Granulocyte-macrophage colony stimulating factor (GM-CSF, sargramostim/immunex corp.) after tcell- depleted allogeneic bmt for myelodysplastic syndromes (MDS): preliminary results of a phase ii trial

Paul V. O'Donnell, Douglas Gladsone, Stephen J. Noga, Richard J Jones

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Based on preliminary data that GM-CSF may have anti-leukemic activity for myeloid malignancies in vitro and in vivo we initiated a Phase II trial of GM-CSF administration after allogeneic BMT for MDS. Patients received a T-cell-depleted allograft prepared by counterflow centrifugal elutriation of a bone marrow mononuclear cell preparation with recovery of CD34+cells from the lymphocyte-rich elutriation fractions [O'Donnell, et al., BMT 22: 947 ( 1998)]. Patients received GM-CSF at a dose of 250 mcg/mVd beginning at day 5 until ANC>2000/uL for 3 d at which time the dose was decreased to 125 mcg/m2/d until day 60. Standard GVHD prophylaxis with Cyclosporine A was given until day 180. To date, 8 patients with advanced MDS [median age 51 yr (range: 33-64 yr)] have been transplanted with a short median follow-up of 15 mo. Post-BMT GM-CSF was well-tolerated without grade 3 or 4 toxicity attributable to study drug. All patients have completed the course of study drug. There were 2 deaths, one from aspergillus infection in the setting of Grade IV GVHD and one from failure-to-engraft. There was no other clinically significant GVHD. The remaining patients are alive and disease-free. Comparison was made to a historical control group of 28 patients with advanced MDS [median age 46 yr (range: 29-65 yr)] transplanted at this center since 1992 on the same transplant protocol except for administration of GM-CSF post-BMT. Actuarial event-free survival (EPS) of this control group of patients is 29% at 5 yr. Actuarial EPS for the study group is 71 % at 1 yr but there have been only two events in this small group of patients compared to 20 events in the larger group. Comparison of the two groups by Cox proportional hazards analysis shows no significant difference in EPS (p=0.316). Increased patient accrual and longer follow-up will be required to determine if treatment of patients with GM-CSF post-BMT leads to improved event-free and overall survival.

Original languageEnglish (US)
Issue number11 PART II
Publication statusPublished - 2000


ASJC Scopus subject areas

  • Hematology

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