Abstract
Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm diagnosed de novo or developed from essential thrombocythemia (ET) or polycythemia vera (PV). Average survival of a patient with MF is 5-7 years. Disease complications include fatigue, early satiety, pruritus, painful splenic infarcts, infections and leukemic transformation. Allogeneic hematopoietic stem cell transplant (HSCT) is the only potentially curative option for MF, but carries a risk of treatment-related mortality and is reserved for the few high-risk patients fit enough to endure the procedure. Other traditional therapies are palliative and supported by few randomized, controlled trials; thus, novel treatment strategies are needed. Discovery of the Janus kinase 2 (JAK2) gain-of-function mutation, JAK2V617F, in the majority (50-60%) of patients with MF led to increased understanding of the biology underlying MF and the development of JAK2 inhibitors to treat MF. Recent Food and Drug Administration (FDA) approval of the first JAK2 inhibitor, ruxolitinib, signaled a new era for treatment of MF. Additional JAK2 inhibitors, such as SAR302503, may become commercially available in the near future, and their distinct pharmacologic and efficacy profiles will help determine their use across the patient population. Data on JAK2 inhibitors, their role in an evolving treatment paradigm, and future directions for treatment of MF are discussed.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 242-251 |
| Number of pages | 10 |
| Journal | Leukemia and Lymphoma |
| Volume | 54 |
| Issue number | 2 |
| DOIs | |
| State | Published - Feb 1 2013 |
| Externally published | Yes |
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Keywords
- Dysplasias
- JAK2V617F
- Janus kinase 2 (JAK2)
- Myelofibrosis
- Myeloid leukemias
- Myeloproliferative disorders
- Signaling therapies
ASJC Scopus subject areas
- Hematology
- Oncology
- Cancer Research
Cite this
The evolving treatment paradigm in myelofibrosis. / Mesa, Ruben.
In: Leukemia and Lymphoma, Vol. 54, No. 2, 01.02.2013, p. 242-251.Research output: Contribution to journal › Review article
}
TY - JOUR
T1 - The evolving treatment paradigm in myelofibrosis
AU - Mesa, Ruben
PY - 2013/2/1
Y1 - 2013/2/1
N2 - Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm diagnosed de novo or developed from essential thrombocythemia (ET) or polycythemia vera (PV). Average survival of a patient with MF is 5-7 years. Disease complications include fatigue, early satiety, pruritus, painful splenic infarcts, infections and leukemic transformation. Allogeneic hematopoietic stem cell transplant (HSCT) is the only potentially curative option for MF, but carries a risk of treatment-related mortality and is reserved for the few high-risk patients fit enough to endure the procedure. Other traditional therapies are palliative and supported by few randomized, controlled trials; thus, novel treatment strategies are needed. Discovery of the Janus kinase 2 (JAK2) gain-of-function mutation, JAK2V617F, in the majority (50-60%) of patients with MF led to increased understanding of the biology underlying MF and the development of JAK2 inhibitors to treat MF. Recent Food and Drug Administration (FDA) approval of the first JAK2 inhibitor, ruxolitinib, signaled a new era for treatment of MF. Additional JAK2 inhibitors, such as SAR302503, may become commercially available in the near future, and their distinct pharmacologic and efficacy profiles will help determine their use across the patient population. Data on JAK2 inhibitors, their role in an evolving treatment paradigm, and future directions for treatment of MF are discussed.
AB - Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm diagnosed de novo or developed from essential thrombocythemia (ET) or polycythemia vera (PV). Average survival of a patient with MF is 5-7 years. Disease complications include fatigue, early satiety, pruritus, painful splenic infarcts, infections and leukemic transformation. Allogeneic hematopoietic stem cell transplant (HSCT) is the only potentially curative option for MF, but carries a risk of treatment-related mortality and is reserved for the few high-risk patients fit enough to endure the procedure. Other traditional therapies are palliative and supported by few randomized, controlled trials; thus, novel treatment strategies are needed. Discovery of the Janus kinase 2 (JAK2) gain-of-function mutation, JAK2V617F, in the majority (50-60%) of patients with MF led to increased understanding of the biology underlying MF and the development of JAK2 inhibitors to treat MF. Recent Food and Drug Administration (FDA) approval of the first JAK2 inhibitor, ruxolitinib, signaled a new era for treatment of MF. Additional JAK2 inhibitors, such as SAR302503, may become commercially available in the near future, and their distinct pharmacologic and efficacy profiles will help determine their use across the patient population. Data on JAK2 inhibitors, their role in an evolving treatment paradigm, and future directions for treatment of MF are discussed.
KW - Dysplasias
KW - JAK2V617F
KW - Janus kinase 2 (JAK2)
KW - Myelofibrosis
KW - Myeloid leukemias
KW - Myeloproliferative disorders
KW - Signaling therapies
UR - http://www.scopus.com/inward/record.url?scp=84872049837&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84872049837&partnerID=8YFLogxK
U2 - 10.3109/10428194.2012.710905
DO - 10.3109/10428194.2012.710905
M3 - Review article
VL - 54
SP - 242
EP - 251
JO - Leukemia and Lymphoma
JF - Leukemia and Lymphoma
SN - 1042-8194
IS - 2
ER -