Evidence map›Paper›PMID 42137281›Full record

ArticleMolecular therapy. Advances2026

First successful allogeneic hematopoietic stem cell transplantation in STING-associated vasculopathy of infancy-A case report.

Uet Yu, Aiyun Song, Changying Luo, Chengjuan Luo, Xia Qin, Xiaohang Huang, Xinan Wang, Yuchen Lin, Chen Zhou, Manpin Zhang and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 2026. 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
  2. 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

12 authors.

Uet YuBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Aiyun SongBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Changying LuoBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Chengjuan LuoBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Xia QinBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Xiaohang HuangBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Xinan WangBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Yuchen LinBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Chen ZhouBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Manpin ZhangBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Jing ChenBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Xiaodong WangBlood and Marrow Transplantation Center, Zhangjiang Campus, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Stimulator of interferon genes (STING)-associated vasculopathy with onset in infancy (SAVI) is a monogenic autoinflammatory disorder caused by gain-of-function mutations in TMEM173, leading to constitutive STING activation and persistent type I interferon signaling. Affected children develop early-onset cutaneous vasculopathy, systemic inflammation, and progressive interstitial lung disease, often refractory to standard immunosuppressive treatments. Janus kinase (JAK) inhibitors offer partial transient control, with risk of irreversible organ damage. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) could cure SAVI by replacing the mutant immune system, but experience is very limited. We report a 6-year-old boy with SAVI carrying the TMEM173 p.N154S mutation who failed multiple JAK inhibitors, including ruxolitinib, tofacitinib, and baricitinib and developed worsening lung fibrosis and cutaneous ulcers. He underwent allo-HSCT from a fully HLA-matched sibling after myeloablative conditioning. Engraftment was rapid. By 12 months post hematopoietic stem cell transplantation (HSCT), vasculitic skin lesions had healed, lung disease was stable, and inflammatory markers normalized, and cellular and humoral immunity reconstituted, including recovery of CD8

Indexed as

allogeneic hematopoietic stem cell transplantationcase reportinterferonopathypediatricrare diseaseSTING-associated vasculopathy of infancytype I interferon

Identifiers

PMID42137281
PMCPMC13148905

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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.