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Allogeneic hematopoietic stem cell transplantation for adult HLH: a retrospective study by the chronic malignancies and inborn errors working parties of EBMT

Bone Marrow Transplantation volume 57, pages 817–823 (2022)Cite this article

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Abstract

Hemophagocytic lymphohistiocytosis (HLH; hemophagocytic syndrome) is a rare syndrome of potentially fatal, uncontrolled hyperinflammation. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is indicated in primary, recurrent or progressive HLH, but information about its outcomes in the adult population is limited. We obtained data about 87 adult (≥18 years of age) patients retrospectively reported to the EBMT. The median survival time was 13.9 months. The three and five-year overall survival (OS) was 44% (95% CI 33–54%). Among 39 patients with a follow-up longer than 15 months, only three died. Relapse rate was 21% (95% CI 13–30%), while NRM reached 36% (95% CI 25–46%). Younger patients (<30 years of age) had better prognosis, with an OS of 59% (95% CI 45–73%) at three and five years vs 23% (95% CI 8–37%) for older ones. No difference in survival between reduced and myeloablative conditioning was found. To our knowledge, this is the largest report of adult HLH patients who underwent allo-HSCT. Patients who survive the first period after this procedure can expect a long disease-free survival. Both reduced intensity and myeloablative conditioning have therapeutic potential in adult HLH.

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Fig. 1: Outcomes of allogeneic hematopoietic stem cell transplantation in 87 adult HLH patients.
Fig. 2: Overall survival of adult HLH patietns undergoing allogeneic hematopoietic stem cell transplantation.
Fig. 3: Overall survival of patients by the conditioning intensity.

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Author information

Authors and Affiliations

  1. Department of Hematology, Transplantation and Internal Medicine, Medical University of Warsaw, Warsaw, Poland

    Rafal Machowicz & Wieslaw Wiktor-Jedrzejczak

  2. Service d’hématologie adultes, Hôpital Necker-Enfants Malades - AP-HP. Centre Université de Paris Institut Imagine - INSERM UMR 1163 et CNRS ERL 8254 Université de Paris, Paris, France

    Felipe Suarez

  3. EBMT Statistical Unit, Leiden, The Netherlands

    Diderik-Jan Eikema

  4. Department of Biomedical Data Sciences, LUMC, Leiden, The Netherlands

    Liesbeth C. de Wreede

  5. EBMT Data Office Leiden, Leiden, The Netherlands

    Henric-Jan Blok

  6. Umeå University Hospital, Umeå, Sweden

    Cecilia Isaksson

  7. Universitätsklinikum Würzburg, Würzburg, Germany

    Hermann Einsele

  8. Cliniques Universitaires St. Luc, Brussels, Belgium

    Xavier Poiré

  9. Radboud University - Medical Centre, Nijmegen, The Netherlands

    Suzanne van Dorp

  10. Birmingham Heartlands Hospital, Birmingham, UK

    Emmanouil Nikolousis

  11. Sahlgrenska University Hospital, Goeteborg, Sweden

    Jan-Erik Johansson

  12. University Hospital Düsseldorf, Medical Faculty of the Heinrich Heine University Düsseldorf, Düsseldorf, Germany

    Guido Kobbe

  13. Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    Marco Zecca

  14. Charité Universitätsmedizin Berlin, Berlin, Germany

    Renate Arnold & Armin Gerbitz

  15. University of Freiburg, Freiburg, Germany

    Jürgen Finke

  16. Hematology Department, Hospital General Universitario Gregorio Marañon, Instituto de Investigaciónes sanitarias Gregorio Marañon, Medicina, Universidad Complutense, Madrid, Spain

    Jose Luis Díez-Martín

  17. IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy

    Francesca Bonifazi

  18. Queen Elizabeth University Hospital, Glasgow, UK

    Grant McQuaker

  19. Skanes University Hospital, Lund, Sweden

    Stig Lenhoff

  20. Department of Hematology, CHU de Nice, University of Cote d’Azur, Nice, France

    Pierre-Simon Rohrlich

  21. University Medical Center, University Cancer Center, Department of Hematology, Medical Oncology, and Pneumology, Mainz, Germany

    Matthias Theobald

  22. Dept. of Cellular Therapy and Allogeneic Stem Cell Transplantation, Karolinska University Hospital Huddinge, Stockholm, Sweden

    Per Ljungman

  23. Freeman Hospital, Newcastle-Upon-Tyne, Newcastle upon Tyne, UK

    Matthew Collin

  24. Department of Pediatrics, Dr. von Hauner Children’s Hospital, University Hospital, LMU, Munich, Germany

    Michael H. Albert

  25. Universitätsklinikum Dresden, Dresden, Germany

    Gerhard Ehninger

  26. University Hospital, Uppsala, Sweden

    Kristina Carlson

  27. Institute of Hematology and Transfusion Medicine, Warsaw, Poland

    Kazimierz Halaburda

  28. University Hospital Eppendorf, Hamburg, Germany

    Kai Lehmberg & Nicolaus Kröger

  29. University of Heidelberg, Heidelberg, Germany

    Stefan Schönland

  30. CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000, Lille, France

    Ibrahim Yakoub-Agha

  31. Translational and Clinical Research Institute, Newcastle University and Dept Paediatric Immunology + HSCT, Great North Childrens’ Hospital Newcastle upon Tyne, Newcastle upon Tyne, UK

    Andrew R. Gennery

  32. Willem-Alexander Children’s Hospital, Paediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, The Netherlands

    Arjan C. Lankester

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  35. Nicolaus Kröger
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Contributions

RM conceived and designed the study, analyzed and interpreted data, designed the questionnaire and drafted the manuscript, NK designed the study, analyzed and interpreted data and edited the manuscript, FS designed the questionnaire, provided clinical data and edited the manuscript, WWJ assisted with the study design and data interpretation and edited the manuscript, DJE and LdW performed statistical analyses and edited the manuscript, HJB performed data collection and edited the manuscript, CI, HE, XP, SvD, EN, JEJ, GK, MZ, RA, AG, JF, JLDM, FB, GMQ, SL, PSR, MT, PL, MC, MA, GE, KC, KH, KL, SS provided clinical data and edited the manuscript, AL, AG and IYA assisted with the study design and data interpretation and edited the manuscript.

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Correspondence to Rafal Machowicz.

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Machowicz, R., Suarez, F., Wiktor-Jedrzejczak, W. et al. Allogeneic hematopoietic stem cell transplantation for adult HLH: a retrospective study by the chronic malignancies and inborn errors working parties of EBMT. Bone Marrow Transplant 57, 817–823 (2022). https://doi.org/10.1038/s41409-022-01634-5

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