Re: First Patient Enrolls in STOPSC Registry

[Guest guest]

>

> In addition to the major support from the Foundation, the

> funding to begin the DNA repository portion of STOPSC was donated by

> PSC Partners Seeking a Cure (www.pscpartners.org). PSC Partners

> Seeking a Cure is a national PSC patient support organization founded

> in 2005 to raise funds to research the causes and cure for PSC,

> promote PSC and organ donation awareness and provide education and

> support to PSC patients and their families.

:

Thank you for helping to provide this info to us.

The PSC patient support group is truly on the map and the many others, from this

group, who have forged this relationship with the Foundation are Amazing

with your vision and drive. THANKS.

DEE

>

[Guest guest]

Thank you for posting this Dave.I think it is great for everyone to see this progress and to know that weare truly moving forward.I am so encouraged by this.Together in the fight, whatever it takes.LeeNews From the LeBonheur Children's Medical Centerhttp://www.lebonheur.org/portal/site/lebonheur/First Patient Enrolls in STOPSC Registry On Monday, July 16, Sledge, a 10-year-old from Water Valley, Miss., was the first patient to be enrolled in the STOPSC registry, a multi-center database and DNA repository for patients with primary sclerosing cholangitis (PSC). The registry is sponsored by the Musette and , Jr. Foundation for the Study of Primary Sclerosing Cholangitis, headquartered at Le Bonheur Children's Medical Center.

[Guest guest]

,

I'd like to show this to my doctor so that I can be registered in this

program. Is there any particular advice as to how to get Kaiser involved in

this study? If my doctor won't do it, how can I go about getting registered?

-Marie

>News From the LeBonheur Children's Medical Center

>

>http://www.lebonheur.org/portal/site/lebonheur/

>

>First Patient Enrolls in STOPSC Registry

>

>On Monday, July 16, Sledge, a 10-year-old from Water Valley,

>Miss., was the first patient to be enrolled in the STOPSC registry, a

>multi-center database and DNA repository for patients with primary

>sclerosing cholangitis (PSC). The registry is sponsored by the

>Musette and , Jr. Foundation for the Study of Primary

>Sclerosing Cholangitis, headquartered at Le Bonheur Children's

>Medical Center.

>

>PSC is a rare disease that causes the bile ducts inside and outside

>of the liver to become scarred, narrowed and eventually blocked.

>Often the only effective treatment for this debilitating disease is a

>liver transplant. While children are waiting for a liver transplant,

>they suffer with such symptoms as severe abdominal pain, jaundice,

>growth failure and a profound lack of energy.

>

>The STOPSC (Studies of Primary Sclerosing Cholangitis) registry

>includes clinical information on children and adults with PSC,

>autoimmune hepatitis and PSC/autoimmune hepatitis overlap syndrome.

>Especially in children, there appears to be a relationship between

>PSC and autoimmune hepatitis. The registry, which is based at Le

>Bonheur Children's Medical Center, will comprise a database of

>patient information and DNA samples for genetic studies. The STOPSC

>project will facilitate collaborative, hypothesis-driven, multi-

>center research on this rare disease. Le Bonheur is one of 13 major

>North American medical centers encompassing 18 pediatric and adult

>hepatology programs participating in the registry. The EMMES

>Corporation in Rockville, MD, has been contracted to serve as the

>STOPSC Data Coordinating Center.

>

>Partners in this registry include:

>

>University of Colorado, Denver

>University of California, San Francisco

>University of Cincinnati

>Northwestern University, Chicago

>Hospital for Sick Children, Toronto

>Harvard, Boston

>Mayo Clinic, Rochester

>Mt. Sinai, New York

>Le Bonheur Children's Medical Center and University of Tennessee

>Health Science Center

>Virginia Commonwealth, Richmond

>Tufts, Boston

>University of California,

>University of Pittsburgh, Children's Hospital of Pittsburgh

>

>Dr. Dennis Black is Co-Director of the Foundation and Study

>Chair for STOPSC, as well as Le Bonheur Vice President for Research

>and Director of the Children's Foundation Research Center at Le

>Bonheur. " I'm excited that will help kick off the STOPSC

>registry. This represents a major milestone in research for this

>devastating disease. We hope that Le Bonheur sets the tone for this

>collaborative relationship with our other partners across the US and

>Canada, " Black said.

>

>In addition to the major support from the Foundation, the

>funding to begin the DNA repository portion of STOPSC was donated by

>PSC Partners Seeking a Cure (www.pscpartners.org). PSC Partners

>Seeking a Cure is a national PSC patient support organization founded

>in 2005 to raise funds to research the causes and cure for PSC,

>promote PSC and organ donation awareness and provide education and

>support to PSC patients and their families.

>

>The Foundation, founded in 2004 by Musette and ,

>Jr., sponsors and facilitates basic and clinical research to discover

>new treatments and ultimately a cure for primary sclerosing

>cholangitis. Dr. Gene Whitington, a Le Bonheur pediatric

>gastroenterologist who cares for the s' son, Worth, who has

>PSC, was the driving force for the creation of the Foundation

>and serves as its Director.

>

>Posted: July 16, 2007

>

>For more information please contact: Parris,

>

>__________________

>

>

>Dave

>(father of (22); PSC 07/03; UC 08/03)

>

>

>

_________________________________________________________________

http://liveearth.msn.com

[Guest guest]

Hi Marie;

Please visit the following web page for details of inclusion and

exclusion criteria in the STOPSC registry:

https://web.emmes.com/study/psc/about/about.html

For example, one of the exclusion criteria is " A diagnosis of large

duct PSC, small duct PSC, AIH, or PSC/AIH overlap more than five

years prior to evaluation for eligibility. " I don't know whether you

were diagnosed more than 5 years ago, but if so, this may already

exclude you from the registry?

If you have already been enrolled in the Mayo Clinic study

on " Unraveling the Genetic Predilection to Primary Sclerosing

Cholangitis (PSC) " :

http://clinicaltrials.mayo.edu/

trial # 100124

then you may already be eligible for entry into this study because

Mayo Clinic is one of the centers involved in STOPSC. Similarly, if

you were already enrolled in the PSC genetic study at UC with

Dr. Bowlus, you may already be eligible for entry into the

STOPSC registry. Well, at least this is what I understood when I

asked about this last summer at the Foundation scientific

advisory board meeting.

When you say " how do I get Kaiser involved " , do you mean the health

insurance company, Kaiser? It is not clear to me that enrollment in

STOPSC will be via insurance companies, but rather via hepatologists

at each of the 13 participating centers in North America. Eventually

they would like to add more centers, but they are starting with 13 to

get the registry launched.

I hope this answers your question?

Best regards,

Dave R.

> I'd like to show this to my doctor so that I can be registered in

this program. Is there any particular advice as to how to get Kaiser

involved in this study? If my doctor won't do it, how can I go about

getting registered?

[Guest guest]

Dear Dee;

In our lifetime we will likely not see another chance to make

significant progress in finding the causes and cure(s) of PSC than this

collaborative effort among North American centers (STOPSC). So we can't

let this initiative fail. It is funded exclusively by PSC caregivers

and patients right now, with several million dollars from Musette and

(their son Worth has PSC and Crohn's), and $20,000 from

PSC Partners Seeking a Cure. Hopefully our organization can continue

with funding of the registy in the future? The key is getting as many

patients enrolled as possible. This will enable more powerful

statistical analysis of environmental and genetic risk factors

associated with PSC. Lessons from recent genome-wide scanning for

genetic factors associated with inflammatory bowel disease are that you

need 1,000 to 2,000 patients to identify genetic components

unambiguosly. It was never possible to get such large numbers of

patients in trials/studies at individual centers for a rare disease

like PSC. So the key is collaboration among centers and pooling of

information via a central database, accessible by all participating

centers. Our PSC Partners Seeking a Cure logo says " working together to

provide research, education, and support for people affected by Primary

Sclerosing Cholangitis " , and so it seems appropriate that we

work " together " with the Foundation to get this done. Thanks

should go to everyone who has contributed their time, energy and $ to

get this registy going. Dr. Black and deserve special

thanks from all of us!

Best regards,

Dave

(father of (22); PSC 07/03; UC 08/03)

> :

>

> Thank you for helping to provide this info to us.

>

> The PSC patient support group is truly on the map and the many

others, from this group, who have forged this relationship with the

Foundation are Amazing with your vision and drive. THANKS.

>

> DEE

[Guest guest]

Dear Dave,I want to echo your thanks to everyone helping us !! I also want to send my thanks to the s and Dr. Black.I'm wondering how we can collectively thank them? Maybe at next years conference we can come up with an idea.Something that we can all sign and send to them? I will have to think about it.Question, I think this was asked before, but I can't remember the answer.For those of us who have already participated in the Mayo kit, will we enter the registry through Mayo?Also, at next years conference would it be possible to have the ville Mayo take samples to enter people into the data base with the Rochester Mayo?Especially since Dr. Gores will be there?That would add a lot of people to the data base.Just wondering.Lee Thanks should go to everyone who has contributed their time, energy and $ to get this registy going. Dr. Black and deserve special thanks from all of us!Best regards,Dave (father of (22); PSC 07/03; UC 08/03)

[Guest guest]

Hi Lee;

I'll find out more about methods of enrollment on Monday at the

Foundation scientific advisory board meeting in Memphis. I will ask

again about entry via the Mayo Clinic study. As I understood it last

year, if you were already enrolled in the Mayo Clinic genetic study

then you would be eligible for STOPSC enrollment via Mayo.

I'm not sure whether they would be able to conduct enrollments at a

conference .... I suspect not because they have a lot of inclusion and

exclusion criteria that will require consultation of detailed medical

records.

I shall pass on your thanks to Dr. Black, and to and Musette

, if I see them on Sunday or Monday?

Best regards,

Dave R.

[Guest guest]

What about PSC patients that are eligible but live abroad?

Chaim Boermeester, Israel

[Guest guest]

Please do !!Thanks Dave!LeeI shall pass on your thanks to Dr. Black, and to and Musette , if I see them on Sunday or Monday?Best regards,Dave R.__

[Guest guest]

Hi Chaim;

They are starting in North America (U.S. and Canada) because they have

to start somewhere. They want to begin small, and grow only after

initial " teething " problems are encountered and solved. It is not yet

clear whether they have plans to include centers in other countries in

the future?

Dave R.

>

> What about PSC patients that are eligible but live abroad?

>

> Chaim Boermeester, Israel

>

[Guest guest]

One criteria (at least for the pediatric arm) is that the dx has to

have been made within the last 5 years.

The criteria, however, don't address situations in which someone was

dx, then transplanted, then dx with recurrence. The people coordinating

the trial at our hospital said there's going to be a national

conference call among centers on Monday. So the answer to that, and

others, should be forthcoming.

My understanding that the trial itself involves mostly data collection,

and a DNA sample. So from a patient perspective, signing up is a no-

brainer.

The Web site given previously lists the participating hospitals.

Pam

(mom to Quantell, 17, dx 1996, tx 2001, dx recurrence with AIH overlap.)

[Guest guest]

--- In , " "

> They are starting in North America (U.S. and Canada)...

-

If no one has asked... I believe with the UCD and/or Mayo study,

folks from other countries could participate if they were visiting US

and could complete the lab tests, etc here. I wonder if the STOPSC

Registry might be open to participation as long as the entry criteria

was met while someone was visiting in US???

Joanne H

(, Ca., mom of 17, UC/PSC 2-06; JRA 1998)

[Guest guest]

Hi Joanne;

I don't know the answer to this question, but I'll ask on Monday at the

Foundation scientific advisory board meeting.

Best regards,

Dave R.

> I wonder if the STOPSC Registry might be open to participation as

long as the entry criteria was met while someone was visiting in US???

[Guest guest]

>

> -

>

> If no one has asked... I believe with the UCD and/or Mayo study,

> folks from other countries could participate if they were visiting US

> and could complete the lab tests, etc here. I wonder if the STOPSC

> Registry might be open to participation as long as the entry criteria

> was met while someone was visiting in US???

>

> Joanne H

> (, Ca., mom of 17, UC/PSC 2-06; JRA 1998)

>

This is what Ellen arranged for me for a study at UC . When on

vacation in the US my blood was drawn in a clinic in LA, I had to fill

out a questionaire and the whole package was sent to . I would be

more than happy to repeat this procedure if it can be arranged the

next time I visit the US.

[Guest guest]

and all.... STOPSC Question: My partner is 50 years old and was diagnosed with PSC in 2000 (7 years ago). He is interested in being included in the study. Does the 5 year exclusion means he is not eligible or is that pediatrics alone? It would seem to me that in a study of genetics it wouldn't matter how long one has had this, so I am unclear about the reasoning of the criteria. High Cholesterol Question: After diagnosis and for the past seven years he has done a daily raw vegetable juice regime and his liver tests have all come back in normal ranges. ERCPs are normal. He was taking asacol for a few years at the beginning but then stopped. He has no symptoms. Together we juice organic carrots, beets, broccoli, chard, kale, collards,

spinach, dandelion greens, cabbage using a Norwalk Juicer. We hike, camp, fish and have a great life. Several months ago his cholesterol edged up and the doctor put him on VYTORIN, which is a statin drug...with warnings for those with liver disease. He immediately began telling me how he could feel his liver being activated, energy increase, etc. and wondered why the doctor would put him on that drug (or any statin) knowing he has liver disease. Within 6 weeks (blood tests every two weeks to check status) his liver enzymes began to climb and his liver began to ache constantly. Then a UC attack flared up. He has had UC since age 14. The doctor suggested he take prednisone to control the UC but wanted him to continue on the VYTORIN. Well, he stopped taking the VYTORIN anyway sensing that it was initial catalyst for the entire episode. He then had to increase the prednisone from 10mg to 20 mg and was on it for

a few weeks until the UC flare-up settled down. Then he tapered off the prednisone and has now stopped taking that. In the meantime he is back on asacol. His liver has stopped aching and all the side effects from prednisone (and withdrawal) have ceased too. He feels energetic again and his general mood (cranky, impatient, intolerant, agitated while on the drug) has returned to his normal congenial self. What have others done to deal with elevated cholesterol issues besides diet and exercise? Is there any medication that works well for those who have PSC and high cholesterol? Maybe having slightly elevated cholesterol is ok versus running the risk of setting PSC in motion that appears not to be progressing? Thanks so much for being here. I have been lurking for a while trying to learn and you have given me so much hope for this man whom I dearly love. My heart goes out to Ken and and others

who are in the midst of transplants. Thanks, Bluesky in California

Take the Internet to Go: Yahoo!Go puts the Internet in your pocket: mail, news, photos more.

[Guest guest]

Hi Bluesky;

These are good questions. I'll try to get answers to them on Monday.

As I understand it the STOPSC registry is going to be more than just

a genetic study; they are also going to be following patients over

the long term for disease progression in relation to treatments. This

will require annual follow-up. As I understand it, each of the

centers involved will have a data co-ordinator who will be

responsible for entering all data [and there will be many bits of

data to enter!] at patient entry, and during follow-up. They have to

have standardized forms for data entry and retrieval! As I

understand it, the main cost of the registry is to pay these data co-

ordinators ($20,000 per year per center, $200 for each patient at

entry, and $100 for each patient each year during follow-up) ....

some rough calculations .... 13 centers x $20,000 = $260,000 per

year; 1,000 patients at entry = $200,000 one time cost; 1,000

patients at yearly follow-up = $100,000 per year.

Best regards,

Dave R.

>

> and all....

>

> STOPSC Question:

> My partner is 50 years old and was diagnosed with PSC in 2000 (7

years ago). He is interested in being included in the study. Does the

5 year exclusion means he is not eligible or is that pediatrics

alone? It would seem to me that in a study of genetics it wouldn't

matter how long one has had this, so I am unclear about the reasoning

of the criteria.

>

[Guest guest]

Hi , Thanks for your replies and for checking on answers tomorrow. This study may be the one that really makes a difference, the one that finds some solutions for PSC. Does this mean that it would cost a person this much to particpate in the study? >>>>As I understand it the STOPSC registry is going to be more than just a genetic study.....the main cost of the registry is to pay these data co-ordinators ($20,000 per year per center, $200 for each patient at entry, and $100 for each patient each year during follow-up) .... some rough calculations .... 13 centers x $20,000 = $260,000 per year; 1,000 patients at entry = $200,000 one time cost; 1,000 patients at yearly follow-up = $100,000 per year. Thanks, Bluesky

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[Guest guest]

I know Dr. Shneider said on Noah's return visit he will have labs drawn

for this purpose too.

He always very carefully words it... it won't help Noah... but might

help people in the future. ( Noah's only 9... thanks a lot.

But still, I am all for it. I just feel he doesn't need to say

THAT!!! Makes a mom want to weep.