Phase III Evaluation of EPO With or Without G-CSF Versus Supportive Therapy Alone in the Treatment of Myelodysplastic Syndromes
- 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
- 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
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.
Allocation: Randomized, Primary Purpose: Treatment
Kenneth B. Miller, MD
Beth Israel Deaconess Medical Center
United States: Federal Government
|CCOP - Missouri Valley Cancer Consortium||Omaha, Nebraska 68131|
|CCOP - Southern Nevada Cancer Research Foundation||Las Vegas, Nevada 89106|
|CCOP - Illinois Oncology Research Association||Peoria, Illinois 61602|
|CCOP - Carle Cancer Center||Urbana, Illinois 61801|
|CCOP - Iowa Oncology Research Association||Des Moines, Iowa 50309-1016|
|CCOP - Kalamazoo||Kalamazoo, Michigan 49007-3731|
|CCOP - Metro-Minnesota||Saint Louis Park, Minnesota 55416|
|Veterans Affairs Medical Center - East Orange||East Orange, New Jersey 07018-1095|
|CCOP - Northern New Jersey||Hackensack, New Jersey 07601|
|CCOP - Michigan Cancer Research Consortium||Ann Arbor, Michigan 48106|
|West Michigan Cancer Center||Kalamazoo, Michigan 49007-3731|
|Medical College of Wisconsin Cancer Center||Milwaukee, Wisconsin 53226|
|CCOP - Cedar Rapids Oncology Project||Cedar Rapids, Iowa 52403-1206|
|CCOP - Merit Care Hospital||Fargo, North Dakota 58122|
|CCOP - Sioux Community Cancer Consortium||Sioux Falls, South Dakota 57105-1080|
|NYU School of Medicine's Kaplan Comprehensive Cancer Center||New York, New York 10016|
|CCOP - Columbus||Columbus, Ohio 43206|
|MetroHealth's Cancer Care Center at MetroHealth Medical Center||Cleveland, Ohio 44106|
|CCOP - Geisinger Clinic and Medical Center||Danville, Pennsylvania 17822-2001|
|CCOP - Scott and White Hospital||Temple, Texas 76508|
|Veterans Affairs Medical Center - Lakeside Chicago||Chicago, Illinois 60611|
|CCOP - St. Vincent Hospital Cancer Center, Green Bay||Green Bay, Wisconsin 54301|
|MBCCOP - LSU Health Sciences Center||New Orleans, Louisiana 70112|
|Cancer Institute of New Jersey||New Brunswick, New Jersey 08901|
|CCOP - Colorado Cancer Research Program, Incorporated||Denver, Colorado 80224|
|Tufts - New England Medical Center||Boston, Massachusetts 02111|
|CCOP - Marshfield Clinic Research Foundation||Marshfield, Wisconsin 54449|
|Robert H. Lurie Comprehensive Cancer Center at Northwestern University||Chicago, Illinois 60611|