Systemic scleroderma in children is very rare and is considered similar to adult-onset disease. In adults, new etiopathogenetic and therapeutic approaches have emerged in recent years. For instance, it has been shown that microchimerism could play a role in disease pathogenesis and that immunoablation followed by stem cell rescue could be of potential therapeutic benefit. There is also evidence that these new approaches can be of value for childhood-onset disease.
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References and Recommended Reading
- 1.Cassidy JT, Petty RE: Textbook of Pediatric Rheumatology. Philadelphia: WB Saunders; 1995.Google Scholar
- 11.Evans PC, Lambert N, Maloney S, et al.: Long-term fetal microchimerism in peripheral blood mononuclear cell subsets in healthy women and women with scleroderma. Blood 1999, 93:2033–2037. A study showing persistent fetal microchimerism in peripheral blood mononuclear cell subsets in healthy women and women with systemic scleroderma.PubMedGoogle Scholar
- 12.Lambert NC, Evans PC, Hashizumi TL, et al.: Persistent fetal microchimerism in T lymphocytes is associated with HLADQA1* 0501: implications in autoimmunity. J Immunol 2000, 164:5545–5548. This study suggests an additional route by which HLA genes may contribute to susceptibility to autoimmune diseases.PubMedGoogle Scholar
- 15.Artlett CM, Cox LA, Jimenez SA: Detection of cellular microchimerism of male or female origin in systemic sclerosis patients by polymerase chain reaction analysis of HLA-Cw antigens. Arthritis Rheum 2000, 43:1062–1067. The study identifies the presence of microchimerism in male and female patients with systemic scleroderma and provides support for the hypothesis that persistent microchimerism plays a role in the pathogenesis of the disease.PubMedCrossRefGoogle Scholar
- 18.Seely JM, Jones LT, Wallave C, et al.: Systemic sclerosis: using high-resolution CT to detect lung disease in children. Am J Roentgenol 1998, 170:691–697.Google Scholar
- 20.Clements PJ, Furst DE, Wong WK, et al.: High-dose versus low-dose D-penicillamine in early diffuse systemic sclerosis: analysis of a two-year, double-blind, randomized, controlled clinical trial. Arthritis Rheum 1999, 42:1194–1203. A controlled trial questioning the efficacy of D-penicillamine in the treatment of systemic sclerosis.PubMedCrossRefGoogle Scholar
- 21.Black CM, Silman AJ, Herrick AI, et al.: Interferon-alpha does not improve outcome at one year in patients with diffuse cutaneous scleroderma: results of a randomized, doubleblind, placebo-controlled trial. Arthritis Rheum 1999, 42:299–305. A controlled trial showing that interferon-alpha is of no value in the treatment of systemic scleroderma.PubMedCrossRefGoogle Scholar
- 27.McKown KM, Carbone LD, Bustillo J, et al.: Induction of immune tolerance to human type I collagen in patients with systemic sclerosis by oral administration of bovine type I collagen. Arthritis Rheum 2000, 43:1054–1061. Describes the use of oral type I collagen in patients with systemic scleroderma to induce tolerance to collagen type I. Induction of tolerance to collagen type I is worth investigating as a possible new treatment of systemic sclerosis.PubMedCrossRefGoogle Scholar
- 28.Badesch DB, Tapson VT, McGoon MD, et al.: Continuous intravenous epoprostenol for pulmonary hypertension due to the scleroderma spectrum of disease: a randomized, controlled trial. Ann Intern Med 2000, 132:425–434. An important study showing the efficacy of intravenous epoprostenol in the treatment of pulmonary hypertension in systemic scleroderma.PubMedGoogle Scholar
- 29.Klings ES, Hill NS, Ieong MH, et al.: Systemic sclerosisassociated pulmonary hypertension: short- and long-term effects of epoprostenol (prostacyclin). Arthritis Rheum 1999, 42:2638–2645. Another study supporting the efficacy of epoprostenol in the treatment of pulmonary hypertension in systemic scleroderma.PubMedCrossRefGoogle Scholar
- 30.Seibold Jr, Korn JH, Simms E, et al.: Recombinant human relaxin in the treatment of scleroderma: a randomized, double-blind, placebo-controlled trial. Ann Intern Med 2000, 132:871–819. Another important study in which administration of recombinant human relaxin, a pregnancy-related hormone that has tissue remodeling and antifibrotic effect, was associated with reduced skin thickening, improved mobility, and improved function.PubMedGoogle Scholar
- 33.Martini A, Maccario R, Ravelli A, et al.: Marked and sustained improvement two years after autologous stem cell transplantation in a girl with systemic sclerosis. Arthritis Rheum 1999, 42:807–811. The first autologous hemopoietic stem cell transplant electively performed in childhood for the treatment of an autoimmune disease.PubMedCrossRefGoogle Scholar
- 34.White B, Moore WC, Wigley FM, 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 showing that lung function outcomes and survival are improved in patients with alveolitis who received cyclophosphamide.PubMedGoogle Scholar