Treosulfan based reduced toxicity conditioning followed by allogeneic stem cell transplantation in patients with myelofibrosis

Simone Claudiani, Sarah Marktel, Simona Piemontese, Andrea Assanelli, Maria Teresa Lupo-Stanghellini, Matteo Carrabba, Elena Guggiari, Fabio Giglio, Tiago De Freitas, Magda Marcatti, Massimo Bernardi, Consuelo Corti, Jacopo Peccatori, Francesca Lunghi, Fabio Ciceri

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Allogeneic transplantation is the only potentially curative strategy for myelofibrosis, even in the era of new drugs that so far only mitigate symptoms. The choice to proceed to allogeneic transplantation is based on several variables including age, disease phase, degree of splenomegaly, donor availability, comorbidities and iron overload. These factors, along with conditioning regimen and time to transplantation, may influence the outcome of ASCT. We report 14 patients affected by myelofibrosis with a median age of 57years (range, 41-76) receiving a treosulfan-fludarabine based reduced toxicity conditioning. Patients (pts) received a stem cell transplantation from an HLA identical (n=10) or matched unrelated donor (n=4). All pts had a complete myeloablation followed by engraftment and in 12 out of 13 evaluated pts donor chimerism was 100% at 1month. In most cases a reduction of splenomegaly and a reduction (or resolution) of bone marrow fibrosis was observed. After a median follow-up of 39months (range, 3-106), the 3-year probability of overall survival and disease free survival was 54+/-14% and 46+/-14%, respectively. The cumulative incidence of non-relapse mortality at 2years was 39+/-15%. Causes of non-relapse mortality were: infection (n=2), GvHD (n=2) and haemorrhage (n=1). We can conclude that a treosulfan and fludarabine based conditioning has a potent myeloablative and anti-disease activity although non-relapse mortality remains high in this challenging clinical setting.

Original languageEnglish
JournalHematological Oncology
DOIs
Publication statusAccepted/In press - 2015

Fingerprint

treosulfan
Primary Myelofibrosis
Stem Cell Transplantation
Splenomegaly
Homologous Transplantation
Mortality
Tissue Donors
Unrelated Donors
Chimerism
Iron Overload
Disease-Free Survival
Comorbidity
Transplantation
Hemorrhage
Survival
Incidence
Infection
Pharmaceutical Preparations

Keywords

  • Bone marrow transplantation
  • Hematopoietic stem cell transplantation
  • Myeloproliferative disease
  • Primary myelofibrosis
  • Reduced-toxicity conditioning
  • Treosulfan

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

Treosulfan based reduced toxicity conditioning followed by allogeneic stem cell transplantation in patients with myelofibrosis. / Claudiani, Simone; Marktel, Sarah; Piemontese, Simona; Assanelli, Andrea; Lupo-Stanghellini, Maria Teresa; Carrabba, Matteo; Guggiari, Elena; Giglio, Fabio; De Freitas, Tiago; Marcatti, Magda; Bernardi, Massimo; Corti, Consuelo; Peccatori, Jacopo; Lunghi, Francesca; Ciceri, Fabio.

In: Hematological Oncology, 2015.

Research output: Contribution to journalArticle

Claudiani, Simone ; Marktel, Sarah ; Piemontese, Simona ; Assanelli, Andrea ; Lupo-Stanghellini, Maria Teresa ; Carrabba, Matteo ; Guggiari, Elena ; Giglio, Fabio ; De Freitas, Tiago ; Marcatti, Magda ; Bernardi, Massimo ; Corti, Consuelo ; Peccatori, Jacopo ; Lunghi, Francesca ; Ciceri, Fabio. / Treosulfan based reduced toxicity conditioning followed by allogeneic stem cell transplantation in patients with myelofibrosis. In: Hematological Oncology. 2015.
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AU - Claudiani, Simone

AU - Marktel, Sarah

AU - Piemontese, Simona

AU - Assanelli, Andrea

AU - Lupo-Stanghellini, Maria Teresa

AU - Carrabba, Matteo

AU - Guggiari, Elena

AU - Giglio, Fabio

AU - De Freitas, Tiago

AU - Marcatti, Magda

AU - Bernardi, Massimo

AU - Corti, Consuelo

AU - Peccatori, Jacopo

AU - Lunghi, Francesca

AU - Ciceri, Fabio

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AB - Allogeneic transplantation is the only potentially curative strategy for myelofibrosis, even in the era of new drugs that so far only mitigate symptoms. The choice to proceed to allogeneic transplantation is based on several variables including age, disease phase, degree of splenomegaly, donor availability, comorbidities and iron overload. These factors, along with conditioning regimen and time to transplantation, may influence the outcome of ASCT. We report 14 patients affected by myelofibrosis with a median age of 57years (range, 41-76) receiving a treosulfan-fludarabine based reduced toxicity conditioning. Patients (pts) received a stem cell transplantation from an HLA identical (n=10) or matched unrelated donor (n=4). All pts had a complete myeloablation followed by engraftment and in 12 out of 13 evaluated pts donor chimerism was 100% at 1month. In most cases a reduction of splenomegaly and a reduction (or resolution) of bone marrow fibrosis was observed. After a median follow-up of 39months (range, 3-106), the 3-year probability of overall survival and disease free survival was 54+/-14% and 46+/-14%, respectively. The cumulative incidence of non-relapse mortality at 2years was 39+/-15%. Causes of non-relapse mortality were: infection (n=2), GvHD (n=2) and haemorrhage (n=1). We can conclude that a treosulfan and fludarabine based conditioning has a potent myeloablative and anti-disease activity although non-relapse mortality remains high in this challenging clinical setting.

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