Different outcomes of allogeneic hematopoietic stem cell transplant in a pair of twins affected by juvenile myelomonocytic leukemia

Simone Cesaro, Paola De Filippi, Annamaria Di Meglio, Anna Leszl, Svetlana Donska, Ada Zaccaron, Claudia Cagioni, Roberta Galavotti, Cesare Danesino, Fiorenza Aprili, Chiara Cugno, Geertruy Te Kronnie, Marco Zecca, Silvia Bresolin

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5 Citations (Scopus)

Abstract

A twin pair affected by juvenile myelomonocytic leukemia (JMML) with the same somatic PTPN11 mutation and abnormal chromosome 7 in bone marrow samples but distinct prognostic gene expression signatures, received a matched-unrelated donor and matched-unrelated cord blood transplant, respectively. Both twins fully engrafted, but after 6 months, the twin with an acute-myeloid-like (AML-like) signature at diagnosis rejected the graft and had an autologous reconstitution. A second transplant with an unrelated 5/6-HLA-matched-loci cord blood performed after 4 months from rejection was unsuccessful. After 25 months from diagnosis, the twin with the AML-like gene expression signature died of liver failure while on progression of his JMML. The other twin, who had a non-acute-myeloid-like (non-AML-like) gene expression signature at diagnosis is in complete hematological remission with full donor chimera. This observation suggests a biological diversity of JMML also in patients with a common genetic background.

Original languageEnglish
Pages (from-to)208-212
Number of pages5
JournalInternational Journal of Hematology
Volume99
Issue number2
DOIs
Publication statusPublished - 1 Feb 2014

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Keywords

  • Gene expression-based diagnostic classification
  • Hematopoietic stem cell transplant
  • Juvenile myelomonocytic leukemia
  • Twins

ASJC Scopus subject areas

  • Hematology

Cite this

Cesaro, S., De Filippi, P., Di Meglio, A., Leszl, A., Donska, S., Zaccaron, A., Cagioni, C., Galavotti, R., Danesino, C., Aprili, F., Cugno, C., Te Kronnie, G., Zecca, M., & Bresolin, S. (2014). Different outcomes of allogeneic hematopoietic stem cell transplant in a pair of twins affected by juvenile myelomonocytic leukemia. International Journal of Hematology, 99(2), 208-212. https://doi.org/10.1007/s12185-013-1489-3