Biomarkers in Predicting Response to Treatment in Bone Marrow Samples From Young Patients With Acute Myeloid Leukemia

Validation of a Classifier for Induction Response Prediction Using Single Cell Network Profiling (SCNP) Assays for Childhood AML

RATIONALE: Studying samples of bone marrow from patients with cancer in the laboratory may help doctors predict how well patients will respond to treatment.

PURPOSE: This research trial is studying biomarkers in predicting response to treatment in bone marrow samples from young patients with acute myeloid leukemia.

Study Overview

• Status: Completed
• Conditions: Leukemia
• Intervention / Treatment: Other: laboratory biomarker analysis; Other: study of socioeconomic and demographic variables

Detailed Description

OBJECTIVES:

Primary

• To validate the accuracy of a pre-specified acute myeloid leukemia (AML) induction response classifier or the My Profile™ AML Induction Therapy Assay (run in a Good Laboratory Practice [GLP] laboratory using Good Manufacturing Practice [GMP] reagents) in predicting response to cytarabine-based induction chemotherapy in pediatric patients with non-M3 AML.

Secondary

• To validate the continuous score from the pre-specified induction response classifier as a predictor of response to induction chemotherapy, after controlling for the simultaneous effects of clinical variables at base-line and/or pre-induction therapy (e.g., age, WBC, and percentage [%] of blasts), and tests commonly available after the start of induction therapy (e.g., cytogenetics and molecular markers).
• To validate the accuracy of the pre-specified induction response classifier as a binary predictor of induction response using a pre-specified cutpoint to assign patients to no response (NR) or complete response (CR) groups.
• To validate the accuracy of the pre-specified induction response classifier as a binary predictor of induction response using a pre-specified cutpoint to assign patients to NR or CR groups, after controlling for the simultaneous effects of clinical variables at base-line (pre-induction therapy) (e.g., age, WBC, % blasts) and clinical variables, including tests commonly available after start of induction therapy (e.g., age, WBC, cytogenetics).

OUTLINE: Cryopreserved bone marrow mononuclear cell specimens are analyzed by cell networking profiling assays. Results are then compared with patient outcomes and demographics from COG-AAML03P1 and COG-AAML0531 studies.

Molecular markers analyzed include Flt3ITD, NPM1, CEBPA, and MRA.

• Study Type: Observational
• Enrollment (Anticipated): 97

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Diagnosis of acute myeloid leukemia.

Description

DISEASE CHARACTERISTICS:

• Diagnosis of acute myeloid leukemia (AML)

  • Non-M3 AML

• Cryopreserved bone marrow mononuclear cell specimens from patients enrolled on COG-AAML03P1 and COG-AAML0531 collected prior to cytarabine-based induction therapy

  • Patient samples from COG-AAML0531 must come from the control arm (i.e., no gemtuzumab ozogamicin induction therapy)

PATIENT CHARACTERISTICS:

• No Down syndrome

PRIOR CONCURRENT THERAPY:

• See Disease Characteristics

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Accuracy of induction response prediction

Secondary Outcome Measures

Outcome Measure
Duration of complete response
Event-free survival and overall survival
Status (dead vs living)
Date of last follow up

Collaborators and Investigators

• Sponsor: Children's Oncology Group
• Collaborators: National Cancer Institute (NCI)
• Investigators: Principal Investigator: Norman J. Lacayo, MD, Stanford University

Study record dates

Study Major Dates

• Study Start: March 1, 2011
• Primary Completion (Actual): May 1, 2016

Study Registration Dates

• First Submitted: March 22, 2011
• First Submitted That Met QC Criteria: March 22, 2011
• First Posted (Estimate): March 23, 2011

Study Record Updates

• Last Update Posted (Estimate): May 18, 2016
• Last Update Submitted That Met QC Criteria: May 17, 2016
• Last Verified: May 1, 2016

More Information

Terms related to this study

• Keywords: childhood acute myeloid leukemia in remission; recurrent childhood acute myeloid leukemia; childhood acute erythroleukemia (M6); childhood acute megakaryocytic leukemia (M7); childhood acute minimally differentiated myeloid leukemia (M0); untreated childhood acute myeloid leukemia and other myeloid malignancies; childhood acute myeloblastic leukemia without maturation (M1); childhood acute myeloblastic leukemia with maturation (M2); childhood acute myelomonocytic leukemia (M4); childhood acute monoblastic leukemia (M5a); childhood acute monocytic leukemia (M5b)
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia
• Other Study ID Numbers: AAML11B8 (Other Identifier: Children's Oncology Group); COG-AAML11B8 (Other Identifier: Children's Oncology Group); NCI-2011-02851 (Registry Identifier: CTRP (Clinical Trial Reporting Program))