To evaluate the safety and effectiveness of transitioning between different complement inhibitors in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Approach:
Study Design: Anonymized data were collected from hematologists regarding patients aged 16 and older diagnosed with PNH who switched complement inhibitors.
Inclusion Criteria: Patients on proximal complement inhibitors who changed treatment for any reason were included.
Data Collected: Information included indications for complement inhibition, patient demographics, treatment regimens, and complications.
Key Findings:
Sixty-five patients were included, with a median age at diagnosis of 40 years and a median hemoglobin level of 85 g/L.
Indications for complement inhibition included hemolysis (78%), hemolysis and thrombosis (9%), and thrombosis (5%).
A total of 149 changes in complement inhibitors were reported, with 75 terminal-to-proximal, 45 proximal-to-proximal, and 29 proximal-to-terminal switches.
Eight patients experienced hemolytic events within 14 days of switching, with five requiring blood transfusions.
Interpretation:
Limitations:
The study is based on a relatively small cohort of 65 patients.
Data were collected from multiple centers, which may introduce variability in treatment protocols.
Conclusion:
The findings indicate a risk of breakthrough hemolysis associated with transitioning between complement inhibitors.
by Morag Griffin, Bruno Fattizzo, Jong Wook Lee, Richard J. Kelly, Roochi Trikha, Yasutaka Ueda, Jun-ichi Nishimura, Christopher J. Patriquin, Alexander Röth, Petra Muus, Jens Panse, Miguel Gómez Álvarez, Alexandra Pike, Talha Munir, Shreyans Gandhi, Austin Kulasekararaj
