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

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The document discusses the evaluation criteria for Bone Marrow Transplant (BMT) in patients with Refractory Systemic Juvenile Idiopathic Arthritis (SJIA) and Macrophage Activation Syndrome (MAS).
It presents a patient perspective, highlighting the shared decision-making process with medical teams and the impact of the patient's quality of life on treatment choices.
The patient, Beatriz, had SJIA, MAS, and underwent a BMT in 2018, achieving remission by 2024 with minimal medication.
Key factors in the BMT decision included failed other medications, severity of illness, and availability of a good donor match.
It explores possible treatment options, classical approaches, and current research on treating different clinical situations associated with SJIA, MAS, and other complications.

#Stillsdisease #Sjiafoundation #Sjia #Bmt #Curesjia

$Refractory SJIA & MAS BMT evaluation criteria: Patient Perspective Luciana Pereira$

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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

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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.

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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

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

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