prospective, multicenter, open label and single-arm study of darbepoetin alfa for anemia in myelodisplastic syndrome patients.

Conditions: Myelodysplastic Syndrome

Summary: this is an open-label, single-arm, multicentre, prospective study of darbepoetin alfa to treat anaemia in patients with low and intermediate-1 ipss risk mds. the study will consist of a 14-day screening period followed by a maximum 24-week treatment period and a final visit.

this is an open-label, single-arm, multicentre, prospective study of darbepoetin alfa to treat anaemia in patients with low and intermediate-1 ipss risk mds. the study will consist of a 14-day screening period followed by a maximum 24-week treatment period and a final visit. darbepoetin alfa will be initiated at a dose of 300 mcg qw sc over a period of 8 weeks. after 8 weeks, erythroid response will be evaluated, and treatment algorithm adapted to it. the study treatment period will last for a maximum of 24 weeks. the treatment will end at the start of week 24. if the scheduled 24-week treatment period is not completed, it will end during the week of the last administration of the study drug. the follow-up period will last for a minimum of 4 weeks and a maximum of 8 weeks after the last dose of darbepoetin alfa. subjects will be stratified at enrolment according to ipss (low risk versus intermediate-1 risk).

Eligibility criteria:
inclusion criteria: * age ³ 18 years * low or intermediate-1 risk mds according to ipss, and fab classification of ra, rars, or raeb with blasts £ 10% * predictive variables of good response (serum erythropoietin levels \< 500 iu/l and transfusion requirements \< 2 packed rbc/month over the preceding 2 months) * anaemia (hb £ 10 g/dl), confirmed in the 14 days before day 1 of the study * life expectancy of at least 6 months * ecog performance status score of 0, 1, or 2 * subject must sign and date the informed consent (approved by a clinical research ethics committee - crec), before any study-specific procedure is performed exclusion criteria: * known history of convulsive disorders * poorly controlled hypertension (diastolic blood pressure \> 100 mmhg) at screening * inadequate liver function (total bilirubin \> two times the upper limit of the normal range (uln), and liver enzymes (alt, ast) \> two times uln) * inadequate renal function (serum creatinine concentration \> 2 mg/dl) * ferritin \< 100 ng/ml or transferrin saturation index (tsi) \< 16%; vitamin b12 deficiency (\< 200 pg/ml) or folate deficiency (\< 2 ng/ml) * clinically-relevant haemorrhages * haemolytic anaemia * cardiac condition: uncontrolled angina, congestive heart failure, or uncontrolled cardiac arrhythmia * clinically significant systemic infection or chronic inflammatory disease present at time of screening * any concomitant therapy used to treat mds (including other growth factors than those described as part of this protocol, chemotherapy, antibody-based cancer treatment, hormonal therapy, interferon, and interleukins) * treatment with rhuepo or darbepoetin alfa over the 4 weeks prior to day 1 of the study * more than 2 rbc transfusions over the 28 days prior to day 1 of the study * pregnant or breast feeding women * subjects of childbearing-potential who do not take adequate contraceptive measures, in the opinion of the investigator * known hypersensitivity to any mammal-derived recombinant product

Keywords: myelodysplastic syndrome