Efficacy.
DISEASE, REMISSION
As shown in At baseline, all patients were on glucocorticoids and most of them were on 1 or 2 steroid-sparing agents, such as calcineurin inhibitors (cyclosporine A or tacrolimus) and an antiproliferative agent (mycophenolate mofetil) (In a post hoc analysis, we created a scoring system to integrate in a single measurement the cumulative effects of reducing the number of relapses in the previous and subsequent 12-month periods and the number of immunosuppressive drugs needed to maintain remission at time of BM-MSC infusions and at +12 months. To this end, we simply cumulated these 2 values for each patient. We found that the median cumulative score was significantly reduced at 12 months after BM-MSC treatment compared with the previous 12 months in the study group considered as a whole (3 [IQR 2–4] vs. 5 [IQR 4–5], Thus, autologous BM-MSC treatment was effective in reducing disease activity (i.e., delaying relapse while reducing immunosuppressive therapy) in children with severe steroid-dependent, multi-relapsing INS, but not in adults.In order to determine whether BM-MSC treatment was effective in improving patient well-being, we evaluated quality of life questionnaires collected at time of BM-MSC infusions (baseline) and at +1, +6, and +12 months after BM-MSC infusions in children for whom we obtained complete data (9 out of the 10 included in the study). Despite the hospitalizations required per protocol both for BM harvesting and for BM-MSC infusions, no child had a worsening in the overall score of well-being and the median score of the parameter representing the physical health conditions significantly increased from 66 [IQR 25–84] to 100 [IQR 84–100] (We did not find any significant posttreatment change in height, weight, BMI, systolic and diastolic blood pressure (