Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases

A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach.

Inclusion Criteria:

1. Age 8 ≤ 25 years at time of enrollment.
2. Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
3. Adequate organ function status
4. No active, untreated infections.

Exclusion Criteria:

1. Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant
2. Pregnancy
3. Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
4. Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy