Stem Cell Transplantation In Juvenile Systemic Sclerosis
Ronald M. Laxer, MD, FRCPC
Vice President, Clinical and Affairs, The Hospital for Sick Children
Professor of Pediatrics and Medicine, University of Toronto
The treatment of juvenile systemic sclerosis has not been one of medicine’s major areas of success, as opposed to that of many other rheumatic diseases. While rheumatologists have become better at treating many of the symptoms associated with juvenile systemic sclerosis (e.g. reflux, Raynaud’s phenomenon), many treatments aimed at the basic mechanisms leading to the sclerodermatous process have not been proven to be effective. Examples include corticosteroids, cyclosporine, Interferon, relaxin and penicillamine. Methotrexate and penicillamine have both been studied in randomized trials, and neither have proven definitively to be effective. Nevertheless, both penicillamine and methotrexate continue to be used for some patients, perhaps with limited success.
Because of the severity of some cases of juvenile systemic sclerosis and the lack of proven effective treatment, new approaches to the treatment of juvenile systemic sclerosis have been developed. One of these new methods is called High Dose Immunotherapy with Stem Cell Rescue (also known as Stem Cell Transplant), a form of treatment which has proven to be effective in some forms of cancer in adults as well as in children.
The rationale behind this treatment is as follows: the scleroderma process is set in motion by the body’s immune system, which for some reason has escaped normal control mechanisms and proceeds to stimulate cells to make excessive amounts of collagen which get deposited in the skin, blood vessels and many vital organs such as the lungs, heart, kidneys and gastrointestinal tract, leading to progressive “scarring” of these organs. If these “out of control” cells could be removed from the body before too much damage has occurred, then the scleroderma process might be able to be kept in check.
The basic mechanism behind the process of High Dose Immunotherapy with Stem Cell Rescue is that stem cells, the originators of cells that lead to blood forming and immune cells, are taken from the patient and stored (harvested). Patients are then treated with high doses of chemotherapy and the immune system and blood forming elements are essentially wiped out. This serves the purpose of getting rid of the immune cell(s) which may have initially triggered the scleroderma process. The patient’s stored stem cells are them given back to the patient intravenously, and the body’s immune system and blood-forming cells are then “put back together”, or reconstituted. This is a very intensive process, which may be somewhat risky for patients, especially when their blood counts are very low, as they are at a higher risk of infection. It must be performed under very controlled situations and in centers with extensive experience with such procedures.
This treatment has been used in probably fewer than 100 adults with severe systemic sclerosis, and in only a handful of children. It is important to time the treatment appropriately. To be effective, it must be done before irreversible organ damage has taken place. On the other hand, it must not be done too early, as one would not want to use such an aggressive therapy in a patient whose ultimate course is not known, as the treatment may be worse than the disease. Therefore, it is very important to have predictive factors as to the ultimate outcome, and while there are some in adults with scleroderma, this area has not been studied in children.
The short-term results with High Dose Immunotherapy with Stem Cell Rescue in adults appear to be promising. However, the very long term outcomes are still not available, and some patients, despite initial improvement, have had a relapse. Several children have been treated with this approach, with success in a few.
For patients with progressive juvenile systemic sclerosis and no other therapeutic options, this does appear to be a valid and rational approach to the treatment. Protocols are underway for children with rheumatic diseases
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For more information on Juvenile Scleroderma, contact:
Juvenile Scleroderma Network, Inc.
1204 W. 13th Street, San Pedro, CA 90731
Tel: (310)519-9511 (Pacific Time)
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