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Cited by 110Immunodeficiency and Autoimmune DisordersHematopoietic Stem Cell TransplantationBlood disorders and treatments

The DOI matches its Crossref record; 1 of the 1 references it cites were found in a registry or an index.

Hematopoietic stem cell transplantation in 29 patients hemizygous for hypomorphic IKBKG/NEMO mutations

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Miot, Charline, Imai, Kohsuke, Imai, Chihaya, Mancini, Anthony J., Kucuk, Zeynep Yesim, Kawai, Tokomki, Nishikomori, Ryuta, Ito, Etsuro, Pellier, Isabelle, Dupuis Girod, Sophie, Rosain, Jeremie, Sasaki, Shinya, Chandrakasan, Shanmuganathan, Pachlopnik Schmid, Jana, Okano, Tsubasa, Colin, Estelle, Olaya-Vargas, Alberto, Yamazaki-Nakashimada, Marco, Qasim, Waseem, Espinosa Padilla, Sara, Jones, Andrea, Krol, Alfons, Cole, Nyree, Jolles, Stephen, Bleesing, Jack, Vraetz, Thomas, Gennery, Andrew R., Abinun, Mario, Güngör, Tayfun, Costa-Carvalho, Beatriz, Condino-Neto, Antonio, Veys, Paul, Holland, Steven M., Uzel, Gulbu, Moshous, Despina, Neven, Benedicte, Blanche, Stéphane, Ehl, Stephan, Döffinger, Rainer, Patel, Smita Y., Puel, Anne, Bustamante, Jacinta, Gelfand, Erwin W., Casanova, Jean-Laurent, Orange, Jordan S., Picard, Capucine·2017·Blood
DOI 10.1182/blood-2017-03-771600PMC PMC5609334
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Abstract

Key Points Global survival rate was 74% at a median follow-up after HSCT of 57 months. Preexisting mycobacterial infection and colitis were associated with poor HSCT outcome.

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Crossref · 10.1182/blood-2017-03-771600

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  • Allogenic hematopoietic stem cell transplantation is the only curative modality for HED-ID at present, with a 74% survival rate.

    “Allogenic hematopoietic stem cell transplantation (HSCT) is the only curative modality at present, with a 74% survival rate.[23]”

    From Ectodermal Dysplasia – An Overview and Update (2024) · cites this paper as [23]

Citations

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Metadata

Authors
Miot, C., Imai, K., Imai, C., Mancini, A. J., Kucuk, Z. Y., Kawai, T., Nishikomori, R., Ito, E., Pellier, I., Dupuis Girod, S., Rosain, J., Sasaki, S., Chandrakasan, S., Pachlopnik Schmid, J., Okano, T., Colin, E., Olaya-Vargas, A., Yamazaki-Nakashimada, M., Qasim, W., Espinosa Padilla, S., Jones, A., Krol, A., Cole, N., Jolles, S., Bleesing, J., Vraetz, T., Gennery, A. R., Abinun, M., Güngör, T., Costa-Carvalho, B., Condino-Neto, A., Veys, P., Holland, S. M., Uzel, G., Moshous, D., Neven, B., Blanche, S., Ehl, S., Döffinger, R., Patel, S. Y., Puel, A., Bustamante, J., Gelfand, E. W., Casanova, J.-L., Orange, J. S., & Picard, C.
Affiliations
  • Hôpital Necker-Enfants Malades (FR)
  • Tokyo Medical and Dental University (JP)
  • Niigata University (JP)
  • Northwestern University (US)
  • Cincinnati Children's Hospital Medical Center (US)
  • Kyoto University (JP)
  • Hirosaki University (JP)
  • Centre Hospitalier Universitaire d'Angers (FR)
  • Hospices Civils de Lyon, Genetic Unit, School of Medicine, University Lyon 1, Bron, France;
  • Study Center for Immunodeficiencies, Assistance Publique–Hopitaux de Paris, Necker Hospital for Sick Children, Paris, France;
  • Division of Immunology and
  • Clinical Immunology Department and Program of Hematopoietic Stem Cell Transplantation, National Institute of Pediatrics, Mexico City, Mexico;
  • University College London (GB)
  • National Jewish Health (US)
  • Oregon Health & Science University (US)
  • Starship Children's Health (NZ)
  • University Hospital of Wales (GB)
  • University of Freiburg (DE)
  • Newcastle University (GB)
  • Newcastle upon Tyne Hospitals NHS Foundation Trust (GB)
  • University of Zurich (CH)
  • Department of Pediatrics and
  • Department of Immunology, Institute of Biomedical Sciences, Federal University of São Paulo, São Paulo, Brazil;
  • Great Ormond Street Hospital (GB)
  • National Institutes of Health (US)
  • Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD;
  • Imagine Institute, Paris Descartes University, Paris, France;
  • National Institute for Health and Care Research (GB)
  • Baylor College of Medicine (US)
ORCID
Venue
Blood
Publisher
American Society of Hematology
Published
2017-09-21
Volume
130
Issue
12
Pages
1456-1467
ISSN
  • 0006-4971 (ISSN-L)
  • 1528-0020
DOI
10.1182/blood-2017-03-771600
PMC
PMC5609334
OpenAlex
W2727852098
Cited by
110
References
1
Citations by year
2026: 5 · 2025: 10 · 2024: 13 · 2023: 12 · 2022: 8 · 2021: 14 · 2020: 19 · 2019: 13 · 2018: 13 · 2017: 3
FWCI
5.94
Type
journal article
Language
en
Open access
Locations
  • Blood (publishedVersion)
  • Europe PMC (PubMed Central) (publishedVersion)
  • PubMed Central (publishedVersion)
  • PubMed (publishedVersion)
  • HAL (submittedVersion)
  • ORCA Online Research @Cardiff (Cardiff University) (submittedVersion)
  • UNIFESP Institutional Repository (Universidade Federal de São Paulo) (submittedVersion)
  • Zurich Open Repository and Archive (University of Zurich) (submittedVersion)
Funders
  • St. Giles Foundation
  • National Institute for Health and Care Research (RP-2014-05-007)
  • Institut National de la Santé et de la Recherche Médicale
  • Bundesministerium für Bildung und Forschung (01EO1303, BMBF 01EO1303)
  • Université Paris Descartes
  • Japan Society for the Promotion of Science (15K09640, 15H04876, 26253061)
Topics
  • Life Sciences › Immunology and Microbiology › Immunology › Immunodeficiency and Autoimmune Disorders
  • Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
  • Health Sciences › Medicine › Hematology › Blood disorders and treatments
Keywords
hematopoietic stem cell transplantation, hypomorphic mutation, primary immunodeficiency, survival outcomes, mycobacterial infection, colitis
MeSH terms
Child, Preschool, Heterozygote, Humans, Infant, Infant, Newborn, Inflammation, Mutation, Phenotype, Tissue Donors, Inflammatory Bowel Diseases, Cohort Studies, Signal Transduction, Survival Analysis, NF-kappa B, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Transplantation Conditioning, I-kappa B Kinase

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