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Hematol Oncol Clin North Am. 2015 Jun;29(3):561-82. doi: 10.1016/j.hoc.2015.01.009. Epub 2015 Apr 4.

Current and future pharmacologic complement inhibitors.

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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:


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.


C3; C5; Complement therapeutics; Compstatin; Eculizumab; PNH; aHUS

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