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.
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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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