Pediatric Rheumatology Online Journal → August 2003 → Newer Treatments → Bone Marrow/Stem Cell Transplantation → Abstract #163

DISAPPEARENCE OF DIFFUSE CALCINOSIS AFTER AUTOLOGOUS STEM CELL TRANSPLANTATION WITH T CELL DEPLETION IN A CHILD WITH AUTOIMMUNE DISEASE

R. Brik, R. El-hassid

¹Pediatric Rheumatology and Hematology, Meyer, Children's Hospital, Haifa, Israel

High dose immunoablation has been investigated as a theraputic alternative for patients with severe autoimmune diseases that are refractory to conventional therapy. Our patient, 16-year-old girl at the time of transplant, presented at the age 12 with arthritis, myalgia, anemia and a positive ANA. Her uncle suffered from SLE, no other family members had history of autoimmune diseases. During the following years she developed recurrent episodes of pulmonary hemmorage, thrombocytopenia, central and peripheral CNS involvement, skin ulcers and diffuse calcinosis mainly in her pelvis which disabled her severely. During the year prior to transplantation she developed secondary anticardiolipin syndrome with pulmonary hypertension and recurrent episodes of digital ischemia.Repeat laboratory tests did not reveal the presence of autoantibodies except for positive low titer ANA, and later on positive IgG and IgM anticardiolipin. The girl had only partial response to different treatment modalities including corticosteroids, plaqunill, cyclophosphamid, azathioprine, methotrexate and thalidomide. Despite these vigorus immunosuppressive therapies, the child became bedridden, and when she developed pulmonary hypertension, a PBSCT was offered.
Peripheral stem cells were harvested after cyclophosphamid 4gr/mand G-CSF induction. T cell depletion was done by positive selection of CD34 cells by immunomagnetic beads. On day 0 she recieved CD34. Engraftment occur after day 13. Gradually, her mobility improved with ability to perform daily activity. A month after transplantation liquefication of calcinosis nodules started to occur with improvement of hip's range of motion. During the next months most of the calcinosis disappeared as was documented by serial bone scans. The patient is now 18 months since transplant, with no signs of disease activity.
In summary, a girl suffering from an autoimmune disease with severe systemic complications including massive calcinosis achieved complete clinical remission after PBSCT with total removal of calcinosis.