Use of recombinant human erythropoietin in the treatment of myelodysplastic syndromes


Myelodysplastic syndromes (MDSs) are clonal proliferations of multipotent bone marrow stem cells which retain their capacity to differentiate but do so in an inefficient manner, so that mature blood cells are variably reduced (Gallagher et al. 1997; Aul et al. 1998; Vallespí et al. 1998). With time there is a progressive impairment in the capacity of stem cells to differentiate and an increasingly high risk of evolution to overt acute myeloid leukemia. Although the clinical course of these disorders is highly variable from patient to patient, the International Prognostic Scoring System (Greenberg et al. 1997) provides an improved method for evaluating prognosis in individual MDS patients (Sanz et al. 1998).


Myelodysplastic Syndrome International Prognostic Scoring System Serum Erythropoietin Erythroid Response Mature Blood Cell 


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  1. 1.
    Aul C, Bowen DT, Yoshida Y (1998) Pathogenesis, etiology and epidemiology of myelodysplastic syndromes. Haematologica 83: 71–86PubMedGoogle Scholar
  2. 2.
    Bessho M, Jinnai I, Hirashima K et al (1994) Trilineage recovery by combination therapy with recombinant human granulocyte colony-stimulating factor and erythropoietin in patients with aplastic anemia and refractory anemia. Stem Cells 12: 604–615PubMedCrossRefGoogle Scholar
  3. 3.
    Cazzola M, Anderson JE, Ganser A, Hellström-Lindberg E (1998) A patient-oriented approach to treatment of myelodysplastic syndromes. Haematologica 83: 910–935PubMedGoogle Scholar
  4. 4.
    Cazzola M, Guarnone R, Cerani P, Centenara E, Rovati A (1998) Red cell precursor mass as an independent determinant of serum erythropoietin level. Blood 91: 2139–2145PubMedGoogle Scholar
  5. 5.
    Cazzola M, Mercuriali F, Brugnara C (1997) Clinical use of recombinant human erythropoietin outside the setting of uremia. Blood 89: 4248–4267PubMedGoogle Scholar
  6. 6.
    Cazzola M, Ponchio L (1992) Subcutaneous erythropoietin for treatment of refractory anemia in hematologic disorders [letter]. Blood 80: 841–843Google Scholar
  7. 7.
    Gallagher A, Darley RL, Padua R (1997) The molecular basis of myelodysplastic syndromes. Haematologica 82: 191–204PubMedGoogle Scholar
  8. 8.
    Greenberg P, Cox C, LeBeau MM et al (1997) International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 89: 2079–2088PubMedGoogle Scholar
  9. 9.
    Hellström-Lindberg E (1995) Efficacy of erythropoietin in the myelodysplastic syndromes: a meta-analysis of 205 patients from 17 studies. Br J Haematol 89: 67–71PubMedGoogle Scholar
  10. 10.
    Hellström-Lindberg E, Ahlgren T, Beguin Y et al (1998) Treatment of anemia in myelodysplastic syndromes with granulocyte colony-stimulating factor plus erythropoietin: results from a randomized phase II study and long-term follow-up of 71 patients. Blood 92: 68–75PubMedGoogle Scholar
  11. 11.
    Hellström-Lindberg E, Birgegârd G, Carlsson M et al (1993) A combination of granulocyte-colony-stimulating factor and erythropoietin may synergistically improve the anaemia in patients with myelodysplastic syndromes. Leuk Lymphoma 11: 221–228PubMedCrossRefGoogle Scholar
  12. 12.
    Hellström-Lindberg E, Negrin R, Stein R et al (1997) Erythroid response to treatment with G-CSF plus erythropoietin for the anaemia of patients with myelodysplastic syndromes: proposal for a predictive model. Br J Haematol 99: 344–351PubMedCrossRefGoogle Scholar
  13. 13.
    Imamura M, Kobayashi M, Kobayashi S et al (1994) Failure of combination therapy with granulocyte colony-stimulating factor and erythropoietin in myelodysplastic syndromes. Ann Hematol 68: 163–166PubMedCrossRefGoogle Scholar
  14. 14.
    Italian Cooperative Study Group for rHuEpo in Myelodysplastic Syndromes (1998) A randomized double-blind placebo-controlled study with subcutaneous recombinant human erythropoietin in patients with low-risk myelodysplastic syndromes. Br J Haematol 103: 1070–1074CrossRefGoogle Scholar
  15. 15.
    Mantovani L, Lentini G, Hentschel B et al (2000) Treatment of anaemia in myelodysplastic syndromes with prolonged administration of recombinant human granulocyte colony-stimulating factor and erythropoietin. Br J Haematol 109: 367–375PubMedCrossRefGoogle Scholar
  16. 16.
    Musto P, Falcone A, Carotenuto M et al (1994) Granulocyte colony-stimulating factor and erythropoietin for the anemia of myelodysplastic syndromes: A real improvement with respect to erythropoietin alone? Blood 84: 1687–1688PubMedGoogle Scholar
  17. 17.
    Musto P, Sanpaolo G, D’Arena G et al (2001) Adding growth factors or interleukin-3 to erythropoietin has limited effects on anemia of transfusion-dependent patients with myelodysplastic syndromes unresponsive to erythropoietin alone. Haematologica 86: 44–51PubMedGoogle Scholar
  18. 18.
    Negrin RS, Stein R, Doherty K et al (1993) Treatment of the anemia of myelodysplastic syndromes using human granulocyte-CSF in combination with erythropoietin. Blood 82: 737–743PubMedGoogle Scholar
  19. 19.
    Negrin RS, Stein R, Doherty K et al (1996) Maintenance treatment of the anemia of myelodysplastic syndromes with recombinant human granulocyte colony-stimulating factor and erythropoietin: evidence for in vivo synergy. Blood 87: 4076–4081PubMedGoogle Scholar
  20. 20.
    Rose EH, Abels RI, Nelson RA, McCullough DM, Lessin L (1995) The use of rHuEpo in the treatment of anaemia related to myelodysplasia ( MDS ). Br J Haematol 89: 831–837Google Scholar
  21. 21.
    Sanz GF, Sanz MA, Grenberg PL (1998) Prognostic factors and scoring systems in myelodysplastic syndromes. Haematologica 83: 358–368PubMedGoogle Scholar
  22. 22.
    Stenke L, Wallvik J, Celsing F, Hast R (1993) Prediction of response to treatment with human recombinant erythropoietin in myelodysplastic syndromes. Leukemia 7: 1324–1327PubMedGoogle Scholar
  23. 23.
    Vallespí T, Imbert M, Mecucci C, Preudhomme C, Fenaux P (1998) Diagnosis, classification, and cytogenetics of myelodysplastic syndromes. Haematologica 83: 258–275PubMedGoogle Scholar

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© Springer-Verlag/Wien 2002

Authors and Affiliations

  1. 1.Department of HematologyUniversity of Pavia School of Medicine, IRCCS Policlinico S. MatteoPaviaItaly

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