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dc.contributor.author
Bergsten, Elisabet
dc.contributor.author
Horne, AnnaCarin
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Hed Myrberg, Ida
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Aricó, Maurizio
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Astigarraga, Itziar
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Ishii, Eiichi
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Janka, Gritta
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Ladisch, Stephan
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Lehmberg, Kai
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McClain, Kenneth L.
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Minkov, Milen
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Nanduri, Vasanta
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Rosso, Diego
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Sieni, Elena
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Winiarski, Jacek
dc.contributor.author
Henter, Jan Inge
dc.date.available
2022-05-05T01:44:42Z
dc.date.issued
2020-08-11
dc.identifier.citation
Bergsten, Elisabet; Horne, AnnaCarin; Hed Myrberg, Ida; Aricó, Maurizio; Astigarraga, Itziar; et al.; Stem cell transplantation for children with hemophagocytic lymphohistiocytosis: results from the HLH-2004 study; American Society of Hematology; Blood Advances; 4; 15; 11-8-2020; 3754-3766
dc.identifier.issn
2473-9537
dc.identifier.uri
http://hdl.handle.net/11336/156554
dc.description.abstract
We report the largest prospective study thus far on hematopoietic stem cell transplantation (HSCT) in hemophagocytic lymphohistiocytosis (HLH), a life-threatening hyperinflammatory syndrome comprising familial/genetic HLH (FHL) and secondary HLH. Although all patients with HLH typically need intensive anti-inflammatory therapy, patients with FHL also need HSCT to be cured. In the international HLH-2004 study, 187 children aged ,18 years fulfilling the study inclusion criteria (5 of 8 diagnostic criteria, affected sibling, or molecular diagnosis in FHL-causative genes) underwent 209 transplants (2004-2012), defined as indicated in patients with familial/genetic, relapsing, or severe/persistent disease. Five-year overall survival (OS) post-HSCT was 66% (95% confidence interval [CI], 59-72); event-free survival (EFS) was 60% (95% CI, 52-67). Five-year OS was 81% (95% CI, 65-90) for children with a complete response and 59% (95% CI, 48-69) for those with a partial response (hazard ratio [HR], 2.12; 95% CI, 1.06-4.27; P 5 .035). For children with verified FHL (family history/genetically verified, n 5 134), 5-year OS was 71% (95% CI, 62-78) and EFS was 62% (95% CI, 54-70); 5-year OS for children without verified FHL (n 5 53) was significantly lower (52%; 95% CI, 38-65) (P 5 .040; HR, 1.69; 95% CI, 1.03-2.77); they were also significantly older. Notably, 20 (38%) of 53 patients without verified FHL had natural killer cell activity reported as normal at diagnosis, after 2 months, or at HSCT, suggestive of secondary HLH; and in addition 14 (26%) of these 53 children had no evidence of biallelic mutations despite having 3 or 4 FHL genes analyzed (natural killer cell activity not analyzed after 2 months or at HSCT). We conclude that post-HSCT survival in FHL remains suboptimal, and that the FHL diagnosis should be carefully investigated before HSCT. Pretransplant complete remission is beneficial but not mandatory to achieve post-HSCT survival.
dc.format
application/pdf
dc.language.iso
eng
dc.publisher
American Society of Hematology
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dc.rights
info:eu-repo/semantics/openAccess
dc.rights.uri
https://creativecommons.org/licenses/by-nc-sa/2.5/ar/
dc.subject
HLH
dc.subject
Children
dc.subject
Stem Cell Transplantation
dc.subject.classification
Hematología
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dc.subject.classification
Medicina Clínica
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dc.subject.classification
CIENCIAS MÉDICAS Y DE LA SALUD
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dc.title
Stem cell transplantation for children with hemophagocytic lymphohistiocytosis: results from the HLH-2004 study
dc.type
info:eu-repo/semantics/article
dc.type
info:ar-repo/semantics/artículo
dc.type
info:eu-repo/semantics/publishedVersion
dc.date.updated
2022-04-26T17:05:50Z
dc.identifier.eissn
2473-9529
dc.journal.volume
4
dc.journal.number
15
dc.journal.pagination
3754-3766
dc.journal.pais
Estados Unidos
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dc.journal.ciudad
Washington DC
dc.description.fil
Fil: Bergsten, Elisabet. Karolinska Huddinge Hospital. Karolinska Institutet; Suecia
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Fil: Horne, AnnaCarin. Karolinska Huddinge Hospital. Karolinska Institutet; Suecia
dc.description.fil
Fil: Hed Myrberg, Ida. Karolinska Huddinge Hospital. Karolinska Institutet; Suecia
dc.description.fil
Fil: Aricó, Maurizio. Children Hospital Giovanni XXIII; Italia
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Fil: Astigarraga, Itziar. Universidad del País Vasco; España
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Fil: Ishii, Eiichi. Ehime University; Japón
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Fil: Janka, Gritta. Universitat Hamburg; Alemania
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Fil: Ladisch, Stephan. Children’s National Medical Center; Estados Unidos
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Fil: Lehmberg, Kai. Universitat Hamburg; Alemania
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Fil: McClain, Kenneth L.. Baylor College of Medicine; Estados Unidos
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Fil: Minkov, Milen. Universidad de Viena; Austria
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Fil: Nanduri, Vasanta. Watford General Hospital; Reino Unido
dc.description.fil
Fil: Rosso, Diego. Gobierno de la Ciudad de Buenos Aires. Hospital General de Niños Pedro Elizalde (ex Casa Cuna); Argentina. Universidad de Buenos Aires. Facultad de Medicina. Hospital de Clínicas General San Martín; Argentina. Consejo Nacional de Investigaciones Científicas y Técnicas; Argentina
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Fil: Sieni, Elena. Universitaria A. Meyer Children Hospital; Italia
dc.description.fil
Fil: Winiarski, Jacek. Karolinska Huddinge Hospital. Karolinska Institutet; Suecia
dc.description.fil
Fil: Henter, Jan Inge. Karolinska Huddinge Hospital. Karolinska Institutet; Suecia
dc.journal.title
Blood Advances
dc.relation.isreferencedin
info:eu-repo/semantics/reference/url/http://hdl.handle.net/11336/41228
dc.relation.alternativeid
info:eu-repo/semantics/altIdentifier/doi/http://dx.doi.org/10.1182/bloodadvances.2020002101
dc.relation.alternativeid
info:eu-repo/semantics/altIdentifier/url/https://ashpublications.org/bloodadvances/article/4/15/3754/461773/Stem-cell-transplantation-for-children-with
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