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Stem cell transplant - when to use - experiences


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#1 anonym4444

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Posted 17 February 2008 - 02:37 AM

Hi to all who had stemm cell transplantation,

I'm fairly new to this disease (have it about 10 month) and have met many people in the course - especially in Germany (where I live).

I have recently met a young family with a kid of 9 years whose father has severe SSc (diffuse PSS). He can barely move in the house, has a haertpacer at age 40 and his lung is also involved. He can barely move up the stairs into their bedroom and he has been spending rougly 1/4 of the last year in hospitals without real help. He has had the disease for 4.5 years now. His wife is pretty much left with all the burden of earning the family income, caring for the kid, the house, the cars and the husband...and is slowly but surely running out of steam and feels more and more helpless.

I have spoken to her and was amazed that she even had the nerve still to give me (who has almost no problems yet compared to that) some advice. I have talked to her about stemm cell treatment as I've read many stories about it myself especially on english sites.

However in Germany the treatment of SSc is far behind and also there are no real specialized centers - it's a bit of a mess to get you diagnosed first and then there is also total confusion about how to treat it. So stemcell transplantation is not often used here and so there is little experience and also they tell you a pretty bad survival rate (<50%). I have suggested to her to post a few questions here in this forum to return her a favor (and as I lived for 7 years in CA...I'm a bit more fluent in medical terms too than she would be).

So: what are the experiences of those who had stemm cell transplantation here? - Did you have it also ONLY with such a severe condition as described for her husband above - she is obviously worried, that her husband is already too weak to survive it - however one women here in Germany did tell her, this wouldn't really matter, as anyway first the whole immune system is killed by chemicals, so that the condition doesn't really matter....
When is stemm cell transplantation used in the US (or elsewhere)? Early? only with fast progression? Or only late with severe organ involvement, if anyway there's nothing to loose anymore? What are the experiences in under which conditions?
It would be especially good, if somebody, who had it under similar bad conditions could tell her his experiences. (I'm offering translation services to the best of my abilities)

It would be nice to get some answers as she is really pretty much at the end of all other options (none of the tried out medicines have helped so far...). And you can clearly see, that it is wearing her out after all the disappointed hopes of the past few years.

Thanks for your answers,
K.

#2 janey

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Posted 17 February 2008 - 05:05 AM

K,
Welcome to you and your friend! It's so wonderful to research on your friend's behalf even when you are dealing with some of the same issues as her husband. But then getting information for her, will only help you as well.

I can't speak from personal experience about SCTs, but I'm sure you will hear from the few that have had them. It's not the normal treatment and is usually for people that have a fast progressing diffuse scleroderma. I can provide a link to our Stem Cell Transplant page. It should give you some information that will help.

Also, I did find a list of hospitals in Germany that are part of the Scleroderma Clinical Trial Consortium. There are four hospitals listed. Hope this helps.

Again Welcome! I'm sure you'll hear from others throughout the week.

Big Hugs to you all,


Janey Willis
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#3 lizzie

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Posted 17 February 2008 - 05:46 AM

Hi K
I have no experience of stem cell transplant - but just wanted to say that the ASTIS trial is evaluating it in a european wide trial that includes centres in Germany. One member on this forum has recentlytaken part in this trial and has had a very agood outcome. I think that there should be an ASTIS trial website which gives more details about it.
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#4 jefa

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Posted 17 February 2008 - 05:52 AM

Hi, Lizzie. We do have a page on the ASTIS Study for those that are interested.


Warm wishes,
Jefa

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#5 GocartMoz

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Posted 17 February 2008 - 07:25 AM

Hello k:

I am sorry to learn of your diagnosis, as well as that of your friend. I can tell you that in the United States, stem cell transplants are only offered currently as part of a clinical study. I had one in March of 2007 as part of the SCOT study. As others have mentioned, they are offered in Europe as part of the ASTIS trial, which I have heard has had some wonderful results. I can tell you that in the SCOT study, they are offering SCT's to those only in the early stages of the disease and only for those who have diffuse scleroderma. In addition, they are looking for those that have a rapidly progressing disease process. I know of no study that is offering the transplant to those in the late stages of the disease, especially with severe organ involvement. I can tell you that I have had a wonderful result from the SCT and have spoken to many others that have gone through the process, only with wonderful results. The road is a difficult one, but in the end all those I have spoken with who have undergone the procedure have had a great recovery. It is my hope that once the clinical trials have concluded, the procedure will be approved for all who endure this dreaded disease.

As far as the survival rate, I too was told when originally diagnosed that the survival rate for systemic patients is <5 years, in 50% of the patients. That being said, I have come to not put much faith in that statistic. There are many on this board who have survived 4 or 5 times that. Please don't obsess with those numbers. It sounds like you are doing everything you can for both your friend and yourself. Unfortunately with this disease, in addition to finding the right doctor, it is so important to be proactive. I think that is exactly what you are doing. If you have any other questions, please don't hesitate to ask or e-mail me privately. I would be happy to help.



#6 PrincessB

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Posted 17 February 2008 - 09:57 PM

I recently had a stem cell transplant as part of the ASTIS trial. If you want to read about my experiences, do a search on posts by PrincessB and read the ones with that in the title. For me it has been successful so far, so I am a big fan!

I see one major stumbling block for your friend's husband getting on to the trial and that is that one of the eligibility criteria is having had the disease for four years or less. Or rather, it says "</= 4 years since development of the skin thickening". He should definitely ask to be assessed to see whether he's eligible, the sooner the better.

Also, because it is a trial, there is only a 50% chance that he will be given a stem cell transplant, because they are comparing the efficacy of stem cell transplants vs. the traditional treatment of chemo only once a month for a year. The link to the ASTIS Clinical Trial page is very useful and has all the info you need.

If you want to ask me anything else, just mail me. You don't say too much about your own condition, but maybe you are eligible for the trial as well?
Diagnosed diffuse systemic scleroderma December 2005 (on my 30th birthday, as if turning 30 wasn't enough?!)

#7 peanut

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Posted 18 February 2008 - 12:33 AM

I’m going to have a stem cell transplant very soon now. :D I have the very rare super aggressive form and nothing is slowing the lung progression. True, with stem cell transplants they use chemotherapy & immunotherapy to get rid of the broken immune system but methodologies are progressing toward less toxic methods. Right now it really just depends on if you meet the qualifications of the study, :huh: so where you are in the progression doesn’t always play a part. Mine is super progressive, yet I know others who only have mild progression in body, lungs or/and skin.

I chose stem cell transplant because it is the only aggressive I have choice left. My lungs are quite poorly due to scleroderma and have progressed extremely rapidly over a year. I am 28; a year ago I lead a normal life and now I’m on federal disability and on full time oxygen. On paper my lungs are beyond the qualifications of the study, but my heart is healthy so I think I’m being treated, but off protocol – or at least I think that’s why the phrase “off protocol” is on my paper. The doctor told me straight out I have very few options and I am high risk, higher than normal with a mortality rate of 5%, yet I am willing because the benefits out weight the risks. I fight to live a better quality of life even if it only means I gain a few years. I would rather fight and go out passionately than give in to a slow and painful outlook.

It is important to remember for as much as we hope this is the “cure” stem cell transplants are still considered experimental – internationally. There are many questions left unanswered, like if it truly works, what exactly is the best application and methodologies, long-term effects, etc. And it can be quite expensive… well maybe not so much in Europe.

For me it wasn’t a question of if I should do it. It has clearly become a question of when can I get on the plane and get it started.

Best of luck, peanut

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#8 anonym4444

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Posted 18 February 2008 - 12:41 AM

Hi,

I did read your recent post - big congratulations...

Yes, I'm certainly also on the lookout for myself...but I guess as I'm not even diagnosed yet, this would be no option yet.

But I definitely would urge for it. I read your from Strassbourg...so maybe you are even German spoken. Never mind...

You can read a bit more about my conditions in my first post earlier (around January).

My problem is, that if I compare myself to many others in the forums in Germany, then I clearly have it...I have read enough by now to be almost certain - I would even be rather certain that it's PSS....

However I'm one of either the lucky or unlucky persons, that do not have ANAs (yet?) no Raynauds (yet?) and I do not have sclerodactily (yet?) nor the special mouth changes (but those should come late only anyway).

So: should I call myself lucky as I seem to have an uncommon (and probably slower start) or call myself lucky as I seem to recognize many signs before having sever problems with organs...? Or should I call myself unlucky as so far I get treated as hypochonder from some of the doctors. Or should I call myself even more unlucky and compare myself to those poor guys, that even after 10 years have no ANAs and such absolutely unique signs, that doctors still are not really certain how to diagnose (despite them having clear organ involvemts)....

I don't know...rather frustrating and I'm almost at the point of giving up....count on that on top of it, that I'm male (which doesn't make your situation simpler at a doctors office and also seems to have more severe progression) and count on that the fact, that I'm the income provider in our family in Munich with quite a number of bills to pay and two young and wonderful kids to take into consideration and then you can probably (well you can for sure, as you've been through it) imagine, what it means to sleep well at nights...

I'm really going between pushing another doctors visit and giving up for now - nevertheless I clearly have some of the clear indicators - just most of the rheumatologists don't look for thos more subtle beginning signs - but the skin of my hands has clearly changed in appearance, texture, color and also in blood circulation and behaviour when cold and it has changed by seeing the shining through veins and it is dry, feels different, looses the natural fingerprints - and get's folds in the other direction.....add that to a ton of other symtptmos (like tendon problems in the spine) and gastrointestinal problems and much more sleep needed (despite the fact, that I can't sleep after max of 7 hours) and you will probably agree to have somebody in front of you, who will in a few years from now maybe be a candidate for stem cell treatment....I'll see...

K.

#9 PrincessB

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Posted 18 February 2008 - 09:48 PM

K,

I would suggest that you take photos of your hands and face, obviously you will already have some of your face so you will be able to do a comparison and also show your doctors. In England (where I was diagnosed) the specialist didn't rely at all on blood test results because as you are finding out, they are not necessarily conclusive, and symptomatic evidence is sometimes the only way to make a diagnosis. My doctors here in France asked me to provide photos when I had my baseline examination. My SCL-70 result is sometimes positive and sometimes negative (is that an ANA? one of the problems of living in a foreign country is not always knowing the medical terms!).

Keep on pushing for an answer - no one will ever care as much about your health as you, so don't give up!

B.

P.S. I understand German pretty well, but I can't speak it that well, am having lessons at the moment. I'm a French and Italian speaker.
Diagnosed diffuse systemic scleroderma December 2005 (on my 30th birthday, as if turning 30 wasn't enough?!)

#10 anonym4444

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Posted 18 February 2008 - 10:47 PM

Hi,

thanks.

Yes, SCL-70 is (to my understanding) a specific type of ANA heavily indicating PSS (SSc).

ANA = Antinuclear Antibodies supposedly are existent in 95% of the scleroderma patients...however also some healty people have them and they supposedly are existent early on (but practical cases in forums show a different picture).
Whereas the specific ones (like SCL-70) are only detectable in about 40% of the diffuse scleroderma patients (and they are normally used to categorize "which" exact form you have).

However for ANAs there even seem no "hard" limit...so some doctors and labs claim a solution thinned with 1:40 is negative, others claim 1:80, again others claim only 1:160 is starting to be positive....but in my case, I'm anyway <1:80..so I guess here I seem definitvely negative and also the specific testing seems to be negative for the major Antibodies that are involved (however as the specific ones only show up for max. 50% of the patients anyhow, this really doesn't mean much). The ANA would mean more, but yet I could be the 5% that never have those....or not yet...

It's interesting, that you went to UK to get diagnosed - where to? I also read many interesting articles from UK and it seems they are more advanced and more centralized experience then the German doctors.

Let me know, "where"...I'd be willing to almost go anywhere to find a final conclusion as early as possible.

How long did it take you from first signs until diagnosed....?

Pictures: well I do certainly also compare that myself...but also that is sometimes hard to see for others in the early stages...do you see some small wrinkles around the mouth that you may have in the beginning - no, you wouldn't.
Do you see that strange dent in the checks, that forms if you try to smile....(which was much smaller earlier on)....not even I myself could for sure tell...left alone others.

And try to show that to a German doctor: and you'll be stampmarked hypochondric...
Yes, I did photograph my hands (and probably should do even more often).

Howeer that doesn't show, that for instance if you try to open your mouth wide, there is a tight sensation around the lips...it doesn't show some small strange wrinkles and the sensation you have which is different....

And it has a hard time showing tendon problems of the spine and sicca...which are my main symptoms (apart from hand skin changes and feet skin changes).

If doctors look at my hands, (if they even do that), then they "can't see a difference"....however I do (and some of my buddies on German forums with similar problems and similar appearance can as well see it and I can see their strange vein appearance)...

#11 PrincessB

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Posted 18 February 2008 - 11:09 PM

I am English and was still living in England when I was diagnosed. The Royal Free in London is apparently a great centre for scleroderma, I guess if you pay you can go there as a private patient - but it's expensive to go private in England! Strasbourg is a centre de référence for autoimmune disorders, one of only four in France, so I was really lucky to have ended up living here. Did you check out the link Lizzie (I think) said to look at to see where the centres are in Germany?

I'm sorry you're so frustrated, try not to get too stressed though, I'm sure it can only make it worse.
Diagnosed diffuse systemic scleroderma December 2005 (on my 30th birthday, as if turning 30 wasn't enough?!)

#12 anonym4444

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Posted 18 February 2008 - 11:41 PM

Hi,

thanks once more...yes, I know, that Stress for sure is no good...Nevertheless...try to reduce stress in such a situation?...

You did not answer my question, how long it took for you from the first signs (that you yourself recognized) and the point you were finally diagnosed at the end of Decmeber of 2005...? Which were your very first signs?

Thanks...I hope I'm not too pushy here...
K.

#13 PrincessB

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Posted 19 February 2008 - 05:41 AM

Hi K,

First of all I got Raynaud's, so I went to see my general practitioner. She referred me to a secondary specialist for him to monitor me. This was in January 2003. During 2005, I noticed that the skin on my hands and face was changing and I think I also got my first ulcer around that time, so I went back to see him and he decided based on the circumstantial evidence that I had scleroderma. I can't remember if I had a positive SCL-70 that time, I think he used two labs actually and one said yes and one said no.

So my Raynaud's was the first sign, but of course only a very small percentage of people who have it go on to develop a secondary condition.
Diagnosed diffuse systemic scleroderma December 2005 (on my 30th birthday, as if turning 30 wasn't enough?!)

#14 peanut

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Posted 19 February 2008 - 06:04 AM

Hi K,
My delay was much longer. I remember having raynauds when I was a teenager and what they thought was asthma. It probably wasn't asthma but I wasn't diagnosed then. I wouldn't get diagnosed for 10 years and by then my condition had moved from inactive to exploding.

If you're trying to find a timeline there is none. We are all different. Some people stay mild their whole lives while others flare up then fizzle out. Sclero can also randomly go into remission too. There's no telling when or what will happen and no answers to why either. While answers are few, sclero can be stabilized and slowed by getting proper managed care and treatment.

peanut

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#15 anonym4444

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Posted 19 February 2008 - 07:18 AM

Hi,

thanks for your answers...yes I kind of know that...the only thing that really freaks me out...is that everybody says: you can at least somehow try to stop it with proper treatment..and early diagnosis...but if you can not get diagnoses (Raynauds people immediately look for the other signs...but without Raynauds...is difficult - and without ANAs).

So what I found from the German forum...there are many examples who did get diagnosed wrongly for years and they are actually in pretty bad shape right now...and there are other examples which more or less by accident and a good doctor got diagnosed early and they actually have little impact to their lives...so early detection is key to a diseases where at most you can hope to stop the progress and freeze it's current impact....

However the longer you wait with that...the worse your symptoms get...
On the other hand, only if you have the first severe symptoms some doctors will be able to diagnose....

Doesn't this sound like a vicious circle??? To me, it does...

I mean if you clearly (by circumstantial evidence) recognize more and more, that it can only be this...and then you go to a doctor and he tells you, that he doesn't see the big stuff yet....interesting...

Today I got told by the Munich University, that my Xray of the spine (which is heavily the victim of my tendon problems) is beginning to degrade...perfect at age 38...but they told me: common with young man - most likely Morbus Scheuermann...however in my case, this is not Morbus Scheuermann...it's the tendons pulling and causing the degrading spine...and they only do this in the last 10 month...worrysome if they already succeeded so much as to be able to see this in the Xray (I can anyway feel it - I felt it after only 2 month -actually it was the worst back then).

So that is, what drives me nuts...how do you find a doctor, that puts together the pieces of the puzzle and starts treating you BEFORE you have lung fibrosis and heart problems and kidney problems....I'd like to go on with my job..I actually have to...with two kids and house to pay for. And on top of that: I like my job and I don't want to hang at home and be frustrated with my life for the rest of my life and feel pitty for myself...(sorry, I don't want to hurt anybody and as I'm new, I may be still in denial...but as male and income provider with young kids this is also a bit more severe situation, than if I was 20 or 15 years older and the kids could well do without me as well...currently however they are fully dependent.)

K.

#16 netta

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Posted 19 February 2008 - 08:29 AM

Hi K,

Maybe if you were examined by specialists in scleroderma you'd have better luck. The following are German centers for the ASTIS trail, and the doctors associated with them.
Alles Gute!
netta

Klinikum der Johann Wolfgang Goethe-Universität, Frankfurt
Prof. Dr. J.P. Kaltwasser, M. Thun, Dept. of Rheumatology
Prof. Dr. S. Klein, Dept. of Hematology
Phone: 00 49 696 301 118
e-mail: [email protected]; [email protected]; [email protected]

Albert-Ludwigs-Universität, Freiburg
Prof. Dr. HH Peter, Dept. of Rheumatology
Phone: 00 49 7612703448,
e-mail: [email protected]

Medizinische Universitätsklinik und Poliklinik Tübingen
Dr. I. Koetter, Dept. of Internal Medicine
Phone: 00 49 7071 29 85 138
e-mail: [email protected]

Medizinische Universitätsklinik und Poliklinik Würzburg
Prof. Dr. H. -P. Tony, Dr. S. Kleienert, Dept. of Rheumatology
Phone 00 49 931 201 70 24
e-mail [email protected]

Marienhospital Mitte, Universitätsklinik der Rhur-Universität Bochum
Hölkeskampring 40, 44625 Herne
Medizinische Klinik I
Prof. L.C. Rump, Dr. S. Weiner
Phone: 0049 2323 4991671
E-mail: [email protected], [email protected]

#17 anonym4444

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Posted 23 February 2008 - 11:34 PM

Hi Netta,

I don't know if you know the German health system...

I know most of these centers by now...but you don't get there unless you General Physician refers you to a Rheumatologist and he sends you there....

Now try to go to general practitioner you statistically once in his life sees (any) Sceroderma patient and try to show him your hand changes and then if you convinced him (or he simply sends you there to get rid of you) try to convince the rheumatologist who looks for Raynaud (don't have) takes blood and looks for ANA (don't have it - but many don't - especially in the beginning) and who looks for Anti-Scl70 (don't have it - but only 40% have it)....and then he looks for the other ARA criteria - ulcers (don't have it after 10 month), sclerodactyly (yes, my skin hurts when stretching but currently I can still stretch my fingers despite that and as this is one thing I CAN fight, I will fight it)...and Teleagicstasias (well many people have them much later only) and problems swallowing...(don't have it).

That's it...-> Hypochonder...

Nobody cares about Sicca, nobody cares about tendon problems (massive in spince) - even now with a diagnoses starting degenerative spine diagnosed they don't see any connection to scleroderma and no need to examine this by somebody who may have seen a scleroderma patient a few times in his life...

So it's really not about knowing the right addresses - it's about finding somebody early in the "filter system" to refer you there.

But many thanks to all of you for the support. I by now find another women here in Germany who is almost a copy of my case and has the similar problems getting diagnosed. That also helps. I will fight on...

K.

#18 ladyhawke

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Posted 24 February 2008 - 04:26 PM

Janey....is there any clinical trials available in Canada? - Thanks - Lisa
Life is NOT meant to be a struggle. Life is meant to be joyously abundant.