ObjectiveTo determine the short-term effectiveness safety of baricitinib in children with refractory and/or severe juvenile dermatomyositis (rsJDM) in a real-world setting.MethodsThis was a single-center retrospective study, including 20 children with rsJDM. They were all treated using baricitinib combined with steroids and other immunosuppressive agents. The childhood myositis assessment scale (CMAS) and PRINTO remission criteria were used to evaluate the disease severity and treatment outcome at 0, 4, 12, and 24 weeks after initiation of baricitinib.ResultsThe skin rash improved in 95% of patients (19/20) at week 24, with a significant decrease of skin-DAS at weeks 12 (6.0 vs. 2.0, p < 0.05] and week 24 [6.0 vs. 1.0, p < 0.05) by median statistics. The CMAS score increased significantly at week 12 (41.0 [29.0, 44.0] vs. 46.0 [42.0, 52.0], p < 0.05) and week 24 (41.0 [29.0, 44.0] vs. 50.0 [45.0, 52.0], p < 0.05), as did the manual muscle testing (MMT)-8 score at week 24 (73.0 [610, 76.0] vs. 79.0 [77.0, 80.0], p < 0.05). At 24 weeks, the complete response (CR) and partial response (PR) were achieved in 75% (15/20) and 15% (3/20), respectively. The dose of corticosteroids (CS) decreased by 37% from the baseline (0.53 [0.42, 1.00] mg/kg) to week 12 (0.33 [0.18, 0.40] mg/kg) (p < 0.05), and by 49% at week 24 (p < 0.05). No serious side effects were observed.ConclusionBaricitinib combined with traditional immunosuppressants treatment was efficacious in rsJDM. Add-on therapy of baricitinib was helpful for tapering CS dose. No serious side effects were observed in this study.

BackgroundTakayasu arteritis (TA) is a chronic granulomatous vasculitis with unknown pathophysiology. TA with severe aortic obstruction has a poor prognosis. However, the efficacy of biologics and appropriate timing of surgical intervention remain controversial. We report a case of tuberculosis (TB)-associated TA with aggressive acute heart failure (AHF), pulmonary hypertension (PH), thrombosis, and seizure, who failed to survive after surgery.Case presentationA 10-year-old boy who developed a cough with chest tightness, shortness of breath, hemoptysis with reduced left ventricular ejection fraction, PH, and increased C-reactive protein and erythrocyte sedimentation rate was hospitalized at the pediatric intensive care unit of our hospital. He had strongly positive purified protein derivative skin test and interferon-gamma release assay result. Computed tomography angiography (CTA) showed occlusion of proximal left subclavian artery and stenosis of descending aorta and upper abdominal aorta. His condition did not improve after administration of milrinone, diuretics, antihypertensive agents, and intravenous methylprednisolone pulse followed by oral prednisone. Intravenous tocilizumab was administered for five doses, followed by two doses of infliximab, but his HF worsened, and CTA on day 77 showed complete occlusion of the descending aorta with large thrombus. He had a seizure on day 99 with deterioration of renal function. Balloon angioplasty and catheter-directed thrombolysis were performed on day 127. Unfortunately, the child's heart function continued to deteriorate and died on day 133.ConclusionTB infection may be related to juvenile TA. The biologics, thrombolysis, and surgical intervention failed to achieve the anticipated effect in our case with aggressive AHF due to severe aortic stenosis and thrombosis. More studies are needed to determine the role of biologics and surgery in such dire cases.

Objective To evaluate the efficacy and safety of low dose baricitinib in children with refractory or severe juvenile dermatomyositis (JDM) in a real-world setting. Methods A monocentric retrospective real-world study was conducted, in which fourteen refractory and one severe newly diagnosed JDM patients were included. These patients were all treated by low dose baricitinib (below the recommended dose) combined with corticosteroids and or immunosuppressive agents. Clinical data were collected at the baseline and 4, 12, 24 weeks after baricitinib implication. Treatment response (complete response (CR), Partial response (PR) and non-response (NR)) was evaluated using both the Paediatric Rheumatology International Trials Organization (PRINTO) remission criteria and skin Disease Activity Score (DAS). All the adverse events (AEs) were recorded. Results After baricitinib treatment, all 15 patients showed improvement of skin involvement, including 14 patients with recurrent skin rashes and one newly diagnosed JDM. Calcinosis stabilized in two patients (2/3) and partially regressed in one. Four patients (4/15) had interstitial lung disease (ILD), which normalized in one, improved in two and stabilized in one. One patient complicated with macrophage activation syndrome (MAS) achieved clinical remission. CR was achieved in 3/15 patients, ranging from 4 to 12 weeks after baricitinib initiation. Five patients (5/15) got PR 4 to 24 weeks after baricitinib use. Daily steroid dosage was decreased from 0.632 mg/kg to 0.357 mg/kg (P = 0.043) at 24 weeks in all responders. However, there was no statistically difference in muscle improvement. One patient was stopped using baricitinib because of varicella zoster virus infection, while no other serious side effect was observed in this study. Conclusion Low dose baricitinib had efficacy and was safe to applied in refractory or severe JDM patients, especially for recurrent skin rashes. Baricitinib may also be helpful for JDM complicated with ILD and MAS.

Objective To evaluate the efficacy and safety of low dose baricitinib in children with refractory or severe juvenile dermatomyositis(JDM) in a real-world setting. Methods A monocentric retrospective real-world study was conducted, in which fourteen refractory and one severe newly diagnosed JDM patients were included. These patients were all treated by low dose baricitinib (below the recommended dose) combined with corticosteroids and or immunosuppressive agents. Clinical data were collected at the baseline and 4, 12, 24 weeks after baricitinib implication. Treatment response (complete response, CR, Partial response, PR and non-response,NR) was evaluated using both the Paediatric Rheumatology International Trials Organization (PRINTO) remission criteria and skin Disease Activity Score (DAS). All the adverse events (AEs) were recorded. Results After baricitinib treatment, all 15 patients showed improvement of skin involvement, including 14 patients with recurrent skin rashes and one newly diagnosed JDM. Calcinosis stabilized in two patients (2/3) and partially regressed in one. Four patients (4/15) had interstitial lung disease (ILD), which normalized in one, improved in two and stabilized in one. One patient complicated with macrophage activation syndrome (MAS) achieved clinical remission. CR was achieved in 3/15 patients, ranging from 4 to 12 weeks after baricitinib initiation. Five patients (5/15) got PR 4 to 24 weeks after baricitinib use. Daily steroid dosage was decreased from 0.632 mg/kg to 0.357 mg/kg (P = 0.043) at 24 weeks in all responders. However, there was no statistically difference in muscle improvement. One patient was stopped using baricitinib because of varicella zoster virus infection, while no other serious side effect was observed in this study. Conclusion Low dose baricitinib had efficacy and was safe to applied in refractory or severe JDM patients, especially for recurrent skin rashes. Baricitinib may also be helpful for JDM complicated with ILD and MAS.