Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Survival without evidence of relapse or disease progression

o Pulmonary function: Change in forced vital capacity (FVC), total lung capacity (TLC) or diffusing capacity of the lung for carbon monoxide (DLCO) > 10%

o Skin condition: An improvement is indicated by a decrease on modified Rodan Skin Score (mRSS) of > 5 points

o Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) < 4

o Complete remission off therapy (BILAG D/E only or SLEDAI=0 and no SLE treatment except hydroxychloroquine)

o Serologic response: presence of positive ANA, anti-dsDNA and anticardiolpin antibody titers

o Serologic response: abnormal complement C3 and C4 levels

Overall survival will be considered as time from transplant to death from any cause

Events include death, and significant persistent organ damage

o An event based on organ dysfunction must be documented on at least two occasions, at least three months apart and include: respiratory failure (resting O2 saturation < 88%), renal failure (chronic dialysis) and cardiomyopathy (clinical congestive heart failure New York Class III or IV, left ventricular ejection fraction (LVEF) < 30% by echocardiogram despite therapy)

Defined as death from non-disease related causes in the 100 days from stem cell infusion

• Achieving an absolute neutrophil count (ANC) > 500 cells/uL and an unsupported platelet count of > 20,000 cells/uL for three consecutive days

• Quality of life will be measured based on the Patient-Reported Outcomes measurement Information System (PROMIS) that evaluates physical, mental and social health in adults and children.
• patient reported outcome measurement information system (PROMIS) will be administered to each patient (or proxy) prior to autologous stem cell transplant (ASCT) and three times/year for the first two years post-transplant and then annually until five years post-transplant.