oru.sePublications
Change search
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association-8th-edition
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf
Confirmed efficacy of etoposide and dexamethasone in HLH treatment: long-term results of the cooperative HLH-2004 study
Childhood Cancer Research Unit, Department of Women’s and Children’s Health, Karolinska Institute, Stockholm, Sweden; Theme of Children’s and Women’s Health, Karolinska University Hospital, Stockholm, Sweden.
Childhood Cancer Research Unit, Department of Women’s and Children’s Health, Karolinska Institute, Stockholm, Sweden; Theme of Children’s and Women’s Health, Karolinska University Hospital, Stockholm, Sweden.
Direzione Generale, Azienda Sanitaria Provinciale, Ragusa, Italy.
Servicio de Pediatria, BioCruces Health Research Institute, Hospital Universitario Cruces, Universidad del Pais Vasco–Euskal Herriko Unibertsitatea (UPV/EHU), Barakaldo, Spain.
Show others and affiliations
2017 (English)In: Blood, ISSN 0006-4971, E-ISSN 1528-0020, Vol. 130, no 25, p. 2728-2738Article in journal (Refereed) Published
Abstract [en]

Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening hyperinflammatory syndrome comprising familial/genetic HLH (FHL) and secondary HLH. In the HLH-94 study, with an estimated 5-year probability of survival (pSu) of 54% (95% confidence interval, 48%-60%), systemic therapy included etoposide, dexamethasone, and, from week 9, cyclosporine A (CSA). Hematopoietic stem cell transplantation (HSCT) was indicated in patients with familial/genetic, relapsing, or severe/persistent disease. In HLH-2004, CSA was instead administered upfront, aiming to reduce pre-HSCT mortality and morbidity. From 2004 to 2011, 369 children aged <18 years fulfilled HLH-2004 inclusion criteria (5 of 8 diagnostic criteria, affected siblings, and/or molecular diagnosis in FHL-causative genes). At median follow-up of 5.2 years, 230 of 369 patients (62%) were alive (5-year pSu, 61%; 56%-67%). Five-year pSu in children with (n = 168) and without (n = 201) family history/genetically verified FHL was 59% (52%-67%) and 64% (57%-71%), respectively (familial occurrence [n = 47], 58% [45%-75%]). Comparing with historical data (HLH-94), using HLH-94 inclusion criteria, pre-HSCT mortality was nonsignificantly reduced from 27% to 19% (P = .064 adjusted for age and sex). Time from start of therapy to HSCT was shorter compared with HLH-94 (P =020 adjusted for age and sex) and reported neurological alterations at HSCT were 22% in HLH-94 and 17% in HLH-2004 (using HLH-94 inclusion criteria). Five-year pSu post-HSCT overall was 66% (verified FHL, 70% [63%-78%]). Additional analyses provided specific suggestions on potential pre-HSCT treatment improvements. HLH-2004 confirms that a majority of patients may be rescued by the etoposide/dexamethasone combination but intensification with CSA upfront, adding corticosteroids to intrathecal therapy, and reduced time to HSCT did not improve outcome significantly.

Place, publisher, year, edition, pages
American Society of Hematology , 2017. Vol. 130, no 25, p. 2728-2738
National Category
Hematology
Identifiers
URN: urn:nbn:se:oru:diva-63902DOI: 10.1182/blood-2017-06-788349ISI: 000418895900008PubMedID: 28935695Scopus ID: 2-s2.0-85039156751OAI: oai:DiVA.org:oru-63902DiVA, id: diva2:1171243
Funder
Swedish Childhood Cancer FoundationSwedish Research CouncilCancer and Allergy FoundationThe Karolinska Institutet's Research Foundation
Note

Funding Agencies:

Märta and Gunnar V Philipson Foundation

Stockholm Country Council (ALF-project) 

Available from: 2018-01-06 Created: 2018-01-06 Last updated: 2018-09-12Bibliographically approved

Open Access in DiVA

No full text in DiVA

Other links

Publisher's full textPubMedScopus

Authority records BETA

Montgomery, Scott

Search in DiVA

By author/editor
Montgomery, Scott
By organisation
School of Medical Sciences
In the same journal
Blood
Hematology

Search outside of DiVA

GoogleGoogle Scholar

doi
pubmed
urn-nbn

Altmetric score

doi
pubmed
urn-nbn
Total: 59 hits
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association-8th-edition
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf