Evidence map›Paper›PMID 38722537›Full record

ArticleAdvances in therapy2024

A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results.

Yuwu Jiang, Yi Wang, Hui Xiong, Wenhui Li, Rong Luo, Wenxiong Chen, Fei Yin, Junlan Lü, Jianmin Liang, Wan-Jin Chen and 9 more

Registry-linked trialAbstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04419233 (A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China), which is not on this map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 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.

NCT04419233 completednot on this map

A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China

TypeobservationalSponsorBiogenRan2020 to 2023Enrolled50ConditionsMuscular Atrophy, SpinalArmsNusinersen Sodium Injection
3 · Its place in the literature

Who cites it

5 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Review
  4. Article
  5. Review
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

19 authors.

Yuwu JiangPeking University First Hospital, Beijing, China.
Yi WangChildren's Hospital of Fudan University, Shanghai, China.
Hui XiongPeking University First Hospital, Beijing, China.
Wenhui LiChildren's Hospital of Fudan University, Shanghai, China.
Rong LuoWest China Second University Hospital, Sichuan University, Chengdu, Sichuan, China.
Wenxiong ChenGuangzhou Women and Children's Medical Center, Guangdong, China.
Fei YinXiangya Hospital of Central South University, Changsha, Hunan, China.
Junlan LüBeijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Jianmin LiangThe First Bethune Hospital of Jilin University, Changchun, Jilin, China.
Wan-Jin ChenThe First Affiliated Hospital of Fujian Medical University, Fuzhou, Fujian, China.
Xinguo LuShenzhen Children's Hospital, Shenzhen, China.
Hua WangShengjing Hospital of China Medical University, Shenyang, Liaoning, China.
Jihong TangChildren's Hospital of Soochow University, Suzhou, Jiangsu, China.
Michael MonineBiogen, 225 Binney St, Cambridge, MA, 02142, USA.
Corinne MakepeaceBiogen, Maidenhead, Berkshire, UK.
Xin JinCytel, Cambridge, MA, USA.
Richard FosterBiogen, Maidenhead, Berkshire, UK.
Russell ChinBiogen, 225 Binney St, Cambridge, MA, 02142, USA.
Zdenek BergerBiogen, 225 Binney St, Cambridge, MA, 02142, USA. Zdenek.berger@biogen.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionSpinal muscular atrophy (SMA) is a rare, autosomal recessive, neuromuscular disease that leads to progressive muscular weakness and atrophy. Nusinersen, an antisense oligonucleotide, was approved for SMA in China in February 2019. We report interim results from a post-marketing surveillance phase 4 study, PANDA (NCT04419233), that collects data on the safety, efficacy, and pharmacokinetics of nusinersen in children with SMA in routine clinical practice in China.

methodsParticipants enrolled in PANDA will be observed for 2 years following nusinersen treatment initiation. The primary endpoint is the incidence of adverse events (AEs)/serious AEs (SAEs) during the treatment period. Efficacy assessments include World Health Organization (WHO) Motor Milestones assessment, the Hammersmith Infant Neurological Examination (HINE), and ventilation support. Plasma and cerebrospinal fluid (CSF) concentrations of nusinersen are measured at each dose visit.

resultsFifty participants were enrolled as of the January 4, 2023, data cutoff: 10 with infantile-onset (≤ 6 months) and 40 with later-onset (> 6 months) SMA. All 50 participants have received at least one dose of nusinersen; 6 have completed the study. AEs were experienced by 45 (90%) participants and were mostly mild/moderate; no AEs led to nusinersen discontinuation or study withdrawal. Eleven participants experienced SAEs, most commonly pneumonia (n = 9); none were considered related to study treatment. Stability or gain of WHO motor milestone was observed and mean HINE-2 scores improved in both subgroups throughout the study. No serious respiratory events occurred, and no permanent ventilation support was initiated during the study. Pre-dose nusinersen CSF concentrations increased steadily through the loading-dose period, with no accumulation in plasma after multiple doses.

conclusionNusinersen was generally well tolerated with an acceptable overall safety profile, consistent with the known safety of nusinersen. Efficacy, safety, and nusinersen exposure are consistent with prior observations. These results support continuing PANDA and evaluation of nusinersen in Chinese participants with SMA.

trial registrationClinicalTrials.gov identifier, NCT04419233.

Indexed as

OligonucleotidesProduct Surveillance, PostmarketingChild, PreschoolChinaFemaleHumansInfantMaleMuscular Atrophy, SpinalSpinal Muscular Atrophies of ChildhoodTreatment OutcomenusinersenOligonucleotidesEfficacyNusinersenPharmacokineticsPhase IVSafetySpinal muscular atrophy

Identifiers

PMID38722537
PMCPMC11213752

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Registered trials

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.