Compositions and Methods for the Treatment of HSCT-Related Thrombotic Microangiopathy

摘要

PROBLEM TO BE SOLVED: To inhibit a complement pathway for use for treating HSCT-related thrombotic microangiopathy (also referred to as HSCT-TMA, TA-TMA) in a patient who has undergone hematopoietic stem cell transplantation (HSCT). To provide a drug that can be used. In certain embodiments, the agent may have an agent or antibody capable of blocking the complement pathway. In one aspect, the antibody may have a monoclonal antibody capable of inhibiting the complement pathway. In other embodiments, the agent may have a humanized monoclonal antibody capable of blocking the complement pathway. In certain embodiments, the agent may be eculizumab, available from Alexion Pharmaceuticals and sold under the trade name Soliris. [Selection diagram] Fig. 1