Diagnosisandtreatment of paroxysmal nocturnal haemoglobinuria

Aim

To evaluate the results of screening for paroxysmal nocturnal haemoglobinuria (PNH) and to assess the efficacy and safety of eculizumab in patients with PNH.

Material and methods

.Screening for PNH clone was conducted by cytofluorometric method using FLAER in peripheral blood of 40 patients.

Results

PNH was diagnosed in 7 patients (5 female and 2 male, mean age was 41.9±10.0 years). There were three patients with the classic PNH, two patients with PNH in the setting of the aplastic anemia and two patients with the subclinical PNH. Three patients with the classic PNH were treated with eculizumab for the median duration of 20 months. The treatment with eculizumab resulted in a significant suppression of hemolysis in all three cases. There were no thrombotic events. Renal function remained normal in all three patients. Transfusion requirement was minimum in two of three patients. Treatment with eculizumab was not associated with serious adverse events.

Conclusion

Eculizumab demonstrated a high efficacy in the treatment of PNH. It suppresses the symptoms of intravascular hemolysis, significantly reduces the dependence on blood transfusions, prevents thrombotic complications.

Key words

Paroxysmal nocturnal hemoglobinuria, eculizumab, screening.