INTRODUCTION: Juvenile dermatomyositis/juvenile polymyositis (JDM/JPM), juvenile systemic scleroderma (JSCL-SYST), and juvenile mixed connective tissue disease (JMCTD) are rare in childhood. OBJECTIVE: The objective of this study was to evaluate the prognosis of the rare connective tissue diseases (RCTDs) in children, METHODS: We reviewed the medical charts of children with a diagnosis of RCTD since 1989 and a minimum follow-up of 5 years. RESULTS: Twenty-four (16 female, 8 male) children with JDM/JPM, JSCL-SYST, and JMCTD were studied. The age at disease onset ranged from 4 to 13 years. The follow-up duration was 5 to 12 years. Sixteen children had JDM, and 2 had JPM. Four had JSCL-SYST, and 2 had JMCTD. Until now, 13 children have reached clinical remission, lasting >3 years after stopping drug therapy. Twelve children had JDM/JPM, and 1 had JMCTD. Persistent disease activity was noted in 11 children: 4 with JSCL-SYST, 6 with JDM/JPM, and 1 with JMCTD. Severe pulmonary disease developed in 3 children: 2 with JSCL-SYST and 1 with JMCTD. None of the children with JDM had pulmonary disease. Pulmonary hypertension (PH) was found in 2 children with JMCTD or JSCL-SYST. The child with JSCL-SYST and PH died. Persistent scleroderma pattern by wide-field capillaroscopy was noted in 4 children who had JDM and had had skin ulcerations and have developed subcutaneous calcifications. One of them has also had marked muscle atrophy and severe contractures. CONCLUSIONS: Persistent activity and/or severe pulmonary involvement may be present during the clinical course of RCTD. The presence of PH indicates very poor prognosis in JSCL-SYST/JMCTD cases. Capillaroscopy may identify children who have JDM and are candidates for aggressive therapy.
A girl, 3 years and 9 months old, suffered from idiopathic thrombocytopenic purpura (ITP), which was evidenced with extensive petechiae and ecchymoses at the onset of a segmental pneumonia of the left lung. High doses of intravenous c -globulin (IVIG: 1 g/kg day ¡ 1 £ 2 days) were administered to control severe epistaxis without achieving a signi® cant increase in platelet count. Over the following 11 months the child remained severely thrombocytopenic and presented with frequent hemorrhagic manifestations (petechiae, ecchymoses, hematuria). Therapeutic attempts with IVIG, prednisolone in conventional doses (2 mg/kg day ¡ 1 ), ascorbic acid, or high doses of methylprednisolone [1] failed to induce a signi® cant and permanent increase in platelets. The short duration of ITP and the patients young age restricted us from preceding to a selective splenectomy. From experience we know that a small number of children with chronic problematic ITP may remit spontaneously or after therapeutic intervention several years from the onset of the disease (even 10 years later) [2]. Besides, splenectomy is known to be associated with a higher incidence of fulminant septicemia in children less than 5 years of age than in older children or adults [3,4]. For those reasons and to give our patient a better chance of remission, we decided to treat her with a course of a -interferon (a -IFN) subcutaneously at a dosage of 3,000,000 IU/m 2 SQ 3 times a week for 4 weeks [5] and subsequently once a week. A prompt increase in platelets was ® rst observed the fourth day from
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