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Objective: 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., Methods: 76 consecutive patients with PNH diagnosed in Peking Union Medical Colleague Hospital from 1997 - 2011 retrospectively., Results: 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)., Conclusions: 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.

Objective: To evaluate the efficacy of porcine anti-human lymphocyte globulin (P-ALG) plus cyclosporine A (CsA) therapy for severe aplastic anemia (SAA)., Methods: Forty-eight SAA patients (31 males, 17 females) including 17 very severe aplastic anemias (vSAA) were treated with ALG plus CsA between 1999 to 2009 in our hospital and the outcomes were analyzed retrospectively for early mortality, response rate and quality, survival rate, toxicity and complications., Results: The median age was 28 (13 - 64) years. The interval from diagnosis to treatment was 45 days. The median neutrophil count at diagnosis was 0.178 × 10(9)/L. Overall response was 83.3% (54.2% complete, 29.2% partial) with a median time of 90 (23 - 380) days. 10.4% died of infection within 30 days mainly of fungi infection. Only 1 patient relapsed 2 years after treatment. No clonal disease was found. The 1.5-year survival rate was 87.5%. vSAAs had less response, higher early mortality and less survival (64.7%, 29.4% and 51.8%, respectively) compared to that of SAA (93.5%, 0, 100%, respectively, P < 0.05). Grouped patients with different age, gender, intervals between diagnosis and treatment and pre-existing infections had similar response. The main side effects were fever and skin rash (52.1%), serum sickness (16.7%), impaired liver function (60.4%) and hemorrhage (2.1%). No treatment-related mortality was found., Conclusion: P-ALG plus CsA is an ideal and well tolerated treatment for SAA but not for vSAA.