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Treatment of familial hemophagocytic lymphohistiocytosis with antithymocyte
globulins, steroids, and cyclosporin A
JL Stephan, J Donadieu, F Ledeist, S Blanche, C Griscelli and A Fischer
Departement de Pediatrie, Hopital des Enfants Malades, Paris, France.
Familial hemophagocytic lymphohistiocytosis (FHL) is a potentially fatal
disease characterized by diffuse infiltration by histiocytes and T
lymphocytes. Treatment with myelotoxic drugs, such as etoposide, brings
about remission in most patients, but problems of toxicity remain, and the
development of disease resistance can cause secondary relapses. We have
used an alternative approach, based on the suggested primary role of T-cell
activation in FHL, comprising combined treatment with steroids (2 to 5
mg/kg/d methylprednisolone intravenously, followed by progressive tapering)
and rabbit antithymocyte globulins (10 mg/kg/d for 5 days), followed by
maintenance therapy with cyclosporine A (CSA). In a pilot study of six
patients (four with a family history of FHL), all showed systemic remission
within 7 days, which was complete in five cases; despite treatment with
intrathecal methotrexate, one patient died of severe brain involvement. Two
patients received T-cell--depleted HLA--non-identical bone marrow
transplants, which was successful in one case. The other three patients,
who have been on CSA maintenance therapy for periods of 6 to 24 months, are
in complete remission. We have observed no side-effects (there has been no
persisting T-cell immunodeficiency). These results suggest that
nonmyelotoxic treatments for FHL may be safe, effective, and worthy of
further investigation; they also support the key role of T lymphocytes in
the disease.
Volume 82,
Issue 8,
pp. 2319-2323,
10/15/1993
Copyright © 1993 by The American Society of Hematology
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