| Autor: |
Fishman J; Apellis Pharmaceuticals, Inc., Waltham, MA 02451, USA., Kuranz S; TriNetX, LLC, Cambridge, MA 02140, USA., Yeh MM; Apellis Pharmaceuticals, Inc., Waltham, MA 02451, USA., Brzozowski K; TriNetX, LLC, Cambridge, MA 02140, USA., Chen H; TriNetX, LLC, Cambridge, MA 02140, USA. |
| Jazyk: |
angličtina |
| Zdroj: |
Hematology reports [Hematol Rep] 2023 Apr 21; Vol. 15 (2), pp. 266-282. Date of Electronic Publication: 2023 Apr 21. |
| DOI: |
10.3390/hematolrep15020027 |
| Abstrakt: |
Paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematologic disorder, can be treated with C5 inhibitors (C5i) such as eculizumab or ravulizumab. This retrospective study is the first to describe real-world treatment patterns and changes in hematologic PNH-monitoring laboratory tests among C5i-treated US patients. Data were extracted from TriNetX Dataworks Network and included patients with a PNH diagnosis between 1 January 2010, and 20 August 2021. Patients were stratified into three cohorts based on their C5i usage: eculizumab, ravulizumab (prior eculizumab), and ravulizumab (eculizumab naïve). Hematological markers (hemoglobin [Hb], lactate dehydrogenase [LDH], and absolute reticulocyte count [ARC]) and relevant clinical events (e.g., breakthrough hemolysis [BTH], complement-amplifying conditions [CAC], thrombosis, infection, and all-cause mortality) were captured any time within 12 months post-index treatment. Of the 143 (eculizumab), 43 (ravulizumab, prior eculizumab), and 33 (ravulizumab, eculizumab naïve) patients, mean age across cohorts was 42-51 years, 55-61% were female, 63-73% were White, and 33-40% had aplastic anemia. Among all cohorts 12 months post-C5i treatment, 50-82% remained anemic, 8-32% required ≥1 transfusion, and 13-59% had BTH, of which 33%-54% had CACs. Additionally, thrombosis was seen in 7-15% of patients, infection in 20-25%, and mortality in 1-7%. These findings suggest many C5i-treated patients experience suboptimal disease control. |
| Databáze: |
MEDLINE |
| Externí odkaz: |
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