Title

Comparing Three Standard Treatments in Older Adult Patients with Acute Non-Lymphocytic Leukemia and Studying the Effect of GM-CSF to See Whether It May Improve Initial Response to Chemotherapy (A Phase III Trial)

Sponsor

Eastern Cooperative Oncology Group www.ecog.org through the NCI-sponsored Cancer Cooperative Group Program

Purpose of the Study

To determine whether one of three chemotherapy drugs (daunorubicine, idarubicin and mitoxantrone) is superior when used in the treatment of acute non-lymphocytic leukemia in adults over age 55 and to see whether receiving GM-CSF, a stimulator of blood cell production in the bone marrow, before the start of treatment can improve a patient's response to chemotherapy.

Results

No difference was observed in the complete response rate (the disappearance of signs of cancer in response to treatment) among the three drugs studied. Patients who received GM-CSF before the chemotherapy did not show improved response to therapy compared with placebo. Patients who did not participate in the GM-CSF part of the study started chemotherapy several days sooner than the GM-CSF group of participants and showed an improved overall response (50 percent versus 38 percent). Delaying therapy in older adults may lead to lower complete remission rates.

Start Date

April 1993

Stop Date

February 1997

Number of Participating Patients

362 in Induction Therapy Study

245 in GM-CSF Study

Eligibility Requirements

Patients who participated in this study were older than 55 years, had a confirmed diagnosis of acute myeloid leukemia, had no previous chemotherapy treatment for a malignancy, and no severe cardiac disease.

Study Design

Two days prior to the start of treatment, 245 patients received either GM-CSF or placebo. Then 362 patients received an intravenous infusion of cytarabine continuously for seven days, together three days of intravenous treatment with one of three drugs, determined by randomization. Participants in all randomized groups, who achieved a complete response, then received 12 identical treatments with high-dose cytarabine.

Side Effects

The most common side effects for the drugs used in the study were an increased risk of infection, nausea, vomiting, mouth ulcers, liver damage, anemia and temporary hair loss. Side effects related to the GM-CSF were mild and short-lived. Some patients experienced fever, chills, nausea, vomiting, diarrhea, fatigue, weakness and headache.

References

2192:Rowe JM, Neuberg D, Friedenberg WR, Bennett JM, Paietta EM, Makary AZ, Liesveld J, Abboud CN, Dewald G, Hayes F, Tallman MS, Wiernik P. A Phase 3 Study of Three Induction Regimens and of Priming with GM-CSF in Older Adults with Acute Myeloid Leukemia: A Trial of the Eastern Cooperative Oncology Group. Blood 2004 Jan 15;103(2):479-85. (E3993) [Manuscript published 1/15/2004]

National Cancer Institute Patient Summary

Not Available.

Reviewed by

Jacob Rowe, MD, Cheryl Rutledge, BSN