究之间的年度进展率。
数据综合了 15 项研究，这些研究中未经治疗的 ATTRv-PN 或其他周围神经病患者至少两次评估了 NIS 和/或 NIS-LL 总分，两次评估之间间隔≥12 周。荟萃分析模型表明，ATTRv-PN 和 CMT 研究的 NIS 总分年度进展率与零有显著差异（11.77 和 1.41；均 P < 0.001），但 DPN 研究则没有（-1.96；P = 0.147）。荟萃回归模型显示，ATTRv-PN 研究的年度进展更快，NIS 每年增加 12.45 分，NIS-LL 每年增加 6.96 分，均显著高于其他周围神经病（均 P < 0.001）。
与其他周围神经病相比，ATTRv-PN 患者的周围神经功能恶化更快。这些发现可能有助于更好地了解 ATTRv-PN 神经病变的自然史，促进早期诊断，并指导专门针对这种使人衰弱的疾病神经病变进展的评估工具和治疗方法的开发。
