Monday, February 11, 2013

Steady, As She Goes


It's been a long time since I updated my blog, but so much is happening in my life and in the world of scleroderma, that I felt it was time to share the news.

First and foremost, Dr. Burt has now published two articles on me, or at least me and my cohorts. :) Both articles were published in Lancet, a very well-respected journal. The most recent just came out this month. So, I want to post the links here in case you want to access and read these articles. The data, in summary, show very encouraging results. Those with aggressive forms of scleroderma are living longer and living better, thus far, following autologous stem cell transplantation. Relapse rate after transplant is about 30%, and 5 year survival is 78%. For a disease which typically can take 7-12% of patients each year in its aggressive form, the form which requires stem cell treatment, these results are encouraging. Those who did best received their transplants early, prior to any cardiac involvement. Dr. Burt is continuing his research in scleroderma and stem cell transplants, but there will no longer be a control group. He is comparing two different conditioning regimens (the chemotherapy used to eliminate the faulty immune system will be different in the two groups) but both groups will receive stem cell transplant to reboot the immune system.

Here is the most recent article, discussing all 90 patients transplanted so far between Dr. Burt and his colleague in Brazil. This includes patients who were older and had more long standing disease than within the ASSIST trial.

Here is the article which discusses the 19 patients in the ASSIST trial, in which I participated.

I, unfortunately, meet relapse criteria. However, it's not as bad as it sounds. The criteria are very strict, if you will. Because my skin began to tighten again about 14 months after the transplant, my local doctors tried new therapy for me. I did not tolerate the treatment, and developed complications. Hence, I have really not been on any immunosuppressants for any extended period of time... perhaps 60 days total. But, using any new treatment at all qualifies as a "relapse."

At my most recent appointment with Dr. Burt, we discussed the use of immunosuppressants, and he feels that my situation is so stable that there is no need. My lung function is stable year over year for the first time. My cardiac status is stable. My kidneys, after a brief decline in function last year, are once again running full steam. All of the potentially life-threatening complications are seemingly stable. I am enjoying riding my horse, and my biggest health issue at present is a dumb back injury I got falling in my garden. I love normal problems. :)

I got my second set of teal streaks in my hair recently... so much fun. I'm streaking for scleroderma. I continue to work for scleroderma awareness, I continue in my volunteer capacity with the Scleroderma Foundation, I'm planning my third education day (April 27) and just loving time with my kids and family.

NOW! Hair aplenty!

I'm looking forward to reading the ASTIS trial--I contacted the investigators this month, and they are hoping to go to press this year, too. This European stem cell transplant trial in scleroderma is larger than ASSIST and will definitely add substantially to our understanding of the treatment's potential. The SCOT trial, the multi-center US trial, is not yet published, and I look forward to those results, as well. In the meanwhile, some of the SCOT investigators have started a new trial which does not include total body irradiation, and is enrolling in Colorado and Seattle. This trial, called STAT, also will not have a control arm.

I am so encouraged that my own experience with the control arm, as painful as it was to lose cardiac and pulmonary function, may have contributed to medical science. Dr. Burt says "no;" he believes that it was never necessary to have a control arm. But, I understand the need for solid science. I just always thought I'd be on the other side of the bench.

Monday, September 19, 2011


“Politics is supposed to be the second oldest profession. I have come to realize that it bears a very close resemblance to the first.” - Ronald Reagan
I had the honor of visiting Washington, D.C., this past week on behalf of the Scleroderma Foundation. The work we do locally to provide support and education to those with scleroderma is vital. Our local fundraisers provide "seed" money for researchers, who can do early studies on a good idea, but we cannot raise the kind of funds we need to do big clinical trials. That money, in general, comes from the NIH (National Institutes of Health).
The NIH is a large organization, but the research money is allocated across the nation. Much of the "bench" research in the United States is funded with NIH dollars, as is a great deal of clinical research.
As you are no doubt aware, scleroderma research does receive some funding from the NIH. Our goal in visiting Washington, D.C., was really two-fold. First, we tried to impress upon our representatives the importance of NIH funding. We talked about high tech jobs, advancement of science, and the health of our citizens. Second, we are hoping that some of the Congressional leaders with whom we spoke will join us on a bill, the "Scleroderma Research and Awareness Act." This bill, which asks for no additional funding, does ask that the NIH fund scleroderma research and that the CDC (Centers for Disease Control) promote awareness of scleroderma. The ultimate goal is that those affected will be diagnosed more quickly (because their doctors recognize the disease) and will have treatments available (because the research has been done). Ideally, when the cause is eventually known, the disease may even be preventable or curable.
So, we spent an exhausting couple of days marching through the House and Senate buildings, discussing our message and our "ask." It was very interesting to see the back side of politics, where the deals are made and the decisions are influenced. I came to realize that the saying "the halls of power" is quite accurate--many of our meeting took place in hallways, as staff members have limited space available to them (the buildings are gorgeous but staffs have grown substantially since they were built).
One of the unexpected highlights of my trip was meeting with the Senior Policy Advisor to Congressman Brian Bilbray, Gary Kline. (photo above) From our leadership within the Foundation, I learned that Congressman Bilbray is a huge supporter of the NIH--he has an incredible reputation on the "hill" for his emphasis on funding of the NIH. In addition, Gary felt that the Congressman would be interested in supporting the bill. So, I'm holding my breath (maybe not, lungs won't tolerate that) and hoping that he was right. Regardless, it felt great to think that my trip was worthwhile and quite heartwarming to know that my own Congressman was such a great supporter of medical research.
OK, next blog will review the results of my one year follow-up in Chicago. But, wanted to share this rather more exciting information first! :)
ADDENDUM: Congressman Bilbray is, indeed, co-sponsoring the Scleroderma Research and Awareness Act. Thank you, Congressman!

Sunday, August 14, 2011

Radio, Radio

Dr. Burt was recently interviewed on air regarding the ASSIST study, and a link is provided here for those interested. The interview once again reviews the phenomenal results this study has shows as compared to any other prior intervention with aggressive systemic scleroderma.

I return to Chicago late this month for a reevaluation. I'm feeling pretty well overall, although I still have symptoms of disease. The transplant didn't "cure" me but my life is vastly improved. My biggest complaints remain my shortness of breath and my joint pain.

I'm heading to my first horse show in more than a year this week. I'm pretty nervous, as Costa and I have not really figured each other out. It will be a lot of work to get there, I'm sure, but I want to enjoy this opportunity to go out and have some fun, see my friends, and share in the excitement of a horse show.

Then it's on to Yosemite to remember the life of Steve's brother, Dale, who passed away from a brain tumor nearly 10 years ago. We call it, "Lunch with Dale," our annual pilgrimage to his favorite site. Nothing like perspective to make you appreciate the gift of a good day.

Here's hoping you are enjoying one, as well. :)

Wednesday, August 10, 2011

Happy Birthday to Me!

Can you believe it's been a year?

Today is the one year anniversary of my stem cell transplant. It's amazing to be where I am.

I had a rather shocking moment at the Scleroderma National Convention this year. Dr. Burt said during his lecture that the average life expectancy of someone with systemic scleroderma and a TAPSE score of less than 1.8 was one year. Well, one year ago my TAPSE was 1.6, but today I enjoyed a wonderful ride on my new horse, Costa, and I plan to attend a horse show next week. :)

I am so lucky to have enjoyed this ride, as well, with all of you. Thanks for all of the love and support of the past year. My fight continues, but it doesn't feel like a fight every day anymore. It feels like a magical and wonderful life.

I am so grateful, and I hope that we will continue to make progress in the treatment of scleroderma.

Video of Holly and Costa

Thursday, July 21, 2011


I am excited to report that the results of the ASSIST study are about to be published, and you fans get a sneak peak at the abstract! :)

I am one of the 9 patients who got control, and one of the 7 who went on to stem cell transplant, just in case you are wondering about my 15 minutes. It's big news, and I think should be a game changer in the treatment of scleroderma. Due to these results, Dr. Burt has started a new study which does not include control, and is comparing two different types of non-myeloablative stem cell transplant.

The Lancet, Early Online Publication, 20 July 2011doi:10.1016/S0140-6736(11)60982-3Cite or Link Using DOI
Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial

Dr Richard K Burt MD a , Sanjiv J Shah MD b, Karin Dill MD c, Prof Thomas Grant FACR c, Prof Mihai Gheorghiade MD d, James Schroeder MD e, Prof Robert Craig MD f, Prof Ikuo Hirano MD f, Karin Marshall RN c, Eric Ruderman MD e, Borko Jovanovic PhD g, Francesca Milanetti MD a h, Sandeep Jain MRCP a, Kristin Boyce RN a, Amy Morgan CNP a, James Carr MD c, Prof Walter Barr MD e ‡

Non-randomised studies of haemopoietic stem-cell transplantation (HSCT) in systemic sclerosis have shown improvements in lung function and skin flexibility but high treatment-related mortality. We aimed to assess safety and efficacy of autologous non-myeloablative HSCT in a phase 2 trial compared with the standard of care, cyclophosphamide.
In our open-label, randomised, controlled phase 2 trial, we consecutively enrolled patients at Northwestern Memorial Hospital (Chicago, IL, USA) who were aged younger than 60 years with diffuse systemic sclerosis, modified Rodnan skin scores (mRSS) of more than 14, and internal organ involvement or restricted skin involvement (mRSS <14) but coexistent pulmonary involvement. We randomly allocated patients 1:1 by use of a computer-generated sequence with a mixed block design (blocks of ten and four) to receive HSCT, 200 mg/kg intravenous cyclophosphamide, and 6·5 mg/kg intravenous rabbit antithymocyte globulin or to receive 1·0 g/m2 intravenous cyclophosphamide once per month for 6 months. The primary outcome for all enrolled patients was improvement at 12 months' follow-up, defined as a decrease in mRSS (>25% for those with initial mRSS >14) or an increase in forced vital capacity by more than 10%. Patients in the control group with disease progression (>25% increase in mRSS or decrease of >10% in forced vital capacity) despite treatment with cyclophosphamide could switch to HSCT 12 months after enrolment. This study is registered with, number NCT00278525.
Between Jan 18, 2006, and Nov 10, 2009 we enrolled 19 patients. All ten patients randomly allocated to receive HSCT improved at or before 12 months' follow-up, compared with none of nine allocated to cyclophosphamide (odds ratio 110, 95% CI 14·04—∞; p=0·00001). Eight of nine controls had disease progression (without interval improvement) compared with no patients treated by HSCT (p=0·0001), and seven patients switched to HSCT. Compared with baseline, data for 11 patients with follow-up to 2 years after HSCT suggested that improvements in mRSS (p<0·0001) and forced vital capacity (p<0·03) persisted.
Non-myeloablative autologous HSCT improves skin and pulmonary function in patients with systemic sclerosis for up to 2 years and is preferable to the current standard of care, but longer follow-up is needed.