Survival in primary hemophagocytic lymphohistiocytosis 2016-2021: etoposide is better than its reputation
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F24%3A10471275" target="_blank" >RIV/00064203:_____/24:10471275 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11130/24:10471275
Result on the web
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=pJfW8nnd9a" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=pJfW8nnd9a</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1182/blood.2023022281" target="_blank" >10.1182/blood.2023022281</a>
Alternative languages
Result language
angličtina
Original language name
Survival in primary hemophagocytic lymphohistiocytosis 2016-2021: etoposide is better than its reputation
Original language description
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 50-59% 5-year survival. Contemporary data are lacking. We evaluated 88 pHLH patients documented in the international HLH Registry between 2016-2021 with follow-up until 6/2023. In 12/88 patients, the diagnosis was made without HLH activity, based on index siblings or partial albinism. Major HLH-directed drugs (etoposide, ATG, alemtuzumab, emapalumab, ruxolitinib) were given to 66/76 symptomatic patients (86% first-line etoposide); 16/57 etoposide-treated and 3/9 patients with other first-line treatment received salvage therapy. HSCT was performed in 75 patients, 7 symptomatic patients died before HSCT. 3-year probability of survival (pSU) was 82% (CI 72%-88%) for the entire cohort and 77% (CI 64-86%) for symptomatic patients receiving first-line etoposide. Compared to the HLH-2004 study, both pre-HSCT survival (83% to 91%) and post-HSCT survival of patients receiving first-line etoposide improved (70% to 88%). Differences to HLH-2004 included preferential use of reduced-toxicity conditioning and reduced time from diagnosis to HSCT (148 to 88 days). 3-year pSU was lower with haploidentical (44%, 4/9 patients) than with other types of donors (94%, 4/66, p<0.001). Importantly, also in this study, early HSCT of asymptomatic patients resulted in excellent survival (100%), emphasizing the potential benefit of newborn screening. This contemporary standard-of-care study of pHLH patients reveals that first-line etoposide-based therapy is better than previously reported, providing a benchmark for novel treatment regimes.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30205 - Hematology
Result continuities
Project
—
Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2024
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Blood
ISSN
0006-4971
e-ISSN
1528-0020
Volume of the periodical
143
Issue of the periodical within the volume
10
Country of publishing house
US - UNITED STATES
Number of pages
10
Pages from-to
872-881
UT code for WoS article
001214252900001
EID of the result in the Scopus database
2-s2.0-85184047930