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High Dose Cyclophosphamide Without Stem Cell Rescue In Scleroderma


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#1 janey

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Posted 19 November 2007 - 06:35 AM

High dose cyclophosphamide without stem cell rescue in scleroderma.

High dose cyclophosphamide without stem rescue can lead to clinically significant improvement in skin score and measures of disease severity in patients with diffuse cutaneous scleroderma. PubMed. Ann rheumatologist Dis. 2007 Nov 1. (Also see: Diffuse Scleroderma, Clinical Trials, and Medications)

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#2 peanut

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Posted 19 November 2007 - 09:04 AM

This study is based on 6 people. Hum. Don't mean to be skeptical but that doesn't seem like enough to quantify a conclusion?

peanut

You can deprive the body but the soul needs chocolate
my HMO makes me wear a helmet...

#3 janey

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Posted 19 November 2007 - 10:04 AM

Peanut,
I totally agree, but it does take the small trials (Phase I or even Phase 0 in this case) to show success before the FDA will give approval to larger trials. No, it isn't a "conclusion" and isn't meant to be. It's just a reporting of results of a small trial.

My rheumatologist prefers to base his treatment decisions only on Phase II and III trials. However, I was able to convince him to let me try out Cellcept based on a couple of Phase I trials. There hasn't been a phase II trial with cellcept and pulmonary fibrosis because of the problems associated with getting "the numbers.

I posted an article recently talking about clinical trials with scleroderma.

Clinical trials in systemic sclerosis: lessons learned and outcomes

It states "A frequent problem encountered in rare diseases such as SSc is the difficulty in obtaining adequate patient numbers. As a result, compromises are made in terms of study entry criteria and patient management, which can complicate interpretation of the results. Another important aspect to consider in trials of SSc is the extremely slow progression of disease in many patients and the fact that spontaneous improvement is observed in many. This makes it difficult to show benefits of therapy over placebo."

What a conundrum. It's amazing when they can actually get quantifiable results from any trial with scleroderma. Thanks for mentioning this. I think it's a great discussion. It emphasizes the need for more participation in clinical trials. I checked out the SCOT Trial a couple of years ago, but was rejected from my inquiry email because I told them I had a pacemaker. So apparently, strict criteria is also a problem.

Big Hugs,
Janey Willis
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(Retired) ISN Assistant Webmaster
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(Retired) ISN Technical Writer for Training Manuals
International Scleroderma Network (ISN)

#4 peanut

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Posted 19 November 2007 - 11:43 AM

Janey,
My rheumatologist told me there are different kinds of trials. For instance, SCOT has a strict criteria so their conclusions are more specific. In Chicago there are lesser known, smaller stem cell clinical trials with more open criteria. Thus, they can conclude more generally, like "yes this works and we know some specifics but not detailed ones.

I too do not qualify for SCOT, but I may qualify for the trials in Chicago - hopefully.

My husband asks me about scleroderma research and development of a treatment or cure. I explained to him that 1 or 2 in 10,000 have scleroderma, and of those even less who wish to participate in a study, and of those less who actually qualify for the trial. It takes a certain number of people to move on to the next level of research and development - it is quite the challenge to meet the numbers.

I guess I am concerned since people may receive and take hope in incomplete information because they read studies whose conclusions are based on small numbers. I suppose it is a good idea to track such these types of studies as potential on the horizon... I dunno

babble, babble, babble...
peanut

You can deprive the body but the soul needs chocolate
my HMO makes me wear a helmet...

#5 Gidget

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Posted 19 November 2007 - 02:49 PM

Peanut -- I'm with you. The doctors like to quote how effective Cellcept is vs. cyclophosphamide -- however the study group was also small. And as we all know with sclero, we are all different. So the 10 people studied with Cellcept might not have the same condition as me or might be at different stages of the disease. What would we every do without the internet. Have fun. Gidget