Mixed phenotype acute leukemia in a child associated with a NUP98-NSD1 fusion and NRAS p.Gly61Arg mutation

Cancer Rep (Hoboken). 2021 Aug;4(4):e1372. doi: 10.1002/cnr2.1372. Epub 2021 Mar 30.

Abstract

Background: Mixed phenotype acute leukemia (MPAL) is a rare subset of acute leukemia in the pediatric population associated with genetic alterations seen in both acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML).

Case: We describe a patient with MPAL with a NUP98 (nucleoporin 98)-NSD1 gene fusion (nuclear receptor binding SET domain protein1) and NRAS (neuroblastoma RAS viral oncogene homolog mutation) p.Gly61Arg mutation who was treated with upfront AML-based chemotherapy, received hematopoietic stem cell transplant (HSCT), but unfortunately died from relapsed disease.

Conclusion: This case highlights the challenges faced in choosing treatment options in MPAL patients with complex genomics, with predominant myeloid features.

Keywords: NRAS; NUP98-NSD1; mTOR inhibitors; mixed phenotype acute leukemia (MPAL).

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Biopsy
  • Bone Marrow / pathology
  • Fatal Outcome
  • Female
  • GTP Phosphohydrolases / genetics*
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Leukemia, Myeloid, Acute / diagnosis*
  • Leukemia, Myeloid, Acute / genetics
  • Leukemia, Myeloid, Acute / pathology
  • Leukemia, Myeloid, Acute / therapy
  • Membrane Proteins / genetics*
  • Mutation
  • Neoadjuvant Therapy
  • Neoplasms, Complex and Mixed / diagnosis*
  • Neoplasms, Complex and Mixed / genetics
  • Neoplasms, Complex and Mixed / pathology
  • Neoplasms, Complex and Mixed / therapy
  • Oncogene Proteins, Fusion / genetics*
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / diagnosis*
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / pathology
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy

Substances

  • Membrane Proteins
  • NUP98-NSD1 protein, human
  • Oncogene Proteins, Fusion
  • GTP Phosphohydrolases
  • NRAS protein, human