CLINICAL TRIALS AND OBSERVATIONS Brief report Disease characteristics and clinical outcome in young adults with essential thrombocythemia versus early/prefibrotic primary myelofibrosis

نویسندگان

  • Tiziano Barbui
  • Jürgen Thiele
  • Alessandra Carobbio
  • Francesco Passamonti
  • Elisa Rumi
  • Maria Luigia Randi
  • Irene Bertozzi
  • Alessandro M. Vannucchi
  • Heinz Gisslinger
  • Bettina Gisslinger
  • Guido Finazzi
  • Marco Ruggeri
  • Francesco Rodeghiero
  • Alessandro Rambaldi
  • Naseema Gangat
چکیده

1Ospedali Riuniti di Bergamo, Bergamo, Italy; 2University of Cologne, Cologne, Germany; 3Ospedale di Varese, Varese, Italy; 4University of Pavia, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico, Policlinico San Matteo, Pavia, Italy; 5University of Padua, Padua, Italy; 6University of Florence, Florence, Italy; 7Medical University of Vienna, Vienna, Austria; 8Institute of Pathology, Medical University of Vienna, Vienna, Austria; 9S. Bortolo Hospital, Vicenza, Italy; and 10Mayo Clinic, Rochester, MN

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CLINICAL TRIALS AND OBSERVATIONS Thrombocythemia and polycythemia in patients younger than 20 years at diagnosis: clinical and biologic features, treatment, and long-term outcome

Sixty-four patients < 20 years of age, investigated for a suspicion of Philadelphianegative myeloproliferative disease (MPD), were retrospectively evaluated to characterize the different forms and to examine the treatments used and longterm outcome. JAK2 mutations, endogenous erythroid colony growth, and clonality were investigated in 51 children. Mutations of thrombopoietin, the thrombopoietin...

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CLINICAL TRIALS AND OBSERVATIONS Refractory anemia with ringed sideroblasts associated with marked thrombocytosis (RARS-T), another myeloproliferative condition characterized by JAK2 V617F mutation

JAK2 V617F mutation recently was identified as a pathogenic factor in typical chronic myeloproliferative diseases (CMPD). Some forms of myelodysplastic syndromes (MDS) show a significant overlap with CMPD (classified as MDS/MPD), but the diagnostic assignment may be challenging. We studied blood or bone marrow from 270 patients with MDS, MDS/MPD, and CMPD for the presence of JAK2 V617F mutation...

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Regular Article CLINICAL TRIALS AND OBSERVATIONS Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: the ANAHYDRET Study, a randomized controlled trial

Medical University of Vienna, Department of Internal Medicine I, Division of Hematology and Blood Coagulation, Vienna, Austria; Institute of Hematology, Clinical Center of Serbia, Belgrade, Serbia; Silesian Medical University, Department of Hematology and BMT, Katowice, Poland; University Hospital Brno, Department of Clinical Hematology, Brno, Czech Republic; Institute of Pathology, University ...

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Molecular Pathogenesis and Clinical Significance of Driver Mutations in Primary Myelofibrosis: A Review

Primary myelofibrosis (PMF) is a rare chronic BCR-ABL1-negative myeloproliferative neoplasm characterized by progressive bone marrow fibrosis, inefficient hematopoiesis, and shortened survival. The clinical manifestations of PMF include splenomegaly, consequent to extramedullary hematopoiesis, pancytopenias, and an array of potentially debilitating constitutional symptoms. The diagnosis is base...

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CLINICAL TRIALS AND OBSERVATIONS Hydroxyurea in essential thrombocythemia: rate and clinical relevance of responses by European LeukemiaNet criteria

A definition of response by cytoreductive therapy in essential thrombocythemia was recently provided by the European LeukemiaNet (ELN). Complete, partial, or no clinicohematologic responses were defined on the bases of platelet count, disease-related symptoms, spleen size, and white blood cell count. To provide estimates and clinical correlation of responses according to these criteria, we retr...

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تاریخ انتشار 2012