Ursolfalk - success as a drug

Ursofalk - how effective is it?

My 14 year old son has a definite diagnosis of Ulcerative colitis with query PSC and autoimmune hepatitis. Over the past 12 months he has been well (apart from increasing tiredness) however recent blood tests show elevated GGT which is leading our Consultant to believe PSC is the issue. Previous Cholangiogram did not show beading of the ducts but scans did show sludge in the gallbladder.

He has been prescribed Usrofalk ahead of a Cholangiogram in July. Does anyone have experience of the drug as part of their regime and has it been effective in reducing GGT levels?

Many thanks

Adamsmum

Hi Adamsmum,

I have just started taking Ursofalk. I have been diagnosed with PSC for 7 years now and thankfully have only had one episode a couple of months ago. Like your son, I also have raised GGT's, around 124 at my last blood test, all the rest of my liver blood tests are low. My specialist has said that taking Ursofalk should start to normalise my blood test results, and in the long run Ursofalk has been shown to increase the time before transplant, if it ever gets to that stage, hopefully not. So hopefully your sons GGT's start to come down.

From speaking to my specialist it seems like you dont have much to loose by taking Ursofalk, or not that they would let on. The side effects listed are standard for any antibiotic or drug.

I have asked on the comments board on here last week, see http://www.livingwithpsc.org/forum/topics/treatment-ursofalk

but it doesnt seem like many people are taking it, see the , its mostly Vancomycin.

I am only 3 days into taking the Ursofalk so have not had any blood tests to compare to previous, but I will keep the PSC'ers updated with any news and results as I get them.

Cheers

Monkey1

Hi Adamsmum, I was diagnosed with PSC in 1998 after many years of Crohn’s disease. and have been taking Ursofalk 250mg x 6 daily for the past 10 years. Recently I was diagnosed with PBC. Ursofalk has been benificial for my condition and has kept my liver function tests ‘abnormally normal’ if that makes sense. I am fortunate to enjoy reasonably good health, after some difficult episodes of Cholangitis. I wish your son all the very best. X

I have been taking Urso (Usrolfalk) 1250mg daily since my PSC diagnosis about 2 1/2 years ago. I haven't had any issues with the medication and it has brought all my levels down to within normal ranges. My doctor has me do annual MRIs to track progression and blood tests every few months. Once thing my doc did mention is they know the Urso brings down the levels they don't know if that actually stops or slows damage to the liver. Hopefully you have prescription coverage as it costs about $160/month

Hi Adamsmum,

I've been taking urso now for a few years and it does seem to help reduce the lft levels. Its not, for me, done much for the Alkaline Phosphatase or GGT but it has reduced the ALT levels so it looks like it helps in some way with reducing inflammation within the liver. None of my levels are within normal limits but all have now been reasonably stable. So its clearly had some benefit. As has been said its not a cure, i've still developed cirrhosis, but if it helps reduce inflammation it should delay the rate at which fibrosis and scar tissue forms. This then means that the progress of the disease should also be slowed.

Urso is generally well tolerated and whilst its efficacy in treating psc is still apparently a little debateable it does have benefits in treating billiary disease in general. Hence it seems to be a course of treatment that docs are happy to prescribe as it does have benefits, even if its only in reducing lfts.

Hope thats helpful.

K

Our 12 year old son was diagnosed with Crohn's colitis with autoimmune hepatitis and PSC in February. His GGT at the highest was 381, the prednisone brought it down to 91 and then it started to go back up to 136. He was put on Ursodiol and it, in combination with the prednisone and Imuran that he was already on, brought his GGT down to 56 in a short time. It seems to have helped our son, so I'd say go for it.

For the latest info, consult the 2014 PSC conference with Dr. Greg Everson (Current Perspectives in the Treatment of PSC)

http://www.pscpartners.org/PSCConf14/index.htm

I take Urso, but a few years back my dosage was reduced from 6X250 mg to 4X250 mg per day. Some evidence suggests that it can be harmful at the high doses, but all right at the lower dosage 15mg/kg/day. I have no side effects.

The reference range I have listed for GGT is that it should be under 55. Mine was 193 in 1998 (prior to urso) and 64 in 2009. I have very few test results in between.

Is Ursofalk the same as Ursodiol just another name?

Ursofalk is name brand Ursodiol.

Urso has been a part of many PSC studies over the years. It tends to lower LFTs compared to control groups, but does not seem to extend the time before a patient reaches an "endpoint." Typically lower LFTs are associated with a longer period of time before reaching an endpoint, so it is thought that Urso masks the numbers rather than actually improving the chemistry. For the longest time it was commonly prescribed because lower LFTs are usually a good thing and there was no known downside to using the drug. Anecdotally, many people have reported that it reduces/eliminates many PSC symptoms.

Now there is a rather drastic divide between hepatologists who will or will not prescribe the drug due to a long-term study on high-dose Ursodiol that was released in 2009 (http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2758780/). Simply put, the risk of reaching an endpoint was 2.3 times greater in the Ursodiol group compared to the group taking a placebo. Most hepatologists no longer prescribe high-dose Urso because of this trial and many are unwilling to prescribe it even at lower doses given the perceived risk and the lack of clinical trials showing any long-term benefits for PSC patients.

Urso has also been studied to see if it lowers the risk of colon cancer for UC patients, but I'm not sure what the current consensus is on this point.

For a pediatric patient, I strongly recommend getting in contact with Dr. Cox at Stanford. His Vancomycin trials have been very successful at treating UC and PSC.

Based on the evidence jtb cites, my son's doc let us decide about Urso. He said he basically didn't have an opinion because the evidence is unclear. We decided to treat with ercp, prednisone tapered off to imuran, and vanco (my son also has UC, PSC, AIH). He is doing well based on labs. One year mrcp still to be read by radiologist, but the only symptom at this point is periodic nausea and some fatigue, but he is able to participate in his life again - graduated high school this year, accepted to university, etc.

Please do read up on the vanco treatment (and be sure you are reading about oral vanco). Our thinking was that urso had mixed, potentially negative results. For pediatric patients, vanco has minimal data, but that minimal data is very positive. We thought since neither had compellingly strong evidence, we'd go with the treatment that offered the best hope of a positive outcome. Dr. Cox will assist remotely and speak with your doctor if the doctor is resistant or doesn't know about the treatment.

Here's a good place to start. http://www.childrenspsc.org/

Good luck! I hope your son starts to feel better!! Feel free to contact me through messaging if you want to speak more specifically... or ask questions about our experience with the vanco and convincing our doc to try it.