Sökning: onr:"swepub:oai:DiVA.org:oru-63902" >
Confirmed efficacy ...
Confirmed efficacy of etoposide and dexamethasone in HLH treatment : long-term results of the cooperative HLH-2004 study
-
- Bergsten, Elisabet (författare)
- Karolinska Institutet
-
- Horne, AnnaCarin (författare)
- Karolinska Institutet
-
- Aricó, Maurizio (författare)
- Direzione Generale, Azienda Sanitaria Provinciale, Ragusa, Italy
-
visa fler...
-
- Astigarraga, Itziar (författare)
- Servicio de Pediatria, BioCruces Health Research Institute, Hospital Universitario Cruces, Universidad del Pais Vasco–Euskal Herriko Unibertsitatea (UPV/EHU), Barakaldo, Spain
-
- Egeler, R. Maarten (författare)
- Division of Hematology & Oncology, The Hospital for Sick Children, Toronto ON, Canada
-
- Filipovich, Alexandra H. (författare)
- Division of Bone Marrow Transplant and Immune Deficiency, Cincinnati Children’s Hospital Medical Center, Cincinnati OH, USA
-
- Ishii, Eiichi (författare)
- Department of Pediatrics, Graduate School of Medicine, Ehime University, Ehime, Japan
-
- Janka, Gritta (författare)
- Pediatric Hematology and Oncology, University Medical Center Hamburg, Hamburg, Germany
-
- Ladisch, Stephan (författare)
- Center for Cancer and Immunology Research, Children’s Research Institute, Children’s National Medical Center, Washington DC, USA
-
- Lehmberg, Kai (författare)
- Pediatric Hematology and Oncology, University Medical Center Hamburg, Hamburg, Germany
-
- McClain, Kenneth L. (författare)
- Texas Children’s Cancer Center, Baylor College of Medicine, Houston TX, USA
-
- Minkov, Milen (författare)
- University Clinic of Pediatrics, St. Anna Children’s Hospital, Medical University of Vienna, Vienna, Austria
-
- Montgomery, Scott, 1961- (författare)
- Karolinska Institutet,Örebro universitet,Institutionen för medicinska vetenskaper,Clinical Epidemiology Unit, Department of Medicine, Solna, Karolinska Institutet, Stockholm, Sweden; Department of Epidemiology and Public Health, University College London, London, United Kingdom,Clinical Epidemiology and Biostatistics
-
- Nanduri, Vasanta (författare)
- Department of Pediatrics, Watford General Hospital, Watford, United Kingdom
-
- Rosso, Diego (författare)
- Department of Pediatric Hematology and Oncology, Hospital de Niños Dr Pedro De Elizalde, Buenos Aires, Argentina; Department of Pediatrics, Hospital de Clinicas Jose de San Martin, Buenos Aires, Argentina; Consejo Nacional de Investigaciones Científicas y Técnicas (CONICET), Buenos Aires, Argentina
-
- Henter, Jan-Inge (författare)
- Karolinska Institutet
-
visa färre...
-
(creator_code:org_t)
- American Society of Hematology, 2017
- 2017
- Engelska.
-
Ingår i: Blood. - : American Society of Hematology. - 0006-4971 .- 1528-0020. ; 130:25, s. 2728-2738
- Relaterad länk:
-
https://ashpublicati...
-
visa fler...
-
https://urn.kb.se/re...
-
https://doi.org/10.1...
-
http://kipublication...
-
visa färre...
Abstract
Ämnesord
Stäng
- 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.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Publikations- och innehållstyp
- ref (ämneskategori)
- art (ämneskategori)
Hitta via bibliotek
-
Blood
(Sök värdpublikationen i LIBRIS)
Till lärosätets databas
- Av författaren/redakt...
-
Bergsten, Elisab ...
-
Horne, AnnaCarin
-
Aricó, Maurizio
-
Astigarraga, Itz ...
-
Egeler, R. Maart ...
-
Filipovich, Alex ...
-
visa fler...
-
Ishii, Eiichi
-
Janka, Gritta
-
Ladisch, Stephan
-
Lehmberg, Kai
-
McClain, Kenneth ...
-
Minkov, Milen
-
Montgomery, Scot ...
-
Nanduri, Vasanta
-
Rosso, Diego
-
Henter, Jan-Inge
-
visa färre...
- Om ämnet
-
- MEDICIN OCH HÄLSOVETENSKAP
-
MEDICIN OCH HÄLS ...
-
och Klinisk medicin
-
och Hematologi
- Artiklar i publikationen
-
Blood
- Av lärosätet
-
Örebro universitet
-
Karolinska Institutet