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 language | English |
|---|---|
| Journal | Hematological Oncology |
| DOIs | |
| Publication status | Accepted/In press - 2015 |
Fingerprint
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 journal › Article
}
TY - JOUR
T1 - Treosulfan based reduced toxicity conditioning followed by allogeneic stem cell transplantation in patients with myelofibrosis
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
PY - 2015
Y1 - 2015
N2 - 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.
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.
KW - Bone marrow transplantation
KW - Hematopoietic stem cell transplantation
KW - Myeloproliferative disease
KW - Primary myelofibrosis
KW - Reduced-toxicity conditioning
KW - Treosulfan
UR - http://www.scopus.com/inward/record.url?scp=84920854715&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84920854715&partnerID=8YFLogxK
U2 - 10.1002/hon.2183
DO - 10.1002/hon.2183
M3 - Article
C2 - 25469485
AN - SCOPUS:84991207326
JO - Hematological Oncology
JF - Hematological Oncology
SN - 0278-0232
ER -