In utero origin of myelofibrosis presenting in adult monozygotic twins

Nat Med. 2022 Jun;28(6):1207-1211. doi: 10.1038/s41591-022-01793-4. Epub 2022 May 30.

Abstract

The latency between acquisition of an initiating somatic driver mutation by a single-cell and clinical presentation with cancer is largely unknown. We describe a remarkable case of monozygotic twins presenting with CALR mutation-positive myeloproliferative neoplasms (MPNs) (aged 37 and 38 years), with a clinical phenotype of primary myelofibrosis. The CALR mutation was absent in T cells and dermal fibroblasts, confirming somatic acquisition. Whole-genome sequencing lineage tracing revealed a common clonal origin of the CALR-mutant MPN clone, which occurred in utero followed by twin-to-twin transplacental transmission and subsequent similar disease latency. Index sorting and single-colony genotyping revealed phenotypic hematopoietic stem cells (HSCs) as the likely MPN-propagating cell. Furthermore, neonatal blood spot analysis confirmed in utero origin of the JAK2V617F mutation in a patient presenting with polycythemia vera (aged 34 years). These findings provide a unique window into the prolonged evolutionary dynamics of MPNs and fitness advantage exerted by MPN-associated driver mutations in HSCs.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Calreticulin
  • Humans
  • Janus Kinase 2 / genetics
  • Mutation / genetics
  • Myeloproliferative Disorders* / genetics
  • Primary Myelofibrosis* / genetics
  • Twins, Monozygotic / genetics

Substances

  • Calreticulin
  • Janus Kinase 2