The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.
Patient, parent, or legal guardian must have given written informed consent. For patients ≥ 168 years of age who are developmentally able, assent or affirmation will be obtained.
Age 8-24, inclusive, at time of consent.
Diagnosed with Systemic Sclerosis (SSc) at the age of ≤19.
Progression of skin thickening over the past 6 months or Modified Rodnan skin score (mRSS) ≥ 20
New York Heart Association classification of heart failure ≥3.
Left ventricular ejection fraction (LVEF) <50% as determined by cardiac MRI.
Significant pulmonary hypertension, for subjects ≥ 18 years of age, defined as mean PASP ≥30 mmHg determined by right heart catheterization, or for subjects ≤ 17 years of age, defined as mean PASP >45 mmHg, determined by echocardiogram.
Atrial tachycardia, atrial fibrillation or atrial flutter of ≥1-minute duration, determined by electrocardiogram (EKG) or, cardiac event monitor and/or implanted loop recorder (if applicable), or on anti-arrhythmic therapy for the arrhythmias listed above.
Paul Szabolcs
Lead sponsor
University of Pittsburgh
Sponsor institution