Illuminating novel biological aspects and potential new therapeutic approaches for chronic myeloproliferative malignancies

Hematol Oncol. 2020 Dec;38(5):654-664. doi: 10.1002/hon.2771. Epub 2020 Sep 4.

Abstract

This review reflects the presentations and discussion at the 14th post-American Society of Hematology (ASH) International Workshop on Chronic Myeloproliferative Malignancies, which took place on the December 10 and 11, 2019, immediately after the 61st ASH Annual Meeting in Orlando, Florida. Rather than present a resume of the proceedings, we address some of the topical translational science research and clinically relevant topics in detail. We consider how recent studies using single-cell genomics and other molecular methods reveal novel aspects of hematopoiesis which in turn raise the possibility of new therapeutic approaches for patients with myeloproliferative neoplasms (MPNs). We discuss how alternative therapies could benefit patients with chronic myeloid leukemia who develop BCR-ABL1 mutant subclones following ABL1-tyrosine kinase inhibitor therapy. In MPNs, we focus on efforts beyond JAK-STAT and the merits of integrating activin receptor ligand traps, interferon-α, and allografting in the current treatment algorithm for patients with myelofibrosis.

Keywords: BCR-ABL1 mutants; genomic risk scores; interferon-alpha; luspatercept; non-JAK-STAT therapies; single-cell genomics.

Publication types

  • Review

MeSH terms

  • Anemia / diagnosis
  • Anemia / etiology
  • Anemia / therapy
  • Biomarkers
  • Biomarkers, Tumor
  • Combined Modality Therapy / adverse effects
  • Combined Modality Therapy / methods
  • Disease Management
  • Disease Susceptibility*
  • Drug Development
  • Humans
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / complications
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / diagnosis
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / etiology*
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / therapy*
  • Molecular Diagnostic Techniques
  • Molecular Targeted Therapy
  • Myeloproliferative Disorders / complications
  • Myeloproliferative Disorders / diagnosis
  • Myeloproliferative Disorders / etiology*
  • Myeloproliferative Disorders / therapy*
  • Prognosis
  • Single-Cell Analysis / methods
  • Translational Research, Biomedical
  • Treatment Outcome

Substances

  • Biomarkers
  • Biomarkers, Tumor