Evidence map›Paper›PMID 40540800›Full record

ArticleBlood advances2025

Allogeneic hematopoietic stem cell transplantation for STAT3 hyper-IgE syndrome: a worldwide study.

Christo Tsilifis, Johannes Raedler, Joanna Renke, Michael Medinger, Alexandra Laberko, Ásgeir Haraldsson, Niraj Patel, Peter Ciznar, Melanie Wong, Steven J Keogh and 31 more

Abstract readMulticenter Study
In one paragraph

Article in Blood advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Observational
  2. Fungal Infections in Disorders of Inborn Errors of Immunity.Clinical reviews in allergy & immunology · 2026
    Review
  3. Review
  4. How I Treat: STAT3 hyper IgE syndrome.Journal of human immunity · 2026
    Review
  5. Case Report:Frontiers in pediatrics · 2026
    Article
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

41 authors.

Christo TsilifisPaediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.ORCID 0000-0002-1368-104X
Johannes RaedlerDepartment of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, Ludwig Maximilian University Munich, Munich, Germany.ORCID 0000-0003-0979-7274
Joanna RenkeDepartment of Pediatrics, Hematology and Oncology, Medical University of Gdańsk, Gdańsk, Poland.ORCID 0000-0002-5389-9190
Michael MedingerDepartment of Hematology, University Hospital Basel, Basel, Switzerland.ORCID 0000-0003-0427-7747
Alexandra LaberkoDepartment of Hematopoietic Stem Cell Transplantation, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.ORCID 0000-0002-2354-2588
Ásgeir HaraldssonChildren's Hospital Iceland, Landspitali - University Hospital, Reykjavík, Iceland.
Niraj PatelDivision of Allergy and Immunology, Department of Pediatrics, Duke University, Durham, NC.
Peter CiznarPediatric Department, Comenius University Medical Faculty, National Institute of Children's Diseases, Bratislava, Slovakia.
Melanie WongAllergy and Immunology, Children's Hospital at Westmead, Westmead, NSW, Australia.
Steven J KeoghCancer Centre for Children, Children's Hospital at Westmead, Westmead, NSW, Australia.
Paul GrayAllergy and Immunology, Sydney Children's Hospital, Sydney, NSW, Australia.ORCID 0000-0003-2057-3968
Richard MitchellKids Cancer Centre, Sydney Children's Hospital, Randwick, NSW, Australia.
Venetia BigleyTranslational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.
Suzanne ElcombeDepartment of Immunology, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.ORCID 0009-0005-7455-9477
Fabian HauckDepartment of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, Ludwig Maximilian University Munich, Munich, Germany.ORCID 0000-0001-9644-2003
Michael H AlbertDepartment of Pediatrics, Dr. von Hauner Children's Hospital, University Hospital, Ludwig Maximilian University Munich, Munich, Germany.ORCID 0000-0001-9143-3263
Eleni TholouliDepartment of Haematology, Manchester Royal Infirmary, Manchester, United Kingdom.
Archana HerwadkarDepartment of Clinical Immunology, Salford Care Organisation, Northern Care Alliance NHS Trust, Salford, United Kingdom.
Shuayb ElkhalifaCentre for Musculoskeletal Research, Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, United Kingdom.ORCID 0000-0002-6292-6795
Chris KosmidisNational Aspergillosis Centre, Manchester University NHS Foundation Trust, Manchester, United Kingdom.ORCID 0000-0003-0662-1265
Giorgio CallistiInfectious Diseases Department, Manchester University NHS Foundation Trust, Manchester, United Kingdom.ORCID 0000-0003-2351-4656
Lauri M BurroughsFred Hutchinson Cancer Research Center, Department of Pediatrics, University of Washington, Seattle Children's Hospital, Seattle, WA.ORCID 0000-0003-1124-4740
Karin ChenDivision of Immunology, Department of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle, WA.ORCID 0000-0001-5593-436X
Ben CarpenterUniversity College London Institute of Immunity and Transplantation, University College London, London, United Kingdom.ORCID 0000-0002-7914-0799
Thomas A FoxUniversity College London Institute of Immunity and Transplantation, University College London, London, United Kingdom.ORCID 0000-0002-1163-2295
Emma C MorrisUniversity College London Institute of Immunity and Transplantation, University College London, London, United Kingdom.ORCID 0000-0003-4834-1130
Ramya UppuluriPaediatric Haemato-Oncology, Apollo Hospitals, Chennai, India.ORCID 0000-0001-5771-8214
Revathi RajPaediatric Haemato-Oncology, Apollo Hospitals, Chennai, India.
Masakatsu YanagimachiDepartment of Pediatrics, Yokohama City University, Yokohama, Japan.ORCID 0000-0002-1055-6497
Emilie P BuddinghPediatric Stem Cell Transplantation Program, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.ORCID 0000-0002-2755-9408
Christina OikonomopoulouStem Cell Transplant Unit, Aghia Sofia Children's Hospital, Athens, Greece.ORCID 0000-0003-2053-0191
Corina GonzalezImmune Deficiency Cellular Therapy Program, National Cancer Institute, National Institutes of Health, Bethesda, MD.
Dimana DimitrovaCenter for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0002-4868-6852
Jennifer A KanakryCenter for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.
Danielle ArnoldImmune Deficiency Cellular Therapy Program, National Cancer Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0001-7501-806X
Sung-Yun PaiImmune Deficiency Cellular Therapy Program, National Cancer Institute, National Institutes of Health, Bethesda, MD.ORCID 0000-0002-0158-8147
Mary A SlatterPaediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
Mark S PearcePopulation Health Sciences Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.ORCID 0000-0002-0583-3779
Austen WorthDepartment of Immunology and Gene Therapy, Great Ormond Street Hospital for Children, London, United Kingdom.ORCID 0000-0001-6803-7385
Alexandra F FreemanLaboratory of Clinical Immunology and Microbiology, National Institutes of Allergy and Infectious Disease, National Institutes of Health, Bethesda, MD.
Andrew R GenneryPaediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

abstractSignal transduction and activator of transcription 3 hyperimmunoglobulin E syndrome (STAT3-HIES) is a multisystem disorder causing recurrent skin and respiratory infection with bronchiectasis, pneumatoceles, and aspergillosis; lymphoma; and extraimmune manifestations including fractures and vasculopathy. Published data on immune and extraimmune hematopoietic stem cell transplant (HSCT) outcomes focus on case reports or small cohorts. We conducted an international multicenter retrospective study of HSCT in STAT3-HIES. Primary end points were overall survival (OS) and event-free survival (EFS; events were death, graft failure, chronic graft-versus-host disease [GVHD]). We identified 41 patients over a 28-year period. HSCT indication was infection (93%) or lymphoma (7%). Median age at HSCT was 14 years (range, 4-45). Most patients had pre-HSCT respiratory disease (93%), including parenchymal lung disease (68%), and prior suspected/confirmed pulmonary fungal infection (32%). Patients received peripheral blood stem cells (51%) or marrow (49%) from HLA 10/10-matched unrelated donors (44%), matched family donors (44%), mismatched family donors (10%), or 1 9/10-mismatched unrelated donor (2%). Conditioning regimens were predominantly treosulfan-based (59%; with thiotepa, 34%); other patients received busulfan-based (24%) or melphalan-based (17%) regimens. Median follow-up for surviving patients was 5 years (0.8-28). The 5-year OS was 93%, and 5-year EFS 90%. Cumulative incidence of grade 2 to 4 acute GVHD was 22%. Median whole blood donor chimerism at latest follow-up was 100%. Eighty-seven percent of patients have reduced or no bacterial or fungal respiratory infection. After HSCT, 20% developed new skeletal fractures. This worldwide study expanded data on HSCT for STAT3-HIES to 41 patients; despite significant pre-HSCT pulmonary morbidity, OS was high, and patients have improved skin and respiratory disease though the impact on extraimmune manifestations appears limited.

Indexed as

Hematopoietic Stem Cell TransplantationJob SyndromeSTAT3 Transcription FactorAdolescentAdultChildChild, PreschoolFemaleGraft vs Host DiseaseHumansMaleMiddle AgedRetrospective StudiesTransplantation, HomologousTreatment OutcomeYoung AdultSTAT3 protein, humanSTAT3 Transcription Factor

Identifiers

PMID40540800
PMCPMC12359223

What Socratic holds

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LicenceCC BY-NC-ND
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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 Socratic graph.