January - April 2006 Issue
Scleroderma Trial Offers HopeEvery day, Mary Ellen Csuka, MD, Medical College of Wisconsin rheumatologist and director of the Osteoporosis Program, sees the benefit of being part of one of the top academic medical centers in the country. She treats patients with systemic sclerosis also known as scleroderma, an uncommon, sometimes fatal rheumatologic disorder. These patients need care from other specialists throughout Froedtert & Medical Collegeof Wisconsin. Being a part of a large academic center also brings her opportunities for leading-edge research that is helping transformthe treatment of this disease.
“I offer a specialty clinic for patients with scleroderma,” says Dr. Csuka. “But because we have a world-renowned, highly respected stem cell program, with physicians like Dr. James Wade, chief of Neoplastic Diseases and Related Disorders, we are participating in a national trial evaluating high dose immunosuppression therapy for the treatment of systemic sclerosis. This is very exciting.”
The trial, Dr. Csuka explains, is known as SCOT, which stands for “Scleroderma: Cyclophosphamide or Transplantation.” Cyclophosphamide is a chemotherapy drug often used to treat autoimmune diseases as well as cancer, and transplantation refers to hematopoietic stem cell transplantation, which uses the patient’s own stem cells.
“While the majority of patients with systemic sclerosis do well,” says Dr. Csuka, “there’s a subset of patients with diffuse systemic cutaneous sclerosis that have very rapid onset of skin thickening and evidence of inflammation in their lungs. This subset of scleroderma patients has a mortality rate of high as 50% in 5 years. This is the group that we feel should be offered high-dose immunosuppressive therapy. The theory is that if we treat their disease aggressively, we will be able to reverse it and stop it in its tracks.”
Dr. Csuka says a recent study using low dose oral cyclophosphamide showed some promise, although the results were not dramatic. “It is encouraging to see that there was improvement with low doses of immunosuppressive therapy. Now the question is if you give very high doses of immunosuppressive therapy, can you really alter the course? Were all previous efforts at treating this disease unsuccessful just because we were not bold enough? Were we more afraid of the medications than of the disease? So this is really a very bold step to try to answer the question. That’s the goal of the SCOT trial.”
When she is not involved in innovative clinical research, Dr. Csuka is treating and reassuring patients. “I see a lot of these patients, and I know their stories,” she notes. “It is comforting to talk to a doctor who says, ‘Yes, I have seen that.’ As opposed to ‘I don’t know.’ It benefits a patient with an uncommon disease to not have the doctor have to step out and look it up.”
Dr. Csuka also shares her specialized expertise by helping other doctors diagnose and understand scleroderma. “I serve as a consultant to local rheumatologists and my colleagues in the community will refer patients to see me for a second opinion and for reassurance,” she explains. “I do not necessarily have to take over their care, but often it is very helpful for them to see me and hear me say, ‘Your doctor is doing an excellent job, he/she is covering all the bases. If I have a study I will let you know.’ So I stay in touch with other doctors, and I am a resource for them. I am getting more and more referrals, which is great. They are coming from farther and farther away. Patients get on the Web, and think they will have to go to Chicago, but they can skip Chicago and come to us,” Dr. Csuka says.
Because of the complexity of the disease and the many different courses it can take, Dr. Csuka treats all her patients as individuals. “The most intensive time to be evaluating scleroderma is during the first five years of onset for diffuse cutaneous, because that’s when things are really changing and the pattern of the disease is going to set itself. This is not an infectious disease where you take antibiotics for two weeks and you are done. This is one of the reasons why I and my colleagues like rheumatology, because we establish long term relationships with people.”
The Scleroderma Clinic is part of the Rheumatology Clinic on the 5th Floor of the East Clinics building, and Dr. Csuka’s patients benefit from the diverse resources available at Froedtert & Medical College of Wisconsin. For example, patients with gastrointestinal symptoms, common in scleroderma, can be seen at the Dysphasia Institute. “We have expertise in managing the gastrointestinal complications, which to me is another great boon, because when I talk to my scleroderma colleagues, some do not bother consulting a gastroenterologist because of their disinterest in the scleroderma patients’ problems unless an endoscopy is necessary,” she explains.
For patients with lung complications, Dr. Csuka says, “we are extremely fortunate at Froedtert to have a Pulmonary Artery Hypertension Center that is headed by Dr. Francisco Soto. Pulmonary hypertension is another aspect of the disease where we now have several treatment options. Prior to the development of these therapies, patients who developed pulmonary hypertension had a mortality rate of about 50 percent per year. In the community you may see a pulmonologist who has never seen or diagnosed scleroderma. The availability of subspecialists who understand scleroderma and how it manifests in their organ of interest means we have something special to offer patients with this disease.”
Source: Every Day
Date: January - April 2006 Issue