Eculizumab
Eculizumab binds to complement component C5, thereby blocking its cleavage and the subsequent formation of the C5b-C9 membrane attack complex, the final common pathway effector of the complement system. Approved indications for eculizumab are paroxysmal nocturnal hemoglobinuria,
atypic hemolytic uremic syndrome (aHUS), myasthenia gravis and neuromyelitis optica spectrum disorder. Besides the prevention and treatment of aHUS and thrombotic microangiopathy (TMA), in organ transplantation there are reports from its use for desensitization, prevention and as supplement in the treatment of AMBR [30, 50-52]. Although these studies are too small to provide definitive conclusions on the role of eculizumab in these situations, there is already an example of how eculizumab concentration measurements combined with a PK/PD model can be used for model-informed precision dosing in the treatment of TMA in children undergoing HSCT [27].
Although no formal economic cost-benefit analysis was reported, for an expensive drug like eculizumab it is worth to notice the remarks from the authors that by using MIPD in their study each patient only received the required therapy course which minimizes the risk of over treating and reduces the clinical and financial burden of eculizumab therapy [26]. The approach that was used in this study could also serve to suggest how to tailor the application of eculizumab and other biologicals in solid organ transplant recipients.