Politics

“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!

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. :)

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

HUGE NEWS!

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 ‡
Summary

Background
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.
Methods
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 ClinicalTrials.gov, number NCT00278525.
Findings
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.
Interpretation
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.
Funding
None

The Boys of Summer

Summer is here!

June was a CRAZY month for me. I ran my first Education Day for the San Diego Chapter of the Scleroderma Foundation. I think it was a success, based on the feedback and evaluations I received. It was a lot of work, and I admit that I feel quite exhausted! Jake was a big help to me, though, and even spoke at the event.

The boys have been off school for about a week now, and that is terrific. After last summer's adventures, I really want to spend a lot of time with my boys this summer. Jake entered a robot competition at the Del Mar Fair and earned 3rd prize. He and Luke are playing lots of tennis and doing some fun learning activities with me, as well.

Luke is also spearheading the "Luke Laps" team for the annual Scleroderma Foundation fundraiser, so don't forget to visit his page at Firstgiving. He is 89% to goal, and with just a few short weeks left, he is excited to be a part of this process.

I have been deeply touched by the commitment of my boys to helping me with these projects. They have been amazing. I had been hesitant to include them in much--their lives are already impacted enough by this disease. But, they expressed interest and enthusiasm, and I am very proud of their efforts.

A Difficult Patient

As I prepare for the Linda Lee Wells Memorial Annual Education Day (what a mouthful!), I have spent more time reading the comments and questions on "the scleroderma sites," particularly on www.inspire.com. Here, the Scleroderma Foundation has joined with Team Inspire to create a safe place for patients to communicate with one another, to share their stories of success and failure, and to question their doctors' advice.

I find that last area is, in fact, where a lot of posts focus. Perhaps it's just the discussions that I am drawn to opening and reading... but a lot of patients are wondering why their doctors have so much difficulty relating to them as people, listening to their concerns, considering them as partner, and approaching them with compassion. I admit it is hard for me to see the medical community lambasted at times, but my perspective has definitely shifted over these past 4 years.

When I first became a scleroderma blog voyeur, I was offended by the vitriol often blasted at the medical field. But, having now lived in the world of "patient" for some time, I admit I could blast a few doctors, myself. I have become, quite probably, a difficult patient. I refuse to return to doctors who offend me, who don't listen, who don't believe, or who patronize rather than work with me as a partner. It's hard--especially as a woman, and someone who loves to be liked and hates conflict--to stand up for myself. So, mostly I don't. I just don't schedule the return visit. But, driving home I have the best comments and most brilliant comebacks for all the injustices of my latest medical experience.

Becoming a patient is never a choice. I certainly don't relish the "sick role." But, if I have to be a person with scleroderma, I'm going to fight the disease and fight for the right kind of care. I'm a difficult patient. I think that we all need to be difficult patients. Not angry patients, not abusive patients--but patients who challenge our doctors' advice, who read the medical literature, who ask the right questions and demand the answers. Patients who understand the disease, want to know the options, accept the limitations of science, and can partner with their doctors. And, I can also assure you, doctors are out there who welcome these partnerships, who don't label this behavior as difficult, and who thrive on the challenge. Those are my fabulous doctors. I owe them a debt of gratitude I can never repay.

The philosophy of my health care team, whether they know it or not, is called "Patient-centered care." In this context it means "treating patients as partners, involving them in planning their health care and encouraging them to take responsibility for their own health." (AAFP 1998) Research also shows that patients who help to shape their treatment plans are far more likely to adhere to the plan. Difficult? I think not!

I read a brilliant essay on being a difficult patient which inspired my post today. Written by a scientist and a mother who died a few years back from scleroderma, I highly recommend it if you or a family member are struggling with any kind of disease.

Article on Being a Difficult Patient

Pump It Up

Ah, if only I were "This Year's Model."

But, given that I had my own little rebirth in 2010, I am trying to "Pump it Up" a bit. When I saw Dr. Furst last month he advised me to participate in a formal cardiac rehab program. However, my cardiologist suggested that I just go ahead and exercise at home, which seemed much easier to me.

I started back on my elliptical, and increased my program by one minute per day. Today, I reached my first goal of 20 minutes on the elliptical. Now, I know to most of you this seems fairly pathetic, but I am pretty proud of my progress. I plan to spend at least a month at 20 minutes now, as this is a decent start, and see how my joints and other symptoms take the new program.

I can hardly believe the progress I've made in a year, though! Whoo hoo!

Our team is coming together for the scleroderma walk in July, and now I am a bit more confident that I can be ready. Don't forget to check the site (see the last post) to sign up to walk with us! :)

Kickboxing Rides Again!

"There is no use trying," said Alice; "one can't believe impossible things."
"I dare say you haven't had much practice," said the Queen. "When I was your age, I always did it for half an hour a day. Why, sometimes I've believed as many as six impossible things before breakfast."
- Lewis Carroll

FirstGiving

Kickboxing for Scleroderma began as a team. A team inspired by my recent diagnosis of scleroderma, and committed to finding a cure, we did our first fundraising walk in 2007. This team led to my blog, and my involvement with the Scleroderma Foundation... I know that I have enjoyed tremendous support through this process, and I hope that I have given back even a fraction of that which I have gained. Now that my strength is improved, I am committed to giving back even more.

You are part of this team, and I am so grateful.

My sons, Jake and Luke, has stepped up this year as team leaders.

Luke is our fund raiser, and you must see the adorable (no bias here!) video that he has created for our annual walk. Check the link above. Luke has lofty goals, and plays the task master both on his video and here at home, making sure we properly promote our team: LUKE LAPS! Please visit the page. :)

Jake--home today with me and recovering from a nasty oral surgery--is the head of the "envelope stuffing committee" as he calls it with all of his wonderful humor. He is technically one of 3 people running the annual Linda Lee Wells Memorial Education Day, a completely free event (you are all welcome to come!), run and staffed by an all volunteer group, to provide a top notch educational event.

See the link below, and join us!

Eventbrite

Work in Progress

I had a busy couple of weeks, seeing several doctors and collecting opinions on my progress. And, the general consensus seems to be that I am, in fact, a work in progress.

My new cardiologist, a specialist in heart failure, seems to think that my heart may be recovering. Additional formal testing is scheduled.

My pulmonologist seems very happy with the improvement in my lung CT, and was not horrified by the tiny drop in my pulmonary function testing. She thinks that the slight difference may reflect the difference in machines between Chicago and San Diego, or may reflect my underlying connective tissue tightness. Overall, however, she is happy with my progress.

My rheumatologist is also pleased with my progress. On substantially less medication than I was on pre-transplant, I do have some arthritis pains. My Raynauds symptoms are worse. But, again, overall I feel better, have more energy, and she is satisfied.

Regardless, everyone seems to agree that I have done what I can. The stem cell transplant can take up to 2 years to see full benefit, so now we wait. :)

I am sorry to report that Alexander needs a lay up to recover from a ligament injury, but I plan to find a way to keep riding. Stay tuned!

A Rough Week

Sometimes it seems like life throws you curve balls, and this has been one of those weeks. It started when I foolishly (again) left my purse in my car during Luke's tennis match down at Barnes Tennis Center. I returned to a bashed in car and a stolen purse, along with many of my other personal belongings. It's hard already, but without an income, losing all that stuff was even harder.

Then our beautiful horse Alexander became sore after a too tough dressage training session, I think. Hopefully that's all it is, but the poor guy is just not right. We were planning our first show on him this weekend, but that's been scratched. :( I am disappointed.

But, the light on my horizon was the thought that I might have found a new, young hunter prospect who looked absolutely perfect. Sold today, of course, and I was to see him Monday.

The last straw was my pulmonary function testing. It put me over the edge today. Cranky all day anyway, and then my numbers are down into the 50th percentiles for the first time. Don't understand, can't explain... don't have any answers but I do have another full week of doctor's appointments next week. Wish I could just walk away from it all sometimes.

Not my week.

Follow Your Passion

I had a lovely "coffee" with a friend today, and she got me thinking about how stem cell therapy for autoimmune diseases began. I came home and found this video on YouTube of Dr. Burt, the man who did my stem cell transplant, discussing how he began his work.

Hope you enjoy it. :) There is a part 2 and a part 3, as well, which you can also find on YouTube.

How to Save a Life

Success! I am home from Chicago and feeling quite well, despite a, ah, pneumomediastinum as a rather unusual complication of my heart catheterization. Seems to be healing up fine, though!

The best news continues to be the dramatic drop in my pulmonary artery pressure. In addition, however, the CT scan of my lungs also showed substantial improvement. It would appear that the stem cell transplant was a resounding success!

I am quite hopeful that the damage already done to my body by scleroderma can be controlled. My heart testing, in particular my echocardiogram, showed continued decline in the function of my heart. My TAPSE (tricuspid annular plane systolic excursion) has dropped from above 3.0 cm less than a year ago to 1.6 cm on this study (with a level of 2.1 cm in August 2010). This value can estimate right ventricular ejection fraction (RVEF), and a level of less that 1.96 cm correlates with a RVEF of less than 40%. This, in turn, predicts big problems. Also, my right ventricle continues to become more hypokinetic--it doesn't move as well or pump as strongly.

Dr. Burt is eager to publish the findings of his study, and showed me several graphs of his results. The bottom line is that Cytoxan does not seem to work. The stem cell transplant works quite well, but is far more effective done earlier, before Cytoxan, than when it is delayed until after Cytoxan as was my situation. He intends to begin a new trial comparing two variations of stem cell transplant and will no longer use a control arm with Cytoxan based on these results.

How to Save a Life: if you have aggressive, early scleroderma, please talk to experts about getting a stem cell transplant early.

Oh, and try to have some fun in the meantime. Is this horse the cutest thing you have EVER seen? It was so much fun to ride him the other day! And enjoy your friends and your family, and travel often, and kiss your mother with that mouth, after all. :)

About A Girl

Had a wonderful evening with my stem cell transplant buddies. After spending a month cooped up together in the hospital, we were looking forward to an actual dinner out! So, we arranged to have our 6 month follow-up visits on the same day--tomorrow--and planned a night out in the big city for tonight.

We went to a fabulous restaurant in Chicago. It was very nice to just sit in peace, eating great food and chatting a bit about our lives "on the outside." Neither of my stem cell buddies has scleroderma, so their experiences have been quite different from mine. (Dr. Burt does stem cell transplants for several autoimmune diseases.) All the same, we have shared something very unique and it was nice to see how well we are all recovering.

I had a lot more testing today and will get my official results tomorrow. I do have my results from the pulmonary function testing, which is stable. That's good, but after yesterday I had even begun to hope for improvement. I'll try not to get too greedy... :)

Valentine's Surprise

I was pretty hopeful that having a heart catheterization on Valentine's Day had to be a good omen. My preliminary results show incredible improvement. I am stunned beyond belief.

My mean pulmonary artery pressure dropped from 26 pre-transplant to 14 today. Normal.

Still lots of follow-up testing to be done tomorrow, but honestly I'm almost numb from the good news. I always steel myself a little, ready myself for that news I've become accustomed to... I'm not sure exactly how excited I can get just yet. But, here's hoping the good news keeps coming tomorrow and I think in time I will figure out the right way to celebrate! :)

One of Us

Someday, the light will shine like a sun through my skin & they will say, What have you done with your life? & though there are many moments I think I will remember, in the end, I will be proud to say, I was one of us.
-Brian Andreas

I attended my first scleroderma support group this past Saturday. It is a strange and comforting and disturbing experience: I see around the room the old me, the present me, and the future me. Kind of "A Christmas Carol" moment. :) But, more importantly, I enjoyed just meeting new friends and laughing about some of the unusual shared issues faced by those with this disease.

I am also a week away from the answer to the question that seems to be most concerning to me of late: what is my pulmonary artery pressure? I will celebrate Valentine's Day with my cardiology team in the cath room and hope for good news there!

Just hoping the weather's not too ugly in Chicago next week... we have enjoyed, as my friend Marcy says, Chamber of Commerce weather the past few days in San Diego. Gorgeous, sunny, crisp and clear. I had a wonderful ride on Xander this morning and was even able to sneak in a little trip to Disneyland with my children, husband and parents last week!

I feel my arthritis slowly but steadily creeping back up on me. I didn't miss it while it gave me a brief respite. Just hoping the big news is good news. Wish me luck!

Gearing up for Progress... or Failure

It's funny how someone sends you the right thing at the right time, so often. I bit off more than I can possibly chew this week, without a doubt. Still struggling with C. diff, and still not able to spend a lot of time on the computer, etc, I agreed to a leadership position with the Scleroderma Foundation, Greater San Diego Chapter. I am trying to arrange our Education Day on June 18 (please come!!!) and have great aspirations and dreams of success. These were somewhat dampened at my visit with Dr. Furst this week when he cautioned me that starting a new event like this is going to take time. Expect 30 people or so, he said, and I cringed inside. All this work for 30 people? Wouldn't that feel like failure?

And then my wonderful friend, Sarah, sent out a little link on Facebook to a story about a man who started a nonprofit in Rhode Island meant to foster creativity. An audience member at a recent talk asked him what the best way is to go about that. And he answered:

So much of our existence is mediated. We're getting billions of messages every day telling us what we should wear, what we should eat, what we should drive, how we should look, who we should date, you know, whatever, and you come to AS220 and someone starts playing a guitar solo, and they fail.
And they start again.
That's human.
We could all identify with that.
It's amazing.
Okay?
And it's empowering to us to have that experience and make that connection. As opposed to being fed perfection. Something we cannot identify with. That's humbling - not humbling, it's, it's, it's, demoralizing -- you know? Because we can never live up to those expectations. So we keep buying more, to try to achieve these expectations that are false.
So I think [the answer is] creating environments where people feel safe to fail, and other people have the opportunity to experience that.

So, it's given me pause, these messages that I have been gently getting this week. I can't always expect perfection and will try to be satisfied with progress, slow and steady. And I will try to see my many failures in this new light.

The (Kick)Boxer

In the clearing stands a boxer
And a fighter by his trade
And he carries a reminder
Of every glove that laid him down
Or cut him 'till he cried out
In his anger and his shame
"I am leaving, I am leaving"
But the fighter still remains

-Paul Simon

As 2011 begins, I am reminded of the importance of the fight. I know that everyone has causes near and dear to their hearts, so this isn't just about scleroderma. But, today I found myself thinking about my friends who have lost their fight against this disease, and I just wanted to acknowledge their fight and their impact, in the hope that the blows they threw will collectively result in a knockout in the not too distant future.

My new year's goal is to become a better fighter. I am going to become more active with the Scleroderma Foundation now that the bulk of my treatment is behind me. I have Bonnie as my muse. :) Today is her birthday, so she's on my mind, too.

Unfortunately, my main fight will be delayed by the warm up act: a (hopefully) brief third round against C. Diff. I knew this was coming, so it's no surprise, and quick action from my doctor has hopefully staved off the worst of it this time.

And my fight will enjoy many interruptions of the best kind, if 2011 goes my way. We head to the desert to watch the boys play tennis again this weekend, and hope to hit plenty of horse shows in the year ahead, too! But, I am looking forward to focusing much of my attention and time on the fight... that is after I find my brain and my ambition, both of which seemed to have taken a much needed (?) hiatus in 2010. For the record, it was my brain that made me type "much needed" in the prior sentence. When it acts, it acts on its own these days.