Evidence map›Paper›PMID 40913624›Full record

ArticleAnnals of hematology2025

A cohort of allogeneic hematopoietic stem cell transplantation for pediatric rare diseases (allo-HSCT-PRD): design and preliminary results.

Shiwen Hu, Ping Wang, Zifeng Li, Wenjin Jiang, Lian Liu, Wenjuan Ma, Ying Huang, Xiaochuan Wang, Xiaonan Du, Xiaowen Qian and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Annals of hematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

14 authors.

Shiwen Hu *Department of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Ping Wang *Department of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Zifeng Li *Department of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Wenjin JiangDepartment of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Lian LiuDepartment of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Wenjuan MaManagement of Science and Technology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Ying HuangDepartment of Gastroenterology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Xiaochuan WangDepartment of Clinical Immunology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Xiaonan DuDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Xiaowen QianDepartment of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Hongsheng WangDepartment of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Yi WangDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Yunhui ZhangKey Laboratory of Health Technology Assessment, National Health Commission of the People's Republic of China, Fudan University, Shanghai, China. yhzhang@shmu.edu.cn.
Xiaowen ZhaiDepartment of Hematology and Oncology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China. xwzhai@fudan.edu.cn.

Funding

National Key Research and Development Program of China 2023YFC2706301Science and Technology Commission of Shanghai Municipality 21Y31900302Shanghai Shenkang Hospital Development Center SHDC12023109
6 · The paper itself

Abstract

Rare diseases in children have attracted widespread attention worldwide due to their rarity and difficulty in diagnosis and treatment. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is emerging as a promising and curable therapy for multiple rare diseases. However, rare disease research in China is relatively backward, prompting us to construct the first cohort of allo-HSCT for pediatric rare diseases (allo-HSCT-PRD) involving those who underwent allo-HSCT at the Children's Hospital of Fudan University from 1 January 2014 to 31 October 2024. We concurrently developed a standard data collection, management, and follow-up process, and a dedicated multidisciplinary team to support the work. The allo-HSCT-PRD consisted of 480 participants who were categorized into inborn errors of immunity (IEI) and inborn errors of metabolism (IEM), with 428 and 52 cases, respectively. Among all subjects, the engraftment rate was 84.6%, and the 5-year overall survival (OS) rate was 78.5% (95%CI 74.9% - 82.4%). In particular, patients in the IEM group had greater age and higher implantation and 5-year OS rate. Our next step is to extend our work experience to build allo-HSCT-PRD into a multi-center cohort to boost the development of disease models, exploration of pathogenesis, and clinical trials for new medications.

Indexed as

Hematopoietic Stem Cell TransplantationRare DiseasesAdolescentAllograftsChildChild, PreschoolChinaCohort StudiesFemaleFollow-Up StudiesHumansInfantMaleMetabolism, Inborn ErrorsSurvival RateTransplantation, HomologousAllogeneic hematopoietic stem cell transplantationChildrenCohortRare disease

Identifiers

PMID40913624
PMCPMC12552309

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Registered trials

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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.