Management of Myelofibrosis: from Diagnosis to New Target Therapies.

Opinion Statement: Myelofibrosis (MF) is a clonal disorder of the pluripotent hematopoietic stem cell, whose clinical manifestations can be extremely heterogeneous, including cytopenias, organomegaly, constitutional symptoms, and cachexia. Median survival ranges from approximately 3.5 to 5.5 years;...

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Publicado en:Current Treatment Options in Oncology Vol. 21; no. 6; pp. 1 - 15
Autores principales: Iurlo, Alessandra, Cattaneo, Daniele, Bucelli, Cristina
Formato: review Journal Article
Publicado: Springer Nature Jun2020
Acceso en línea:Ver este registro en EBSCOhost
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      pub: Springer Nature
      place: New York, New York
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        atl: Management of Myelofibrosis: from Diagnosis to New Target Therapies.
      aug:
        au:
          Iurlo, Alessandra
          Cattaneo, Daniele
          Bucelli, Cristina
        affil: Hematology Division, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Francesco Sforza 35, 20122, Milan, Italy
      sug:
        subj:
          Primary Myelofibrosis Diagnosis
          Primary Myelofibrosis Therapy
          Treatment Outcomes
          Drug Therapy Methods
          Primary Myelofibrosis Etiology
          Disease Susceptibility
          Disease Management
          Combined Modality Therapy Methods
          Protein Kinase Inhibitors Adverse Effects
          Protein Kinase Inhibitors Therapeutic Use
          Drug Therapy, Combination
          Drug Therapy Adverse Effects
          Protein Kinase Inhibitors Administration and Dosage
          Algorithms
          Impact of Events Scale
          Scales
          Ferrans and Powers Quality of Life Index
      ab: Opinion Statement: Myelofibrosis (MF) is a clonal disorder of the pluripotent hematopoietic stem cell, whose clinical manifestations can be extremely heterogeneous, including cytopenias, organomegaly, constitutional symptoms, and cachexia. Median survival ranges from approximately 3.5 to 5.5 years; while the most frequent cause of death is the evolution to acute myeloid leukemia, also other conditions such as progression without transformation, complications due to cytopenias including infections or bleeding, and cardiovascular events may be fatal. Myelofibrosis is still orphan of curative treatments: allogeneic hematopoietic stem cell transplant (HSCT), the only therapeutic approach that has clearly demonstrated an impact on disease progression, is associated with relevant morbidity and mortality and only a minority of patients is eligible for such an intensive procedure. While the discovery of the crucial role of JAK2 mutations and the consequent clinical use of JAK inhibitors has led to a dramatic improvement of symptoms control and quality of life, yet these drugs do not significantly modify the natural history of the disease. A better understanding of the molecular pathogenesis will hopefully foster the development of new targeted therapies aimed at improving MF prognosis. Herein, we review the most recent advances about JAK inhibitors and other molecules which are under investigation.
      pubtype: Academic Journal
      doctype:
        review
        Journal Article
      ougenre: Article
    language: English
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