Mini Review ARTICLE
Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis
- 1Division of Paediatric Stem Cell Transplantation and Immunology, University Medical Center Hamburg-Eppendorf, Germany
- 2Department of Immunohematology, Necker-Enfants Malades Hospital, France
- 3Imagine Institut, Inserm U 1163, Université Paris Descartes, France
- 4Department of Paediatric Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, United Kingdom
Review: Haematopoietic stem cell transplantation currently remains the only curative treatment of primary forms of haemophagocytic lymphohistiocytosis (HLH). Rapid diagnosis, efficient primary treatment of hyperinflammation, and conditioning regimens tailored to this demanding condition have substantially improved prognosis in the past 40 years. However, refractory hyperinflammation, central nervous system involvement, unavailability of matched donors, susceptibility to conditioning-related toxicities, and a high frequency of mixed chimaerism remain a challenge in a substantial proportion of patients. Gene therapeutic approaches for several genetic defects of primary HLH are being developed at pre-clinincal and translational levels.
Keywords: Macrophage Activation Syndrome (MAS), Haematopoietic stem cell transplantation (HSCT), hemophagocitic lymphohistiocytosis, gene tharapy, Veno-occlusive disease (PVOD), Mixed chimerísm, reduced toxicity conditioning
Received: 10 Jul 2019;
Accepted: 07 Oct 2019.
Copyright: © 2019 Lehmberg, Moshous and Booth. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: MD. Kai Lehmberg, University Medical Center Hamburg-Eppendorf, Division of Paediatric Stem Cell Transplantation and Immunology, Hamburg, Germany, email@example.com