Clinical features of 76 Chinese patients with paroxysmal nocturnal haemoglobinuria / 中华血液学杂志

ABSTRACT

<p><b>OBJECTIVE</b>To further analyse the relationship between the new technology and clinical characteristics in paroxysmal nocturnal haemoglobinuria (PNH) patients, and summarize the data of PNH during the past 15 years in China.</p><p><b>METHODS</b>76 consecutive patients with PNH diagnosed in Peking Union Medical Colleague Hospital from 1997 - 2011 retrospectively.</p><p><b>RESULTS</b>Most of the patients were diagnosed based on flow cytometric data. There were 46 male and 30 female patients. The median age at diagnosis was 40 (10 - 74). 46 (60.5%) patients presented with classical PNH, 16 (21.1%) pancytopenia, and 14 (18.4%) thrombosis. Anatomic locations of first thrombosis were intra abdominal in 7 patients, lower extremities in 3 patients, intracerebral in 2 patients, and pulmonary thrombosis in 2 patients. The size of PNH clone at first determination (shown by CD55 and CD59 negative percentage) was (61.23 ± 27.47)% and (60.24 ± 25.59)% on neutrophils; (34.24 ± 25.50)% and (32.22 ± 23.12)% on erythrocytes, respectively. The mean LDH level was (1199.2 ± 893.5) U/L. In our cohort, 13(17.0%) patients suffered from renal deficiency, 12 (15.8%) patients cholecystolithiasis, 10 (13.2%) patients hemorrhage and 9 (11.8%) patients infections. In a median of 7-year (range 0.5 - 20 years) follow-up (68 patients), 2 (2.9%) patients developed into myelodysplastic syndromes/ acute myeloid leukemia, 1(1.5%) patient ovary cancer, 11(14.5%) patients died. Patients with thrombosis had higher percentage of CD59 negative neutrophils \[(73.45 ± 22.32)%\] compared with those without thrombosis \[(58.3 ± 20.2)%\] (P < 0.05).</p><p><b>CONCLUSIONS</b>The cohort had higher percentage of classical hemolysis, thrombosis and renal dysfunction compared with previous reports in China. Patients with thrombotic events had higher percentages of CD55 and CD59 negative neutrophils.</p>