Novel combinations yield promising results for leukemia patients with poor prognoses

(Press-News.org) (SAN FRANCISCO, DECEMBER 7, 2014) - Recognizing that leukemia cannot be conquered with a "one-size-fits-all" approach, researchers are pursuing novel targeted therapies and combinations of existing treatment regimens with new agents for patient populations with historically poor prognoses, according to data presented today during the 56th American Society of Hematology (ASH) Annual Meeting and Exposition. In recent years, outcomes for patients with leukemia have steadily improved with the emergence of numerous therapies that target specific genetic drivers of disease, as well as potent combination regimens. While overall outcomes for patients with leukemia are improving as a result of these new therapies, some more vulnerable subgroups of patients, including elderly patients and patients with aggressive genetic mutations, have not experienced the same positive results.

Studies presented today reflect dedicated efforts to better understand the course of disease among these higher-risk patients to enable the development of new treatments or more effective treatment combinations that will improve survival outcomes. Two studies suggest that combining new targeted agents with standard chemotherapy regimens may be effective in treating newly diagnosed young or elderly patients with acute leukemias. Further evidence suggests that adolescents and young adults may respond better to treatment with a chemotherapy regimen pioneered in pediatric patients rather than with standard adult regimens. Additional studies evaluate an investigational, targeted therapy for certain blood cancers fueled by a recently discovered genetic mutation and examine the use of a specific combination of chemotherapies to treat a rare type of pediatric leukemia.

"This group of studies represents important progress on our continued quest to develop new therapies and to maximize use of existing therapies to yield improved results for patients with leukemias, particularly those with aggressive disease that is resistant to current approaches," said David Steensma, MD, moderator of the press conference and faculty member in the adult leukemia program at Dana-Farber Cancer Institute in Boston. "Our challenge moving forward will be to better understand how distinct subtypes of these diseases can affect patients in different ways and to use that insight to tailor our treatment approaches to each biological type of leukemia, ultimately improving long-term outcomes."

This press briefing will take place at 9:30 a.m. PST on Sunday, December 7, in rooms 236-238 of Moscone South, East Mezzanine.

Children with Rare Leukemia Associated with Poor Prognosis Have Better-Than-Expected Outcomes After Tailored Treatment

T-Lymphoblastic Leukemia (T-ALL) Shows Excellent Outcome, Lack of Significance of the Early Thymic Precursor (ETP) Immunophenotype, and Validation of the Prognostic Value of End-Induction Minimal Residual Disease (MRD) in Children's Oncology Group (COG) Study AALL0434 [1]

Relapse after therapy is a major challenge for children and young adults with T-lymphocytic leukemia (T-ALL), a subset of leukemia sometimes associated with a poor prognosis. Previous research has indicated that patients with a specific protein signature of T-ALL, called the early thymic precursor (ETP) immunophenotype, have particularly poor outcomes. To better understand how the presence of this immunophenotype among pediatric T-ALL patients influences their response to therapy, researchers enrolled 1,144 T-ALL patients in this Phase III trial, the largest ever conducted among patients with this leukemia subtype. Included in the trial were children with the ETP immunophenotype (11.3%), children with a nearly identical type of T-ALL known as "near-ETP" (17%), and children without the ETP immunophenotype (71.6%).

After administering the same standard initial regimen to all children enrolled in the study, investigators measured levels of "minimal residual disease" (MRD - i.e., disease that is still detectable by sensitive techniques, but not by the pathologists' traditional light microscope) in each patient, placing children into groups at low-, intermediate-, and high-risk of relapse (low risk END