-
Something wrong with this record ?
Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation
S. Böhm, K. Wustrau, J. Pachlopnik Schmid, S. Prader, M. Ahlmann, J. Yacobovich, R. Beier, C. Speckmann, W. Behnisch, M. Ifversen, M. Jordan, R. Marsh, N. Naumann-Bartsch, C. Mauz-Körholz, M. Hönig, A. Schulz, I. Malinowska, M. Hines, KE....
Language English Country United States
Document type Journal Article
NLK
Free Medical Journals
from 1946 to 1 year ago
Freely Accessible Science Journals
from 1946 to 1 year ago
Open Access Digital Library
from 1946-01-01
Open Access Digital Library
from 1946-01-01
ROAD: Directory of Open Access Scholarly Resources
- MeSH
- Etoposide therapeutic use MeSH
- Humans MeSH
- Lymphohistiocytosis, Hemophagocytic * drug therapy diagnosis MeSH
- Lymphoproliferative Disorders * etiology MeSH
- Infant, Newborn MeSH
- Hematopoietic Stem Cell Transplantation * methods MeSH
- Treatment Outcome MeSH
- Check Tag
- Humans MeSH
- Infant, Newborn MeSH
- Publication type
- Journal Article MeSH
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.
Center for Pediatric and Adolescent Medicine Frankfurt University Hospital Frankfurt Germany
Clinic for Children and Adolescents University Clinic Erlangen Erlangen Germany
Department of Pediatrics and Adolescent Medicine Mannheim University Hospital Mannheim Germany
Department of Pediatrics University Medical Center Ulm Ulm Germany
Department of Pediatrics University Medical Center Utrecht Utrecht The Netherlands
Division of Pediatric Hematology and Oncology Göttingen University Medical Center Göttingen Germany
Medical Faculty Martin Luther University of Halle Wittenberg Halle Germany
Paediatric Haematology Oncology IWK Health Centre Halifax NS Canada
Pediatric Hematology and Oncology Hannover Medical School Hannover Germany
Pediatric Hematooncology University Children's Hospital Giessen Giessen Germany
Pediatric Immunology University Children's Hospital Zurich Eleonorenstiftung Zürich Switzerland
Princess Máxima Center Utrecht The Netherlands
Schneider Children's Medical Center of Israel Petah Tikva Israel
Tel Aviv Medical School Tel Aviv University Tel Aviv Israel
Theme of Children's Health Karolinska University Hospital Stockholm Sweden
References provided by Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc24006781
- 003
- CZ-PrNML
- 005
- 20240423155507.0
- 007
- ta
- 008
- 240412s2024 xxu f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1182/blood.2023022281 $2 doi
- 035 __
- $a (PubMed)37992218
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a xxu
- 100 1_
- $a Böhm, Svea $u Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Eppendorf, Hamburg, Germany
- 245 10
- $a Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation / $c S. Böhm, K. Wustrau, J. Pachlopnik Schmid, S. Prader, M. Ahlmann, J. Yacobovich, R. Beier, C. Speckmann, W. Behnisch, M. Ifversen, M. Jordan, R. Marsh, N. Naumann-Bartsch, C. Mauz-Körholz, M. Hönig, A. Schulz, I. Malinowska, M. Hines, KE. Nichols, J. Gil-Herrera, JA. Talano, B. Crooks, R. Formankova, N. Jorch, S. Bakhtiar, I. Kühnle, M. Streiter, M. Nathrath, A. Russo, M. Dürken, P. Lang, C. Lindemans, JI. Henter, K. Lehmberg, S. Ehl
- 520 9_
- $a 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.
- 650 _2
- $a novorozenec $7 D007231
- 650 _2
- $a lidé $7 D006801
- 650 _2
- $a etoposid $x terapeutické užití $7 D005047
- 650 12
- $a lymfohistiocytóza hemofagocytární $x farmakoterapie $x diagnóza $7 D051359
- 650 _2
- $a výsledek terapie $7 D016896
- 650 12
- $a transplantace hematopoetických kmenových buněk $x metody $7 D018380
- 650 12
- $a lymfoproliferativní nemoci $x etiologie $7 D008232
- 655 _2
- $a časopisecké články $7 D016428
- 700 1_
- $a Wustrau, Katharina $u Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Eppendorf, Hamburg, Germany $u Department of Pediatrics, University Medical Center Ulm, Ulm, Germany
- 700 1_
- $a Pachlopnik Schmid, Jana $u Division of Immunology and Children's Research Center, University Children's Hospital Zürich, Zürich, Switzerland $u Pediatric Immunology, University Children's Hospital Zurich-Eleonorenstiftung, Zürich, Switzerland $1 https://orcid.org/0000000266539047
- 700 1_
- $a Prader, Seraina $u Division of Immunology and Children's Research Center, University Children's Hospital Zürich, Zürich, Switzerland $u Pediatric Immunology, University Children's Hospital Zurich-Eleonorenstiftung, Zürich, Switzerland
- 700 1_
- $a Ahlmann, Martina $u Department of Pediatric Hematology and Oncology, University Children's Hospital Münster, Münster, Germany
- 700 1_
- $a Yacobovich, Joanne $u Schneider Children's Medical Center of Israel, Petah Tikva, Israel $u Tel Aviv Medical School, Tel Aviv University, Tel Aviv, Israel $1 https://orcid.org/0000000337635694
- 700 1_
- $a Beier, Rita $u Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany $1 https://orcid.org/0000000275688749
- 700 1_
- $a Speckmann, Carsten $u Division of Pediatric Hematology and Oncology Faculty of Medicine, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany $u Center for Chronic Immunodeficiency, Institute for Immunodeficiency, Medical Center-University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany
- 700 1_
- $a Behnisch, Wolfgang $u Center for Pediatrics and Adolescent Medicine, Hematology/Oncology, Heidelberg University Hospital, Heidelberg, Germany
- 700 1_
- $a Ifversen, Marianne $u Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
- 700 1_
- $a Jordan, Michael $u Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
- 700 1_
- $a Marsh, Rebecca $u Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
- 700 1_
- $a Naumann-Bartsch, Nora $u Clinic for Children and Adolescents, University Clinic Erlangen, Erlangen, Germany $1 https://orcid.org/0000000156069822
- 700 1_
- $a Mauz-Körholz, Christine $u Pediatric Hematooncology, University Children's Hospital Giessen, Giessen, Germany $u Medical Faculty, Martin-Luther University of Halle-Wittenberg, Halle, Germany $1 https://orcid.org/0000000282058665
- 700 1_
- $a Hönig, Manfred $u Department of Pediatrics, University Medical Center Ulm, Ulm, Germany
- 700 1_
- $a Schulz, Ansgar $u Department of Pediatrics, University Medical Center Ulm, Ulm, Germany $1 https://orcid.org/0000000277987996
- 700 1_
- $a Malinowska, Iwona $u Department of Oncology, Pediatric Hematology, Clinical Transplantology and Pediatrics, Medical University of Warsaw, Warsaw, Poland
- 700 1_
- $a Hines, Melissa $u Division of Critical Care, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, TN
- 700 1_
- $a Nichols, Kim E $u Division of Critical Care, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, TN $1 https://orcid.org/0000000255816555
- 700 1_
- $a Gil-Herrera, Juana $u Immunology, Hospital General Universitario and Instituto de Investigación Sanitaria "Gregorio Marañón," Madrid, Spain
- 700 1_
- $a Talano, Julie-An $u Pediatric Hematology/Oncology/Blood and Marrow Transplant Division, Medical College of Wisconsin, Madison, WI
- 700 1_
- $a Crooks, Bruce $u Paediatric Haematology/Oncology, IWK Health Centre, Halifax, NS, Canada $1 https://orcid.org/0000000347154536
- 700 1_
- $a Formankova, Renata $u Department of Pediatric Haematology and Oncology, University Hospital Motol Prague, Prague, Czech Republic
- 700 1_
- $a Jorch, Norbert $u Bielefeld University, University Clinic for Pediatrics, Evangelisches Klinikum Bethel, Bielefeld, Germany
- 700 1_
- $a Bakhtiar, Shahrzad $u Center for Pediatric and Adolescent Medicine, Frankfurt University Hospital, Frankfurt, Germany
- 700 1_
- $a Kühnle, Ingrid $u Division of Pediatric Hematology and Oncology, Göttingen University Medical Center, Göttingen, Germany
- 700 1_
- $a Streiter, Monika $u Clinic for Pediatrics and Adolescent Medicine, Hematology/Oncology, Klinikum am Gesundbrunnen Heilbronn, Heilbronn, Germany
- 700 1_
- $a Nathrath, Michaela $u Pediatric Hematology and Oncology, Psychosomatics and Systemic Diseases, Kassel Hospital, Kassel, Germany $u Department of Pediatrics and Children's Cancer Research Center, Klinikum rechts der Isar, Technical University of Munich, Munich, Germany
- 700 1_
- $a Russo, Alexandra $u Center for Pediatric and Adolescent Medicine, Pediatric Hematology/Oncology, University of Mainz, Mainz, Germany
- 700 1_
- $a Dürken, Matthias $u Department of Pediatrics and Adolescent Medicine, Mannheim University Hospital, Mannheim, Germany
- 700 1_
- $a Lang, Peter $u Clinic for Children and Adolescents, Department I Hematology/Oncology, Tübingen University Hospital, Tübingen, Germany
- 700 1_
- $a Lindemans, Caroline $u Department of Pediatrics, University Medical Center Utrecht, Utrecht, The Netherlands $u Princess Máxima Center, Utrecht, The Netherlands
- 700 1_
- $a Henter, Jan-Inge $u Department of Women's and Children's Health, Childhood Cancer Research Unit, Karolinska Institutet, Stockholm, Sweden $u Theme of Children's Health, Karolinska University Hospital, Stockholm, Sweden $1 https://orcid.org/0000000206292126
- 700 1_
- $a Lehmberg, Kai $u Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Eppendorf, Hamburg, Germany
- 700 1_
- $a Ehl, Stephan $u Center for Chronic Immunodeficiency, Institute for Immunodeficiency, Medical Center-University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany $1 https://orcid.org/0000000292652721
- 773 0_
- $w MED00000807 $t Blood $x 1528-0020 $g Roč. 143, č. 10 (2024), s. 872-881
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/37992218 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y - $z 0
- 990 __
- $a 20240412 $b ABA008
- 991 __
- $a 20240423155504 $b ABA008
- 999 __
- $a ok $b bmc $g 2081018 $s 1216548
- BAS __
- $a 3
- BAS __
- $a PreBMC-MEDLINE
- BMC __
- $a 2024 $b 143 $c 10 $d 872-881 $e 20240307 $i 1528-0020 $m Blood $n Blood $x MED00000807
- LZP __
- $a Pubmed-20240412
