A team of researchers from Indiana University and Cook General Biotechnology has successfully co-transplanted bone marrow and marrow-derived mesenchymal stem/stromal cells (MSC) in a patient with Franconi anemia (FA). The team presented their work at the 2012 AABB Conference in Boston, MA. FA patients are often treated with stem cell therapy, but this can lead to complications including engraftment failure and increased mortality from graft-vs-host disease (GVHD). MSC had not previously been used to treat FA patients, but had been shown to enhance engraftment and prevent and/or treat GVHD. In the presentation, the research team reported that the MSC infusions were well-tolerated without complications, hematopoetic engraftment occurred rapidly (12 days following stem cell transplantation), and no donor MSC engrafting occurred. Four months post-transplantation, the patient had normal blood counts, no serious infections, no GVHD, and did not require immunosuppression. The study authors suggest that trials to assess the role of MSC therapy in improving engrafting and preventing GVHD are warranted.
1. Goebel W, Nalepa G, Shadday MD, Helman R, Hanenberg H, Hawkins TB, Duffy LJ, Reeves L, Woods EJ, Robertson KA. Co-transplantation of Matched Related Donor Marrow and Marrow-derived Mesenchymal Stem/stromal Cells for the Treatment of Franconi Anemia AABB Conference 2012. Boston, MA, 2012. Transfusion 2012;52 Suppl 3: 11A-284A.