The following questions were answered by:
James Neuberger, DM, FRCP
Question 1
Should a transplant recipient refer to PBC in the present or past tense (i.e. 'I have PBC' or 'I had PBC')? Any statistics about reoccurrence? If reoccurrence does occur, is disease progression faster?
Answer
Most studies suggest that PBC does recur in the graft. The diagnosis is made by examining the histology of the graft. The rate of recurrence varies and may be affected by many factors including the choice of immunosuppression. In nearly all cases, the recurrence has no impact on the functioning or survival of the graft. The effect in the very long term is not yet known; whether urso affects recurrence is not known. We do not know whether the progression of recurrence is greater after transplantation: PBC has a great variation of progression and it may be that those who have a more rapid progression are those who get transplanted.
Question 2
So many with PBC complain of sleep problems. Is this a common symptom or could it be related to the medications such as ursodiol?
Answer
There are many causes of poor sleep and people with PBC are not immune to them! I am not aware that sleeplessness is more common in PBC (unless itching or other symptoms will awaken people) and I am not aware that Urso affects the pattern of sleep.
Question 3
What type of a doctor should one see for Sjogrens Syndrome? Is Sjogrens Syndrome a completely different disease than the dry eyes/mouth symptoms associated with PBC? Besides the eyes, what other organs does Sjogrens Syndrome affect?
Answer
If specialist treatment is needed for sicca syndrome, in the UK, we would refer either to a rheumatologist or a specialist centre
Question 4
What is the cause of varcies? Is blood and or mucus on the stool a symptom of varcies? Is this common in PBC patients? How often do you recommend a endoscopy for your patients?
Answer
Varices can be seen in any one with portal hypertension (high pressure in the veins from the bowel and spleen to the liver). Those with PBC may get varices before the onset of cirrhosis. Varices are seen in those with advancing liver disease and the doctors will usually screen for these. Often varices are asymptomatic but may bleed giving rise to either vomiting of blood or passage of blood in the bowel (then the motions are usually black and tarry). This is a medical emergency and the person must seek immediate medical help. Passage of mucus is not a symptom of varices.
Question 5
Although asked many times, could you review the etiology of itching in PBC and the more advanced therapies such as Rifampan to curtail the itching. Also, if itching remains a problem along with fatigue, can a patient be sent for a transplant evaluation due to quality of life issues or do you have to wait until the lab tests meet criteria?
Answer
Although asked many times, could you review the etiology of itching in PBC and the more advanced therapies such as Rifampan to curtail the itching. Also, if itching remains a problem along with fatigue, can a patient be sent for a transplant evaluation due to quality of life issues or do you have to wait until the lab tests meet criteria?
The cause of itching is not entirely clear. The older view that itching was due to retained bile acids is no longer widely held; there is now a great deal of interest in the concept that naturally occurring opioid-like substances may be contributing to the itch. Cholestyramine remains the main-stay of treatment but in some, the drug cannot be tolerated or is ineffective. In such cases, a number of other options may be tried. Rifampicin may be effective but can be hepatotoxic and may interact with the metabolism of some other drugs. In others, naltrexone may be helpful but this should be used with caution as there may be significant side-effects. Other treatments that have been suggested include urso (usually of little benefit for the itch), prednisolone, ultra-violet light and some steroids such as stanozolol (this too may have major side-effects). There has been some interest in the use of a form of dialysis called MARS and plasmaphoresis; these treatments involve major interventions and I have found some benefit in some people. Liver transplantation is an effective form of therapy. The system of allocation of donor livers will vary between countries. I can speak only for the UK where we do not have laboratory criteria (such as MELD) to get onto the waiting list or to get a liver. This is not true for all other countries. Just as PBC may recur in the graft, so may itching come back after transplantation
Question 6
I read ia patient is diagnosed early and with proper medication most will never need a transplant. Is this true? What is considered "early?" Is it within the first five or ten years of the disease or a certain stage?
Answer
I have read on the PBCers website that if a patient is diagnosed early and with proper medication most will never need a transplant. Is this true? What is considered "early?" Is it within the first five or ten years of the disease or a certain stage?
It is certainly true that many people with PBC will never need a transplant. It is believed that those who show a good response to Urso will have a slower progression but I am not yet convinced that one can say that transplantation will never be required. PBC can be diagnosed as early on a variety of criteria: several factors have been identified which predict prognosis: serum bilirubin is the most important but other factors, such as serum albumin, will also predict survival. The histology is a rough guide. The presence or severity of symptoms such as itching or lethargy bear little correlation with prognosis.