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).
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.
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).
Uncontrolled Infection: Presence of a severe, uncontrolled infection at time of enrollment (e.g. sepsis, pneumonia not responding to therapy).
Cardiac: EF < 40%
Renal: GFR < 30 ml/hr
Active Malignancy: Concurrent cancer diagnosis or history of malignant disease requiring active treatment.
Children and young adults \<35 years of age with longstanding Systemic Juvenile Idiopathic Arthritis diagnosed in childhood will be enrolled if they meet the eligibility criteria below. Patients with Systemic Juvenile Idiopathic Arthritis associated lung disease are included and will be specifically evaluated.
Cohort
Prospective
Not listed
Overall Survival at 1 year
sJIA clinical remission
Proportion achieving clinical remission/ inactive diasease at 1 year
Corticosteroid and biologic independence
Time to discontinuation of corticosteroids and biologic therapy following HCT.
Graft Failure
Incidence of primary and secondary graft failure following HCT.
Acute and Chronic Graft-Versus-Host Disease (GVHD)
Incidence and severity of acute and chronic GVHD following HCT.
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