My daughter (6) was just diagnosed with PSC. The doc (pediatric gi who specializes in liver diseases) told us there was no treatment for it. Then I got online and found some articles about oral Vancomycin as a treatment in the journal of pediatric gastroenterology and nutrition. Does anyone have experience with this drug? Is there a reason my doctor would not consider it a valid treatment?
Many kids and adults, myself included, have had success at stopping disease progression (both PSC and PSC IBD) using oral Vancomycin. Your doctor probably isn't aware of the oral Vanco trials. Most liver doctors aren't up to date with the latest and greatest PSC research and you can't really blame them when they have 100+ fatty liver/hep C patients and only 1 PSC patient. Oral Vanco isn't part of the PSC guidelines because, among other reasons, it is still in the trial phase. We have only had small albeit promising Oral Vanco trials and are awaiting results on a larger trial that just finished up this year.
What you'll find with a rare disease like PSC is that you need to be the expert. The doctor is there to check your work and keep you out of trouble, but you need to be the one to initiate the process in getting things to happen. Do the research and become a subject matter expert. If the treatment resonates with you, brief your doctor and provide the data.
1 Like
Indeed. Oral vanco phase 3 trial at Stanford University has been completed, results are very good and will be published soon. FDA approval is not coming anytime soon, since FDA requires expensive double blind studies etc. Stanford does not have money to do those easily (vanco was identified at Stanford >20 years ago to be able to stop psc in children and results have been great). It does not work for everybody (20%?). Stanford is trying to study why is that.
In any case, many good doctors will prescribe it and many insurance companies will pay for it. Maybe worth contacting some reputable hospital eg Mayo clinic or Stanford, UC Davis etc
Thanks! Soā¦ I guess I am feeling nervous about telling the (learned and experienced) doctor that I would like her to consider trying a treatment that I read about onlineāeven in a reputable journal. Should I contact Stanford or another hospital or ask her to?. Is there a specific etiquette to requesting this?
My doc didnāt know anything about vanco either. He is famous doc in the field, teaching at top school, two of his colleagues have won Nobel prizeā¦and Iām the one explaining vanco to him. To his credit, he agreed to check it out and agreed that it looks good. Vanco was used in the same department for pscā¦big school, lots of people ā¦
Hilary,
I know there are parents on this site that have kids taking vanco, but if you are comfortable with it I would join the Facebook PSC Partners group. There is an open group and a closed group. There are lots of parents on FB and maybe they could help you through this journey. Also, they may know of a doctor in your area that understands vancomycin as a treatment for PSC.
Also, if you can make it work, you may want to schedule an appointment with Dr. Cox at Stanford or Dr. Davies in Sacramento. A lot of PSCers travel to see an "expert" doctor once a year and then have their local doctors oversee their treatment the rest of the year.
I'm sorry to hear about your child receiving this diagnosis. Good luck and take care.
Crewmom
Ted, vancomycin works for 20% or doesnāt work for 20%?
I dont know current details, it seems to work for most, but not for everybody. Maybe it does not work for 20% or so. Hopefully phase 3 result publication has more details and more info on brand name vs. generics, dosage etc.
1 Like
Hilary - I was also very nervous about asking the specialist about vanco. Our kids' treatment is in their hands and we don't want to insult or imply lack of confidence in them (nor do we want to lose confidence in them!) When the first course of treatment was prescribed, I simply asked if vanco was an option. The doc did know about it, so that made it easier, but he did not prescribe it, nor had it been used at the hospital for PSC, nor did he think it was a good idea. We deferred - however, in the meantime I started doing my homework - reading the studies, asking for references on the protocol that he was using. After deciding that I wanted to pursue vanco, I contacted Dr. Cox and asked if he would see us. (We travel to CA often to visit family.) He agreed, and he also made a call to our doctor to explain the protocol and his experience with it. I was so nervous about letting our specialist know we were going to take our kid to get a second opinion - but I asked some of my doctor friends and they said any good doctor should understand a parent's desire to get a 2nd opinion, especially with a serious diagnosis. When I finally screwed up the courage to let him know, he was incredibly kind and even provided a letter in medical speak to bring to Dr. Cox to save everybody time. Once we decided we wanted to start vanco, we did have a few conversations with our dr about it - he needed to be sure that we understood the absence of randomized double blind studies and what that meant. So, do your homework, read the studies, ask questions if you don't understand, make an informed decision about what you think you want to pursue, ask your doctor to discuss the possible downsides with you, and if you decide you want to pursue vanco, make sure you communicate to your doctor that you understand the risk you could be taking by pursuing an unproven therapy. It seems to be working for my son, but we'll never know how he would have done without it! And this is a long-term game (hopefully!), so we might have another story to tell about our decision to use vanco in 10 years, or 20 years... I'm so sorry about your child and this diagnosis. And I'm so sorry that these decisions can be so hard. Don't hesitate to ask questions here!
My son was diagnosed with PSC in November of 2016. Our liver doctor did not hesitate to put him on Vancomycin. His liver enzymes have dropped and continue to drop with this medication. The doctor told us that if he had a child with PSC this is the way he would go. So far so good.
1 Like
Just to say thank you very much for this forum, all of you are strong, amazing peopleļ. I thought that it is my time to share my daughterās journey. I apologise in advance for my lengthy post but the details might be of help to othersā¦ We are living in Ireland. Our daughter was diagnosed in 2013 (12yrs old back then) with both UC (colonoscopy) and PSC -Stage 2 (liver biopsy). She was put straight on a series of traditional meds: corticosteroids, followed by sulfasalazine, and immunosuppressive. She was extremely sick, losing weight, not being able to walk for 10 minutes, lying down on the couch, pain in the upper right quadrant of the stomach and extreme itching. We even tried the SCD (Specific Carbohydrate diet) diet for 8 months. The UC symptoms improved but the PSC did not: the LFTs were up to the roof (ie. GGT 700-1000, ALT 300, ALKPHOS 500). The doctors told us that we just have to accept this and deal with itā¦
I went on the internet, I have researched all the research papers that I could find on these two conditions and I came across Dr. Coxās successful research on the 14 kids (UC and PSC) treated with Vancomycin. I contacted him via the email provided on one of the research papers and he kindly offered to help us with info on the Vanco protocol. The doctors in Ireland did not want to cooperate with him, nor to prescribe Vanco to us, justifying that Vanco is just in the research phase. We found a doctor in my birth country who was willing to help us. We started our daughter on Vanco in 2014 (Kabi IV compound) 500mg, 3 times per day. In three months her LFTs normalized, she started to feel much better, no clinical symptoms of UC or PSC. She was gaining 1kg in weight and 1 cm in height every month for 8 months. Her LFTs remained stable for 1 year. Dr. Cox changed the dose to 500mg, 2 times per day. She continued on that dose until today, LFTs are normal. In 2015 we have done a Fibroscan test (which measure the stiffness of the liver) and the value came up very high, basically she was border cirrhosis. The MRCP test (MRI scan of the liver and billiary ducts) was showing improvements though: very mild beading of the intrahepatic ducts and normal spleen and gallbladder. We continued the treatment and in 2017 we have repeated the Fibroscan and the results showed that she has a normal liver now. It took almost three years of Vanco treatment for her liver to normalize. She is a normal 16 years old teenager now, thriving and wanting to do Medicineļ.
Andā¦when the doctors here in Ireland have seen that the liver is normal now, they started to prescribe Vanco for her. We got our daughter back, she is a legacy of Dr. Coxās effort to fight this difficult disease. Andā¦JTB thank you very much for your info and presence on this forum, your knowledge about this disease and the Vanco treatment is very helpful. Daniela
3 Likes
Daniela,
Thank you for sharing your daughters story. This is amazing news and we are all so glad that she is doing well! I hope this continues for her in the days and years ahead. Please stay up on regular visits with her hepatologist. He may still want to do annual MRCPās to keep an eye on the PSC to make sure itās not advancing. Weāve heard some very promising reports from others as well about the Vanco treatment. Iām very glad itās working for your daughter. We wish her all the very best and hope she lives a very full and active life.
Mark
Hi Mark,
Thank you for your kind words. Just forgot to mention that my daughter is only on Vanco now (Kabi IV compound) 500mg, 2 times per day, Lactobacillus acidophilus probiotics (6 billion CFU) - 1 capsule per day, Omega3 - 1000mg per day and Vitamin D - 1 tab per day (800IU).
We do MRCP and Fibroscan annually and she is in the care of a GI (public health system) and a hepatologist (private). The GI doctors here did not hear about MRCP and Fibroscan, I found about these tests from reading research papers (Pubmed is a good resource on that) and we have to go private to do the tests. Good luck to everyone fighting this disease. All the best wishes. Daniela
1 Like
Daniela, this is awesome! Iām glad to hear she is doing so well and thanks so much for sharing.
Do you know if her doctors would entertain the possibility of treating other PSC patients with oral vancomycin on a case by case basis?
Hi JTB,
I donāt think so, in fact it was extremely hard to get the Irish paediatric consultant to prescribe her Vanco (almost 4years). He is an amazing person, very open to research. After he saw that her liver normalized, he started to prescribe it (the doctor who prescribed up until now was from my birth country and he basically was doing it only for us, being impressed by our storyā¦). We have to pay for it privately as it is not covered by the insurance (public or private): 400euro/month in Ireland and 600euro/month in my birth country. My daughter just transitioned to adults now, and we still have to see if the new consultant here in Ireland is willing to prescribe it as well. In Europe, it is almost impossible to get a doctor to prescribe Vanco long term. The battle continuesā¦Also, what I found during all these years, the treatment is sensitive to the brand. To us, Kabi IV compound worked quite well (ALKPhos started to decrease dramatically on it). Sorry JTB, I wished I could have been of more helpā¦
1 Like
Sorry JTB, I really cannot go into the details of the battle that we had here with the system to get our daughter on Vanco. It was, still is a battle. Probably, in a couple of years time things will change in Europe (re prescribing Vanco for PSC). We hopeā¦
Thanks for the info, Daniela, and sorry to hear it has been such a struggle. I too noticed a wide variation in results between different generics and also found IV Kabi to be effective. What other IV generics has your daughter used and how did the results compare? I hope your daughterās transition to an adult consultant goes through without a hitch.
Teva IV, Mylan IV, Demo IV, Kabi IV (the Vanco tablets are not available in pharmacies) . Out of all of them Kabi IV seemed to have made a real impact on the ALKPhos values. I know that the ALKPhos value is not relevant in children(GGT is the one to watch for kids, as high values of ALKPhos in kids could be purely an indication of bone growth rather than liver disease), but we have noticed that since we started on Kabi IV (1 1/2 years ago) her ALKPhos continuously decreased and now for the first time in 3 years it reached a normal value. Also the Fibroscan showed a normal liver in Feb. 2017.
1 Like
Hello, I am new member in this group. I have my sister she is 19 years old and she has PSC confirmed on December 2016 by MRCP. Since that time we did a lot of work up, 3 months back she started on vancomycin, brand name (vancolon) 500mg tid. Last blood test was done yesterday unfortunately, her investigations worsen and for the first time her bilirubin reached 2 mg/dl.
How can I get vancomycin, brand name ( kapi ) knowing that we are living in Saudi Arabia.
Thank you
Hi there. Please ask your pharmacist if they know a supplier that would provide Kabi for you. Sometimes the pharmacists will go far and beyond to keep the customers in.
1 Like