Erythropoietin (EPO)+/- Filgrastim (G-CSF) vs. Supportive Therapy Alone for Patients With Myelodysplastic Syndromes
Phase III Evaluation of EPO With or Without G-CSF Versus Supportive Therapy Alone in the Treatment of Myelodysplastic Syndromes
3 other identifiers
interventional
118
1 country
28
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Mar 1998
Longer than P75 for phase_3
28 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 4, 1998
CompletedFirst Submitted
Initial submission to the registry
November 1, 1999
CompletedFirst Posted
Study publicly available on registry
January 27, 2003
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2008
CompletedResults Posted
Study results publicly available
December 6, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2014
CompletedJune 29, 2023
June 1, 2023
10.4 years
November 1, 1999
September 11, 2013
June 14, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Proportion of Patients Free of Transfusion at 4 Months
Whether a patient required transfusion or not at 4 months was recorded.
Assessed at 4 months
Secondary Outcomes (2)
Overall Survival
Assessed every 3 months for 2 years, every 6 months for 3 subsequent years, and annually thereafter
Quality of Life- Total Functional Assessment of Cancer Therapy - General (FACT-G) Score at 4 Months
Assessed at 4 months
Study Arms (2)
Supportive Care
ACTIVE COMPARATORPatients received red cell and platelet transfusions for symptoms or to maintain hematocrit at or above 25% by volume. Patients who required transfusion support for symptomatic anemia prior to entering the study and who developed an increase in their transfusion requirement of \>= 50% shall cross over to the Erythropoietin treatment arm, after at least four months on the supportive therapy arm.
Erythropoietin
EXPERIMENTALErythropoietin was administered at 150 units/kg subcutaneously every day. If patients stopped responding, they were subsequently treated with Erythropoietin (150 units/kg) and filgrastim and then Erythropoietin (300 units/kg) and filgrastim.
Interventions
Administered at 150 units/kg subcutaneously every day. Rotating sites should be used. The dose should be rounded off to the nearest 1000 U. The dose should be adjusted based on hematocrit.
G-CSF should start at a dose of 1 mcg/kg per day or 2.5 mcg/kg three times a week subcutaneously. Rotating sites should be used The dose should be rounded off to the nearest 10 mcg.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (28)
CCOP - Colorado Cancer Research Program, Incorporated
Denver, Colorado, 80224, United States
Veterans Affairs Medical Center - Lakeside Chicago
Chicago, Illinois, 60611-4494, United States
Robert H. Lurie Comprehensive Cancer Center at Northwestern University
Chicago, Illinois, 60611, United States
CCOP - Illinois Oncology Research Association
Peoria, Illinois, 61602, United States
CCOP - Carle Cancer Center
Urbana, Illinois, 61801, United States
CCOP - Cedar Rapids Oncology Project
Cedar Rapids, Iowa, 52403-1206, United States
CCOP - Iowa Oncology Research Association
Des Moines, Iowa, 50309-1016, United States
MBCCOP - LSU Health Sciences Center
New Orleans, Louisiana, 70112, United States
Tufts - New England Medical Center
Boston, Massachusetts, 02111, United States
CCOP - Michigan Cancer Research Consortium
Ann Arbor, Michigan, 48106, United States
CCOP - Kalamazoo
Kalamazoo, Michigan, 49007-3731, United States
West Michigan Cancer Center
Kalamazoo, Michigan, 49007, United States
CCOP - Metro-Minnesota
Saint Louis Park, Minnesota, 55416, United States
CCOP - Missouri Valley Cancer Consortium
Omaha, Nebraska, 68106, United States
CCOP - Southern Nevada Cancer Research Foundation
Las Vegas, Nevada, 89106, United States
Veterans Affairs Medical Center - East Orange
East Orange, New Jersey, 07019, United States
CCOP - Northern New Jersey
Hackensack, New Jersey, 07601, United States
Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903, United States
NYU School of Medicine's Kaplan Comprehensive Cancer Center
New York, New York, 10016, United States
CCOP - Merit Care Hospital
Fargo, North Dakota, 58122, United States
MetroHealth's Cancer Care Center at MetroHealth Medical Center
Cleveland, Ohio, 44109, United States
CCOP - Columbus
Columbus, Ohio, 43206, United States
CCOP - Geisinger Clinic and Medical Center
Danville, Pennsylvania, 17822-2001, United States
CCOP - Sioux Community Cancer Consortium
Sioux Falls, South Dakota, 57104, United States
CCOP - Scott and White Hospital
Temple, Texas, 76508, United States
CCOP - St. Vincent Hospital Cancer Center, Green Bay
Green Bay, Wisconsin, 54307-3453, United States
CCOP - Marshfield Clinic Research Foundation
Marshfield, Wisconsin, 54449, United States
Medical College of Wisconsin Cancer Center
Milwaukee, Wisconsin, 53226-3596, United States
Related Publications (3)
Cherry AM, Brockman SR, Paternoster SF, Hicks GA, Neuberg D, Higgins RR, Bennett JM, Greenberg PL, Miller K, Tallman MS, Rowe J, Dewald GW. Comparison of interphase FISH and metaphase cytogenetics to study myelodysplastic syndrome: an Eastern Cooperative Oncology Group (ECOG) study. Leuk Res. 2003 Dec;27(12):1085-90. doi: 10.1016/s0145-2126(03)00104-8.
PMID: 12921944BACKGROUNDGreenberg PL, Sun Z, Miller KB, Bennett JM, Tallman MS, Dewald G, Paietta E, van der Jagt R, Houston J, Thomas ML, Cella D, Rowe JM. Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996). Blood. 2009 Sep 17;114(12):2393-400. doi: 10.1182/blood-2009-03-211797. Epub 2009 Jun 29.
PMID: 19564636RESULTMiller KB, Kim HT, Greenberg P, et al.: Phase III prospective randomized trial of EPO with or without G-CSF versus supportive therapy alone in the treatment of myelodysplastic syndromes (MDS): results of the ECOG- CLSG trial (E1996). [Abstract] Blood 104 (11): A-70, 2004.
RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Statistician
- Organization
- ECOG Statistical Office
Study Officials
- STUDY CHAIR
Kenneth B. Miller, MD
Beth Israel Deaconess Medical Center
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 1, 1999
First Posted
January 27, 2003
Study Start
March 4, 1998
Primary Completion
August 1, 2008
Study Completion
May 1, 2014
Last Updated
June 29, 2023
Results First Posted
December 6, 2013
Record last verified: 2023-06