抗 NXP2 抗体相关中国幼年特发性肌炎患儿的临床特征及不良预测因素。
Clinical characteristics and poor predictors of anti-NXP2 antibody-associated Chinese JDM children.
机构信息
Department of Rheumatology and Immunology, The Affiliated Children's Hospital, Capital Institute of Pediatrics, 2 Yabao Road, Chaoyang District, Beijing, 100020, China.
出版信息
Pediatr Rheumatol Online J. 2021 Jan 6;19(1):6. doi: 10.1186/s12969-020-00492-z.
BACKGROUND
Juvenile dermatomyositis (JDM) is a rare and sometimes fatal disease in children. The anti-NXP2 antibody is one of the most common antibodies and muscle ischaemia associated with NXP2 autoantibodies was a severe subtype of JDM. Further information is needed regarding clinical characteristics and factors associated with poor prognosis. But there are no reports about clinical characteristics and high risk factor of poor prognosis. For the first time, we introduced the clinical characteristics and poor predictors of anti-NXP2 antibody-associated juvenile dermatomyositis in Chinese children.
METHODS
Twenty-six patients with anti-NXP2 antibody-related JDM from 85 JDM Chinese patients were diagnosed from January 2016 to November 2019. Logistic regression was used to analyze the risk factors for refractory cases and mortality.
RESULTS
The ratio of male to female was 1:1.9. The median age of onset was 4.5 (1-13) years. Twenty-four cases (92.3%) had rash and muscle weakness. Treatments included glucocorticoids, immunosuppressive agents, biological agents (7 cases), plasma exchange, Janus kinase inhibitor (7 cases) and autologous stem cell transplant (1 case). Refractory JDM patients (11/26, 42.3%) were associated with edema, skin ulcer, muscle strength<=grade 3, CD4/CD8 ratio < 1.4 and ferritin > 200μg/ml. Among 6 cases (6/26, 23.1%) with severe gastrointestinal involvement, 5 cases died and 1 case survived after autologous stem cell transplant (ASCT). The risk factors for gastrointestinal involvement and mortality were edema, skin ulcer, severe muscle weakness (dysphagia/ hoarseness/ soft voice), BMI < 15 and ANA positive.
CONCLUSIONS
Edema, skin ulcer and severe muscle weakness predicted refractory disease, GI involvement, and mortality in anti-NXP2 antibody-positive JDM of Chinese children. Decreased CD4/CD8 ratio and high ferritin related with refractory cases, and very low BMI and ANA (+) are probably, associated with gastrointestinal involvement and mortality.
TRIAL REGISTRATION
背景
青少年皮肌炎（JDM）是一种罕见且有时致命的儿童疾病。抗 NXP2 抗体是最常见的抗体之一，与 NXP2 自身抗体相关的肌肉缺血是 JDM 的一种严重亚型。需要进一步了解与不良预后相关的临床特征和因素。但是，没有关于抗 NXP2 抗体相关青少年皮肌炎不良预后的临床特征和高危因素的报道。我们首次介绍了中国儿童抗 NXP2 抗体相关青少年皮肌炎的临床特征和不良预后预测因素。
方法
从 2016 年 1 月至 2019 年 11 月，从 85 例中国 JDM 患者中诊断出 26 例抗 NXP2 抗体相关 JDM 患者。采用逻辑回归分析难治性病例和死亡率的危险因素。
结果
男女比例为 1:1.9。发病中位年龄为 4.5（1-13）岁。24 例（92.3%）有皮疹和肌无力。治疗包括糖皮质激素、免疫抑制剂、生物制剂（7 例）、血浆置换、Janus 激酶抑制剂（7 例）和自体干细胞移植（1 例）。难治性 JDM 患者（11/26，42.3%）与水肿、皮肤溃疡、肌肉力量<=3 级、CD4/CD8 比值<1.4 和铁蛋白>200μg/ml 相关。在 6 例（6/26，23.1%）严重胃肠道受累的病例中，5 例死亡，1 例在自体干细胞移植（ASCT）后存活。胃肠道受累和死亡的危险因素是水肿、皮肤溃疡、严重肌无力（吞咽困难/声音嘶哑/声音微弱）、BMI<15 和 ANA 阳性。
结论
中国儿童抗 NXP2 抗体阳性 JDM 中，水肿、皮肤溃疡和严重肌无力预测难治性疾病、GI 受累和死亡率。CD4/CD8 比值降低和铁蛋白升高与难治性病例相关，极低 BMI 和 ANA（+）可能与胃肠道受累和死亡率相关。
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