Whole-genome sequencing facilitates patient-specific quantitative PCR-based minimal residual disease monitoring in acute lymphoblastic leukaemia, neuroblastoma and Ewing sarcoma

Br J Cancer. 2022 Feb;126(3):482-491. doi: 10.1038/s41416-021-01538-z. Epub 2021 Sep 1.

Abstract

Background: Minimal residual disease (MRD) measurement is a cornerstone of contemporary acute lymphoblastic leukaemia (ALL) treatment. The presence of immunoglobulin (Ig) and T cell receptor (TCR) gene recombinations in leukaemic clones allows widespread use of patient-specific, DNA-based MRD assays. In contrast, paediatric solid tumour MRD remains experimental and has focussed on generic assays targeting tumour-specific messenger RNA, methylated DNA or microRNA.

Methods: We examined the feasibility of using whole-genome sequencing (WGS) data to design tumour-specific polymerase chain reaction (PCR)-based MRD tests (WGS-MRD) in 18 children with high-risk relapsed cancer, including ALL, high-risk neuroblastoma (HR-NB) and Ewing sarcoma (EWS) (n = 6 each).

Results: Sensitive WGS-MRD assays were generated for each patient and allowed quantitation of 1 tumour cell per 10-4 (0.01%)-10-5 (0.001%) mononuclear cells. In ALL, WGS-MRD and Ig/TCR-MRD were highly concordant. WGS-MRD assays also showed good concordance between quantitative PCR and droplet digital PCR formats. In serial clinical samples, WGS-MRD correlated with disease course. In solid tumours, WGS-MRD assays were more sensitive than RNA-MRD assays.

Conclusions: WGS facilitated the development of patient-specific MRD tests in ALL, HR-NB and EWS with potential clinical utility in monitoring treatment response. WGS data could be used to design patient-specific MRD assays in a broad range of tumours.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Biomarkers, Tumor / genetics*
  • Bone Neoplasms / blood
  • Bone Neoplasms / genetics
  • Bone Neoplasms / pathology
  • Child
  • Child, Preschool
  • Female
  • Gene Rearrangement*
  • Humans
  • Infant
  • Male
  • N-Myc Proto-Oncogene Protein / genetics
  • Neoplasm, Residual / genetics
  • Neoplasm, Residual / pathology*
  • Neuroblastoma / blood
  • Neuroblastoma / genetics
  • Neuroblastoma / pathology*
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / blood
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / pathology*
  • Proto-Oncogene Protein c-fli-1 / genetics
  • Receptors, Antigen, T-Cell / genetics
  • Sarcoma, Ewing / blood
  • Sarcoma, Ewing / genetics
  • Sarcoma, Ewing / pathology*
  • Transcriptional Regulator ERG / genetics
  • Whole Genome Sequencing / methods*

Substances

  • Biomarkers, Tumor
  • ERG protein, human
  • FLI1 protein, human
  • N-Myc Proto-Oncogene Protein
  • Proto-Oncogene Protein c-fli-1
  • Receptors, Antigen, T-Cell
  • Transcriptional Regulator ERG