Chimeric antigen receptor T-cell therapy for marrow and extramedullary relapse of infant acute lymphoblastic leukemia

Pediatr Blood Cancer. 2021 Jan;68(1):e28739. doi: 10.1002/pbc.28739. Epub 2020 Oct 3.

Abstract

Chimeric antigen receptor (CAR) T-cells, engineered autologous T-cells that target antigens found in leukemia, have shown durable remissions in relapsed acute lymphoblastic leukemia (ALL). Infant ALL with KMT2A rearrangements (KMT2Ar) is a rare, aggressive form of leukemia associated with extramedullary disease both at diagnosis and at relapse, and overall outcomes for these patients are dismal. Here we report the successful use of tisagenlecleucel, a CAR T-cell product approved for relapsed/refractory ALL, in a patient with KMT2Ar infant ALL who was treated for combined marrow and extramedullary (renal) relapse.

Keywords: ALL; ALL relapse; immunotherapy; pediatric hematology/oncology.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Bone Marrow Neoplasms / pathology
  • Bone Marrow Neoplasms / therapy*
  • Combined Modality Therapy
  • Female
  • Hematopoietic Stem Cell Transplantation
  • Histone-Lysine N-Methyltransferase / genetics
  • Humans
  • Immunotherapy, Adoptive / methods*
  • Infant
  • Kidney Neoplasms / pathology
  • Kidney Neoplasms / therapy*
  • Mutation
  • Myeloid-Lymphoid Leukemia Protein / genetics
  • Neoplasm Recurrence, Local / genetics
  • Neoplasm Recurrence, Local / pathology
  • Neoplasm Recurrence, Local / therapy*
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / pathology
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy*
  • Prognosis
  • Receptors, Chimeric Antigen / immunology*

Substances

  • KMT2A protein, human
  • Receptors, Chimeric Antigen
  • Myeloid-Lymphoid Leukemia Protein
  • Histone-Lysine N-Methyltransferase