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| Verfasst von: | Böhm, Svea [VerfasserIn]  |
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| | Mauz-Körholz, Christine [VerfasserIn] |
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| | Wustrau, Katharina [VerfasserIn] |
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| | Pachlopnik Schmid, Jana [VerfasserIn] |
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| | Prader, Seraina [VerfasserIn] |
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| | Ahlmann, Martina [VerfasserIn] |
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| | Yacobovich, Joanne [VerfasserIn] |
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| | Beier, Rita [VerfasserIn] |
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| | Speckmann, Carsten [VerfasserIn] |
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| | Behnisch, Wolfgang [VerfasserIn] |
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| | Ifversen, Marianne [VerfasserIn] |
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| | Jordan, Michael [VerfasserIn] |
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| | Marsh, Rebecca [VerfasserIn] |
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| | Naumann-Bartsch, Nora [VerfasserIn] |
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| | Mauz-Körholz, Christine [VerfasserIn] |
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| | Hönig, Manfred [VerfasserIn] |
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| | Schulz, Ansgar [VerfasserIn] |
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| | Malinowska, Iwona [VerfasserIn] |
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| | Hines, Melissa [VerfasserIn] |
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| | Nichols, Kim E. [VerfasserIn] |
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| | Gil-Herrera, Juana [VerfasserIn] |
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| | Talano, Julie-An [VerfasserIn] |
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| | Crooks, Bruce [VerfasserIn] |
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| | Formankova, Renata [VerfasserIn] |
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| | Jorch, Norbert [VerfasserIn] |
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| | Bakhtiar, Shahrzad [VerfasserIn] |
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| | Kühnle, Ingrid [VerfasserIn] |
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| | Streiter, Monika [VerfasserIn] |
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| | Nathrath, Michaela [VerfasserIn] |
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| | Russo, Alexandra [VerfasserIn] |
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| | Dürken, Matthias [VerfasserIn] |
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| | Lang, Peter [VerfasserIn] |
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| | Lindemans, Caroline [VerfasserIn] |
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| | Henter, Jan-Inge [VerfasserIn] |
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| | Lehmberg, Kai [VerfasserIn] |
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| | Ehl, Stephan [VerfasserIn] |
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| Titel: | Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021 |
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| Titelzusatz: | etoposide is better than its reputation |
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| Verf.angabe: | Svea Böhm, Katharina Wustrau, Jana Pachlopnik Schmid, Seraina Prader, Martina Ahlmann, Joanne Yacobovich, Rita Beier, Carsten Speckmann, Wolfgang Behnisch, Marianne Ifversen, Michael Jordan, Rebecca Marsh, Nora Naumann-Bartsch, Christine Mauz-Körholz, Manfred Hönig, Ansgar Schulz, Iwona Malinowska, Melissa Hines, Kim E. Nichols, Juana Gil-Herrera, Julie-An Talano, Bruce Crooks, Renata Formankova, Norbert Jorch, Shahrzad Bakhtiar, Ingrid Kühnle, Monika Streiter, Michaela Nathrath, Alexandra Russo, Matthias Dürken, Peter Lang, Caroline Lindemans, Jan-Inge Henter, Kai Lehmberg, Stephan Ehl |
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| E-Jahr: | 2024 |
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| Jahr: | March 7, 2024 |
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| Umfang: | 10 S. |
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| Fussnoten: | Gesehen am 13.01.2025 |
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| Titel Quelle: | Enthalten in: Blood |
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| Ort Quelle: | Washington, DC : American Society of Hematology, 1946 |
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| Jahr Quelle: | 2024 |
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| Band/Heft Quelle: | 143(2024), 10, Seite 872-881 |
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| ISSN Quelle: | 1528-0020 |
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| 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. |
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| DOI: | doi:10.1182/blood.2023022281 |
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| URL: | Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.
Resolving-System: https://doi.org/10.1182/blood.2023022281 |
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| | DOI: https://doi.org/10.1182/blood.2023022281 |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| K10plus-PPN: | 1883320968 |
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| Verknüpfungen: | → Zeitschrift |
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Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021 / Böhm, Svea [VerfasserIn]; March 7, 2024 (Online-Ressource)
