Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria.

Autor:	Cançado RD; Faculdade de Ciências Médicas da Santa Casa de São Paulo (FCMSCSP), São Paulo, SP, Brazil. Electronic address: rdcan@uol.com.br., Araújo ADS; Hospital de Hematologia e Hemoterapia de Pernambuco (HEMOPE), Recife, PE, Brazil., Sandes AF; Fleury Medicina e Saúde, São Paulo, SP, Brazil; Universidade Federal de São Paulo (UNIFESP), Sao Paulo, SP, Brazil., Arrais C; Universidade Federal de São Paulo (UNIFESP), Sao Paulo, SP, Brazil; Hospital Sírio-Libanês, São Paulo, SP, Brazil., Lobo CLC; Instituto de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO), Rio de Janeiro, RJ, Brazil., Figueiredo MS; Escola Paulista de Medicina, Universidade Federal de São Paulo (Unifesp), São Paulo, SP, Brazil., Gualandro SFM; Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HCFMUSP), São Paulo, SP, Brazil., Saad STO; Centro de Hematologia e Hemoterapia, Universidade Estadual de Campinas, (HEMOCENTRO-UNICAMP), Campinas, SP, Brazil; Instituto Nacional de Ciência e Tecnologia do Sangue (INCT-S), Campinas, SP, Brazil., Costa FF; Centro de Hematologia e Hemoterapia, Universidade Estadual de Campinas, (HEMOCENTRO-UNICAMP), Campinas, SP, Brazil; Instituto Nacional de Ciência e Tecnologia do Sangue (INCT-S), Campinas, SP, Brazil.
Jazyk:	angličtina
Zdroj:	Hematology, transfusion and cell therapy [Hematol Transfus Cell Ther] 2021 Jul-Sep; Vol. 43 (3), pp. 341-348. Date of Electronic Publication: 2020 Jul 06.
DOI:	10.1016/j.htct.2020.06.006
Abstrakt:	Paroxysmal nocturnal hemoglobinuria is a chronic, multi-systemic, progressive and life-threatening disease characterized by intravascular hemolysis, thrombotic events, serious infections and bone marrow failure. Paroxysmal nocturnal hemoglobinuria results from the expansion of a clone of hematopoietic cells that due to an inactivating mutation of the X-linked gene PIG-A are deficient in glycosylphosphatidylinositol-linked proteins. Early diagnosis, using flow cytometry performed on peripheral blood, the gold standard test to confirm the diagnosis of paroxysmal nocturnal hemoglobinuria, is essential for improved patient management and prognosis. The traditional therapy for paroxysmal nocturnal hemoglobinuria includes blood transfusion, anti-thrombosis prophylaxis or allogeneic bone marrow transplantation. The treatment that has recently become available is the complement blockade by the anti-C5 monoclonal antibody eculizumab. In this consensus, we are aiming to review the diagnosis and treatment of the paroxysmal nocturnal hemoglobinuria patients, as well as the early recognition of its systemic complications. These procedures express the opinions of experts and have been based on the best available evidence and international guidelines, with the purpose of increasing benefits and reducing harm to patients. (Copyright © 2020. Published by Elsevier España, S.L.U.)
Databáze:	MEDLINE
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