Selected references related to Apellis’ science and areas of focus


Complement C3 and Complement Inhibitors

 

Delanghe JR, Speeckaert R, Speeckaert MM. Complement C3 and its polymorphism: biological and clinical consequences. Pathology. 2014;46(1):1-10.


Holers VM. The spectrum of complement alternative pathway‐mediated diseases. Immunol Rev. 2008;223:300-316.


Mastellos DC, Yancopoulou D, Kokkinos P, Compstatin: a C3-targeted complement inhibitor reaching its prime for bedside intervention. Eur J Clin Invest. 2015;45(4):423-440.


Morgan BP, Harris CL. Complement, a target for therapy in inflammatory and degenerative diseases. Nat Rev Drug Discov. 2015;14(12):857-877.


Ricklin D, Lambris JD. Complement-targeted therapeutics. Nat Biotechnol. 2007;25(11):1265-1275.


Ricklin D, Lambris JD. Compstatin: a complement inhibitor on its way to clinical application. In: Lambris JD, ed. Current Topics in Complement II. New York, NY: Springer; 2008.


Risitano AM. Current and future pharmacologic complement inhibitors. Hematol Oncol Clin North Am. 2015;29(3):561-582.

 


Paroxysmal Nocturnal Hemoglobinuria (PNH)

 

DeZern AE, Brodsky RA. Paroxysmal nocturnal hemoglobinuria: a complement-mediated hemolytic anemia. Hematol Oncol Clin North Am. 2015;29(3):479-494.


Griffin M, Hillmen P, Munir T, et al. Significant hemolysis is not required for thrombosis in paroxysmal nocturnal hemoglobinuria. Haematologica. 2019;104(3):e94-e96.


Hill A, Rother RP, Arnold L, et al. Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization. Haematologica. 2010;95(4):567-573.


Risitano AM, Notaro R, Marando L, et al. Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009;113(17):4094-4100.


Risitano AM, Ricklin D, Huang Y, et al. Peptide inhibitors of C3 activation as a novel strategy of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria. Blood. 2014;123(13):2094-2101.


Risitano AM. Dissecting complement blockade for clinic use. Blood. 2015;125(5):742-744.


Sica M, Rondelli T, Ricci P, et al. Eculizumab treatment: stochastic occurrence of C3 binding to individual PNH erythrocytes. J Hematol Oncol. 2017;10(1):126.


Thurman JM. New anti-complement drugs: not so far away. Blood. 2014;123(13):1975-1976.

 


Geographic Atrophy (GA) and Age-related Macular Degeneration (AMD)

Anderson DH, Mullins RF, Hageman GS, Johnson LV. A role for local inflammation in the formation of drusen in the aging eye. Am J Ophthalmol. 2002;134(3):411-431.


Anderson DH, Radeke MJ, Gallo NB, et al. The pivotal role of the complement system in aging and age-related macular degeneration: hypothesis re-visited. Prog Retin Eye Res. 2010;29(2):95-112.


Boyer DS, Schmidt-Erfurth U, van Lookeren Campagne M, et al. The pathophysiology of geographic atrophy secondary to age-related macular degeneration and the complement pathway as a therapeutic target. Retina. 2017;37(5):819-835.


Chakravarthy U, Bailey CC, Johnston RL, et al. Characterizing disease burden and progression of geographic atrophy secondary to age-related macular degeneration. Ophthalmology. 2018;125(6):842-849.


Friedman DS, O'Colmain BJ, Munoz B, et al. Prevalence of age-related macular degeneration in the United States. Arch Ophthalmol. 2004;122(4):564-572.


Holz FG, Strauss EC, Schmitz-Valckenberg S, van Lookeren Campagne M. Geographic atrophy: clinical features and potential therapeutic approaches. Ophthalmology. 2014;121(5):1079-1091.


Scholl HP, Charbel Issa P, Walier M, et al. Systemic complement activation in age-related macular degeneration. PLoS One. 2008;3(7):e2593.


Sunness JS, Gonzalez-Baron J, Applegate CA, et al. Enlargement of atrophy and visual acuity loss in the geographic atrophy form of age-related macular degeneration. Ophthalmology. 1999;106(9):1768-1779.

 


C3 Glomerulopathy (C3G)

 

Servais A, Fremeaux-Bacchi V, Lequintrec M, et al. Primary glomerulonephritis with isolated C3 deposits: a new entity which shares common genetic risk factors with haemolytic uraemic syndrome. J Med Genet. 2007;44(3):193-199.


Servais A, Noel LH, Roumenina LT, et al. Acquired and genetic complement abnormalities play a critical role in dense deposit disease and other C3 glomerulopathies. Kidney Int. 2012;82(4):454-464.


Zipfel PF, Skerka C, Chen Q, et al. The role of complement in C3 glomerulopathy. Mol Immunol. 2015;67(1):21-30.

 


Autoimmune Hemolytic Anemia (AIHA): Cold Agglutinin and Warm Antibodies

Berentsen S. Role of complement in autoimmune hemolytic anemia. Transfus Med Hemother. 2015;42(5):303-310.


Wouters D, Zeerleder S. Complement inhibitors to treat IgM-mediated autoimmune hemolysis. Haematologica. 2015;100(11):1388–1395.