Gestão Prática das Citopenias no Doente com Mielofibrose Adaptada à Realidade em Portugal

Cytopenias are a common clinical manifestation of myelofibrosis, particularly anemia. Although correcting cytopenias is essential to improve prognosis and quality of life of patients with myelofibrosis, there are few effective therapeutic options. Furthermore, there are no national guidelines for the management of cytopenias in myelofibrosis. To address this gap, a panel composed of six hematologists proposes an approach for patients with myelofibrosis and cytopenias adapted to clinical practice in Portugal, based on scientific evidence and clinical experience. Firstly, the etiology of the anemia should be identified. In patients with myelofibrosis-related anemia who have splenomegaly and/or constitutional symptoms, the use of Janus kinase inhibitors should be prioritized: momelotinib as monotherapy, or ruxolitinib in combination with an erythropoiesis-stimulating agent or danazol, depending on erythropoietin levels. In anemic patients without splenomegaly or constitutional symptoms, treatment with an erythropoiesis-stimulating agent, danazol, a combination of an immunomodulator with corticosteroid, or momelotinib, should be considered. In patients with treatment-induced anemia, the use of a Janus kinase inhibitor (with possible dose reduction in the case of ruxolitinib) in combination with an erythropoiesis-stimulating agent or danazol, or momelotinib as monotherapy should be considered. Thrombocytopenic patients eligible for Janus kinase inhibitors may receive ruxolitinib at a reduced dose, or momelotinib, the latter of which does not require dose reduction for thrombocytopenia. In thrombocytopenic patients without splenomegaly, constitutional symptoms, or anemia, the approach consists of monitoring or treatment with danazol, corticosteroids, or a combination of immunomodulator and corticosteroid, according to platelet count and hemorrhagic diathesis. Transfusion should be considered in patients with severe anemia or thrombocytopenia, diathesis, or the need for surgery. Symptomatic and analytical improvement, reduction of splenomegaly, and independence from or a significant decrease in transfusion support indicate a response to treatment. Treatment switch is warranted in cases of toxicity, failure, loss of clinical or hematological response after initial response, or disease progression. The implementation of these recommendations will help to optimize care for patients with myelofibrosis and cytopenias, improving clinical outcomes.