Phase 3
Completed N=118
Erythropoietin (EPO)+/- Filgrastim (G-CSF) vs. Supportive Therapy Alone for Patients With Myelodysplastic Syndromes
Source: ClinicalTrials.gov NCT00003138 ↗Enrolled (actual)
118
Serious AEs
16.8%
Results posted
Dec 2013
Primary outcomePrimary: Proportion of Patients Free of Transfusion at 4 Months — 0.459; 0.714 Proportion of patients
Summary
RATIONALE: Erythropoietin and colony-stimulating factors such as filgrastim stimulate the production of blood cells. It is not yet known whether erythropoietin with or without filgrastim is more effective than standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.
PURPOSE: Randomized phase III trial to compare the effectiveness of erythropoietin with or without filgrastim with that of standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Proportion of Patients Free of Transfusion at 4 Months |
0.459; 0.714 | — |
| SECONDARY Overall Survival |
31; 37 | — |
| SECONDARY Quality of Life- Total Functional Assessment of Cancer Therapy - General (FACT-G) Score at 4 Months |
84.1; 84.33 | — |
Eligibility Criteria
Inclusion Criteria
- At least 18 years of age
- Diagnosis of a myelodysplastic syndrome
- Refractory anemia (RA)
- RA with ringed sideroblasts
- RA with excess blasts (RAEB). RAEB patients must have a bone marrow blast count of less than 20% and less than 5% blast forms on peripheral blood
- Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 3
- Platelet count greater than 30, 000/mm^3 (without platelet transfusions)
- Hematocrit less than 30% (pretransfusion)
- Bilirubin less than 3 mg/dL
- Blood urea nitrogen (BUN) less than 40 mg/dL or Creatinine less than 2.0 mg/dL
- Prior epoetin alfa allowed provided dosage was less than 30,000 units per week for less than 1 month duration
- At least 1 month since prior erythropoietin
- At least 2 months since prior recombinant growth factor
- At least 2 months since prior chemotherapy for other malignancy or autoimmune disease
- At least 2 weeks since prior androgen or steroids for treatment of myelodysplastic syndromes
Exclusion Criteria
- RAEB in transformation
- Chronic myelomonocytic leukemia
- Splenomegaly greater than 6 cm below the left costal margin or greater than 3 times normal size
- Uncontrolled hypertension
- Sensitivity to E. coli-derived proteins
- Sensitivity to epoetin alfa or any of its components (e.g., human albumin)
- Documented iron deficiency. If marrow iron stain is not available, the transferrin saturation must be greater than 20% or ferritin greater than 100 ng/dL
- Active infection or bleeding
- Other uncontrolled malignancy
- Pregnant or nursing. Fertile patients must use effective contraception.
Data sourced from ClinicalTrials.gov (NCT00003138). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.