Phase 3
N=1,070
Combination Chemotherapy With or Without Gemtuzumab in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia
Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT00372593 ↗Enrolled (actual)
1,070
Serious AEs
5.8%
Results posted
Jan 2015
Primary outcome: Primary: Event-free Survival at 3 Years — 46.9; 53.1; 50.0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- asparaginase (Drug); cytarabine (Drug); daunorubicin hydrochloride (Drug); etoposide (Drug); gemtuzumab ozogamicin (Drug); mitoxantrone hydrochloride (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Children's Oncology Group
- Primary completion
- Aug 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Event-free Survival at 3 Years |
46.9; 53.1; 50.0 | — |
| PRIMARY Overall Survival at 3 Years |
65.4; 69.4; 50.0 | — |
| SECONDARY Remission Induction Rate After 2 Courses of Induction Therapy |
0.851324; 0.882716; 0.666667 | — |
| SECONDARY Disease-free Survival (DFS) |
56.6; 63.0; 75.0 | — |
| SECONDARY Mortality |
11; 13; 1 | — |
| SECONDARY Time to Marrow Recovery |
30.56; 30.56; 29.17; 28.54; 28.52; 26.6 | — |
| SECONDARY Toxicities, Including Infectious Complications |
482; 477; 5 | — |
Summary
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Monoclonal antibodies, such as gemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Giving combination chemotherapy together with gemtuzumab may kill more cancer cells. It is not yet known whether combination chemotherapy is more effective with or without gemtuzumab in treating patients with newly diagnosed acute myeloid leukemia.
PURPOSE: This randomized phase III trial is studying combination chemotherapy and gemtuzumab to see how well they work compared with combination chemotherapy alone in treating young patients with newly diagnosed acute myeloid leukemia.
Eligibility Criteria
DISEASE CHARACTERISTICS:
- Newly diagnosed acute myeloid leukemia (AML)
- Meets customary criteria for AML with ≥ 20% bone marrow blasts (by WHO classification)
- Patients with < 20% bone marrow blasts and cytopenia or myelodysplastic syndromes (e.g., chronic myelomonocytic leukemia, refractory anemia (RA), RA with excess blasts, RA with ringed sideroblasts) are eligible provided 1 of the following criteria is met:
- Karyotypic abnormality characteristic of de novo AML (t[8;21][q22;q22], inv[16][p13q22], t[16;16][p13;q22], or 11q23 abnormalities)
- Unequivocal presence of megakaryoblasts (by WHO classification)
- Isolated myeloid sarcoma (i.e., myeloblastoma or chloroma) allowed regardless of bone marrow results
- Infants < 1 month of age with progressive disease* are eligible NOTE: *Infants < 1 month of age with AML may be given supportive care until it is clear that the leukemia is not regressing (i.e., the disappearance of peripheral blasts and the normalization of peripheral blood counts)
- Patients with Down syndrome ≥ 4 years of age are eligible
- No juvenile myelomonocytic leukemia
- No Fanconi's anemia, Kostmann syndrome, Shwachman syndrome, or any other known bone marrow failure syndrome
- No promyelocytic leukemia (M3)
- No secondary or treatment-related AML
- Matched family donor criteria (for patients with intermediate-risk or high-risk disease):
- HLA-A, -B, -C, and beta chain (-DRB1), identical or 1 antigen or allele mismatched by molecular high resolution technique
- All available first-degree family members (parents and siblings) must be HLA typed
- No syngeneic donors
- Matched alternative donor criteria (for patients with high-risk disease):
- HLA-A, -B, -C, and -DRB1, identical or 1 antigen or allele mismatched donor
- HLA-A, -B, and -DRB1 4 of 6 antigen matched unrelated cord blood donor
- Mismatched family member donor with ≥ 1 haplotype match or 5 of 6 antigen phenotypic match
PATIENT CHARACTERISTICS:
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
- No prior chemotherapy, radiation therapy, or any antileukemic therapy
- Topical or inhalation steroids for other conditions allowed
- Intrathecal cytarabine given at diagnosis allowed
- No other prior treatment for AML
- No concurrent peripheral blood stem cell transplantation in patients with matched family donor
Data sourced from ClinicalTrials.gov (NCT00372593). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.