title: Current and future pharmacologic complement inhibitors.
abstract: The availability of anticomplement therapies has been a major achievement for medicine in the last decade. Indeed, eculizumab has changed the treatment paradigm of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome and promises to do the same in several other human complement-mediated diseases. Nowadays, a 10-year experience has also taught us that there are some pitfalls that represent a challenge to improve the current anticomplement treatment. Most of these observations come from paroxysmal nocturnal hemoglobinuria, where unmet clinical needs are emerging, triggering the attention of several investigators and pharmaceutical companies.
authors.affiliation: Bone Marrow Transplantation Clinical Unit, Hematology, Department of Clinical Medicine and Surgery, Federico II University of Naples, Via Pansini 5, Naples 80131, Italy. Electronic address: amrisita@unina.it.
authors.forename: Antonio M
authors.initials: AM
authors.lastname: Risitano
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000378:metabolism / Q000627:therapeutic use*; D065766:Atypical Hemolytic Uremic Syndrome / Q000188:drug therapy*; D003167:Complement Activation / Q000187:drug effects; D003182:Complement C5 / Q000378:metabolism; D051056:Complement Inactivating Agents / Q000378:metabolism / Q000627:therapeutic use*; D004358:Drug Therapy / Q000379:methods / Q000639:trends; D005544:Forecasting; D006457:Hemoglobinuria, Paroxysmal / Q000188:drug therapy*; D006801:Humans; D011485:Protein Binding
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D003182:Complement C5; D051056:Complement Inactivating Agents; C481642:eculizumab
keywords: C3; C5; Complement therapeutics; Compstatin; Eculizumab; PNH; aHUS