J Korean Pediatr Soc.  1994 Mar;37(3):339-346.

The Effect of Alfacalcidol in the Treatment of Idiopathic Myelofibrosis in Children

Abstract

Idiopathic myelofibrosis (IMF), which is characterized by marrow fibrosis, leukoerythroblastic anemia, teardrop poikilocytosis and splenomegaly due to extrumedullary hematopoiesis, has known to have no form of therapy. On the ground of the possibility of reversing collagen deposion in IMF using 1, 25dihydroxycholecalciferol [1, 25(OH)2D3], we report here our observations of 5 patients (M:F=1:4) with IMF before and after treatment with 0.5 microgram/day of alfacalcidol, precursor of 1, 25(OH)2D3. In 3 fo 5 patients the hemoglobin rose and in 4 of 5 the platelet count increased. Follow-up marrow examination revealed that marrow trephine reticulin fibrosis decreased according as the amelioration of clinical and laboratory findings. But these did not persist except one patient in spite of the sustained use of alfacalcidol. Our results suggest that alfacalcidol may have a therapeutic role in some patients with IMF. More extensive studies will be clarify the action of alfacalcidol in IMF.


MeSH Terms

Anemia, Myelophthisic
Bone Marrow
Child*
Collagen
Fibrosis
Follow-Up Studies
Hematopoiesis
Humans
Platelet Count
Primary Myelofibrosis*
Reticulin
Splenomegaly
Collagen
Reticulin
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