Confirmed efficacy of etoposide and dexamethasone in HLH treatment: long-term results of the cooperative HLH-2004 study

Elisabet Bergsten(Karolinska University Hospital), AnnaCarin Horne(Karolinska University Hospital), Maurizio Aricò(Agenzia Regionale Sanitaria della Puglia), Itziar Astigarraga(University of the Basque Country), R. Maarten Egeler(Hospital for Sick Children), Alexandra H. Filipovich(Cincinnati Children's Hospital Medical Center), Eiichi Ishii(Ehime University), Gritta Janka(Universität Hamburg), Stephan Ladisch(Children's National), Kai Lehmberg(Universität Hamburg), Kenneth L. McClain(Baylor College of Medicine), Milen Minkov(St Anna Children's Hospital), Scott Montgomery(Örebro University), Vasanta Nanduri(Watford General Hospital), Diego Rosso(Consejo Nacional de Investigaciones Científicas y Técnicas), Jan‐Inge Henter(Karolinska University Hospital)
Blood
September 22, 2017
Cited by 657Open Access
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Abstract

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.


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