Manuscripts
The following information for US healthcare professionals includes links to Apellis-sponsored clinical trials that have been published in peer reviewed journals. The list of publications below may contain information about doses, uses, formulations and populations different from product labeling or contain information about investigational compounds or uses not approved by a regulatory authority. The following list of publications is not comprehensive or exhaustive as to any particular topic. Apellis does not suggest or recommend the use of its products in any manner other than as described in the Prescribing Information. Please refer to the product’s Full Prescribing Information.
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- Novel Complement Therapeutics in Development as Potential Treatment for Renal Disease
- Kelleher C, Kocinsky H. Novel Complement Therapeutics in Development as Potential Treatment for Renal Disease. Adv Chronic Kidney Dis. 2020;27(2):95-103. doi:10.1053/j.ackd.2020.02.006
- Clinical Safety and Efficacy of Pegcetacoplan in a Phase 2 Study of Patients with C3 Glomerulopathy and Other Complement-Mediated Glomerular Diseases.
- Dixon B, Greenbaum LA, Huang L, et al. Clinical Safety and Efficacy of Pegcetacoplan in a Phase 2 Study of Patients with C3 Glomerulopathy and Other Complement-Mediated Glomerular Diseases. Kidney International Reports. 2023. doi: 10.1016/j.ekir.2023.08.033
- Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials
- Heier JS, Lad EM, Holz FG, et al. Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials. Lancet. 2023;402(10411):1434-1448. doi:10.1016/S0140-6736(23)01520-9.
- Diagnosis and Management of Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Delphi Consensus Exercise
- Singh R, Amoaku W, Bandello F, et al. Diagnosis and Management of Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Delphi Consensus Exercise. Ophthalmic Surg Lasers Imaging Retina 2023;54:589–598 doi: 10.3928/23258160-20230824-01
- Humanistic and Economic Burden of Geographic Atrophy: A Systematic Literature Review
- Sarda, Sujata & Heyes, Anne & Bektas, Meryem & Thakur, Tanvee & Chao, Wendy & Intorcia, Michele & Wronski, Samantha & Jones, Daniel. Humanistic and Economic Burden of Geographic Atrophy: A Systematic Literature Review. Clinical ophthalmology (Auckland, N.Z.). 2021. 4629–4644. 10.2147/OPTH.S338253.
- Characterizing New-Onset Exudation in the Randomized Phase 2 FILLY Trial of Complement Inhibitor Pegcetacoplan for Geographic Atrophy
- Wykoff CC, Rosenfeld PJ, Waheed NK, et al. Characterizing New-Onset Exudation in the Randomized Phase 2 FILLY Trial of Complement Inhibitor Pegcetacoplan for Geographic Atrophy. Ophthalmology. 2021;128(9):1325-1336. doi:10.1016/j.ophtha.2021.02.025
- Pegcetacoplan treatment for geographic atrophy due to age-related macular degeneration: a plain language summary of the FILLY study
- Liao DS, Metlapally R, Joshi P. Pegcetacoplan treatment for geographic atrophy due to age-related macular degeneration: a plain language summary of the FILLY study. Immunotherapy. 2022;14:13, 995-1006. doi:10.2217/imt-2022-0078
- Impact of Baseline Characteristics on Geographic Atrophy Progression in the FILLY Trial Evaluating the Complement C3 Inhibitor Pegcetacoplan
- Steinle NC, Pearce I, Monés J, et al. Impact of Baseline Characteristics on Geographic Atrophy Progression in the FILLY Trial Evaluating the Complement C3 Inhibitor Pegcetacoplan. Am J Ophthalmol. 2021;227:116-124. doi:10.1016/j.ajo.2021.02.031
- Complement C3 Inhibitor Pegcetacoplan for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Phase 2 Trial
- Liao DS, Grossi FV, El Mehdi D, et al. Complement C3 Inhibitor Pegcetacoplan for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Phase 2 Trial. Ophthalmology. 2020;127(2):186-195. doi:10.1016/j.ophtha.2019.07.011
- Exploring patient acceptability of emerging intravitreal therapies for geographic atrophy: A mixed-methods study
- Enoch J, Ghulakhszian A, Sekhon M, et al. Exploring patient acceptability of emerging intravitreal therapies for geographic atrophy: A mixed-methods study. Eye (2023). https://doi.org/10.1038/s41433-023-02571-3
Kolev M, Das M, Gerber M, Baver S, Deschatelets P and Markiewski MM (2022) Inside-Out of Complement in Cancer. Front. Immunol. 13:931273. doi: 10.3389/fimmu.2022.931273
- Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
- Hillmen P, Szer J, Weitz I, et al. (2021) Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 384(11):1028-1037. doi:10.1056/NEJMoa2029073
- Plain language summary of PEGASUS, a study comparing pegcetacoplan with eculizumab for 16 weeks in people with paroxysmal nocturnal hemoglobinuria
- Griffin M, Szer J, Weitz I, et al. (2023) Plain language summary of PEGASUS, a study comparing pegcetacoplan with eculizumab for 16 weeks in people with paroxysmal nocturnal hemoglobinuria. Future Rare Diseases. 3:3. doi: 10.2217/frd-2023-0005
- Pegcetacoplan controls hemolysis in complement inhibitor–naive patients with paroxysmal nocturnal hemoglobinuria
- Wong RSM, Navarro-Cabrera JR, Comia NS, et al. (2023). Pegcetacoplan controls hemolysis in complement inhibitor–naive patients with paroxysmal nocturnal hemoglobinuria. Blood Advances, 7 (11): 2468–2478. doi: https://doi.org/10.1182/bloodadvances.2022009129
- Pegcetacoplan compared with supportive care for 26 weeks for participants with paroxysmal nocturnal hemoglobinuria: a plain language summary
- Wong RSM, Navarro-Cabrera JR, Comia NS, et al. (2023). Pegcetacoplan compared with supportive care for 26 weeks for participants with paroxysmal nocturnal hemoglobinuria: a plain language summary. Future Rare Diseases, Epub ahead of print. doi: 10.2217/frd-2023-0006
- Fatigue and health-related quality of life in paroxysmal nocturnal haemoglobinuria: A post hoc analysis of the pegcetacoplan PEGASUS trial data
- Panse J, Wilson K, Fishman J, et al. (2023). Fatigue and health-related quality of life in paroxysmal nocturnal haemoglobinuria: A post hoc analysis of the pegcetacoplan PEGASUS trial data. Eur J Haematol, 111:72–83. doi: 10.1111/ejh.13969
- Injection Site Reactions with Long-Term Pegcetacoplan Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Brief Report
- Sharma V, Koprivnikar J, Drago K, et al. (2023). Injection Site Reactions with Long-Term Pegcetacoplan Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Brief Report. Adv Ther, 40(11):5115-5129. doi: 10.1007/s12325-023-02653-4
- Descriptive, real-world treatment patterns, resource use, and total cost of care among eculizumab- and ravulizumab-treated members with paroxysmal nocturnal hemoglobinuria
- Broderick KC, Burke JP, Fishman J, & Gleason PP. (2023). Descriptive, real-world treatment patterns, resource use, and total cost of care among eculizumab- and ravulizumab-treated members with paroxysmal nocturnal hemoglobinuria. Journal of Managed Care & Specialty Pharmacy, 29(8), 941–951. doi: 10.18553/jmcp.2023.29.8.941
- Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison
- Bhak RH, Mody-Patel N, Baver SB, et al. (2021) Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison, Current Medical Research and Opinion, 37:11, 1913-1923, DOI: 10.1080/03007995.2021.1971182
- Real-World Healthcare Resource Utilization (HRU) and Costs of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population
- Cheng WY, Sarda SP, Mody-Patel N, et al. (2021) Real-World Healthcare Resource Utilization (HRU) and Costs of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population. Adv Ther. 38(8):4461-4479. doi:10.1007/s12325-021-01825-4
- C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab
- de Castro C, Grossi F, Weitz IC, et al. C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab. Am J Hematol. 2020;95(11):1334-1343. doi:10.1002/ajh.25960
- Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease
- Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease. J Manag Care Spec Pharm. 2020;26(12-b Suppl):S8-S14. doi:10.18553/jmcp.2020.26.12-b.s8