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Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs.
PURPOSE Myeloproliferative neoplasm (MPN) symptoms are troublesome to patients, and alleviation of this burden represents a paramount treatment objective in the development of MPN-directed therapies.Expand
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Thalidomide versus placebo in myeloid metaplasia with myelofibrosis: a prospective, randomized, double-blind, multicenter study.
BACKGROUND AND OBJECTIVES In non-randomized studies, thalidomide appeared to be effective in myeloid metaplasia with myelofibrosis (MMM). We compared thalidomide to placebo for treatment of anemia inExpand
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Ruxolitinib Before Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Patients With myelofibrosis : a Preliminary Descriptive Report Of The JAK ALLO Study, a Phase II Trial Sponsored By
Ruxolitinib (RUXO) is a JAK inhibitor recently approved in France in patients (pts) with myelofibrosis (MF) because of its efficacy on splenomegaly and constitutional symptoms. Although noExpand
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Practical application and clinical impact of the WHO histopathological criteria on bone marrow biopsy for the diagnosis of essential thrombocythemia versus prefibrotic primary myelofibrosis
Brousseau M, Parot‐Schinkel E, Moles M‐P, Boyer F, Hunault M & Rousselet M‐C
(2010) Histopathology56, 758‐767
Practical application and clinical impact of the WHO histopathological criteria on boneExpand
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High response rate and improved exercise capacity and quality of life with a new regimen of darbepoetin alfa with or without filgrastim in lower-risk myelodysplastic syndromes: a phase II study by
Darbepoetin (DAR), with or without granulocyte colony-stimulating factor (G-CSF), has proved effective in treating anemia in patients with lower-risk myelodysplastic syndrome (MDS), but its effectsExpand
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Conventional therapeutic options have limited impact on MPN symptoms: Insights from a prospective analysis of the MPN-SAF TSS
Background. Symptom burden in MPNs is severe and a risk factor for mortality in some disease subtypes (Blood 2010;115(9):1703-8). A recent trial comparing JAK2 treatment to best available therapyExpand
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Symptomatic Profiles of Patients With Polycythemia Vera: Implications of Inadequately Controlled Disease.
PURPOSE Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) associated with disabling symptoms and a heightened risk of life-threatening complications. Recent studies have demonstrated theExpand
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Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients
Immunogenicity of recombinant human acid-alpha glucosidase (rhGAA) in enzyme replacement therapy (ERT) is a safety and efficacy concern in the management of late-onset Pompe disease (LOPD). However,Expand
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Clinical and biological characterization of MPN patients harboring two driver mutations, a French intergroup of myeloproliferative neoplasms (FIM) study
To the Editor: Mutations in “driver” genes (JAK2, MPL, or CALR) are found in the vast majority of Philadelphia-negative myeloproliferative neoplasms (MPN): JAK2V617F or exon 12 mutations inExpand
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Thrombin generation and procoagulant phospholipids in patients with essential thrombocythemia and reactive thrombocytosis
Thrombocytosis is a commonly encountered clinical scenario and can be either a secondary process (reactive thrombocytosis), or due to clonal disorder (i.e., essential thrombocythemia). ThisExpand
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