Stem Cell Transplantation in Patients With Primary Biliary Cirrhosis

There will be no randomization in this study. All subjects who are determined to be
eligible for the study treatment will receive high dose cyclophosphamide, fludarabine and
CAMPATH-1H followed by infusion of allogeneic peripheral blood stem cells. The procedures
the subject will undergo are as follows:

1. Physician visit to determine potential eligibility for the study. Subjects will be
evaluated by a transplant physician. They will have a complete physical examination
and will provide a full medical history at these visits. In addition, subjects will be
asked to complete a quality of life Questionnaire . Finally, the study will be
described in detail by the transplant physician and nurse and the consent form will be
provided to be taken home to read.

2. HLA Testing. In order to proceed on this study, subjects will need to have a HLA
identical matched sibling donor. The subject and his or her siblings will need to
undergo HLA typing in order to determine whether there is a match or Cord blood stem
cells will be obtained from the New York Blood Center Cord Blood Registry which is an
internationally recognized registry or, if a match is not available, from Stemcyte, a
commercial registry that specializes in minority donors. One unit of HLA matched cord
blood unit will be infused on day zero.

3. Insurance verification. Subjects who remain interested in pursuing the study treatment
and who have severe scleroderma that is following a progressive course will proceed to
the insurance verification phase. Third party payment or self-pay must be verified
before subjects can proceed.

4. Consent form. Prior to proceeding, the appropriate signatures will be obtained on the
consent form. Subjects will be given an opportunity to ask further questions of the
attending physician and transplant nurse prior to signing the consent form.

5. Pre-transplant testing. To determine final eligibility for the study, subjects will
undergo a series of tests/procedures. These include: CXR, electrocardiogram,
endoscopy , ultrasound of the liver and gallbladder,transvenous liver biopsy,
lymphopheresis, pulmonary function test, urinalysis, and blood testing to include CBC;
chemistries; liver and kidney function tests; PT; PTT; AMA; viral studies and, for
females, a pregnancy test. Subjects will also be evaluated by a dentist at
Northwestern Memorial Hospital to rule out any potential sources of infection. All
pre-transplant testing is routine medical testing done to verify diagnosis and to
insure adequate organ function and absence of viral illnesses which would preclude a
safe transplant course. Allogeneic donors will also need to undergo testing in order
to determine eligibility to proceed. Testing for donors will include history and
physical, electrocardiogram, CXR, urinalysis, CBC, chemistries, PT, PTT, viral studies
and, for females, a pregnancy test.

6. Allogeneic peripheral blood stem cell collection. Allogeneic donors will undergo a
routine procedure for the mobilization and collection of peripheral blood stem cells.
This includes the subcutaneous administration of G-CSF, to be self-administered as an
outpatient, beginning three days prior to the start of peripheral blood stem cell
harvesting. The peripheral blood stem cells will be collected as an outpatient in the
Blood Center on the fourth day of G-CSF administration. A central line may be placed
for this purpose on the first day of leukopheresis. Leukopheresis will be continued on
a daily basis (up to four consecutive days) until an adequate number of peripheral
blood stem cells have been collected. G-CSF will continue to be administered until
leukopheresis is completed. The pheresis catheter will be discontinued when stem cell
harvesting is completed. Processed cells will be frozen and stored until they are
reinfused after the conditioning regimen.

7. Study treatment. Prior to the administration of the study treatment, subjects will
have a double lumen PICC line placed for the administration of chemotherapy, IV fluids,
blood products and the withdrawal of blood samples. The placement of a central
catheter is a routine medical procedure. Subjects will then undergo conditioning which
will include 4 days of intravenous fludarabine and 4 days of cyclophosphamide and 2
days of CAMPATH-1H. All drugs are FDA approved drugs. The previously collected
allogeneic peripheral blood stem cells will be reinfused following the completion of
the conditioning regimen.

8. Post-transplant follow-up. Subjects will have a history and physical by the transplant
physician at 6 months, 12 months, and yearly for 5 years. In addition, routine
urinalysis and blood testing will be performed at these same intervals to include CBC;
chemistries; kidney and liver function tests; AMA; ultrasound of the liver and
gallbladder, transvenous liver biopsy, lymphopheresis, and patients will also be asked
to complete a SF-36 Questionnaire, NDDK-QA Questionnaire.