Erythropoietin and granulocyte-colony stimulating factor treatment associated with improved survival in myelodysplastic syndrome

• Purpose To assess the effect of erythropoietin (EPO) plus granulocyte-colony stimulating factor (G-CSF) treatment on survival and leukemic transformation in myelodysplastic syndrome (MDS).Patients and Methods We compared the long-term outcome of patients with MDS treated with EPO plus G-CSF (n = 121) with untreated patients (n = 237) with MDS using multivariate Cox regression with delayed entry, for the first time adjusting for all major prognostic variables (WHO classification, karyotype, cytopenias, level of transfusion-need, age, and sex).Results The erythroid response rate to EPO plus G-CSF was 39%, and the median response duration 23 months (range, 3 to 116+). In the multivariate analysis, treatment was associated with improved overall survival (hazard ratio, 0.61; 95% CI, 0.44 to 0.83; P = .002). Interestingly, this positive association was primarily observed in patients requiring fewer than 2 units of RBCs per month. Treatment was not linked to the rate of acute myeloid leukemia in any defined subgroup, including patients with an increase of marrow blasts or an unfavorable karyotype.Conclusion The inherent risk of leukemic evolution in MDS makes the current investigation highly relevant, in light of the recent reports of potential negative effects of EPO treatment on outcome in patients with cancer. We conclude that treatment of anemia in MDS with EPO plus G-CSF may have a positive impact on outcome in patients with no or low transfusion need, while not affecting the risk of leukemic transformation.

Ämnesord

MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Cancer och onkologi (hsv//swe)

MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Cancer and Oncology (hsv//eng)

MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Kirurgi (hsv//swe)

MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Surgery (hsv//eng)

Nyckelord

Aged

Anemia/drug therapy/etiology

Blood Transfusion

Cell Transformation; Neoplastic/*drug effects

Clinical Trials; Phase II as Topic

Erythropoietin/*therapeutic use

Female

Granulocyte-Macrophage Colony-Stimulating Factor/*therapeutic use

Humans

Kaplan-Meiers Estimate

Leukemia; Myeloid; Acute/epidemiology

Male

Middle Aged

Myelodysplastic Syndromes/complications/*drug therapy/mortality

Precancerous Conditions/*drug therapy

MEDICINE

MEDICIN

Oncology

Onkologi

Surgery

Kirurgi

Onkologi

Oncology

Publikations- och innehållstyp

ref (ämneskategori)

art (ämneskategori)