Evidence map›Paper›PMID 39823014›Full record

ArticleTranslational pediatrics2024

Differential genes expression of immune tolerance induction in hemophilia A: an exploratory RNA-seq test from a Chinese hemophilia comprehensive care centre.

Jialu Zhang, Zekun Li, Guoqing Liu, Wanru Yao, Di Ai, Zhengping Li, Zhenping Chen, Runhui Wu

Abstract read
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In one paragraph

Article in Translational pediatrics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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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

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3 · Its place in the literature

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No citing paper in PubMed yet.

4 · The record

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5 · Who and what money

Authors and funding

8 authors.

Jialu ZhangDepartment of Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Zekun LiDepartment of Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Guoqing LiuDepartment of Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Wanru YaoDepartment of Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Di AiDepartment of Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Zhengping LiDepartment of Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Zhenping ChenDepartment of Clinical Laboratory Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Runhui WuDepartment of Hematology Center, National Key Clinical Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: The production of inhibitors is a serious complication that can arise during coagulation factor replacement therapy for hemophilia A (HA). The primary therapeutic strategy to eliminate inhibitors is immune tolerance induction (ITI), which is known to be an extremely challenging, prolonged, and costly treatment. With the widespread use of RNA sequencing (RNA-seq) to analyze differentially expressed genes (DEGs) across various treatment outcomes, there is potential for predicting ITI outcomes. This study aims to use RNA-seq to test differently expressed genes in different outcomes of ITI treatment for HA patients with high-titer inhibitor (HAI), to explore its prediction possibility. Methods: RNA-seq was employed to screen and compare the DEGs between patients in the Success group and those in the Failure group, based on ITI clinical outcomes. DEGs were subjected to Gene Ontology (GO) analysis and enrichment analysis of Kyoto Encyclopedia of Genes and Genomes (KEGG) pathways. Results: Thirteen analyzable HAI cases were collected, comprising seven in the Success group and six in the Failure group. Blood samples were taken before and after ITI. RNA-seq was applied to all samples to screen for expressed genes. In the Success group, a total of 4,967 messenger RNA (mRNA) transcripts were differentially expressed between pre-ITI and post-ITI, with 2,865 being up-regulated and 2,102 down-regulated. In the Failure group, 515 mRNA transcripts were expressed either before or after ITI, showing up-regulation in 68.7% (354/515) and down-regulation in 31.3% (161/515). Conclusions: The increased expression of genes which related to immune system activation suggests a possibly favorable therapeutic outcome of ITI. Future studies should test with a larger cohort to validate these findings.

Indexed as

hemophilia A (HA)immune tolerance induction (ITI)PediatricspredictRNA sequencing (RNA-seq)

Identifiers

PMID39823014
PMCPMC11732629

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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.