Current therapies for Scleroderma
The condition should be regarded as treatable even if a cure is not yet possible. Some treatments can be considered as general therapy (see below). Specific complications such as lung fibrosis, gut disease, kidney damage, fingertip ulcers, or pulmonary hypertension require specific specialist treatment.
Disease modifying
• Vascular
• Immunomodulatory
• Antifibrotic
Symptomatic treatments
Organ-based strategies – targeting gastrointestinal (gut), heart, kidney or lung problems.
This is one of the most successful parts of scleroderma therapy and has benefited from advances in other branches of medicine.
Targeting therapy in Scleroderma
• Accurate diagnosis
• Appropriate subsetting
• Staging disease within subset - disease modifying therapy?
• Risk stratification for major organ-based complications based upon serological, genetic and clinical features
• Screening and early intervention when complications develop - organ-based therapy
Disease-modifying treatments for Scleroderma
Vascular
• Vasodilators (calcium channel blockers, alphablockers, sildenafil)
• Vascular remodelling (ACE inhibitors, angiotensin receptor blockers, serotonin re-uptake inhibitors, bosentan)
• Prostacyclin analogues (iloprost, flolan)
• Antioxidants (probucol, vitamin
supplements)
Immunological
• Methotrexate
• Cyclophosphamide
• Antithymocyte globulin (ATG)
• Azathioprine
• Mycophenolate mofetil
• Stem cell transplant
• Low dose corticosteroids (<10mg/day prednisolone)
Antifibrotic
All current treatments for fibrosis are experimental – at present the most effective way of treating fibrosis in scleroderma seems to be treatment of the vascular or inflammatory processes that drive fibrosis.
In the future it may be possible to block fibrosis with antibodies or other drugs that interfere with the regulation of collagen production. Collagen overproduction is the key problem in fibrosis and scarring
• Minocycline is an antibiotic that has been suggested to be antifibrotic
• Other drugs are in trial
Prof Chris Denton
Royal Free Hospital, London
