Article

Philadelphia-Negative Classical Myeloproliferative Neoplasms: Critical Concepts and Management Recommendations From European LeukemiaNet

Unit of Clinical Epidemiology/Center for the Study of Myelofibrosis, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico S. Matteo, Viale Golgi 19, 27100 Pavia, Italy.
Journal of Clinical Oncology (Impact Factor: 18.43). 02/2011; 29(6):761-70. DOI: 10.1200/JCO.2010.31.8436
Source: PubMed

ABSTRACT We present a review of critical concepts and produce recommendations on the management of Philadelphia-negative classical myeloproliferative neoplasms, including monitoring, response definition, first- and second-line therapy, and therapy for special issues. Key questions were selected according the criterion of clinical relevance. Statements were produced using a Delphi process, and two consensus conferences involving a panel of 21 experts appointed by the European LeukemiaNet (ELN) were convened. Patients with polycythemia vera (PV) and essential thrombocythemia (ET) should be defined as high risk if age is greater than 60 years or there is a history of previous thrombosis. Risk stratification in primary myelofibrosis (PMF) should start with the International Prognostic Scoring System (IPSS) for newly diagnosed patients and dynamic IPSS for patients being seen during their disease course, with the addition of cytogenetics evaluation and transfusion status. High-risk patients with PV should be managed with phlebotomy, low-dose aspirin, and cytoreduction, with either hydroxyurea or interferon at any age. High-risk patients with ET should be managed with cytoreduction, using hydroxyurea at any age. Monitoring response in PV and ET should use the ELN clinicohematologic criteria. Corticosteroids, androgens, erythropoiesis-stimulating agents, and immunomodulators are recommended to treat anemia of PMF, whereas hydroxyurea is the first-line treatment of PMF-associated splenomegaly. Indications for splenectomy include symptomatic portal hypertension, drug-refractory painful splenomegaly, and frequent RBC transfusions. The risk of allogeneic stem-cell transplantation-related complications is justified in transplantation-eligible patients whose median survival time is expected to be less than 5 years.

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    • "Based on European Leukaemia Net recommendations (Barbui et al, 2011), in the absence of sufficient data to recommend any treatment, paediatricians individually tailored the treatment to each patient. "
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    British Journal of Haematology 02/2015; 169(4). DOI:10.1111/bjh.13329 · 4.96 Impact Factor
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    • "There was no particular exclusion criterion. Peg-IFNa-2a was prescribed off-label in accordance with local practice and following French and International recommendations (Barbui et al, 2011). The study was approved by the FIM institutional review board. "
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    • "Symptom response and QOL are relevant in evaluating newer treatments and have indeed been incorporated into treatment response criteria established through the European LeukemiaNet (Barosi et al, 2009). Current therapies and risk stratification for ET and PV as discussed above have changed little over the past two decades (summarized in Table I; Barbui et al, 2011). This has reflected a lack of understanding of the pathogenesis of the disease that could be translated to design newer therapies. "
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