Sensitivity, specificity, and accuracy of molecular profiling on circulating cell-free DNA in refractory or relapsed multiple myeloma patients, results of MM-EP1 study

Leuk Lymphoma. 2024 Jun;65(6):789-799. doi: 10.1080/10428194.2024.2320258. Epub 2024 Mar 3.

Abstract

As a promising alternative to bone marrow aspiration (BMA), mutational profiling on blood-derived circulating cell-free tumor DNA (cfDNA) is a harmless and simple technique to monitor molecular response and treatment resistance of patients with refractory/relapsed multiple myeloma (R/R MM). We evaluated the sensitivity and specificity of cfDNA compared to BMA CD138 positive myeloma plasma cells (PCs) in a series of 45 R/R MM patients using the 29-gene targeted panel (AmpliSeq) NGS. KRAS, NRAS, FAM46C, DIS3, and TP53 were the most frequently mutated genes. The average sensitivity and specificity of cfDNA detection were 65% and 97%, respectively. The concordance per gene between the two samples was good to excellent according to Cohen's κ coefficients interpretation. An increased number of mutations detected in cfDNA were associated with a decreased overall survival. In conclusion, we demonstrated cfDNA NGS analysis feasibility and accuracy in R/R MM patients who may benefit from early phase clinical trial.

Keywords: Refractory/relapsed multiple myeloma; circulating cell-free DNA; liquid biopsy; precision medicine; targeted next-generation sequencing; tumor-derived.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Biomarkers, Tumor* / genetics
  • Cell-Free Nucleic Acids / blood
  • Cell-Free Nucleic Acids / genetics
  • Circulating Tumor DNA* / blood
  • Circulating Tumor DNA* / genetics
  • DNA Mutational Analysis / methods
  • Drug Resistance, Neoplasm / genetics
  • Female
  • High-Throughput Nucleotide Sequencing* / methods
  • Humans
  • Male
  • Middle Aged
  • Multiple Myeloma* / blood
  • Multiple Myeloma* / diagnosis
  • Multiple Myeloma* / genetics
  • Multiple Myeloma* / mortality
  • Mutation*
  • Neoplasm Recurrence, Local / blood
  • Neoplasm Recurrence, Local / genetics
  • Neoplasm Recurrence, Local / pathology
  • Prognosis
  • Reproducibility of Results
  • Sensitivity and Specificity

Substances

  • Biomarkers, Tumor
  • Circulating Tumor DNA
  • Cell-Free Nucleic Acids