Plasma therapy yields mixed results for aHUS patients (to include PI/PE non-responders). Physicians and atypical HUS families in most nations are limited to using plasma therapies such as plasma infusion (PI, usually with units of FFP or ‘fresh frozen plasma’) or therapeutic plasma exchange (aka PE, PEX, TPE, or plasmapheresis) which have been used to treat aHUS for the past several decades. A drug created by Alexion Pharmaceuticals, eculizumab (brand name Soliris ®) is highly effective but expensive and not widely available around the world.
Currently eculizumab is the only drug approved to treat the rare disease atypical hemolytic uremic syndrome (aHUS or atypical HUS). Out of more than 7000 rare diseases, only an estimated 5% have an approved treatment or therapy (Rare Disease Day, FAQs).