HSCT on the Increase

How common is HSCT being performed as a treatment for MS and which countries are using it most? 

You may be surprised by the results. 

Snowden JA, Badoglio M, Labopin M, Giebel S, McGrath E, Marjanovic Z, Burman J, Moore J, Rovira M, Wulffraat NM, Kazmi M, Greco R, Snarski E, Kozak T, Kirgizov K, Alexander T, Bader P, Saccardi R, Farge D. Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases.Blood Adv. 2017 Dec 20;1(27):2742-2755

Between January 1994 and December 2015, there were 2097 transplant procedures in 2056 patients (62% female, 8% pediatric <18 years) registered by 247 participating centers in 40 countries.

The main indications based on patients treated were MS (n = 839), connective tissue disorders (n = 596), inflammatory arthritis (IA, n = 178), vasculitis (n = 46), inflammatory bowel disease (In = 191), haematological immune cytopenia (n = 97).

So in 1995 people were largely progressive, but with the realization that it is most active in Relapsing MS this demographic is changing.

Transplant rates differed substantially among participating European countries. The predominant countries of activity were Italy, Germany, Sweden, the United Kingdom, The Netherlands, Spain, France, and Australia.

With respect to stem cell source, there was a significant trend to increasing use of peripheral blood stem cell (1994-1999, 86.3%; 2000-2004, 92.9%, 2005-2010, 98.2%; and 2011-2015, 99.6%; P < 10−3)

Should there be “Centers of Excellence” where there is both HSCT and specialist expertise working closely in the same hospital? The concept of an experienced team might have benefits not only in terms of transplant skills, but also familiarity with the clinical idiosyncrasies within this patient group

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  • If someone gave me the choice between cancer and MS, I'd chose cancer (and this is coming from someone who's lost his dad and step dad).

    HSCT should not be viewed as extreme or specialist. It is nothing new. Its been around for decades.

    I see HSCT being treating like atheists, always labelled strident or extreme. When nothing could be further from the truth.

    HSCT will, hopefully, one day be something that nurses can administer and help patients recover from.

    Ironically, the most effective treatment for MS, is also one that makes logical sense, even the lay person like me.

    DMD's don't make sense to me. HSCT does. Hit early, hit hard – and you MS need never be the blight it is for many.

    • This whole 'i'd rather have cancer than MS' thing is a bit problematic for me. When I was first diagnosed I immediately felt that way. But 5 years on I feel completely different. Firstly, I have met a number of people with fairly serious MS disability who are still living out lives that, although probably aren't quite as they imagined they would be (whose ever is), still have value and still bring joy to them and those around them. On reflection I was frankly horrified with my initial reaction. To look at their lives and say 'i'd rather death than have that life' is not something i could justify. At best it was flippant. Now I might well feel that way one day, but I need to leave room for my perspective to change.

      Fast forward to now, and I'm having ongoing trouble with ITP post alemtuzumab and have also have to get my thyroid removed due to graves disease. This is not ideal (and maybe something I wouldn't have to deal with after HSCT, i don't know). But my MS has been stable for years now and life very good. When they decided that they needed to do a PET scan to rule out lymphoma I was terrified (it came back completely clear-phew). Now I will happily consider HSCT in the future, but I want it to be evaluated objectively and I won't be so quick to judge the choice between potential death and disability.

  • It's interesting because only this year more than 400 patients were treated privately for MS only, without including the US trial. And the leading european country in transplants was Norway, which appears to be the last one on the list (probably exactly because of that, as well as their high % of MS and their relative economic ability).

    So many medically interesting cases that are left without proper examination, it is such a shame for science really (not to mention the shame for patients who go bankrupt to save their lives).

    Wish scientists will soon wake up and see the knowledge treasure HSCT has to offer in autoimmunity.

    • In the study also mentions "overall survival (86% vs. 51%) also favored transplantation" which probably refers to deaths by the disease, so this is another factor those patients need to take into account.
      Here we are talking about myeloablative chemotherapy, which is not the regimen that is been used in the current protocols for MS. At the private clinics the deaths rates are close to 0,2% (The US trial will confirm that).
      Still it is interesting that myeloablative appears more effective for this autoimmune.

    • No. There are lots of progressive patients who have had disease progression stopped. It carries a reduced risk (as does disease time Ben with RRMS). But is by no means pointless.

      HSCT is so much more effective than any other DMD out there. It's so sad to see the needless suffering due to pharma, lack of humility by neurologists.. all on the merry-go-round. No that is pointless!

    • There are many studies you can find on HSCT and PMS, the success rates are between 50%-80%. From real life experience, you can not predict who will succeed and who will not. SPMS patients with high EDSS have remained stable for many years with even disability improvements and others (few) with only few years in SPMS start to deteriorate 2 years post.
      The reality is that the majority of patients who proceed privately with HSCT are SPMS patients.

    • Yes, in my opinion the risk of the procedure is not worth the reward. Very few people with progressive MS make it past 5 years without relapsing. We need neuroprotectives and myelin repair. While I understand this treatment is very effective for RRMS, we need to focus on other options for PMS. I feel like too many people are misinformed and believe this treatment will cure them, which couldn't be further from the truth in PMS

  • HSCT – the ultimate stupid "solution" for MS, and other things. Akin to having software problems on a computer and just switching it off at the wall, switching it back on, and hoping everything is okay now and that the hard drive hasn't corrupted and failed in the process.

    Heaven help us – particularly those of us with progressive MS – if HSCT becomes the go-to, lazy therapy for all and sundry.

    • I do think it works in RRMS, but I totally agree with you. We need to be turning our attention to different treatments for progressive MS now. It's not fair

    • Hi lz it doesn't matter if your RRMS, or PMS, we are 'll going to convert to progressive. Good news there are 5 treatments on its way. Ocrelizumab, Spinimod, Mastlinab, Ibudalist, Statins. Wait is nearly over for ppms patients. HSCT is like taking a sledge hammer to crack a nut.

    • a. They give Lemtrada to SPMS (not in UK), of course chemotherapy that penetrates brain can offer much more. There are patients with Lemtrada that are 10 years relapse free, imagine with chemotherapy that is not a flawed application as Lemtrada
      b. Lemtrada is also an immune "format", its not any brilliant idea but it works for now and we can learn a lot during
      c. If HSCT lasts less than 5 years it is considered unsuccessful, after the 5 year mark it has the potential to last much longer -of course its not a cure and MS can re-emerge- but it is considered a long lasting treatment. There are already SPMS patients who have been treated with HSCT more than 5 years ago and are stable and improved in EDSS.

      The clear indication is for RRMS, but hopefully someday we will be able to tell who PMS can benefit from the treatment, because the activity (lesions, relapses) is not a reliable indicator from what we see from daily experience.

    • "Ocrelizumab, Spinimod, Mastlinab, Ibudalist, Statins. Wait is nearly over for ppms patients. HSCT is like taking a sledge hammer to crack a nut."

      MD you have created a monster with your "sledge hammer" theories. Now patients believe that they can reverse time.

      The whole point with HSCT is exactly to prevent progressive MS, to halt the disease before the damage is already been done. All the damage, not parts.

    • No monster created. HSCT not a cure. Do you give HSCT to kids? No one wants to have this disease. Why keep pushing for HSCT over experts who have studied this disease all their lives. What makes u and beam greater expert? Have u beam had hstc? Put your money where your mouths are!

  • You're completely missing the point. If hsct was first line treatment. Then there would hardly be any progressive ms.

    We only have that problem now because of decades of ineffective dmd's.

    • There's a lot of wishful thinking going on around HSCT.

      What of those who never had any noticeable relapse activity? I am one of them.

      This blog attracts a lot of fundamentalist aggressive treaters, and HSCT is but the latest very blunt, dumb tool with which to bludgeon the desperate.

    • It is you indulges in wishful thinking. As I've said before, it is this very bluntness, as you put it, that is required.

      You don't adopt the same tone and stance when talking about chemotherapy for cancer do you?

      This 'dumb and blunt' tool, just so happens to be delivering THE best results ever, for pwMS.

      So you should really be asking who is the dumb and blunt tool?

      Bludgeon the desperate? There are more desperate among Dmd's than there are this who had hsct.

    • There is no filter or perspective in the evangelism shown in some of the comments

      However I have never suggested that any treatment turns back time.

      That is why I would advocate dealing with the problems that can be dealt with as soon as possible.
      There may be some natural repair once the inflammatory response is stopped.

    • I am (by your accusation, not my admission) strident in my HSCT opinion, only because the force of the status-quo and laissez-faire approach is harder to defeat and more damaging.

      Change takes change. The self-serving, lethargic, lackluster, illogical and harmful influence of neuroligsits, researchers and the medical class, combined with their antipathy, results in a pernicious, physically painful demise for pwMS.

      Those who need forward progress the most, are a at the mercy of those who's interests are best served by ignoring a treatment that has already proven highly effective.

      If HSCT were first line treatment, flip the pyramid as you say, then a lot of tears would be saved.

    • "So you should really be asking who is the dumb and blunt tool?"

      Predictable comment/attitude. Pointless discussing with your sort.

      MD, as I said, I had no "warning" with my MS, and seem to have had very little relapse activity. Just very, very slow progression. I do not believe that HSCT or even the DMTs would make sense in my case, and I know I'm not the only one. Neuroprotectives are what people like me need, in my opinion.

  • Late to enter this discussion but I was PPMS and had HSCT in 2014. Prior to treatment was deteriorating noticeably. In the 4 years post treatment I have stablised. Who knows if it will come back. So far though the disease has been halted and I only wish I had it done sooner. It is not a cure and I have not had improvements.

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