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! :)