Journal of clinical laboratory analysis · 2026
Analysis of the Recovery of Immune Status in the Patients With Paroxysmal Nocturnal Hemoglobinuria After Treatment With C5 Complement Inhibitor.
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Liu Hui, Che Mengting, Li Liyan, Wang Honglei, Wang Chaomeng, Wu Junshu, Chen Yingying, Liu Zhaoyun, Li Lijuan, Fu Rong
Аннотация
BACKGROUND: Currently, C5 complement inhibitors serve as the first-line therapy for classic paroxysmal nocturnal hemoglobinuria (PNH), effectively controlling hemolysis and enhancing patients' quality of life. However, the impact of this treatment on patients' immune status remains unclear.
METHODS: A retrospective analysis was conducted on 27 PNH patients treated with C5 complement inhibitors, focusing on the dynamic changes and recovery of key immune cell subsets, including T lymphocytes, B lymphocytes, natural killer (NK) cells, and dendritic cells (DCs).
RESULTS: Among the 27 patients, 70.3% (19/27) achieved transfusion independence. Treatment responses were categorized as follows: complete response (7.4%, 2/27), major response (3.7%, 1/27), good response (25.9%, 7/27), partial response (22.2%, 6/27), minor response (29.6%, 8/27), and non-response (11.1%, 3/27). Before treatment, the proportion of CD4+ T lymphocytes was significantly lower in PNH patients than in normal controls [(38.96 ± 8.270)% vs. (48.57 ± 5.760)%, p < 0.001], and it gradually recovered toward normal levels at 12 months [(42.48 ± 5.623)%] and 24 months [(43.71 ± 6.204)%] post-treatment. In contrast, the percentage of CD8+ T lymphocytes was markedly elevated in pre-treatment patients compared with controls [(49.38 ± 7.259)% vs. (41.75 ± 4.744)%, p < 0.01], and it returned to near-normal levels at 12 months [(42.97 ± 7.074)%]. The proportion of B lymphocytes was significantly reduced in pre-treatment PNH patients [(6.535 ± 1.854)% vs. (12.70 ± 2.256)% in controls, p < 0.001] and partially recovered at 6, 12, and 24 months post-treatment [(8.266 ± 1.756)%, (8.626 ± 1.649)%, and (8.559 ± 1.697)% respectively], though it did not reach normal levels. No significant changes were observed in Treg cells, NK cells, and DCs after treatment.
CONCLUSIONS: This is the first study to systematically analyze immune status alterations following C5 complement inhibitor treatment in PNH patients, suggesting that the complement system may be involved in the development of immune escape mechanisms in PNH.
METHODS: A retrospective analysis was conducted on 27 PNH patients treated with C5 complement inhibitors, focusing on the dynamic changes and recovery of key immune cell subsets, including T lymphocytes, B lymphocytes, natural killer (NK) cells, and dendritic cells (DCs).
RESULTS: Among the 27 patients, 70.3% (19/27) achieved transfusion independence. Treatment responses were categorized as follows: complete response (7.4%, 2/27), major response (3.7%, 1/27), good response (25.9%, 7/27), partial response (22.2%, 6/27), minor response (29.6%, 8/27), and non-response (11.1%, 3/27). Before treatment, the proportion of CD4+ T lymphocytes was significantly lower in PNH patients than in normal controls [(38.96 ± 8.270)% vs. (48.57 ± 5.760)%, p < 0.001], and it gradually recovered toward normal levels at 12 months [(42.48 ± 5.623)%] and 24 months [(43.71 ± 6.204)%] post-treatment. In contrast, the percentage of CD8+ T lymphocytes was markedly elevated in pre-treatment patients compared with controls [(49.38 ± 7.259)% vs. (41.75 ± 4.744)%, p < 0.01], and it returned to near-normal levels at 12 months [(42.97 ± 7.074)%]. The proportion of B lymphocytes was significantly reduced in pre-treatment PNH patients [(6.535 ± 1.854)% vs. (12.70 ± 2.256)% in controls, p < 0.001] and partially recovered at 6, 12, and 24 months post-treatment [(8.266 ± 1.756)%, (8.626 ± 1.649)%, and (8.559 ± 1.697)% respectively], though it did not reach normal levels. No significant changes were observed in Treg cells, NK cells, and DCs after treatment.
CONCLUSIONS: This is the first study to systematically analyze immune status alterations following C5 complement inhibitor treatment in PNH patients, suggesting that the complement system may be involved in the development of immune escape mechanisms in PNH.