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Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis

Lehmberg, K; Moshous, D; Booth, C; (2019) Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis. Frontiers in Pediatrics , 7 , Article 435. 10.3389/fped.2019.00435. Green open access

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Abstract

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 (CNS) 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-clinical and translational levels.

Type: Article
Title: Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis
Open access status: An open access version is available from UCL Discovery
DOI: 10.3389/fped.2019.00435
Publisher version: https://doi.org/10.3389/fped.2019.00435
Language: English
Additional information: 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. http://creativecommons.org/licenses/by/4.0/
Keywords: haemophagocytic lymhohistiocytosis, macrophage activation syndrome, haematopoietic stem cell transplantation, gene therapy, veno-occlusive disease, mixed chimerism, reduced toxicity conditioning
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10091601
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