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Related Experiment Videos

Effects of CSFs in preleukemia.

P L Greenberg1, R Negrin, A Nagler

  • 1Hematology Division, Stanford University School of Medicine, CA 94305.

Bone Marrow Transplantation
|July 1, 1990
PubMed
Summary

Granulocyte colony-stimulating factors (G-CSF) and granulocyte-macrophage colony-stimulating factors (GM-CSF) show promise in treating myelodysplastic syndromes (MDS) by improving neutrophil counts and reducing infections, though risks of acute myeloid leukemia (AML) progression exist.

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Area of Science:

  • Hematology
  • Oncology
  • Pharmacology

Background:

  • Pre-clinical studies show granulocyte-colony stimulating factors (G-CSF) and granulocyte-macrophage colony-stimulating factors (GM-CSF) enhance granulocytic proliferation and differentiation.
  • These recombinant human hormones are explored for treating cytopenic patients with myelodysplastic syndromes (MDS).

Purpose of the Study:

  • To review the efficacy and safety of short-term and long-term G-CSF and GM-CSF treatment in patients with myelodysplastic syndromes (MDS).

Main Methods:

  • Review of five studies using short-term GM-CSF (7-14 days, 1-5 courses).
  • Analysis of one longer-term GM-CSF study (2-9 weeks).
  • Evaluation of 18 patients treated with G-CSF for 2 months, including maintenance therapy (6-16 months).

Main Results:

  • Short-term GM-CSF improved neutrophil counts in 38/45 patients; 14 had increased reticulocytes, and 8 had transient platelet increases. Seven patients progressed to acute myeloid leukemia (AML), especially those with >15% marrow blasts.
  • Longer-term GM-CSF showed limited sustained neutrophil improvement, with one patient developing antibodies and one progressing to AML.
  • G-CSF treatment for 2 months normalized neutrophil counts in 16/18 patients with improved marrow maturation. Maintenance therapy sustained neutrophil increases and improved reticulocyte counts in most patients, with decreased infectious episodes. Four patients progressed to AML after 6-16 months of G-CSF therapy.

Conclusions:

  • Both G-CSF and GM-CSF can improve hematologic parameters in MDS patients, including neutrophil counts and red blood cell transfusion requirements.
  • While G-CSF demonstrates sustained efficacy in neutrophil normalization and reduced infections, a subset of patients treated with either G-CSF or GM-CSF may progress to acute myeloid leukemia (AML).
  • Careful patient selection and monitoring are crucial, particularly for those with higher blast counts, due to the risk of AML transformation.

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