DOCTORS PANEL QUESTIONS
& ANSWERS
We wish to thank all the members on our Doctors Panel, for giving us their
time answering PBCers questions. We greatly appreciate all they do
for us.
The following questions were answered by:
Nathan M. Bass, MD, PhD
Professor of Medicine
Medical Director, UCSF Liver Transplantation Service
Division of Gastroenterology
UCSF Medical Center and
2004
Question 1
What are the side effects of long-term usage of
Urso (1250 mg/day) over ten years?
Answer 1
From a great deal of data in hand, there seems to
be no downside to long term Urso. Most side
effects are seen early on in treated patients. Patients who tolerate
the drug without symptoms of diarrhea, itching or other effects in the short
term seem to have no long term adverse effects at all.
Question 2
Have any of the new monoclonal antibodies like
Rituxin
been shown to benefit PBC patients? Is there any future with
these type antibodies in treating PBC?
Answer 2
Not as yet. As we still do not have a good handle on the cause
and mechanism of PBC, the answer to your question would be a hopeful
"maybe". A number of treatments that alter the function of the immune
system have been tried out as treatment for PBC. Some showed promise, but
were limited by unacceptable toxicity. Overall, the story of
immunomodulators in treating PBC has been quite
disappointing.
Question 3
I have been diagnosed with PBC...Is fatigue a common symptom of this
condition? I have been told that there is no connection between PBC
and fatigue...my body tells me different.
Answer 3
Your body wins this one. Fatigue is one of the most
prominent symptoms reported by patients with PBC. In a significant
number of patients, it is felt to be worse than itching.
Fatigue is a common symptom in patients with any type of significant liver
disease, and its mechanism is not well understood. As liver disease advances,
fatigue may become quite disabling.
Factors that may contribute include failure of the liver to perform its
detoxification function, high blood ammonia (hepatic encephalopathy), the
effect of inflammatory molecules on the brain, loss of muscle mass, and
depression. Sjogren's syndrome, which is
common inpatients with PBC, has been found to cause fatigue even in the absence
of PBC, so that other factors may be operating in patients with this additional
complication.
Question 4
Does having fat accumulations in the liver significantly alter the prognosis
for PBC, if diagnosed in stage 1? What is the best method of treatment when
both are present?
Answer 4
Fat most commonly accumulates in the liver as a result of alcohol, overweight,
diabetes, or certain liver-toxic drugs. PBC alone in a patient with
no other risk factors does not cause fat to accumulate in the liver.
Fat in the liver may be relatively benign if not present in a large amount,
but it may signal the presence of other factors that could prove damaging,
such as diabetes, overweight or alcohol. The first step in eliminating
fat from the liver is to look for reasons that put in there in the first
place, and address them. Avoiding alcohol, liver-toxic drugs, and establishing
a healthy weight and good diabetic control are an excellent start.
Urso was found in early studies to reduce fat in
the liver in patients with fatty liver alone, but a recent large study has
not confirmed this.
Question 5
What is known about the relationship of PBC to
peripherial neuropathy? When hand tingling,
burning, and loss of sensation become chronic, what is the best course of
treatment? Can it be a reaction to medications such as URSO?
Answer 5
Peripheral neuropathy is well recognized although believed to be rare in
PBC. Typically, the symptoms are worse in the hands than in the
feet. The typical association is with high cholesterol in the blood
and cholesterol deposits in the skin, usually around the
eye/eylids
(xanthelasma). The classic condition is known
as xanthomatous neuropathy, and can be unpleasantly
symptomatic. There are many other causes of peripheral neuropathy that
may occur in a patient with PBC, however. Medications are a prime suspect,
but thus far, Urso does not seem to cause this
type of problem. There are studies that suggest that peripheral neuropathy
is quite common in PBC, if one looks for it hard enough, and that this is
generally common in liver disease. Deficiency in vitamin E may
play a role in some patients who are not taking any supplements. This
is readily treatable, but is not the cause in all patients.
consultation with a neurologist is strongly indicated
when symptoms like these develop. Sometimes, these
symptoms may be a manifestation of carpal
tunnel syndrome rather than a true peripheral neuropathy.
Question 6
What is the average length of time a PBCer can
stay in Stage 4 (cirrhosis? ) ?
If diagnosed under age 50 in Stage 4 will patient most likely need a liver
transplant?
If diagnosed older does that make it less likely transplant will be needed?
What is the average length of time a patient can live with PBC, after it
is diagnosed?
Answer 6
That is a difficult question, as there is tremendous
variability between individuals. The average life expectancy for even a
completely asymptomatic patient with PBC is considered less that a healthy,
person of the same age, based on extensive follow up studies. Some asymptomatic
patients with histological Stage 4 diseases have been known to live with
little impact of their disease for 5 - 10 years. The presence of severe
symptoms, but especially the presence of an elevated
bilirubin, or fluid retention are more telling
and predictive of progression to the need for a liver transplant in the near
future. The average length of time a patient with PBC can live
before a transplant is mandatory depends not
only on the on the stage of disease, but on the rate of clinical
progression. Life expectancy can be calculated from the well
known Mayo Model or from the MELD score, with just a few biochemical numbers
in hand. These "predictive models", however, just provide a statistical
probability, and not a crystal ball. For the individual, they are more
likely to err on the side of underestimating life expectancy. A high
Mayo or MELD score should definitely not be disregarded either as they provide
some advanced warning of worse to come and an opportunity to prepare for
potentially life-saving transplantation surgery.