Haemophagocytic lymphohistiocytosis in children.
OBJECTIVE
To evaluate the clinical and diagnostic features of children presenting with haemophagocytic lymphohistiocytosis (HLH), evolution of the disease and outcomes in response to treatment.


METHODS
The medical records of 12 children, aged 5 weeks to 13 years at diagnosis, with HLH managed at a single institution were reviewed.


RESULTS
Presenting features were fever, hepatosplenomegaly, pancytopenia and hypertriglyceridemia or hypofibrinogenemia. Nine patients (75%) developed central nervous system (CNS) disease. Only one child with CNS disease survived. Five children had complete responses to therapy (42%), but all relapsed at a median of 1.5 months after starting treatment (range 2 weeks to 5 months). Two of the children treated are long-term survivors (17%), both after allogeneic bone marrow transplantation. All deaths occurred in the context of active disease.


CONCLUSIONS
Haemophagocytic lymphohistiocytosis is a disease with a poor prognosis. Central nervous system complications are common and response to treatment usually is transient. This study provides support for the use of immunomodulatory therapy for remission introduction followed by consideration of allogeneic bone marrow transplantation.
Hallahan
A R
AR
Department of Haematology and Oncology, Sydney Children's Hospital, Randwick, New South Wales, Australia.
Carpenter
P A
PA
O'Gorman-Hughes
D W
DW
Vowels
M R
MR
Marshall
G M
GM
D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use; D001856:Bone Marrow Examination; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003937:Diagnosis, Differential; D018450:Disease Progression; D005260:Female; D015616:Histiocytosis, Non-Langerhans-Cell / Q000150:complications / Q000175:diagnosis* / Q000628:therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008297:Male; D011379:Prognosis; D012074:Remission Induction / Q000379:methods; D012189:Retrospective Studies; D016019:Survival Analysis; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D007166:Immunosuppressive Agents