While patients may not initially experience symptoms of their condition, the ineffective blood cell production that characterizes MDS eventually results in uncomfortable and life-threatening side effects. Low red blood cell production results in anemia and associated fatigue; low white blood cell production causes neutropenia, which increases the risk of infection; and low platelets causes thrombocytopenia and an increased risk of bleeding.
Patients who are at low risk of progressing to acute myeloid leukemia may not have any of these symptoms or they may require supportive treatments in order to prevent the complications associated with low blood counts. Several strategies exist for improving blood cell production and decreasing complications:
Anemia: Patients who are not producing enough red blood cells are anemic and may experience fatigue, difficulty conducting normal activities or tasks, and/or worsening of heart and lung problems, depending on the severity of their anemia.
Anemia can be treated with a red blood cell transfusion or by increasing red blood cell production with a naturally produced protein called erythropoietin. Erythropoietin stimulates the bone marrow to produce more red blood cells. When administered to some patients, it reduces the severity of anemia and can prevent red blood cell transfusions.
Currently, there are two forms of erythropoietin available, epoetin alfa (Epogen® or Procrit®) and darbepoetin alfa (Aranesp®). Aranesp is a longer acting form that can be administered less frequently.
Three clinical trials have shown that Aranesp improves anemia in patients with low or intermediate-risk MDS. 1, 2, 3 Study results indicate that one-third to nearly half of patients with intermediate-risk MDS who were treated with Aranesp experienced a significant increase in red blood cells. Patients who had an increase in red blood cells also experienced an improvement in quality of life.
Patients with MDS appear to benefit fromprolonged treatment with erythropoietin. Results from clinical trials show that patients have a significantly improved response to treatment after more than two years of therapy (26 months) compared to responses after one year of treatment. 4
To learn more, go to Management of Anemia.
Neutropenia: Neutropenia, or a low white blood count, increases a patient’s risk of infection. The white blood count can be temporarily increased with Neupogen®, which stimulates the growth and production of white blood cells. This can decrease the chance of additional or worsening infections. To learn more, go to Management of Neutropenia.
Thrombocytopenia: Thrombocytopenia, or low platelet counts, increases a patient’s risk of bleeding. To learn more, go to Management of Low Platelet Counts.
Mannone L, Gardin C, Quarre MC, et al. For the Groupe Francais des Myelodysplasies (GFM). High response rate to Darbopoetin alfa in “low risk” MDS: results of a phase II study. Proceedings of the American Society of Hematology. Blood
. 2004;104(suppl 1):24a, abstract 69.
2 Stasi R, Abruzzese E, Lanzetta G, Terzoli E, Amadori S. Darbepoetin alfa for the treatment of anemic patients with low- and intermediate-1-risk myelodysplastic syndromes. Annals of Oncology. 2005; 16: 1921-1927.
3 Musto P, Lanza F, Balleari E, et al. Darbopoetin alfa for the treatment of anaemia in low-intermediate risk myelodysplastic syndromes. British Journal of Haematology. 2005;128:204-209.
4 Terpos E, Mougiou A, Kouraklis A, et al. Prolonged administration of erythropoietin increases erythroid response in myelodysplastic syndromes: a phase II trial in 281 patients. British Journal of Haematology. 2002;118:174-180.