Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria

Hematology, Transfusion and Cell Therapy

Aderson da Silva Araújo¹  Sandra Fátima Menosi Gualandro²  Clarisse Lopes de Castro Lobo³  Fernando Ferreira Costa⁴  Rodolfo D. Cançado⁵  Celso Arrais⁶  Alex Freire Sandes⁷  Sara Teresinha Olalla Saad⁸  Maria Stella Figueiredo⁹ 

[1] Corresponding author at: Hemocentro da Santa Casa de São Paulo, Rua Marques de Itu, 579, Vila Buarque, São Paulo, SP, CEP 01223-001, Brazil.;Hospital Sírio-Libanês, São Paulo, SP, Brazil;Universidade Federal de São Paulo (UNIFESP), Sao Paulo, SP, Brazil;Escola Paulista de Medicina, Universidade Federal de São Paulo (Unifesp), São Paulo, SP, Brazil;Faculdade de Ciências Médicas da Santa Casa de São Paulo (FCMSCSP), São Paulo, SP, Brazil;Fleury Medicina e Saúde, São Paulo, SP, Brazil;Hospital de Hematologia e Hemoterapia de Pernambuco (HEMOPE), Recife, PE, Brazil;Instituto de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO), Rio de Janeiro, RJ, Brazil;Universidade Federal de São Paulo (UNIFESP), Sao Paulo, SP, Brazil;

关键词: Hemoglobinuria;    Paroxysmal;    Consensus;    Eculizumab;   

DOI  :  

来源: DOAJ

【 摘 要 】

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.

【 授权许可】

Unknown