Epoetin Alfa With or Without Filgrastim Compared With Blood Transfusions in Treating Patients With Myelodysplastic Syndrome
Recruitment status was Active, not recruiting
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Purpose
RATIONALE: Epoetin alfa and colony-stimulating factors such as filgrastim stimulate the production of blood cells. It is not yet known whether epoetin alfa 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 epoetin alfa 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.
| Condition | Intervention | Phase |
|---|---|---|
|
Anemia Myelodysplastic Syndromes |
Biological: epoetin alfa Biological: filgrastim Procedure: quality-of-life assessment |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Primary Purpose: Treatment |
| Official Title: | Phase III Evaluation of EPO With or Without G-CSF Versus Supportive Therapy Alone in the Treatment of Myelodysplastic Syndromes |
| Estimated Enrollment: | 139 |
| Study Start Date: | November 1997 |
OBJECTIVES:
- Compare the benefit of epoetin alfa vs standard transfusion support in reducing transfusion requirements in patients with myelodysplastic syndromes.
- Compare the clinical response, disease progression, and survival in patients treated with these regimens.
- Compare the toxicity of these regimens in these patients.
- Determine the effect of pretreatment epoetin alfa levels on the response to epoetin alfa in these patients.
- Evaluate whether adding filgrastim (G-CSF) or increasing the epoetin alfa dose will reduce the transfusion requirement in patients who do not respond to epoetin alfa alone.
- Assess quality of life (QOL) of these patients and determine whether either cross-sectional or longitudinal differences in patients' QOL and fatigue are correlated with the use of the growth factors.
OUTLINE: This is a randomized, controlled, multicenter, cross-over study. Patients are stratified according to morphologic subtype (refractory anemia [RA] vs RA with ringed sideroblasts vs RA with excess blasts), transfusion requirement (yes vs no), prior epoetin alfa treatment (yes vs no), and epoetin alfa level (at least 200 mU/mL vs less than 200 mU/mL). Patients are randomized to one of two treatment arms.
- Arm I (standard transfusion support): Patients receive red cell and platelet transfusions for symptoms or to maintain hematocrit level of 25% or above. Patients undergo bone marrow aspirate and biopsy at 4 months and then every year until development of acute leukemia or completion of study. Patients with progressive disease may cross over to arm II after at least 4 months on study and up to 1 year from the time of randomization. Patients who cross over receive epoetin alfa alone.
- Arm II (epoetin alfa support): Patients receive epoetin alfa subcutaneously (SC) or IV daily. Patients undergo bone marrow aspirate and biopsy as in arm I. Treatment continues daily for a maximum of 1 year.
Patients with stable or progressive disease at day 120 receive filgrastim (G-CSF) SC daily or 3 days a week and epoetin alfa SC daily for up to 6 months. Patients with no response to G-CSF and lower-dose epoetin alfa may proceed to a higher dose of epoetin alfa.
Quality of life is assessed at baseline, every 4 months during study, and at study completion.
Patients are followed every 4 months for 2 years, every 6 months for 3 years, and then annually for 5 years.
PROJECTED ACCRUAL: A total of 139 patients will be accrued for this study within 3.6 years.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Histologically proven myelodysplastic syndromes
- 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
- No RAEB in transformation
No chronic myelomonocytic leukemia
- Secondary myelodysplastic syndromes allowed
- No splenomegaly greater than 6 cm below the left costal margin or greater than 3 times normal size
PATIENT CHARACTERISTICS:
Age:
- 18 and over
Performance status:
- ECOG 0-3
Life expectancy:
- Not specified
Hematopoietic:
- See Disease Characteristics
- Platelet count greater than 30,000/mm^3 (without platelet transfusions)
- Hematocrit less than 30% (pretransfusion)
Hepatic:
- Bilirubin less than 3 mg/dL
Renal:
- BUN less than 40 mg/dL OR
- Creatinine less than 2.0 mg/dL
Cardiovascular:
- No uncontrolled hypertension
Other:
- No sensitivity to E. coli-derived proteins
- No sensitivity to epoetin alfa or any of its components (e.g., human albumin)
No 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
- No active infection or bleeding
- No other uncontrolled malignancy
- Not pregnant or nursing
- Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- 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 epoetin alfa
- At least 2 months since prior recombinant growth factor
Chemotherapy:
- At least 2 months since prior chemotherapy for other malignancy or autoimmune disease
Endocrine therapy:
- At least 2 weeks since prior androgens or steroids for treatment of myelodysplastic syndromes
Radiotherapy:
- Not specified
Surgery:
- Not specified
Contacts and Locations
Show 28 Study Locations| Study Chair: | Kenneth B. Miller, MD | Beth Israel Deaconess Medical Center |
More Information
Additional Information:
Publications:
| ClinicalTrials.gov Identifier: | NCT00003138 History of Changes |
| Other Study ID Numbers: | CDR0000065907, ECOG-1996 |
| Study First Received: | November 1, 1999 |
| Last Updated: | March 8, 2012 |
| Health Authority: | United States: Federal Government |
Keywords provided by Eastern Cooperative Oncology Group:
|
anemia refractory anemia refractory anemia with ringed sideroblasts refractory anemia with excess blasts |
de novo myelodysplastic syndromes previously treated myelodysplastic syndromes secondary myelodysplastic syndromes |
Additional relevant MeSH terms:
|
Anemia Myelodysplastic Syndromes Preleukemia Hematologic Diseases Bone Marrow Diseases Precancerous Conditions Neoplasms Epoetin Alfa |
Lenograstim Hematinics Hematologic Agents Therapeutic Uses Pharmacologic Actions Adjuvants, Immunologic Immunologic Factors Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on June 17, 2013