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A Pilot Study of High Dose Busulfan Combined With IL2/GM-CSF Activated Autologous/Syngeneic PBSC, Sequential IL2/GM-CSF Therapy and Alpha Interferon Maintenance Therapy as Treatment of CML

Phase 2
70 Years
Not Enrolling

Thank you

Trial Information

A Pilot Study of High Dose Busulfan Combined With IL2/GM-CSF Activated Autologous/Syngeneic PBSC, Sequential IL2/GM-CSF Therapy and Alpha Interferon Maintenance Therapy as Treatment of CML

OBJECTIVES: I. Determine the toxicity of high-dose busulfan followed by interleukin-2 (IL-2)
and sargramostim (GM-CSF) activated autologous/syngeneic peripheral blood stem cell (PBSC)
transplantation, sequential IL-2 and GM-CSF therapy, and interferon alfa in patients with
chronic myelogenous leukemia. II. Determine engraftment potential of IL-2/GM-CSF activated
PBSC followed by sequential IL-2/GM-CSF therapy in this patient population. III. Assess the
time to cytogenetic and/or morphologic relapse, overall event-free survival, and overall
survival in these patients treated with this regimen.

OUTLINE: Autologous peripheral blood stem cells (PBSC) are harvested and activated with
interleukin-2 (IL-2) and sargramostim (GM-CSF) on another protocol. Patients receive oral
busulfan every 6 hours on days -6 to -3 for a total of 16 doses. IL-2 and GM-CSF-activated
PBSC are reinfused on day 0. Beginning 4 hours after PBSC infusion, patients receive IL-2 IV
continuously for 5 days followed by 2 days of rest for 4 weeks. In addition, GM-CSF is
administered subcutaneously (SC) every Monday, Wednesday, and Friday for 4 weeks. Upon
hematologic recovery, but no earlier than 2 weeks after IL-2 and GM-CSF, patients receive
interferon alfa SC 3 times weekly until clear evidence of disease progression. Patients are
followed every 6 months for 3 years and then annually thereafter.

PROJECTED ACCRUAL: A total of 25 patients will be accrued for this study within 2-3 years.

Inclusion Criteria

DISEASE CHARACTERISTICS: Chronic myelogenous leukemia with previously stored CD34 cells
from FHCRC-928.00 Chronic phase: No evidence of a major response after 6-month course of
interferon alfa OR Initially achieved a major cytogenetic response but subsequently failed
interferon alfa OR Accelerated phase: At least 1 month after collection of peripheral
blood stem cells No blast crisis No CNS involvement Ineligible for or refused allogeneic
conventional or minitransplant protocol

PATIENT CHARACTERISTICS: Age: 70 and under Performance status: Karnofsky 80-100% Life
expectancy: Not specified Hematopoietic: Not specified Hepatic: Bilirubin no greater than
2.0 mg/dL (unless history of Gilbert's disease) AST or ALT no greater than 2.5 times upper
limit of normal No cirrhosis Hepatitis B and C negative Renal: Creatinine no greater than
2.0 mg/dL Cardiovascular: No myocardial infarction within past 12 months No unstable
angina, poorly controlled arrhythmias, or hypertension LVEF greater than 50% Pulmonary:
DLCO at least 50% Alveolar arterial gradient less than 30 at sea level Other: Not pregnant
or nursing Negative pregnancy test Fertile patients must use effective contraception HIV
negative No active infection requiring systemic antibiotics No known allergy to gentamicin
or murine or E. coli proteins or documented prior anaphylactic reaction to sargramostim
(GM-CSF) or interleukin-2

PRIOR CONCURRENT THERAPY: Biologic therapy: See Disease Characteristics Chemotherapy: Not
specified Endocrine therapy: No concurrent ongoing steroids Radiotherapy: Not specified
Surgery: Not specified

Type of Study:


Study Design:

Primary Purpose: Treatment

Principal Investigator

Leona A. Holmberg, MD, PhD

Investigator Role:

Study Chair

Investigator Affiliation:

Fred Hutchinson Cancer Research Center


United States: Federal Government

Study ID:




Start Date:

January 2000

Completion Date:

October 2001

Related Keywords:

  • Leukemia
  • relapsing chronic myelogenous leukemia
  • chronic phase chronic myelogenous leukemia
  • accelerated phase chronic myelogenous leukemia
  • Leukemia
  • Leukemia, Myeloid
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive



Fred Hutchinson Cancer Research Center Seattle, Washington  98109
University of Colorado Cancer Center Denver, Colorado  80262