Your browser doesn't support javascript.
loading
Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation.
Böhm, Svea; Wustrau, Katharina; Pachlopnik Schmid, Jana; Prader, Seraina; Ahlmann, Martina; Yacobovich, Joanne; Beier, Rita; Speckmann, Carsten; Behnisch, Wolfgang; Ifversen, Marianne; Jordan, Michael; Marsh, Rebecca; Naumann-Bartsch, Nora; Mauz-Körholz, Christine; Hönig, Manfred; Schulz, Ansgar; Malinowska, Iwona; Hines, Melissa; Nichols, Kim E; Gil-Herrera, Juana; Talano, Julie-An; Crooks, Bruce; Formankova, Renata; Jorch, Norbert; Bakhtiar, Shahrzad; Kühnle, Ingrid; Streiter, Monika; Nathrath, Michaela; Russo, Alexandra; Dürken, Matthias; Lang, Peter; Lindemans, Caroline; Henter, Jan-Inge; Lehmberg, Kai; Ehl, Stephan.
Afiliação
  • Böhm S; Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Eppendorf, Hamburg, Germany.
  • Wustrau K; Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Eppendorf, Hamburg, Germany.
  • Pachlopnik Schmid J; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
  • Prader S; Division of Immunology and Children's Research Center, University Children's Hospital Zürich, Zürich, Switzerland.
  • Ahlmann M; Pediatric Immunology, University Children's Hospital Zurich-Eleonorenstiftung, Zürich, Switzerland.
  • Yacobovich J; Division of Immunology and Children's Research Center, University Children's Hospital Zürich, Zürich, Switzerland.
  • Beier R; Pediatric Immunology, University Children's Hospital Zurich-Eleonorenstiftung, Zürich, Switzerland.
  • Speckmann C; Department of Pediatric Hematology and Oncology, University Children's Hospital Münster, Münster, Germany.
  • Behnisch W; Schneider Children's Medical Center of Israel, Petah Tikva, Israel.
  • Ifversen M; Tel Aviv Medical School, Tel Aviv University, Tel Aviv, Israel.
  • Jordan M; Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
  • Marsh R; Division of Pediatric Hematology and Oncology Faculty of Medicine, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany.
  • Naumann-Bartsch N; Center for Chronic Immunodeficiency, Institute for Immunodeficiency, Medical Center-University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Mauz-Körholz C; Center for Pediatrics and Adolescent Medicine, Hematology/Oncology, Heidelberg University Hospital, Heidelberg, Germany.
  • Hönig M; Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
  • Schulz A; Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
  • Malinowska I; Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
  • Hines M; Clinic for Children and Adolescents, University Clinic Erlangen, Erlangen, Germany.
  • Nichols KE; Pediatric Hematooncology, University Children's Hospital Giessen, Giessen, Germany.
  • Gil-Herrera J; Medical Faculty, Martin-Luther University of Halle-Wittenberg, Halle, Germany.
  • Talano JA; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
  • Crooks B; Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
  • Formankova R; Department of Oncology, Pediatric Hematology, Clinical Transplantology and Pediatrics, Medical University of Warsaw, Warsaw, Poland.
  • Jorch N; Division of Critical Care, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, TN.
  • Bakhtiar S; Division of Critical Care, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, TN.
  • Kühnle I; Immunology, Hospital General Universitario and Instituto de Investigación Sanitaria "Gregorio Marañón," Madrid, Spain.
  • Streiter M; Pediatric Hematology/Oncology/Blood and Marrow Transplant Division, Medical College of Wisconsin, Madison, WI.
  • Nathrath M; Paediatric Haematology/Oncology, IWK Health Centre, Halifax, NS, Canada.
  • Russo A; Department of Pediatric Haematology and Oncology, University Hospital Motol Prague, Prague, Czech Republic.
  • Dürken M; Bielefeld University, University Clinic for Pediatrics, Evangelisches Klinikum Bethel, Bielefeld, Germany.
  • Lang P; Center for Pediatric and Adolescent Medicine, Frankfurt University Hospital, Frankfurt, Germany.
  • Lindemans C; Division of Pediatric Hematology and Oncology, Göttingen University Medical Center, Göttingen, Germany.
  • Henter JI; Clinic for Pediatrics and Adolescent Medicine, Hematology/Oncology, Klinikum am Gesundbrunnen Heilbronn, Heilbronn, Germany.
  • Lehmberg K; Pediatric Hematology and Oncology, Psychosomatics and Systemic Diseases, Kassel Hospital, Kassel, Germany.
  • Ehl S; Department of Pediatrics and Children's Cancer Research Center, Klinikum rechts der Isar, Technical University of Munich, Munich, Germany.
Blood ; 143(10): 872-881, 2024 Mar 07.
Article em En | MEDLINE | ID: mdl-37992218
ABSTRACT: Primary hemophagocytic lymphohistiocytosis (pHLH) is a life-threatening hyperinflammatory syndrome that develops mainly in patients with genetic disorders of lymphocyte cytotoxicity and X-linked lymphoproliferative syndromes. Previous studies with etoposide-based treatment followed by hematopoetic stem cell transplantation (HSCT) resulted in 5-year survival of 50% to 59%. Contemporary data are lacking. We evaluated 88 patients with pHLH documented in the international HLH registry from 2016-2021. In 12 of 88 patients, diagnosis was made without HLH activity, based on siblings or albinism. Major HLH-directed drugs (etoposide, antithymocyte globulin, alemtuzumab, emapalumab, ruxolitinib) were administered to 66 of 76 patients who were symptomatic (86% first-line etoposide); 16 of 57 patients treated with etoposide and 3 of 9 with other first-line treatment received salvage therapy. HSCT was performed in 75 patients; 7 patients died before HSCT. Three-year probability of survival (pSU) was 82% (confidence interval [CI], 72%-88%) for the entire cohort and 77% (CI, 64%-86%) for patients receiving first-line etoposide. Compared with the HLH-2004 study, both pre-HSCT and post-HSCT survival of patients receiving first-line etoposide improved, 83% to 91% and 70% to 88%. Differences to HLH-2004 included preferential use of reduced-toxicity conditioning and reduced time from diagnosis to HSCT (from 148 to 88 days). Three-year pSU was lower with haploidentical (4 of 9 patients [44%]) than with other donors (62 of 66 [94%]; P < .001). Importantly, early HSCT for patients who were asymptomatic resulted in 100% survival, emphasizing the potential benefit of newborn screening. This contemporary standard-of-care study of patients with pHLH reveals that first-line etoposide-based therapy is better than previously reported, providing a benchmark for novel treatment regimes.
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Linfo-Histiocitose Hemofagocítica / Transtornos Linfoproliferativos Limite: Humans / Newborn Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Linfo-Histiocitose Hemofagocítica / Transtornos Linfoproliferativos Limite: Humans / Newborn Idioma: En Ano de publicação: 2024 Tipo de documento: Article