Dr. Robert Gish: Any sign of cholestatic pruritus should prompt a deeper evaluation.

Dr. Robert Gish: So PFIC has a number of these manifestations, these ways that PFIC presents. And to the patient, most commonly, they're presenting with itching, and itching changes their life. It changes their life in that they can't sleep, it changes their energy level, it changes how they appear to the public. Scratching themselves has a certain amount of stigma associated with it.

Dr. Richard Thompson: Most of us really can't put ourselves into our patient's situation, but we can see the consequences. We can see the scratch marks, we can see the sleep deprivation in the faces and the eyes of the patients and their family, but it has all sorts of manifestations.

Dr. Robert Gish: So in PFIC, in this cholestatic disease world, the patients are very heavily impacted. Pruritus, this itch state, could be just on the palms of their hands or their feet, or it could be their entire body. It could be 10 minutes a day, it could be 24 hours a day.

Dr. Richard Thompson: I've never had intense pruritus: I get the occasional itch. And for most of us, that's exactly how we think about itch, as some slightly annoying thing that happens from time to time. I think that those of us who have seen these children and adults with intense pruritus realize that this is something quite different from a minor irritation. But I think whether you're an adult or a child, the one time of day where you actually don't want to be distracted, you don't want to think about much, you want to go to sleep, and you want to reduce stimulation, that is the time where this awful sensation becomes very dominant in the existence and experience at that time.

Dr. Robert Gish: Some patients had such bad itching that we've gone to the transplant authorities and asked for an exception. You also have these cognitive changes, the way your brain works, this mental—obviously, a mental fatigue, or a mental fogginess that takes place. The consequences of this itch, this fatigue, can go as far as people being suicidal, very depressed. I've seen that happen in a few of my patients. That's a direct, immediate impact on their life.

The second impact is activities of daily living. Having trouble taking care of children, household, partner, parents. If you're this tired, you're just struggling to get through one day at a time. This can have a massive impact on their life, their decisions not to have children, not to be around extended family. In PFIC, pruritus can dominate sleep, work, and overall quality of life.