Characterization of Antibiotic Prophylaxis Risk Mitigation Practices Across Eculizumab and Ravulizumab Adult, Phase III Clinical Trials for Paroxysmal Nocturnal Hemoglobinuria, Atypical Hemolytic Uremic Syndrome, Generalized Myasthenia Gravis, and Neuromyelitis Optica Spectrum Disorder
Ukwen Akpoji1, Shirali Pandya1, Rasha Aguzzi1, Jeannette Stankowski1, Sami Fam1, Arshad Mujeebuddin1, Lokesh Jha1
1Alexion, AstraZeneca Rare Disease
Objective:
To describe the antibiotic prophylaxis (AB-PPx) durations of exposure and classes by indication in Alexion C5 inhibitor therapies (ALXN-C5ITs) adult pivotal studies.
Background:
ALXN-C5ITs, eculizumab and ravulizumab, are indicated for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). As with inherited complement deficiencies, ALXN-C5ITs carry risk for Neisseria meningitidis infection and AB-PPx is advised in patients not up to date with vaccinations. Data gaps exist, however, in AB-PPx drug selection and duration for ALXN-C5IT patients.
Design/Methods:
ALXN-C5IT–exposed patients during PNH, aHUS, gMG, or NMOSD adult trials who received at least 1 dose of AB-PPx against meningococcal infection were included. AB-PPx patterns were stratified by geography and indication for ALXN-C5IT–exposed patients. Demographics, frequencies of AB-PPx durations, and classes utilized were calculated with descriptive statistics.
Results:
The 946 patients enrolled were primarily White (58.7%), female (59%), and on ALXN-C5IT for a mean of 2.81±1.52 years (y). Overall, 38.3% (362/946) received AB-PPx, notably in Europe (64.9%, 235/362), Asia-Pacific (59/362, 16.3%), and the U.S. (32/362, 8.8%). AB-PPx was most utilized in PNH (210/362, 58%), then aHUS (122/362, 33.7%), NMOSD (18/362, 5%), and gMG (12/362, 3.3%). Among U.S. patients, 65.6% (21/32) received AB-PPx ≤30 days, whereas 53% (175/330) of ex-U.S. patients received AB-PPx >1y. Among the AB-PPx classes investigated, penicillins were most used (U.S.: 43.8%; ex-U.S.: 66.4%).
Conclusions:
AB-PPx durations varied at ALXN-C5IT initiation, with the majority of patients receiving either 1-30 days (U.S.) or >1y (ex-U.S.). Penicillin-class AB-PPx was most utilized, regardless of geography, consistent with inherited-complement-deficiency recommendations.
Generative AI Usage
No, did not use generative AI in the drafting or editing in this abstract.
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