• 作者：Montserrat Ló ; pez Rubio^a ; ^{montserratlr@terra.es} ; Marta Morado^b ; Ana Gaya^c ; Dora Alonso Rosa^d ; Emilio Ojeda^e ; Juan Antonio Muñ ; oz^f ; Begoñ ; a Pé ; rez de Mendiguren^g ; Marí ; a Dolores Monteagudo^h ; José ; Marí ; a Durá ; nⁱ ; Rosa Marí ; a Fisac^j ; Damiá ; n Moreno^k ; Ana Marí ; a Villegas^l
• 关键词：Hemoglobinuria paroxí ; stica nocturna ; Eculizumab
• 刊名：Medicina Cl铆nica
• 出版年：2011
• 期刊代码：pca254_00257753
• 类别：med
• 出版时间：11 June 2011
• 卷：137
• 期：1
• 页码：8-13
• 文件大小：185 K

Background and objectives

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal disease characterized by complement-mediated hemolysis, bone marrow failure and thrombosis. Eculizumab is a humanized monoclonal antibody that blocks the cytolytic component of the complement system by binding to complement C5.

Material and Methods

We report the results of eculizumab treatment in 25 PNH patients from different centers in Spain. Statistical analysis was perfomed with a SPSS v15.0 software.

Results

Fifty-eight per cent of the patients achieved transfusional independence after a median of 14 months. Transfusion requirements were reduced in 60%of the remaining cases. Fatigue resolved in 96%of the patients and smooth muscle dystony-related symptoms in all cases. A single case of treatment-related infection was observed.

Conclusions

Eculizumab controls effectively hemolysis and greatly improves clinical symptoms. The drug is safe and well tolerated, without significant adverse effects except meningococcal infection. Patients with suboptimal response to treatment must be assessed for bone marrow insufficiency and extravascular haemolysis.