Failure of recombinant human granulocyte-macrophage colony-stimulating
factor therapy in aplastic anemia patients with very severe neutropenia
C Nissen, A Tichelli, A Gratwohl, B Speck, A Milne, EC Gordon-Smith and J Schaedelin
Preclinical and Clinical Research, Sandoz, Ltd, Basel, Switzerland.
Four patients with very severe aplastic anemia refractory to antilymphocyte
globulin were administered recombinant human granulocyte-
macrophage--colony stimulating factor (GM-CSF). One patient with minimal
residual myelopoiesis responded transiently to two separate courses of
GM-CSF at 4 and 8 micrograms/kg/d administered intravenously and another
course at 4 micrograms/kg/d administered subcutaneously. Septicemia and
bilateral pneumonia that had been resistant to conventional therapy
resolved. Three patients with no evidence of residual myelopoiesis did not
respond to GM-CSF. In one patient, the dose was increased to 32
micrograms/kg/d with no effect on hematopoiesis. Immediate side effects
were minimal at GM-CSF doses up to 16 micrograms/kg/d. GM-CSF may, however,
have been involved in the pathophysiology of thrombosis of the inferior
vena cava in the patient administered 32 micrograms/kg/d. We conclude that
GM-CSF does not induce hematopoiesis in long-standing, severe,
treatment-resistant aplastic anemia with complete myelopoietic failure.
However, in patients with minimal residual myelopoiesis, GM-CSF could be a
promising adjuvant therapy for severe infection.
Volume 72,
Issue 6,
pp. 2045-2047,
12/01/1988
Copyright © 1988 by The American Society of Hematology
