[Eculizumab in patients with paroxysmal nocturnal hemoglobinuria: a real-world study in China].
Zhonghua Xue Ye Xue Za Zhi
; 45(2): 184-189, 2024 Feb 14.
Article
in Zh
| MEDLINE
| ID: mdl-38604796
ABSTRACT
Objective:
To evaluate the efficacy and safety of eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.Methods:
Data from PNH patients who received at least 3 months of full-dose eculizumab and were followed for at least 3 months between December 2022 and July 2023 were retrospectively collected. We evaluated changes in clinical and laboratory parameters after 1, 2, 3, and 6 months of eculizumab treatment. The rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and the occurrence of adverse reactions were also monitored.Results:
The study included nine patients, six males and three females, with a median age of 54 (28-69) years. 5 of the patients had classic PNH, while 4 had PNH/AA. The number of episodes of hemoglobinuria was 5 (1-25) per month before eculizumab. 4 patients required blood transfusion, 5 had thrombosis and one had renal impairment before eculizumab. The median time to eculizumab was 6 (3-7) months and the followup period was 3 (3-6) months after treatment. The number of episodes of hemoglobinuria following eculizumab was 0 (0-1). During the followup period, no additional thrombotic events occurred. LDH at any time after eculizumab was lower than at baseline, and some patients' HGB increased. All transfused patients became transfusion-independent after receiving eculizumab. The FACIT-Fatigue score improved by an average of 17.3 points following treatment. 2 patients developed BTH and improved with symptomatic treatment. There were three adverse events that caused mild symptoms. There are no serious adverse events or deaths.Conclusion:
Eculizumab can effectively control the hemolytic-related symptoms of PNH in China, reducing the need for blood transfusions to some extent, while also demonstrating a higher safety profile.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Thrombosis
/
Antibodies, Monoclonal, Humanized
/
Hemoglobinuria, Paroxysmal
Limits:
Aged
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Female
/
Humans
/
Male
/
Middle aged
Country/Region as subject:
Asia
Language:
Zh
Journal:
Zhonghua Xue Ye Xue Za Zhi
Year:
2024
Document type:
Article
Affiliation country: