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Stem cell transplantation: limits and hopes
  1. K P MACHOLD,
  2. J S SMOLEN
  1. Department of Rheumatology
  2. Internal Medicine III
  3. Vienna General Hospital, University of Vienna
  4. Waehringer Strasse 18–20
  5. A-1090 Vienna, Austria

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The clinical course and severity of inflammatory rheumatic diseases vary considerably. A large proportion of patients have mild to moderate activity of the inflammatory process which can be successfully controlled by conventional therapeutic measures: traditional disease modifying antirheumatic drugs (DMARDs) for rheumatoid arthritis (RA) and some other forms of chronic arthritides, intermediate steroid doses or mild immunomodulatory agents for systemic lupus erythematosus (SLE) and other connective tissue diseases. Also, control of more severe disease is often manageable by more aggressive, established means, such as high dose methotrexate and combination treatment for RA, or pulse cyclophosphamide treatment and steroids in SLE. For most of these therapeutic approaches significant evidence has accumulated in randomised controlled trials.1-3

Without the possibility of making individual predictions, there are many patients whose diseases are not sufficiently responsive to the traditional measures. At least for RA, the armamentarium has recently been significantly enriched by new means of intervention,4-6 among them biological agents which specifically target a key mediator of inflammation, tumour necrosis factor α (TNFα); more targets are currently being studied. The TNF blockers also appear to be quite efficient therapeutic agents for diseases which were often less easy to control, such as psoriatic arthritis,7 or ankylosing spondylitis, which was regarded as intractable when treated with traditional DMARDs.8

However, despite some success of modern antirheumatic treatment, groups of patients exist, familiar to every rheumatologist, whose disease is resistant to therapeutic measures. This is still the case for a significant proportion of patients with RA whose continuing disease activity, refractory to traditional and new DMARDs, combination treatment, and biological agents, leads to a relentless progression of joint destruction; approximately 30–40% of patients with RA do not have clinical responses even when receiving the new agents. This is also the case for a significant number …

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