38 Histiocytoses ______________________________________________________________________________________
7. Histiocytoses
7.3 Hemophagocytic lymphohistiocytosis (HLH)
7.3.1 General considerations
Hemophagocytic lymphohistiocytosis (HLH) is a rare disorder of infancy. It comprises familial
(or hereditary or primary) HLH (= FHL) and secondary HLH (= SHLH), both clinically characterized by fever, hepatosplenomegaly and cytopenia.
FHL is an autosomal recessive disease which is invariably fatal when untreated. It is associated with defective triggering of apoptosis resulting in a widespread accumulation of activated T-lymphocytes and histiocytes in the reticuloendothelial system. Moreover, immune system derangement with prominent secretion of inflammatory cytokines ( hypercytokinemia) and low or absent cytotoxic T- and natural killer cell activity is a consistent feature.
SHLH can be either infection- associated, malignancy-associated or idiopathic.
Regarding the therapeutic strategy, prompt differential diagnosis is essential and choice of treatment should be based on the risk of prognoss. Either cyclosporine A, steroids or i.v. immunoglobulin may be indicated as initial treatment for low-risk patients, and etoposide-containing regimens for high-risk patients. Immuno-chemotherapy and allogeneic hematopoietic stem cell transplantation have contributed significantly to further improved survival or cure of familial and refractory HLH.
Literature: for review e.g.
ARCECI, Eur. J. Cancer 35 (1999): 747 – 769 (incl. A commentary by HENTER)
DÜRKEN et al, Leuk. Lymphoma 41(2001): 89 – 95 (role of bone marrow transplantation)
FADEEL et al, Leuk. Lymphoma 42 (2001): 13 – 20
IMASHUKU et al, Expert Opin. Pharmacother. 2 (2001): 1437 – 1448
7.3.2 Study HLH 94
The treatment protocol includes 8 weeks of initial therapy followed (in case of familial disease or persistent nonfamilial disease) by a continuation therapy and if possible a bone marrow transplantation. Children with resolved nonfamilial disease ceased therapy after 8 weeks and restarted only in case of reactivation.
Histiocytoses 41 _____________________________________________________________________________________
Initial therapy
|
Etoposide 150 mg/m² i.v. twice weekly for 2 wks then weekly till wk 8 |
|
Dexamethasone 10 mg/m² i.v. daily for 2 wks, then 5 mg/m² i.v. daily for 2 wks, then 2.5 mg/m² i.v. daily for 2 wks, then 1.25 mg/m² i.v. daily for 1 wks , then one wk of tapering |
|
Methotrexate * i.t. at a max of 4 doses wks 3 – 6 |
not improved
Continuation therapy ( starting week 9 )
|
Etoposide 150 mg/m² i.v. every alternating wk |
|
Dexamethasone 10 mg/m² i.v. d 1-3, every second wk |
|
Cyclosporin* p.o. daily |
Literature:
HENTER et al, Blood 100 (2002): 2367 – 2373