Evidence map›Paper›PMID 39030456›Full record

ArticleJournal of neurology2024

Long-term impact of nusinersen on motor and electrophysiological outcomes in adolescent and adult spinal muscular atrophy: insights from a multicenter retrospective study.

Ningning Wang, Ying Hu, Kexin Jiao, Nachuan Cheng, Jian Sun, JinXue Tang, Jie Song, Chong Sun, Tao Wang, Kai Wang and 5 more

Abstract readMulticenter Study
PubMed Publisher
In one paragraph

Article in Journal of neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

11 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
  3. Observational
  4. Observational
  5. Article
  6. Article
  7. Review
  8. Observational
  9. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Ningning Wang *Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Ying Hu *Department of Neurology, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui Province, China.
Kexin Jiao *Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Nachuan ChengDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Jian SunDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
JinXue TangQilin District People's Hospital, Qujing, Yunnan Province, China.
Jie SongDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Chong SunDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Tao WangState Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China.
Kai WangDepartment of Neurology, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui Province, China.
Kai QiaoDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Jianying XiDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Chongbo ZhaoDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China.
Liqiang YuDepartment of Neurology, The First Affiliated Hospital of Soochow University, Suzhou, Jiangsu Province, China. yuliqiang@suda.edu.cn.
Wenhua ZhuDepartment of Neurology, Huashan Hospital, Fudan University, Shanghai, China. whzhu@fudan.edu.cn.ORCID http://orcid.org/0000-0001-8011-0274

Funding

Innovative Research Group Project of the National Natural Science Foundation of China 82171398
6 · The paper itself

Abstract

background5q spinal muscular atrophy (SMA) is a progressive autosomal recessive motor neuron disease.

objectiveWe aimed to assess the effects of nusinersen on motor function and electrophysiological parameters in adolescent and adult patients with 5q SMA.

methodsPatients with genetically confirmed 5q SMA were eligible for inclusion, and clinical data were collected at baseline (V1), 63 days (V4), 180 days (V5), and 300 days (V6). The efficacy of nusinersen was monitored by encompassing clinical assessments, including the Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), 6-Minute Walk Test (6MWT), and percent-predicted Forced Vital Capacity in sitting position (FVC%) and Compound Muscle Action Potential (CMAP) amplitude. The patients were divided into "sitter" and "walker" subgroups according to motor function status.

results54 patients were screened, divided into "sitter" (N = 22) and "walker" (N = 32), with the mean age at baseline of 27.03 years (range 13-53 years). The HFMSE in the walker subgroup increased significantly from baseline to V4 (mean change +2.32-point, P = 0.004), V5 (+3.09, P = 0.004) and V6 (+4.21, P = 0.005). The patients in both the sitter and walker subgroup had no significant changes in mean RULM between V1 and the following time points. Significant increases in CMAP amplitudes were observed in both upper and lower limbs after treatment. Also, patients with RULM ≥ 36 points showed significant CMAP improvements. Our analysis predicted that patients with CMAP amplitudes of trapezius ≥ 1.76 mV were more likely to achieve significant motor function improvements.

conclusionsNusinersen effectively improves motor function and electrophysiological data in adolescent and adult patients with SMA. This is the first report on the CMAP amplitude changes in the trapezius after treatment in patients with SMA. The CMAP values effectively compensate for the ceiling effect observed in the RULM, suggesting that CMAP could serve as an additional biomarker for evaluating treatment efficacy.

Indexed as

Muscular Atrophy, SpinalOligonucleotidesAdolescentAdultFemaleHumansMaleMiddle AgedRetrospective StudiesTreatment OutcomeYoung AdultnusinersenOligonucleotidesCompound muscle action potentialElectrophysiological outcomesMotor functionNusinersenSpinal muscular atrophy

Identifiers

PMID39030456

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.