Objective: To observe the real-world efficacy and safety of eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.

Methods: The baseline, symptoms, laboratory tests, and adverse reactions before and after treatment of PNH patients treated with eculizumab in the West China Blood Alliance from January 2024 to the present were collected.

Results: A total of 12 PNH patients were included, consisting of 5 males and 7 females, with a median age of 37 years. Three patients had concomitant aplastic anemia, 7 require blood transfusion, 2 had a history of thromboembolic disease, and 4 had chronic renal insufficiency. None of the 11 patients who were screened with C5 R885 mutation was positive. The proportion of patients with monthly income of less than 5000 yuan was 72.7%. The median Hb was 53.5 g/L, the median reticulocyte count was 12.21%, and the LDH level was 2146 IU/L before treatment. The median percentage of CD55- and CD59-negative neutrophils were 96.8% and 93.8%, respectively. And the percentages of CD55- and CD59-negative red cells were 42.4% and 43.5%, respectively, the median percentage of FLARE-negative granulates was 95.3%, and the median percentage of FLARE-negative monocytes was 91.2%. There were no patients with C5 R885H gene mutation. The median duration of treatment was 14 weeks. After 4 weeks of treatment, the median Hb was 71 g/L, the count of reticulocyte was 7.61%, and the LDH level was 504 IU/L; after 8 weeks of treatment, the median Hb was 80.5 g/L, the reticulocyte count was 9.55%, and the LDH level was 275 IU/L. Five patients became transfusion-independent after 8 weeks, and the frequency of blood transfusion was reduced in 2 patients. Cr levels decreased in 2 patients. The proportion of patients with mildly elevated bilirubin after the last follow-up compared to the lowest value was 85.7%. After treatment, 83.3% of patients with positive Coombs C3 were found. During the treatment, there was one case of respiratory infection and two cases of headache as mild adverse events. One case with suspected intracranial infection was reported as a serious adverse event. No new thrombotic events occurred.

Conclusion: Eculizumab has good control over anemia and hemolysis-related symptoms in PNH patients, and its safety is good. After being included in the medical insurance system, it is applicable to PNH patients in China.

Disclosures

No relevant conflicts of interest to declare.

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2024
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