Erythropoietin (EPO)+/- Filgrastim (G-CSF) vs. Supportive Therapy Alone for Patients With Myelodysplastic Syndromes



Status:Completed
Conditions:Blood Cancer, Blood Cancer, Anemia
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:18 - Any
Updated:4/21/2016
Start Date:December 1997
End Date:May 2014

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Phase III Evaluation of EPO With or Without G-CSF Versus Supportive Therapy Alone in the Treatment of Myelodysplastic Syndromes

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.

OBJECTIVES:

- Compare the benefit of erythropoietin 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.

- Evaluate whether adding filgrastim (G-CSF) or increasing the erythropoietin dose will
reduce the transfusion requirement in patients who do not respond to erythropoietin
alone.

- To compare the benefit of erythropoietin versus supportive care alone on quality of
life (QOL) in persons with myelodysplastic syndromes.

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
erythropoietin treatment (yes vs no), and erythropoietin 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 erythropoietin alone.

- Arm II (Erythropoietin): Patients receive erythropoietin subcutaneously (SC) or
intravenously (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 erythropoietin SC daily for up to 6 months. Patients with no response
to G-CSF and lower-dose erythropoietin may proceed to a higher dose of erythropoietin.

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.

ACTUAL ACCRUAL: A total of 118 patients were accrued for this study.

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.
We found this trial at
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Cedar Rapids, Iowa 52403
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675 N Saint Clair St # 21-100
Chicago, Illinois 60611
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Robert H. Lurie Comprehensive Cancer Center at Northwestern University The cancer center was first established...
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200 North Park Street
Kalamazoo, Michigan 49007
(269) 382-2500
West Michigan Cancer Center In 1994, Borgess Health Alliance and Bronson Healthcare Group opened the...
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5301 East Huron River Drive
Ann Arbor, Michigan 48106
1.877.590.5995
CCOP - Michigan Cancer Research Consortium The Community Clinical Oncology Program (CCOP) is a comprehensive...
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Boston, Massachusetts
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1335 Dublin Road, Suite 124A
Columbus, Ohio 43215
(614) 488-2118
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Danville, Pennsylvania 17822
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300 East Locust St., Ste 350
Des Moines, Iowa 50309
(515) 244-7586
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East Orange, New Jersey 07018
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Fargo, North Dakota 58122
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Hackensack, New Jersey 07601
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Kalamazoo, Michigan 49007
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Marshfield, Wisconsin 54449
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8800 W. Doyne Avenue
Milwaukee, Wisconsin 53226
(414) 805-6840
Medical College of Wisconsin Cancer Center Cancer touches everyone in our community, and for many,...
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New Brunswick, New Jersey 08901
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533 Bolivar Street, Room 420
New Orleans, Louisiana 70112
(504) 568-3435
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Saint Louis Park, Minnesota 55416
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Sioux Falls, South Dakota 57104
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Temple, Texas 76508
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509 W. University Avenue
Urbana, Illinois 61801
(217) 383-3516
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