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

Bergsten, Elisabet ; Horne, AnnaCarin ; Aricó, Maurizio ; Astigarraga, Itziar ; Egeler, R. Maarten ; Filipovich, Alexandra H. ; Ishii, Eiichi ; Janka, Gritta ; Ladisch, Stephan ; Lehmberg, Kai ; McClain, Kenneth L. ; Minkov, Milen ; Montgomery, Scott ; Nanduri, Vasanta ; Rosso, Diego ; Henter, Jan-Inge

Blood, 2017-12, Vol.130 (25), p.2728-2738 [Periódico revisado por pares]

United States: Elsevier Inc

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Título:
Confirmed efficacy of etoposide and dexamethasone in HLH treatment: long-term results of the cooperative HLH-2004 study
Autor: Bergsten, Elisabet ; Horne, AnnaCarin ; Aricó, Maurizio ; Astigarraga, Itziar ; Egeler, R. Maarten ; Filipovich, Alexandra H. ; Ishii, Eiichi ; Janka, Gritta ; Ladisch, Stephan ; Lehmberg, Kai ; McClain, Kenneth L. ; Minkov, Milen ; Montgomery, Scott ; Nanduri, Vasanta ; Rosso, Diego ; Henter, Jan-Inge
Assuntos: Antineoplastic Combined Chemotherapy Protocols - therapeutic use ; Clinical Trials and Observations ; CME ; Cyclosporine - therapeutic use ; Dexamethasone - therapeutic use ; Etoposide - therapeutic use ; Female ; Hematopoietic Stem Cell Transplantation - methods ; Hematopoietic Stem Cell Transplantation - mortality ; Humans ; Infant ; Longitudinal Studies ; Lymphohistiocytosis, Hemophagocytic - drug therapy ; Lymphohistiocytosis, Hemophagocytic - mortality ; Male ; Medicin och hälsovetenskap ; Treatment Outcome
É parte de: Blood, 2017-12, Vol.130 (25), p.2728-2738
Notas: ObjectType-Article-1
SourceType-Scholarly Journals-1
ObjectType-Feature-2
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Descrição: 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. •Early introduction of cyclosporine did not improve HLH outcome in patients treated with the HLH-94 etoposide-dexamethasone backbone (P = .06).•HLH-2004 may be improved by risk-group stratification, less therapy reduction weeks 7 to 8 for verified FHL patients, and earlier HSCT.
Editor: United States: Elsevier Inc
Idioma: Inglês

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

United States: Elsevier Inc

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