Azacitidine Plus Phenylbutyrate in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

• Status: Completed
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

RATIONALE: Azacitidine plus phenylbutyrate may help leukemia cells develop into normal white blood cells.

PURPOSE: Phase I trial to study the effectiveness of combining azacitidine and phenylbutyrate in treating patients who have acute myeloid leukemia or myelodysplastic syndrome.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center; Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
• Collaborator: National Cancer Institute (NCI)
• Treatments: 4-phenylbutyric acid; Azacitidine

Criteria

DISEASE CHARACTERISTICS:

• Histologically or cytologically confirmed myelodysplastic syndrome (MDS) indicating one of the following:

• Refractory anemia (RA)

• Primary refractory leukopenia or thrombocytopenia with MDS morphology

• RA with excess blasts (RAEB)

• RA with ringed sideroblasts (RARS)

• Chronic myelomonocytic leukemia

• RAEB in transformation

• RA or RARS must have at least one of the following:

• Absolute neutrophil count less than 1,000/mm^3

• Untransfused hemoglobin less than 8 g/dL

• Platelet count less than 20,000/mm^3

• Anemia

• Thrombocytopenia requiring transfusion

• High risk chromosomal abnormalities

• Any stage of MDS allowed including:

• Previously untreated MDS

• Refractory MDS allowed if failure to achieve remission following prior intensive chemotherapy of at least 1 month ago

• Relapsed, refractory, or untreated acute myeloid leukemia (AML) with the following:

• WBC less than 30,000/mm^3

• Stable for at least 2 weeks

• Unlikely to require cytotoxic therapy during study

• Untreated AML with poor risk factors for response to standard therapy including:

• Greater than 60 years old

• AML occurs in setting of antecedent hematologic disorder

• High risk chromosomes (e.g., abnormalities of chromosome 5 or 7 or complex cytogenetic abnormalities)

• Medical conditions that preclude cytotoxic chemotherapy as primary therapy

• Refusal of cytotoxic chemotherapy allowed

• No clinical evidence of CNS leukostasis or CNS leukemia

PATIENT CHARACTERISTICS:

Age:

• 18 and over

Performance status:

• Zubrod 0-2

Life expectancy:

• Not specified

Hematopoietic:

• See Disease Characteristics

• Hemoglobin at least 8 g/dL (transfusion allowed)

Hepatic:

• Bilirubin less than 2.0 mg/dL (unless due to hemolysis or Gilbert's disease)

Renal:

• Creatinine less than 2.0 mg/dL

Cardiovascular:

• No disseminated intravascular coagulation

Pulmonary:

• No pulmonary leukostasis

Other:

• No active infection

• Not pregnant or nursing

• Negative pregnancy test

• Fertile patients must use effective contraception 2 weeks prior, during and 3 months after study

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• At least 3 weeks since prior biologic therapy including colony stimulating factors and recovered

Chemotherapy:

• See Disease Characteristics

• At least 3 weeks since prior chemotherapy and recovered

Endocrine therapy:

• At least 3 weeks since prior hormonal therapy and recovered

Radiotherapy:

• At least 3 weeks since prior radiotherapy and recovered

Surgery:

• Not specified