NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 1

NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 1

• 1. Refractory SJIA & MAS BMT evaluation criteria: Patient Perspective Luciana Pereira

• 2. Beatriz, 22 yo, Switzerland ● SJIA, MAS (multiple times), LD ● 2018: Bone Marrow Transplant (BMT) ● 2024: In remission, no SJIA medications ○ Just Vitamin D and Calcium Actual treatment: Complete remission Meds used: Steroids, Methotrexate, Actemra, Canakinumab, Anakinra, Cyclosporin, Tacrolimus

• 3. Our Decision for BMT ● Shared decision with medical team - Rheumatology & BMT Teams ● Beatriz was feeling really sick, quality of life was getting worse everyday. ● ILD + Pulmonary hypertension - bad outcome in a short period of time. ● No other medications available, steroid dependant. ● Good match (unrelated donor, 9/10 match). Note: Beatriz was HLA DRB 1*15 positive, but in 2018, we didn’t know this could be related to LD.

• 4. Possible Treatment Options Quartier P. Systemic Juvenile Idiopathic Arthritis/Pediatric Still's Disease, a Syndrome but Several Clinical Forms: Recent Therapeutic Approaches. J Clin Med. 2022;11(5):1357. Published 2022 Mar 1. doi:10.3390/jcm11051357

• 5. Evaluation for BMT depending on center & meds ● At least 9 BMTs patients that we know from FB had BMT since 2018 ○ Different timing for BMT evaluation ● MAS Episodes (smoldering/full blown) ● Lung Disease/Persistent Arthritis/Liver reactions… ● HLA DRB 1*15 positive ● Meds availability in different countries ● Donor availability Is it possible to have an algorithm or protocol for BMT evaluation in SJIA & MAS refractory cases?

• 6. Thank you!