Evolution in the management of acute liver failure-associated aplastic anaemia in children: a single centre experience.
OBJECTIVE
Bone marrow failure (BMF) is a potentially life-threatening complication of acute liver failure (ALF).


METHODS
To investigate prevalence and evolving management of BMF associated with ALF, we reviewed all cases seen in our centre over 17 years. BMF was classified as: (a) bone marrow hypoplasia, (b) severe aplastic anaemia (SAA) and (c) very severe aplastic anaemia (VSAA), using standard criteria. We compared outcomes in children receiving: (1) medical treatment only with or without immunomodulation (anti-lymphocyte globulin, calcineurin inhibitors, G-CSF); (2) medical treatment with or without immunomodulation plus liver transplantation (LT); (3) haematopoietic stem cell transplantation (HSCT).


RESULTS
Of 213 patients with ALF, 20 [(9.4%); 14 (70%) boys] developed BMF after a median of 1 month (range, 0.5 to 7). Seven had VSAA, 7 SAA and 6 bone marrow hypoplasia. Five children were treated medically, including 3 by immunomodulation; 10 (50%) received LT, with immunomodulation in 6; 5 (25%) received HSCT, in one after LT. Four (20%) children died, only one as consequence of AA. There was no difference in recovery, complication rates or outcome among the three groups.


CONCLUSIONS
Aggressive management of ALF-associated AA, including immunomodulation, HSCT and LT, is successful in most cases. HSCT has the advantage of removing the risk of late clonal disorders.
Hadzić
Nedim
N
Institute of Liver Studies, King's College London School of Medicine at King's College, London, UK. nedim.hadzic@kcl.ac.uk
Height
Susan
S
Ball
Sarah
S
Rela
Mohamed
M
Heaton
Nigel D
ND
Veys
Paul
P
Mieli-Vergani
Giorgina
G
D000293:Adolescent; D000741:Anemia, Aplastic / Q000150:complications* / Q000628:therapy*; D001855:Bone Marrow Diseases / Q000150:complications / Q000473:pathology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000175:diagnosis; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use; D007223:Infant; D007231:Infant, Newborn; D017114:Liver Failure, Acute / Q000150:complications* / Q000628:therapy; D016031:Liver Transplantation; D008297:Male; D012189:Retrospective Studies; D016896:Treatment Outcome
D007155:Immunologic Factors