Patients.
DISEASE
Following regulatory approval in July 2018, between September 2018 and February 2020 18 patients (10 children and 8 adults) were enrolled. Of these, 16 (all 10 children and 6 adults) received the autologous BM-MSC infusions according to protocol. Two adult patients did not receive autologous BM-MSCs, because following enrolment, their disease changed and they were considered “screening failures” due to lack of eligibility.When the COVID-19 pandemic occurred, due to logistical difficulties and for the safety of patients, we had to modify our modality of study assessments, as face-to-face visits were not possible. We obtained complete clinical and laboratory data for 8 out of 10 children, while for 1 child we obtained complete data until 8 months after BM-MSC infusion and subsequently remote clinical and safety data until study completion, and for 1 other we obtained complete data until 8 months after MSC infusion, and then only retrospective clinical information (relating to relapses and immunosuppression) and safety data at 9 and 12 months. Of the 6 adult patients who received the BM-MSC infusions according to study protocol, 5 completed the study. One patient was obliged to skip visits after 8 months from BM-MSC infusion due to the COVID-19 pandemic. The last patient visit occurred on July 14, 2021 (