Skip to main content

Advertisement

Log in

Clinical trials for pediatric scleroderma

  • Published:
Current Rheumatology Reports Aims and scope Submit manuscript

Abstract

Progressive systemic sclerosis (PSS), or scleroderma, is a rare disease in the pediatric population. Many children with PSS have significant involvement of their internal organs, which leads to decreased survival. Because of the infrequency of the condition and delayed diagnosis, there are no large studies to evaluate therapy for PSS in children. Treatment is controversial in the adult literature, and its applicability to children is unclear. Only through collaborative efforts will researchers be able to clearly delineate the etiopathogenesis of PSS, and gather information from multicenter studies to ultimately provide appropriate and effective care for children with PSS.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Similar content being viewed by others

References and Recommended Reading

  1. Uziel Y, Miller ML: Scleroderma in children. Pediatr Clin North Am 1995, 42:1171–1203.

    PubMed  CAS  Google Scholar 

  2. Lehman TJA: Systemic and localized scleroderma in children. Curr Opin Rheum 1996, 8:576–579.

    Article  CAS  Google Scholar 

  3. LeRoy EC, Medsger TA: Criteria for the classification of early systemic sclerosis. J Rheumatol 2001, 28:1573–1576.

    PubMed  CAS  Google Scholar 

  4. Steen VD, Medsger TA: Severe organ involvement in Systemic Sclerosis with diffuse scleroderma. Arthritis Rheum 2000, 43:2437–2444.

    Article  PubMed  CAS  Google Scholar 

  5. Foeldvari I, Zhavania M, Birdi N, et al.: Favorable outcome in 135 children with juvenile systemic sclerosis: results of a multi-national survey. Rheumatology 2000, 39:556–559.

    Article  PubMed  CAS  Google Scholar 

  6. Denton CP, Abraham DJ:Transforming growth factor-[beta] and connective tissue growth factor: key cytokines in scleroderma pathogenesis. Curr Opin Rhuem 2001, 13:505–511.

    Article  CAS  Google Scholar 

  7. Strummvoll GH:Current treatment options in systemic sclerosis (scleroderma). Acta Medica Austraica 2002, 29:14–19.

    Article  Google Scholar 

  8. Martini A: Juvenile systemic scleroderma. Current Rheum Rep 2001, 3:387–390. Outstanding review of pediatric scleroderma. Offers information on the role of microchimerism in the pathogenesis of PSS.

    Article  CAS  Google Scholar 

  9. Clements PJ, Furst DE, Wong WK, et al.: High dose versus low dose D-penicillamine in early diffuse systemic sclerosis: analysis of a 2 year, double blind, randomized controlled clinical trial. Arthritis Rheum 1999, 42:1194–203.

    Article  PubMed  CAS  Google Scholar 

  10. Steen VD, Medsger TA: Improvement in skin thickening in systemic sclerosis associated with improved survival. Arthritis Rheum 2001, 12:2828–2835.

    Article  Google Scholar 

  11. Clements PJ, Hurvitz EL, Furst DE: Skin thickness core as a predictor and correlate of outcome in systemic sclerosis: high dose versus low dose penicillamine trial. Arthritis Rheum 2000, 11:2445–2454.

    Article  Google Scholar 

  12. Jimenez SA, Sigal SH: A 15-year prospective study of the treatment of rapidly progressive systemic sclerosis with D-penicillamine. J Rheumatol 1991, 18:1496–503.

    PubMed  CAS  Google Scholar 

  13. Medsger TA, Lucas M: D-Penicillamine in systemic sclerosis? Yes! Scand J Rheumatol 2001, 30:192–194.

    Article  PubMed  Google Scholar 

  14. Furst DE, Clements PJ: D-penicillamine is not an effective treatment in systemic sclerosis. Scand J Rheumatol 2001, 30:189–191.

    Article  PubMed  CAS  Google Scholar 

  15. Pope JE, Bellamy N, Seibold JR, et al.: A randomized, controlled trial of methotrexate versus placebo in early diffuse scleroderma. Arthritis Rheum 2001, 44:1351–1358. Outstanding review of pediatric scleroderma. Offers information on the role of microchimerism in the pathogenesis of PSS.

    Article  PubMed  CAS  Google Scholar 

  16. Lehman TJA: Methotrexate for the treatment of early diffuse scleroderma. Arthritis Rhem 2002, 46:845.

    Article  Google Scholar 

  17. Clements PJ, Lachenbruch PA, Stertz M, et al.: Cyclosporin in systemic sclerosis: results of a forty eight-week open study in ten patients. Arthritis Rheum 1993, 36:75–83.

    Article  PubMed  CAS  Google Scholar 

  18. Filachi G, Cutolo M, Indiveri F, et al.:Long term treatment of patients affected by systemic sclerosis with cyclosporin A. Rheumatology 2001, 40:1431–1432.

    Article  Google Scholar 

  19. White B, Moore W, Wigley F, et al.:Cyclophosphamide is associated with pulmonary function and survival benefit in patients with scleroderma and alveolitis. Ann Intern Med 2000, 132:947–954. A retrospective study that shows beneficial effects of cyclophosphamide in scleroderma-related interstitial lung disease.

    PubMed  CAS  Google Scholar 

  20. Giacomelli R, Valentini G, Salsano F, et al.:Cyclophosphamide pulse regimen in the treatment of alveolitis in systemic sclerosis. J Rheumatol 2002, 29:731–736.

    PubMed  CAS  Google Scholar 

  21. Pakas I, Ioannidis JPA, Malagari K, et al.:Cyclophosphamide with low or high dose prednisolone for systemic sclerosis lung disease. J Rheumatol 2002, 29:298–304.

    PubMed  CAS  Google Scholar 

  22. Oliver SJ, Moreira A, Kaplan G:Immune stimulation in scleroderma patients treated with thalidomide. Clin Immunol 2000, 97:109–120.

    Article  PubMed  CAS  Google Scholar 

  23. Furst DE:Stem cell transplantation for autoimmune disease: progress and problems. Curr Opin Rheumatol 2002, 14:220–224.

    Article  PubMed  Google Scholar 

  24. Tyndall A, Gratwohl A: Immune ablation and stem-cell therapy in autoimmune disease: a clinical experience. Arthritis Res 2000, 2:276–280.

    Article  PubMed  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Rights and permissions

Reprints and permissions

About this article

Cite this article

Rosenkranz, M.E., Lehman, T.J.A. Clinical trials for pediatric scleroderma. Curr Rheumatol Rep 4, 449–451 (2002). https://doi.org/10.1007/s11926-002-0049-2

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1007/s11926-002-0049-2

Keywords

Navigation