Hillmen P et al. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. NEJM. 2021 (PEGASUS trial)
Pegcetacoplan superior to eculizumab for hemoglobin response
DOI 10.1056/NEJMoa2029073
Peffault de Latour R et al. Oral Iptacopan Monotherapy in PNH. NEJM. 2024 (APPLY-PNH trial)
Iptacopan demonstrated hemoglobin improvement vs placebo
DOI 10.1056/NEJMoa2308695
Lee JW et al. Addition of Danicopan in PNH with Extravascular Hemolysis. Lancet Haematology. 2023 (ALPHA trial)
Danicopan add-on reduced extravascular hemolysis
PMID 38030318
Gurnari C et al. PNH-related thrombosis in the era of novel therapies. Blood. 2024
2,043 patient-year analysis of thrombosis outcomes
PMID 38513233
Meningococcal safety references
Vaccination and infection risk
McNamara LA et al. High risk for invasive meningococcal disease in patients receiving eculizumab despite vaccination. MMWR. 2017
Documented ~1,000-2,000-fold risk increase
PMID 28704351
Schillie S et al. New dosing interval and schedule for Bexsero MenB-4C vaccine. MMWR. 2024
Updated ACIP recommendations for MenB vaccination
PMC11637420
Review articles and epidemiology
Major reviews
Godby RC, Shah S. Paroxysmal Nocturnal Hemoglobinuria. Mayo Clinic Proceedings. 2025
Current comprehensive review
PMID 41171226
Kelly RJ, Holt M, Szer J. Pharmacological therapies in PNH. Drugs. 2025
Focus on complement inhibition pharmacology
PMID 40986193
Devalet B et al. Pathophysiology, diagnosis, and treatment of PNH: a review. European Journal of Haematology. 2015
Foundational review of mechanism and management
DOI 10.1111/ejh.12543
Notaro R, Luzzatto L. Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition. NEJM. 2022
Mechanisms of breakthrough hemolysis on complement inhibitors
DOI 10.1056/NEJMra2201664
Coding standards
ICD-10 and diagnostic codes
ICD-10 D59.5 Paroxysmal nocturnal hemoglobinuria
Primary coding for classic PNH
PNH-AA overlap may additionally code D61.3 (aplastic anemia)
SNOMED CT concept for paroxysmal nocturnal hemoglobinuria
GPI-anchor deficiency disorder concept
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