Abstract
Myelofibrosis shows a progressive clinical course and usually a poor, lethal prognosis. The molecular pathogenesis of this disease largely remains to be fully understood but the identification of the JAK2V617F mutation in more than half of patients was a major improvement in our understanding of the disease biology and may represent the first biologic marker useful for risk stratification, independently from conventional clinical predictors. After many elusive efforts, new effective treatment strategies are becoming available for this disease. Allogeneic transplantation following reduced-intensity conditioning programs, at least in some patients, may induce not only a hematologic response but also a molecular remission, thus supporting the hope of a possible, definitive eradication of the disease. Moreover, new innovative drugs, targeting either the JAK2V617F mutation or more general oncogenic mechanisms, may provide widely applicable, effective treatments to many patients for whom allogeneic transplantation is not feasible.
| Original language | English |
|---|---|
| Pages (from-to) | 83-91 |
| Number of pages | 9 |
| Journal | Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program |
| Publication status | Published - 2008 |
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ASJC Scopus subject areas
- Medicine(all)
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From palliation to epigenetic therapy in myelofibrosis. / Rambaldi, Alessandro; Barbui, Tiziano; Barosi, Giovanni.
In: Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program, 2008, p. 83-91.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - From palliation to epigenetic therapy in myelofibrosis.
AU - Rambaldi, Alessandro
AU - Barbui, Tiziano
AU - Barosi, Giovanni
PY - 2008
Y1 - 2008
N2 - Myelofibrosis shows a progressive clinical course and usually a poor, lethal prognosis. The molecular pathogenesis of this disease largely remains to be fully understood but the identification of the JAK2V617F mutation in more than half of patients was a major improvement in our understanding of the disease biology and may represent the first biologic marker useful for risk stratification, independently from conventional clinical predictors. After many elusive efforts, new effective treatment strategies are becoming available for this disease. Allogeneic transplantation following reduced-intensity conditioning programs, at least in some patients, may induce not only a hematologic response but also a molecular remission, thus supporting the hope of a possible, definitive eradication of the disease. Moreover, new innovative drugs, targeting either the JAK2V617F mutation or more general oncogenic mechanisms, may provide widely applicable, effective treatments to many patients for whom allogeneic transplantation is not feasible.
AB - Myelofibrosis shows a progressive clinical course and usually a poor, lethal prognosis. The molecular pathogenesis of this disease largely remains to be fully understood but the identification of the JAK2V617F mutation in more than half of patients was a major improvement in our understanding of the disease biology and may represent the first biologic marker useful for risk stratification, independently from conventional clinical predictors. After many elusive efforts, new effective treatment strategies are becoming available for this disease. Allogeneic transplantation following reduced-intensity conditioning programs, at least in some patients, may induce not only a hematologic response but also a molecular remission, thus supporting the hope of a possible, definitive eradication of the disease. Moreover, new innovative drugs, targeting either the JAK2V617F mutation or more general oncogenic mechanisms, may provide widely applicable, effective treatments to many patients for whom allogeneic transplantation is not feasible.
UR - http://www.scopus.com/inward/record.url?scp=67651149991&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=67651149991&partnerID=8YFLogxK
M3 - Article
C2 - 19074063
AN - SCOPUS:67651149991
SP - 83
EP - 91
JO - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
JF - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
SN - 1520-4391
ER -