Characterization and use of an antibody detecting the CBFbeta-SMMHC fusion protein in inv(16)/t(16;16)-associated acute myeloid leukemias

Blood. 1998 Mar 15;91(6):1882-90.

Abstract

The inv(16)(p13q22) and t(16;16)(p13;q22) cytogenetic abnormalities occur commonly in acute myeloid leukemia (AML), typically associated with French-American-British (FAB) AML-M4Eo subtype. Reverse transcriptase-polymerase chain reaction (RT-PCR) techniques have been recently developed to detect the presence of several variants of the resultant CBFB-MYH11 fusion gene that encodes a CBFbeta-smooth muscle myosin heavy chain (SMMHC) fusion protein. We have now determined the clinical use of a polyclonal antibody [anti-inv(16) Ab] directed against a junctional epitope of the most common type of CBFbeta-SMMHC fusion protein (type A), which is present in 90% of inv(16)/t(16;16) AML cases. Using flow cytometry, reproducible methods were developed for detection of CBFbeta-SMMHC proteins in permeabilized cells; flow cytometric results were then correlated with cytogenetics and RT-PCR detection methods. In an analysis of 42 leukemia cases with various cytogenetic abnormalities and several normal controls, the anti-inv(16) Ab specifically detected all 23 cases that were cytogenetically positive for inv(16) or t(16;16), including a single AML case that was RT-PCR-negative. In addition to detecting all type A fusions, the anti-inv(16) Ab also unexpectedly identified the type C and type D CBFbeta-SMMHC fusion proteins. Molecular characterization of one RT-PCR-positive and Ab-positive t(16;16) case with a non-type A product showed a novel previously unreported CBFB-MYH11 fusion (CBFB nt 455-MYH11 nt 1893). Flow cytometric results were analyzed using the Kolmogorov-Smirnov statistic D-value and the median value for positive samples was 0.65 (range, 0.35 to 0.77) versus 0.07 (range, -0.21 to 0.18) in the negative group (P < .0001). The overall concordance between cytogenetics and RT-PCR was 97%, whereas the concordance between flow cytometry and cytogenetics was 100%. Thus, using the anti-inv(16) Ab, all cytogenetically positive and RT-PCR-positive AML cases with inv(16) or t(16;16) could be rapidly identified. This study demonstrates the use of this antibody as an investigational tool in inv(16)/t(16;16) AML and suggests that the development of such reagents may have potential clinical diagnostic use.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Acute Disease
  • Adult
  • Aged
  • Aged, 80 and over
  • Antibodies, Monoclonal / immunology*
  • Base Sequence
  • Biomarkers, Tumor / analysis*
  • Child, Preschool
  • Chromosome Inversion*
  • Chromosomes, Human, Pair 16 / genetics*
  • Chromosomes, Human, Pair 16 / ultrastructure
  • Female
  • Flow Cytometry
  • Humans
  • Leukemia, Myeloid / genetics
  • Leukemia, Myeloid / metabolism*
  • Leukemia, Myeloid / pathology
  • Male
  • Middle Aged
  • Molecular Sequence Data
  • Neoplasm Proteins / analysis*
  • Neoplasm Proteins / genetics
  • Neoplasm Proteins / immunology
  • Neoplasm, Residual
  • Oncogene Proteins, Fusion / analysis
  • Oncogene Proteins, Fusion / genetics
  • Oncogene Proteins, Fusion / immunology
  • Polymerase Chain Reaction
  • RNA, Messenger / analysis
  • RNA, Neoplasm / analysis
  • Reproducibility of Results
  • Sensitivity and Specificity
  • Translocation, Genetic / genetics*

Substances

  • Antibodies, Monoclonal
  • Biomarkers, Tumor
  • CBFbeta-MYH11 fusion protein
  • Neoplasm Proteins
  • Oncogene Proteins, Fusion
  • RNA, Messenger
  • RNA, Neoplasm