Pediatric Rheumatology Online Journal → July 2003 → Vasculitides → Miscellaneous Vasculitis Entities → Abstract #140
                                                                August 2003 → Newer Treatments → Bone Marrow/Stem Cell Transplantation → Abstract #140

TWENTY-FOUR MONTH FOLLOW-UP AFTER TREATMENT OF CHILD WITH LUNG VASCULITIS USING NONMYELOABLATIVE ALLOGENEIC BMT

O. Y. Jones,¹ R. A. Good,¹ R. A. Cahill.¹

Pediatrics, University of South Florida/ All Children's Hospital, St. Petersburg, FL

A thirteen year-old white female with overlap syndrome and biopsy proven lung vasculitis, underwent allogeneic BMT. Her mother was a 6/6 HLA-matched donor. Upon failure to induce remission with combination immunosuppressives, including cyclophosphamide (cumulative dose 11 g/m), steroid dependency and iatrogenic liver toxicity, we treated her with a non-myeloablative preparatory regimen. This consisted of fludarabine 25 mg/m QD x 5 (days -7 to -3), cyclophosphamide 50 mg/kg QD x 2 (day -3 to -2), and total body irradiation (200 cGy) on day -1. Our patient was infused with 5 x 10[sup3> / kg donor BM mononuclear cells on day 0. The BM cells were processed only for removal of erythrocytes by centrifugation due to ABO incompatibility (A -- 0). For additional BM mesenchymal cells (MSCs), she was also implanted i.p.with maternal bone chips on day 0 and infused with in vitro with culture expended donor MSCs (1.5 x 10⁵) on day + 13. Posttransplant immunosuppression was with Cyclosporine (6 mg/kg po BID) and Mycophenolate Mofetil (30 mg/kg/day) from day -1 were weaned off by 3 months and 8 months posttransplant, respectively. She tolerated the procedure well without any evidence of GVHD or infections. She has been 95% donor per short tandem repeats (STR) of PBCs in the last year, STR showed 66% donor in core bone biopsy at 9 months posttransplant, and pulmonary functions tests were stable. She switched her blood type from 0+ to A+ at 12 months posttransplant without significant anemia. She was gradually weaned off steroids and has since been taken off all immunosuppressive medications. Patient represents the first pediatric case treated with allogeneic BMT after non-myeloablative conditioning solely for the treatment of an autoimmune disorder. The results of her treatment encourages future clinical trials using similar protocols and in investigating the role of MSCs for future treatment of lung vasculitis and other autoimmune diseases.