Scleroderma - 2001 National Meeting Reports (cont.)

Inflammation of the lungs' tiny air sacs (alveolitis) stabilized or improved in scleroderma patients treated with cyclophosphamide (Cytoxan) and prednisone. 

Dr. Shiel's Perspective: This form of lung disease (as compared to the one described in the report above) actually did respond to Cytoxan. The key element seems to be inflammation. If inflammation is present in the lungs, it may be helped by Cytoxan treatments (typically given as a monthly intravenous infusion). The exact form of lung disease requires a lung biopsy for a precise diagnosis.

Specific genes were found responsible for why some scleroderma patients develop lung disease while others do not. 

Dr. Shiel's Perspective: This has always been a perplexing phenomenon with autoimmune rheumatic diseases - why they present so differently from patient to patient. It may be that environmental differences account for some of this, but it seems that there are also subtle differences genetically in the inherited material of each patient that may be playing a significant role.

Viagra (sildenafil) seemed to help function in scleroderma patients with severe pulmonary hypertension (elevated blood pressure in the artery to the lungs).

Dr. Shiel's Perspective: Because Viagra has an effect on the smooth muscle of the blood vessels, it was tried in these very ill patients who had failed the traditional prostacyclin intravenous infusions. Although Viagra did show favorable results, these are very small preliminary studies. Incidentally, it might be expected that there would be some benefit on the Raynaud's phenomenon in patients treated with this drug, and that is exactly what the researchers saw in all patients. Further research studies are now needed to really determine the exact role and safety of Viagra in patients with scleroderma.

Treatment Trials

L-carnitine seemed to help Raynaud's and ulcers in scleroderma patients as compared to a placebo. 

Dr. Shiel's Perspective: This study was designed to also determine if L-carnitine would help the skin disease and overall patient function, which it did not. According to the researchers, more studies are necessary to confirm whether or not L-carnitine really has benefits. I agree.

Tamoxifen failed to help the skin disease or Raynaud's phenomenon in patients with scleroderma.

Dr. Shiel's Perspective: Again, this was a preliminary study. These studies, even when unsuccessful, are very critical in establishing which are the best candidate treatments for long-term research trials. Tamoxifen is out.

Bowel Disease

Erythromycin helped scleroderma stomach more effectively than the bowel muscle-stimulator drug, metoclopramide.

Dr. Shiel's Perspective: Stomach and bowel muscles can become weakened as a result of scleroderma, thereby resulting in loss of normal function of the bowels. It is a serious condition. Doctors have used bowel muscle-stimulating drugs, such as metoclopramide, in the past. Doctors have also used antibiotics (I use tetracycline or erythromycin). The reason for the antibiotics is that the bowel of scleroderma patients can become overgrown with bacteria, which is associated with slowing the bowel function. The antibiotics seem to improve this condition. This study is a head-to-head comparison of the two options. It is encouraging to see that common antibiotics can be beneficial in this condition.