Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation
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Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation. / Böhm, Svea; Wustrau, Katharina; Pachlopnik Schmid, Jana; Prader, Seraina; Ahlman, Martina; Yacobovich, Joanne; Beier, Rita; Speckmann, Carsten; Behnisch, Wolfgang; Ifversen, Marianne; Jordan, Michael B; Marsh, Rebecca A; Naumann-Bartsch, Nora; Mauz-Koerholz, Christine; Honig, Manfred; Schulz, Ansgar S; Malinowska, Iwona; Hines, Melissa Ruth; 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; Duerken, Matthias; Lang, Peter; Lindemans, Caroline A; Henter, Jan-Inge; Lehmberg, Kai; Ehl, Stephan.
In: BLOOD, Vol. 143, No. 10, 07.03.2024, p. 872-881.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation
AU - Böhm, Svea
AU - Wustrau, Katharina
AU - Pachlopnik Schmid, Jana
AU - Prader, Seraina
AU - Ahlman, Martina
AU - Yacobovich, Joanne
AU - Beier, Rita
AU - Speckmann, Carsten
AU - Behnisch, Wolfgang
AU - Ifversen, Marianne
AU - Jordan, Michael B
AU - Marsh, Rebecca A
AU - Naumann-Bartsch, Nora
AU - Mauz-Koerholz, Christine
AU - Honig, Manfred
AU - Schulz, Ansgar S
AU - Malinowska, Iwona
AU - Hines, Melissa Ruth
AU - Nichols, Kim E
AU - Gil-Herrera, Juana
AU - Talano, Julie-An
AU - Crooks, Bruce
AU - Formankova, Renata
AU - Jorch, Norbert
AU - Bakhtiar, Shahrzad
AU - Kühnle, Ingrid
AU - Streiter, Monika
AU - Nathrath, Michaela
AU - Russo, Alexandra
AU - Duerken, Matthias
AU - Lang, Peter
AU - Lindemans, Caroline A
AU - Henter, Jan-Inge
AU - Lehmberg, Kai
AU - Ehl, Stephan
N1 - Copyright © 2023 American Society of Hematology.
PY - 2024/3/7
Y1 - 2024/3/7
N2 - 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.
AB - 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.
U2 - 10.1182/blood.2023022281
DO - 10.1182/blood.2023022281
M3 - SCORING: Journal article
C2 - 37992218
VL - 143
SP - 872
EP - 881
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 10
ER -
