Precision Medicine in Myeloid Malignancies: Hype or Hope?

Shristi Upadhyay Banskota, Nabin Khanal, Rosalyn I. Marar, Prajwal Dhakal, Vijaya Raj Bhatt

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Purpose of Review: We review how understanding the fitness and comorbidity burden of patients, and molecular landscape of underlying acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) at the time of diagnosis is now integral to treatment. Recent Findings: The upfront identification of patients’ fitness and molecular profile facilitates selection of targeted and novel agents, enables risk stratification, allows consideration of allogeneic hematopoietic cell transplantation in high-risk patients, and provides treatment selection for older (age ≥ 75) or otherwise unfit patients who may not tolerate conventional treatment. The use of measurable residual disease (MRD) assessment improves outcome prediction and can also guide therapeutic strategies such as chemotherapy maintenance and transplant. In recent years, several novel drugs have received FDA approval for treating patients with AML with or without specific mutations. A doublet and triplet combination of molecular targeted and other novel treatments have resulted in high response rates in early trials. Following the initial success in AML, novel drugs are undergoing clinical trials in MDS. Summary: Unprecedented advances have been made in precision medicine approaches in AML and MDS. However, lack of durable responses and long-term disease control in many patients still present significant challenges, which can only be met, to some extent, with innovative combination strategies throughout the course of treatment from induction to consolidation and maintenance.

Original languageEnglish (US)
Pages (from-to)217-227
Number of pages11
JournalCurrent Hematologic Malignancy Reports
Issue number6
StatePublished - Dec 2022


  • Acute myeloid leukemia
  • Geriatric assessment
  • Minimal residual disease
  • Myelodysplastic syndrome
  • Novel drugs
  • Precision medicine

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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