Stem cell transplant or Methotrexate

Hi I'm new here but I would like to know if anyone in the U.K. has had a Stem cell transplant for diffuse systemic sclerosis? I was diagnosed just last year 2016 with no skin activity, but 4 months later my arms were covered and my skin score was 10. Now by February 2017 my skin score is 28 and my body is covered. Thankfully my lungs are still clear but it's looking like my heart may now be infected. I have been offered a choice of Methotrexate or a Stem cell transplant by the Royal Free hospital. I have currently had mmf at the highest dosage possible and I have just finished cyclophosphamide which have both been unsuccessful. My local Rheumatologist has said that the Methotrexate is unlikely to be successful as it's the weakest treatment out of the two ive already tried so thinks the transplant would be better. I would just like to know if anyone has had a successful transplant for this disease in the U.K.? Plus how advanced were you when you had the transplant? Thankyou in advance for just reading my ramblings 🤗

13 Replies

  • Morning Cole57, just to say I am thinking of you but unfortunately I unable to help with the decision. I have had Scleroderma for 30 years but mainly effects my digestive tract plus lots of calcinosis. I was on methotrexate for a while but had to come of it as it didn't agree with me. Could the Royal Free put you in touch with someone who has had a stem cell transplant.? There was a young lady speaking at last years Scleroderma Day in London but she had hers done in America. Also there was another young lady in the audience who was in the middle of her treatment which was being done in England . Maybe if you contact SRUK they may be able to help you find someone to talk to. In the meantime sending lots of love and healing thoughts xxx

  • Thankyou for replying and just hearing you've had the disease for 30 years is inspiration in itself and will make my sister feel better. So many stories are negative and we are both struggling to come to terms with how quickly I have become so sick. One of the problems we've had researching the transplant is most patients have had it done in the states. It seems there are very few that the Royal Free have actually done, and my suspicion could be the risk of mortality rate of 10-15% two years after the procedure. But I will contact SRUK and see if they can help. But thankyou and sending you healing thoughts on your journey xx

  • Hi, you mentioned that methotrexate didnt agree with you, plz tell me, in what way were you affected? I have systemic scleroderma from 2013 and consultant put me on same medication in Oct 2016, 4mnths on and i am just getting over a severe mouth infection. Wondering if the methotrexate caused it!

  • I haven't tried Methotrexate I was actually put on Mmf at the highest dose possible but it didn't work for me as my condition got worse. I was then given 6 pulses of cyclophosphamide and that hasn't worked. I have been offered the Methotrexate by the Royal Free but have been the that it probably won't work either if the other two I tried didn't. So was also offered the transplant. But I do know the Methotrexate has a lot of side effects which can vary from person to person. Like most of these drugs keep notes of the things that happen.

  • Hi, sorry to hear about your mouth infection - methotrexate can cause this but with me it was severe nausea, giddiness and it really effected my digestive tract which was already severely damaged . take care and sending lots of healing thoughts xxx

  • Thank you Monica for your reply and good wishes. I go in morning for oesphagus dilation and have an appointment with my rheumy consultant following day! Have to wait until then to see if he will change methotrexate to something different. God Bless. Keep good. X

  • I'm very sorry you're going through this. If you have not had a favorable response to the drugs already tried, I agree that methotrexate is unlikely to be very effective. Are your seeing a scleroderma specialist, or a general rheumatologist? It's very important to get an assessment by the former with aggressive diffuse SSc if at all possible. Has anyone mentioned rituximab infusions as a possibility?

    A stem cell transplant (HSCT) is the closest thing we have to a cure - when successful it can stop progression but can't reverse all damage already done- but it's also a very serious procedure. The procedure itself carries a mortality rate of anywhere from 4 -10% depending on the center. Additionally there's a percentage of people who relapse, but others have near-miraculous outcomes. HSCT is sometimes contraindicated when there is heart involvement, although Dr. Burt at Northwestern in the US has developed a heart-safe protocol that may have been adopted by other centers by now. A stem cell transplant is a tough procedure to get through, but can be life-saving for good candidates. You have to be "sick enough but not too sick" to qualify. It's a big decision. If possible, get a second qualified opinion and do your homework. Have you seen Dr. Burt's online videos about HSCT? He is the one who pioneered the procedure and has the most experience performing it for SSc. There is also a Facebook group called Scleroderma Stem Cell Pioneers for patients who have gone through the procedure or are researching it. Look through the files in the group, watch the videos, and ask questions, it will help you.

    I wish you all the best and will keep you in my thoughts and prayers. I hope you will keep us posted.

  • Thankyou for the info and reply. I have been offered the transplant by professor Denton at the Royal Free as they think I would be a perfect candidate. Thankfully so far my skin is the main effected area as I'm completely covered and have the usual myopathy. So far my organs are still clear which is why they think it would be good to have the transplant now before I get worse. A lot of patients in America have had the transplant when their disease has progressed a lot further. So in my mind I'm wondering would I have a good outcome with the transplant because it's more about my skin right now? It's just such a huge decision to make especially having children to consider and the recovery afterwards! But I will definitely look into the online videos you have suggested.Research is so important with this disease but thankyou for the advice and I will keep you posted. Wish you all the best to xx

  • Im sorry the MmF didnt work out. Wishing you all the best whatever you decide to do.

  • Thankyou for your kind words. I'm actually on cyclophosphamide again and it was decided I should do the full 12 cycles. I'm currently on cycle 7 and it's thankfully working, especially now I have heart involvement. So the stem cell treatment may now no longer be an option so hopefully when I see The Royal in November they will have a new treatment plan in place for me.

    But I hope everything is going well for you and your treatment process.

  • Cole57, has rituximab been discussed? It has had some encouraging responses in SSc and would be more powerful than methotrexate and less drastic than HSCT. What scleroderma antibody do you test positive for?

  • The Rituximab is the next drug option after the chemotherapy. I tested positive for the diffuse antibody and it's spreading quickly. Them finding it in my heart was unexpected and that was only diagnosed a few weeks ago. So I'm about to have a heart biopsy to see whether the disease is active or non active. But the chemo is the standard treatment for the heart as well anyway. So they hope to see also if chemotherapy works in early stage heart disease patients with scleroderma.

  • There are several scleroderma antibodies that are associated with the diffuse form of SSc, each with different disease characteristics and risk factors. It's important to know which one, do have copies of your labs? SCL-70 is the most common diffuse antibody, followed by RNA polymerase III, but there are others as well.

    I was just started on the new version of rituximab (ocrelizumab) but too soon to gauge results. Best to you.

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