Abstract
Objective: To emphasize the monitoring of PNH by flow cytometric immunophenotyping (CMF) and to evaluate the efficacy of treatment with the eculizumab monoclonal antibody. Methodology: The study is a retrospective analysis of patient records data sent to a reference institute for hematological diseases in the state of Goiás for diagnosis and monitoring of PNH from January 2010 to December 2017. Results: Of the 31 patients diagnosed. with PNH, 5 were selected with complete data in the medical records. In patient 1, there was a significant improvement with treatment, with an increase in the relative number of erythrocyte markers (CD55 and CD59) from 22.7% to 71.7%. For patient 3, the mean variation of markers went from 0.5% to 5.2%. In patient 5 the average variation was between 12.6% and 36.9%. Eculizumab reduced hemolytic status, improving symptoms and stabilizing hemolysis test results. Conclusion: CMF immunophenotyping is a gold standard in the investigation of PNH for evaluating the expression of GPI-anchored proteins with high sensitivity and specificity. This study is the first to demonstrate the effectiveness of eculizumab without disease progression.