NCT07845942招募中不适用
Allogeneic Hematopoietic Cell Transplantation for Refractory Systemic Juvenile Idiopathic Arthritis: A Single-Center Prospective Trial
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Overall Survival at 1 year
研究概览
简要总结
The goal of this study is to collect clinical data and samples and to evaluate the feasibility of allogeneic hematopoietic cell transplantation (HCT) in children and young adults with refractory Systemic Juvenile Idiopathic Arthritis (sJIA), including those with sJIA-associated lung disease (sJIA-LD).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients <35 years of age are eligible, and participants must meet all the following criteria to be eligible for this study:
- •Diagnosis: Systemic Juvenile Idiopathic Arthritis (sJIA) diagnosed prior to age 16, fulfilling ILAR classification criteria for sJIA or the provisional PRINTO criteria for systemic arthritis. The diagnosis and disease course must be reviewed and confirmed by a pediatric rheumatologist.
- •Refractory Disease: sJIA that is refractory to standard therapy, defined as meeting at least one of these conditions despite appropriate treatment:
- •Persistent JIA activity: Ongoing active disease (e.g. uncontrolled arthritis or systemic features such as fever, rash, organ inflammation) or progressive joint damage or liver dysfunction that has not responded to biologic agents (IL-1/IL-6 inhibitors) and conventional therapies, or inability to wean from high-dose corticosteroids (steroid dependence).
- •Recurrent MAS: Two or more episodes of macrophage activation syndrome within a 2-year period, indicating a failure to control the hyperinflammation with medical management. (MAS is defined by clinical and laboratory criteria such as fever, cytopenias, hyperferritinemia, hemophagocytosis, etc., per standard definitions.)
- •sJIA-LD: Presence of lung disease related to sJIA, either symptomatic LD (e.g. chronic cough, tachypnea, hypoxemia) or progressive LD on imaging (worsening interstitial changes), meeting clinical/imaging features such as patchy ground-glass opacities, septal and pleural thickening, or clubbing of digits. sJIA-LD should be confirmed by a pulmonologist/radiologist (e.g. HRCT findings).
排除标准
- •Uncontrolled Infection: Presence of a severe, uncontrolled infection at time of enrollment (e.g. sepsis, pneumonia not responding to therapy).
- •Significant Organ Failure:
- •Cardiac: EF < 40%
- •Renal: GFR < 30 ml/hr
- •Active Malignancy: Concurrent cancer diagnosis or history of malignant disease requiring active treatment.
- •Pregnancy or Lactation: Female patients who are pregnant or breastfeeding. Female participants of childbearing potential must have a negative pregnancy test clinical result at screening (pregnancy testing is done as standard of care for all transplant patients).
结局指标
主要结局
Overall Survival at 1 year
时间窗: 1-year post-transplant
次要结局
- sJIA clinical remission(1-year post-transplant)
- Corticosteroid and biologic independence(Time Frame: 1-year post-transplant)
- Graft Failure(1-year post-transplant)
- Acute and Chronic Graft-Versus-Host Disease (GVHD)(1-year post-transplant)
- Transplant-Related Mortality(1-year post-transplant)
- Pulmonary Outcomes in Participants With sJIA-LD(1-year post-transplant)
研究者
研究点 (1)
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