New and considering stem cell transplant
Posted 21 April 2009 - 06:00 PM
I'm 30 years old now and I was originally diagnosed with limited systemic scleroderma in March of '05. I lived mostly with some thickened skin on my hands and fingers and frequent Raynaud's attacks. I was also getting regular ulcers on my fingers and experiencing mild heartburn.
This past November I called my rheumatologist that I had not seen in almost 2 years after noticing a drastic change in my lung function over just 6-9 months. She ordered a PFT and echo to be done before my visit with her. We found that my basic lung funtion is at about 35%. My diagnosis was officially "upgraded" to diffuse systemic sclerosis in December of '08.
In January I began receiving monthly IV Cytoxan and will conclude that treatment in June. I know everyone responds to this differently, but I have had what I would consider a pretty good experience with Cytoxan. Now, I don't know that it's done anything significant to help yet, but I have tolerated the drug very well. Never really getting nauseated, thanks to drugs and not even losing my hair (although it's thinned).
After much research and talking with a physician at Duke University (which is also where my brother happens to work), my family and I feel I haven't a lot of good options for slowing lung progression and improving my quality of life. At this point I am still able to do most of the things I love, although it's more challenging to go shopping or to sporting events and things where I have to walk a lot or climb stairs. I feel looking into my options for a stem cell transplant is a good choice. I know I do not qualify for the SCOT study because of my lung function, but I have contacted the study being conducted at Northwestern and they seem to think they may be able to help me. My mother and I will be driving the 6 hours next week for an evaluation.
I just want to give myself the opportunity to be as well as I can for as long as I can. So, I guess my point in writing is to ask what would be your thoughts or ideas about treatments. What has worked for you? What have you seen that looks promising? Anything helps.
Posted 22 April 2009 - 04:26 AM
I commend you for taking this step in looking into your options with transplant. Dr. Burt, at Northwestern, is a wonderful doctor and hopefully he and his staff at Northwestern will be able to help you. I have spoken to many who have had transplants there with wonderful success. As you may know, if you have followed previous posts, I had my transplant done at Duke in March of 2007, as part of SCOT, and have had wonderful results. While there are never any guarantees, it has turned my life around and I have never looked back. If you haven't contacted Dr. Sullivan and CJ Paarz-Largay, at Duke, regarding SCOT, you may want to do that. I think, though I am unsure, some of the requirements for entry into SCOT may have changed. As such, you may find out you qualify, despite your lung capacity issues. In any event, both Northwestern and Duke are great options. Best of luck to you and if I can answer any questions for you don't hesitate to ask.
Posted 22 April 2009 - 06:39 AM
Thank you so much for sharing such informative experiences with sclero family. My daughter who is 11 years, cute girl, is receiving cyclophosphomide infusion. She has received 3 cycles. She has Raynaud's and skin thickening. Worst part is she walks on her toes so she is unable to do daily routine because of contractures as well as progression in skin problems . I would like to know how many cycles did you receive? What is the dosage schedule? How is your well being after drug? You can PM or email me if you like.
Thank you so much.
Posted 30 April 2009 - 04:56 PM
Posted 01 May 2009 - 04:47 AM
Thanks for sharing this update with us.
I think many of us wonder if we should do the stem cell transplant. I myself have wondered, but according to my current doctor I'm not bad enough yet.
I hope that you are able to raise the funds that are needed, and please keep us posted on how you're doing.
Posted 01 May 2009 - 12:01 PM
Posted 04 May 2009 - 04:23 PM
Northwestern is a fantastic facility and Dr. Burt has much experience/success with the stem cells--just make sure you understand the risks. Like you, I went for the stem cell but could not get insurance approval. I got my state's health advocate involved and spoke with the state's attorney general. All appeals were denied. At the time it felt like a death sentance.
In the meantime, I did the cytoxan treatments and changed my lifestyle completely by moving to a warm climate, not working and trying to stay active. For the most part this all worked. I also went into the Gleevec clinical trial for a year. In the end, my strategy was to try to "run out the clock" as it is my understanding that Sclero is the most active for the first 36 to 60 months. I hit the 36 month mark this month and my fingers are crossed.
I now have medicare coverage which will cover stem cells. The reason that I am choosing to not have it done is that I think I am relatively stable although my PFTs move around. Like you, I had no problem with the cytoxan -- I only had 6 dosages and I asked and asked to continue getting more -- I argued that if I couldn't have it monthly then at least let me have it quarterly -- I was convinced that I needed it to keep me stable -- I tried to get it from 3 doctors -- in the end, all 3 had the same response -- "the risk outweighs the benefits". Their recommendations were Cellcept/Immuran/Gleevec. So, this is the part I can't get my arms around. Basically the cytoxan I was asking for was a "thimblefull" compared with what is given for the stem cell. So, if 3 doctors won't give me a thimblefull of cytoxan because the "risk outweighs the benefits" -- then how can it be OK to get an ablative dose of cytoxan. When I asked this question, I was told it is a "different procedure in that it is necessary to do for the SCT".
I recently went to Northwestern again to find out how bad my PFT needs to be before they will NOT perform the stem cell. Basically, the DLCO can be at 14% and they still will do the stem cell. The issue is the heart. Your heart needs to be in tip top shape. My DLCO is at 50% -- so I figure I still have time. Basically my take is that I can be dragging an oxygen tank and as long as my heart is good -- I can have the procedure done. So, I am again opting to wait and try to "run out the clock" until the disease peaks if it hasn't already.
I wish you the best. Had the cytoxan not been able to control the lung progression, I would have had the stem cell -- and paid out of pocket. It sounds like you are at the 3 year mark and may be at the disease peak as I count my 3 year mark as when I got the acid reflux as I had the Raynaud's for 2 years before the acid reflux and the exhaustion started. I would guess that while you are raising funds, you will see if your lungs are stabilized or not. And I am assuming that Dr. Varga is recommending the stem cell transplant for you as he is the rheumatologist paired with Dr. Barr. Good Luck. You will be in good hands at Northwestern. Gidget
Posted 06 May 2009 - 05:58 AM
Posted 06 May 2009 - 02:59 PM
Yes, you are right -- stem cell transplant is your only option and you are smart to move forward on it. I will PM you with some info regarding people that may be able to help you with your fund raising.