My two year old daughter was diagnosed in May with aHUS. She responded beautifully to Soliris and continues to do well. Her doctor has suggested using Cellcept to try to eliminate the Factor H auto antibody to eventually stop infusions. Her numbers in May were over 32,000 ppm. Her last three titer levels, without intervention, have been 42, 82, and 76-significantly lower. He thinks it will work since her levels are only about 3x what is considered "negative". Our concern is side effects, pariticulary neurological. She can't communicate well so if her hand is numb we'd only know by watching her try to use it as she always has. He said they are rare, 1 in 1,000, but rare hasn't been on our side. Not only does she have aHUS but she was also born with a rare eye anomaly, Peter's Anomaly. So with the ultra rare designation given to aHUS and the rarity of her eye defect we are very worried about falling into that 1 in 1,000 group. The idea of removing her port and stopping infusions is awesome, but at what cost? She is only two; do we really want to risk permanent nerve damage at this age? Is that a decision we can make for her?
Any success in eliminating Factor H with Cellcept? Side effects experienced?