Evidence map›Paper›PMID 41219744›Full record

ArticleBMC medicine2025

Clinical-genetic profiles and risk prediction model in childhood restrictive cardiomyopathy: a national cohort study of China.

Hao Chen, Shushui Wang, Junjun Quan, Wenhong Ding, Cong Liu, Hua Peng, Juanli Wang, Yanyan Xiao, Zipu Li, Hong Wang and 23 more

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in BMC medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–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

2 citing papers in PubMed.

  1. 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

33 authors.

Hao Chen *Department of Cardiology, Shanghai Children's Medical Center, National Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Shushui Wang *Department of Pediatric Cardiology, Guangdong Provincial People's Hospital (Guangdong Academy of Medical Sciences), Southern Medical University, Guangzhou, 510080, China.
Junjun Quan *Department of Cardiology, Children's Hospital of Chongqing Medical University, Chongqing, 400014, China.
Wenhong Ding *Department of Pediatric Cardiology, Capital Medical University Affiliated Beijing Anzhen Hospital, Beijing, 100029, China.
Cong LiuDepartment of Cardiology, Shenzhen Children's Hospital, Shenzhen, 518026, China.
Hua PengDepartment of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China.
Juanli WangDepartment of Cardiology, Xi'an Children's Hospital, Affiliated Children's Hospital of Xi'an Jiaotong University, Xi'an, 710003, China.
Yanyan XiaoDepartment of Cardiology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, 100045, China.
Zipu LiDepartment of Heart Center, Women and Children's Hospital Affiliated to Qingdao University, Qingdao Women and Children's Hospital, Qingdao, 266011, China.
Hong WangDepartment of Pediatrics, Shengjing Hospital, China Medical University, Shenyang, 110004, China.
Lu GaoDepartment of Cardiology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, 100045, China.
Lin ShiDepartment of Cardiology, Capital Center for Children's Health, Capital Medical University, Beijing, 100020, China.
Yifei LiKey Laboratory of Birth Defects and Related Diseases of Women and Children of Ministry of Education, Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, 610041, China.
Shiwei YangDepartment of Cardiology, Children's Hospital of Nanjing Medical University, Nanjing, 210008, China.
Li ZhangDepartment of Cardiology, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, 510120, China.
Yulu HuangDepartment of Pediatric Cardiology, Guangdong Provincial People's Hospital (Guangdong Academy of Medical Sciences), Southern Medical University, Guangzhou, 510080, China.
Piaohong LiangDepartment of Cardiology, Shenzhen Children's Hospital, Shenzhen, 518026, China.
Qing GuoDepartment of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China.
Jian LiDepartment of Cardiology, Xi'an Children's Hospital, Affiliated Children's Hospital of Xi'an Jiaotong University, Xi'an, 710003, China.
Guangsong ShanDepartment of Heart Center, Women and Children's Hospital Affiliated to Qingdao University, Qingdao Women and Children's Hospital, Qingdao, 266011, China.
Hongwei ZhangDepartment of Cardiology, Capital Center for Children's Health, Capital Medical University, Beijing, 100020, China.
Xiaoliang LiuKey Laboratory of Birth Defects and Related Diseases of Women and Children of Ministry of Education, Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, 610041, China.
Han ZhangDepartment of Cardiology, Children's Hospital of Nanjing Medical University, Nanjing, 210008, China.
Wei LiDepartment of Cardiology, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, 510120, China.
Bing ZhangEngineering Research Center of Techniques and Instruments for Diagnosis and Treatment of Congenital Heart Disease, Institute for Developmental and Regenerative Medicine, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200092, China.
Chengchen ZhangClinical Research Center, Shanghai Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, 200127, China.
Shilu TongNational Institute of Environmental Health, Chinese Center for Disease Control and Prevention, Beijing, 100021, China.
Wenxiu ChanDepartment of Cardiology, Shanghai Children's Medical Center, National Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Rui WangDepartment of Cardiology, Shanghai Children's Medical Center, National Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China.
Hao ZhangHeart Center, Shanghai Institute of Pediatric Congenital Heart Disease, Shanghai Clinical Research Center for Rare Pediatric Diseases, Shanghai Children's Medical Center, National Children's Medical Center, Shanghai Jiaotong University School of Medicine, Shanghai, 200127, China. drzhanghao@126.com.
Ling HanDepartment of Pediatric Cardiology, Capital Medical University Affiliated Beijing Anzhen Hospital, Beijing, 100029, China. hanl6610@sina.com.
Tiewei LvDepartment of Cardiology, Children's Hospital of Chongqing Medical University, Chongqing, 400014, China. ltw200145@163.com.
Lijun FuDepartment of Cardiology, Shanghai Children's Medical Center, National Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, 200127, China. fulijun@scmc.com.cn.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundUnderstanding restrictive cardiomyopathy (RCM) in children is limited, and currently, no prognostic model is available for assessing risk stratification in pediatric RCM. The authors elaborated on the clinical and genetic characteristics of pediatric RCM and developed a prediction model to assess the 1-year risk of major adverse cardiovascular events (MACE), aiding in determining the optimal timing for heart transplantation (HTx).

methodsThis multicenter retrospective cohort study collected data on children with RCM from 14 centers in China, with patient enrollment from January 1, 2013, to December 31, 2022, and follow-up concluding on August 1, 2023. It analyzed clinical and genetic characteristics and followed patients longitudinally for the development of MACE (including cardiac death, HTx, or equivalent events). A logistic regression model was developed to predict MACE one year post-diagnosis, with internal validation using bootstrapping. The model's performance was evaluated in terms of its discrimination, calibration, and clinical utility.

resultsThe study included 185 children with a median diagnosis age of 5.4 years (IQR, 3.1-9.4), and 110 (59%) were male. Significant heart failure was the primary clinical feature. TNNI3 mutations were present in 61% of cases, the most common in pediatric RCM. During the follow-up period, 114 patients (62%) experienced MACE, with the median MACE-free survival time for the entire cohort being 2.1 years post-diagnosis (IQR, 0.6-5.4). A prediction model was developed to estimate the one-year risk of MACE using four easily accessible clinical parameters: heart failure classification, brain natriuretic peptide levels, cardiac troponin levels, and a modified score for ST-segment deviation. Internal validation with bootstrapping confirmed accuracy, showing an optimism-adjusted C statistic of 0.78 (95% CI, 0.72-0.85) and a Brier score of 0.17 (95% CI, 0.14-0.21), with a calibration slope of 0.90 (95% CI, 0.63-1.27). Decision curve analysis indicated high net benefit across HTx treatment thresholds from 8.5% to 78.3%.

conclusionsThis model utilizes accessible clinical parameters to assess individual risk for MACE in pediatric RCM, potentially improving precision in healthcare strategies and supporting more informed clinical decision-making.

Indexed as

Cardiomyopathy, RestrictiveChildChild, PreschoolChinaCohort StudiesFemaleHeart TransplantationHumansMalePrognosisRetrospective StudiesRisk AssessmentHeart transplantationMajor adverse cardiovascular eventsPediatricsRestrictive cardiomyopathyRisk prediction

Identifiers

PMID41219744
PMCPMC12606984

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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.