Opportunities and challenges of personalized therapy of patients with HR ALL
EHA Library. Izraeli S. 06/14/19; 273710
Shai Izraeli
Shai Izraeli
Contributions
Learning Objectives
THIS MANUSCRIPT IS PUBLISHED AS AN OFFICIAL SUPPLEMENT OF HEMASPHERE.

Arend von Stackelberg - Chair introduction

Acute lymphoblastic leukemia (ALL) is the most frequent malignant disease in children. With conventional chemotherapy, prognosis is favorable, however at the cost of acute and long-term side effects. Relapsed and refractory ALL in contrast is associated with poor prognosis. Therefore, in general, there is a medical need for targeted potentially less toxic therapy to replace toxic chemotherapy and overcome resistance.
In B-cell progenitor (BCP) ALL favorable genetic alterations are hyperdiploidy or translocation t(12;21)/ETV6-RUNX1, whereas t(9;22)/BCR-ABL1, KMT2A (MLL) rearrangements, chromosome 21amplification (iAMP21), t(17/19)/TCF3-HLF and hypodiploidy are associated with poor prognosis. Numerous additional coexisting genetic lesions have been detected involving B-cell development, proliferation and differentiation pathways that may serve as prognostic markers or therapeutic targets.
In T-ALL, early T-immunophenotype (ETP) shows an immature genetic profile with stem-cell- and myeloid characteristics. Alterated pathways include NOTCH signaling, cell cycle regulation, PI3K, JAK/STAT, which may serve as therapeutic targets.
Targeted treatment may however be impaired by heteroclonality of the ALL. Prospective controlled trials to prove safety and efficacy of these approaches are largely lacking.

Learning goals of the article
• Genetic alterations are important prognostic factors in ALL.
• Affected intracellular pathways may serve as therapeutic targets.
• Prospective trials to prove the efficacy and safety of targeted therapies are needed.

Learning goals of the presentation
After attending this lecture, the participant will be able to
• realize the importance of including high risk patients with ALL in clinical trials,
• realize the challenges and opportunities of incorporating novel therapies for patients with leukemia, and
• understand the critical necessity to report every patients receiving off-label novel therapies, so that the effectiveness and toxicities of these therapies will be properly collected.

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