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

Research output: Contribution to journalArticle

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 - Feb 2014
Externally publishedYes

Fingerprint

Juvenile Myelomonocytic Leukemia
Hematopoietic Stem Cells
Transplants
Transcriptome
Fetal Blood
Unrelated Donors
Chromosomes, Human, Pair 7
Biodiversity
Liver Failure
Bone Marrow
Tissue Donors
Mutation

Keywords

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

ASJC Scopus subject areas

  • Hematology

Cite this

Different outcomes of allogeneic hematopoietic stem cell transplant in a pair of twins affected by juvenile myelomonocytic leukemia. / Cesaro, Simone; De Filippi, Paola; Di Meglio, Annamaria; Leszl, Anna; Donska, Svetlana; Zaccaron, Ada; Cagioni, Claudia; Galavotti, Roberta; Danesino, Cesare; Aprili, Fiorenza; Cugno, Chiara; Te Kronnie, Geertruy; Zecca, Marco; Bresolin, Silvia.

In: International Journal of Hematology, Vol. 99, No. 2, 02.2014, p. 208-212.

Research output: Contribution to journalArticle

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, vol. 99, no. 2, pp. 208-212. https://doi.org/10.1007/s12185-013-1489-3
Cesaro, Simone ; De Filippi, Paola ; Di Meglio, Annamaria ; Leszl, Anna ; Donska, Svetlana ; Zaccaron, Ada ; Cagioni, Claudia ; Galavotti, Roberta ; Danesino, Cesare ; Aprili, Fiorenza ; Cugno, Chiara ; Te Kronnie, Geertruy ; Zecca, Marco ; Bresolin, Silvia. / Different outcomes of allogeneic hematopoietic stem cell transplant in a pair of twins affected by juvenile myelomonocytic leukemia. In: International Journal of Hematology. 2014 ; Vol. 99, No. 2. pp. 208-212.
@article{4a336672cfa949099612998ec792867d,
title = "Different outcomes of allogeneic hematopoietic stem cell transplant in a pair of twins affected by juvenile myelomonocytic leukemia",
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.",
keywords = "Gene expression-based diagnostic classification, Hematopoietic stem cell transplant, Juvenile myelomonocytic leukemia, Twins",
author = "Simone Cesaro and {De Filippi}, Paola and {Di Meglio}, Annamaria and Anna Leszl and Svetlana Donska and Ada Zaccaron and Claudia Cagioni and Roberta Galavotti and Cesare Danesino and Fiorenza Aprili and Chiara Cugno and {Te Kronnie}, Geertruy and Marco Zecca and Silvia Bresolin",
year = "2014",
month = "2",
doi = "10.1007/s12185-013-1489-3",
language = "English",
volume = "99",
pages = "208--212",
journal = "International Journal of Hematology",
issn = "0925-5710",
publisher = "Springer Japan",
number = "2",

}

TY - JOUR

T1 - Different outcomes of allogeneic hematopoietic stem cell transplant in a pair of twins affected by juvenile myelomonocytic leukemia

AU - Cesaro, Simone

AU - De Filippi, Paola

AU - Di Meglio, Annamaria

AU - Leszl, Anna

AU - Donska, Svetlana

AU - Zaccaron, Ada

AU - Cagioni, Claudia

AU - Galavotti, Roberta

AU - Danesino, Cesare

AU - Aprili, Fiorenza

AU - Cugno, Chiara

AU - Te Kronnie, Geertruy

AU - Zecca, Marco

AU - Bresolin, Silvia

PY - 2014/2

Y1 - 2014/2

N2 - 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.

AB - 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.

KW - Gene expression-based diagnostic classification

KW - Hematopoietic stem cell transplant

KW - Juvenile myelomonocytic leukemia

KW - Twins

UR - http://www.scopus.com/inward/record.url?scp=84894457523&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84894457523&partnerID=8YFLogxK

U2 - 10.1007/s12185-013-1489-3

DO - 10.1007/s12185-013-1489-3

M3 - Article

VL - 99

SP - 208

EP - 212

JO - International Journal of Hematology

JF - International Journal of Hematology

SN - 0925-5710

IS - 2

ER -