NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 4

NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 4

@SJIA_Foundation
@SJIA_Foundation
8 Followers
2 weeks ago 73

NextGen 2024: Bone Marrow Transplantation (BMT) Session Part 4

@SJIA_Foundation2 weeks ago

Long Term outcomes of Allogeneic 
HSCT for severe, refractory Juvenile 
Idiopathic Arthritis
Jul…
1/14
Allo-HSCT for JIA
• Most common cancer in children & most common cause of death 
< 20 years of ag…
2/14
Results
• 12/16 (75%) patients achieved 
CR;
• 2/16 patients died of transplant 
related toxici…
3/14
LTFU UPDATED COHORT
N. centers 9
N. patients 31
Period 2007 - 2022
Type of JIA
• Polyarthritis…
4/14
HSCT CHARACTERISTICS
HLA matching
10/10 MSD 6/31 (19%)
10/10 MUD 18/31 (58%)
9/10 MMUD 5/31 (16…
5/14
LONG TERM RESULTS
N=31 
24/26
CR
1/26
PR
5/31
Died 
14/31
CR
1/2
Relapsed
/
Active 
d…
6/14
CAUSES OF DEATH
 1 Fungal chest infection D+85
 
 1 RSV pneumonitis D+28
 2 Thrombotic microang…
7/14
RELAPSE/REMISSION
Patient JIA
subtype
Time to 
relapse
Type of 
relapse
Chimerism 
at relap…
8/14
RELAPSE/ ACTIVE DISEASE
Patient JIA
subtype
Time to 
relapse
Type of relapse Chimerism at 
re…
9/14
Survival Outcomes
6y OS: 84% 
• Deaths within first 18 m
CR: 19/26 (61%) at last FU
PR: 1/26 (4…
10/14
In summary
•Allogeneic HSCT is an alternative option for patients with 
refractory and/or complic…
11/14
Future perspectives 
• Consensus Statement on indications and timing for transplant
• Prospective…
12/14
Acknowledgements
Teaching Hospital 
Motol, Prague
Petr Sedlacek 
Renata Formankova
GOSH
Persi…
13/14
Thank you!
14/14


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