Zulian Francesco, Dal Pozzolo Roberto, Meneghel Alessandra, Castaldi Biagio, Marcolongo Renzo, Caforio Alida Linda Patrizia, Martini Giorgia
Department of Woman's and Child's Health.
Clinical Immunology, Department of Medicine.
Rheumatology (Oxford). 2020 Dec 1;59(12):3793-3797. doi: 10.1093/rheumatology/keaa193.
Juvenile systemic sclerosis (JSSc) with rapidly progressive course is a life-threatening condition associated with a poor prognosis. Recently, rituximab (RTX) has been shown to be a promising treatment for adult patients with SSc. We present a series of four patients with rapidly progressive JSSc successfully treated with RTX.
Clinical, laboratory and functional parameters were collected from four patients with rapidly progressive JSSc treated with RTX for at least 1 year. All patients underwent four yearly courses of i.v. RTX 375 mg/m2 on day 0 and 14, at 3-month intervals. Low dose oral prednisone and MMF were also administered. Data were recorded at baseline and every 6 months and included pulmonary and myocardial function parameters, muscular, vascular and skin changes. The Juvenile Systemic Sclerosis Severity Score (J4S) estimated the overall disease severity over time.
Four patients (three males, one female), aged 8-17 years, entered the study. Three patients presented with prevalent cardiac involvement, one with severe pulmonary involvement. After 1 year of RTX treatment, all patients showed significant improvement of J4S, Raynaud's phenomenon and cutaneous involvement. Among those with prevalent cardiac involvement, two showed an improvement of the myocardial function (left ventricular ejection fraction [EF] +37% and +19%, respectively) and in the third arrhythmias disappeared. The patient with severe pulmonary involvement showed a significant improvement of the respiratory function (forced vital capacity +46%, forced expiratory volume in 1 s +33%, diffusing capacity of the lung for carbon monoxide [DLCO] +30%). No major side effects were reported.
Our data suggest that a combination of RTX and MMF is effective in arresting the rapid progression of JSSc.
快速进展型青少年系统性硬化症（JSSc）是一种危及生命且预后不良的疾病。最近，利妥昔单抗（RTX）已被证明是治疗成年系统性硬化症（SSc）患者的一种有前景的疗法。我们报告了4例快速进展型JSSc患者经RTX成功治疗的病例。
收集了4例接受RTX治疗至少1年的快速进展型JSSc患者的临床、实验室和功能参数。所有患者在第0天和第14天接受4个年度疗程的静脉注射RTX，剂量为375mg/m²，间隔3个月。同时给予低剂量口服泼尼松和霉酚酸酯（MMF）。在基线和每6个月记录数据，包括肺和心肌功能参数、肌肉、血管和皮肤变化。青少年系统性硬化症严重程度评分（J4S）评估了随时间推移的整体疾病严重程度。
4例患者（3例男性，1例女性），年龄8 - 17岁，进入研究。3例患者主要表现为心脏受累，1例有严重肺部受累。RTX治疗1年后，所有患者的J4S、雷诺现象和皮肤受累均有显著改善。在主要表现为心脏受累的患者中，2例心肌功能改善（左心室射血分数[EF]分别增加37%和19%），第3例心律失常消失。严重肺部受累的患者呼吸功能有显著改善（用力肺活量增加46%，第1秒用力呼气量增加33%，肺一氧化碳弥散量[DLCO]增加30%）。未报告重大副作用。
我们的数据表明，RTX和MMF联合使用可有效阻止JSSc的快速进展。
