Long Term outcomes of Allogeneic HSCT for severe, refractory Juvenile Idiopathic Arthritis

Juliana Silva

Bone marrow Transplant Consultant Great Ormond Street Hospital London, UK

EBMT

Allo-HSCT for JIA

juvenile idiopathic arthritis

· Most common cancer in children & most common cause of death < 20 years of age; Persis J. Amrolia Rachael Hough; Rebecca Marsh, 3,4 Mario Abinun, 6,7 and Paul Lucy Veys

• · 16 patients underwent allo HSCT from 5 different centres
• · Median FU was 2y 5m
• · 4 MSD, 8 MUD and 4 mMUD
• · 10 Flu/Mel/Alemtuzumab and 6 Flu/Treo/ Alemtuzumab

Blood Advances, 2018

Results

• · 12/16 (75%) patients achieved CR;
• · 2/16 patients died of transplant related toxicity.
• · 9/15 FULL donor chimerism
• -1 relapse, 1 PR
• · 5/15 MIXED chimerism
• -1 PR
• · 1/15 AUTOLOGOUS reconstitution
• -CR

LTFU UPDATED COHORT


HSCT CHARACTERISTICS


LONG TERM RESULTS


CAUSES OF DEATH

• 1 Fungal chest infection D+85
• 1 RSV pneumonitis D+28
• 2 Thrombotic microangiopathy both 6/8 m post HSCT
• 1 Sepsis post-surgery for hips replacement 1.5 y, CR
RELAPSE/REMISSION


RELAPSE/ ACTIVE DISEASE


Survival Outcomes

6y OS: 84%

O(S

Relapse: 42% (11/26)

• · Deaths within first 18 m
• · Achieved further CR: 5/11 (45%)
• · Active disease: 6/11 (54%)

CR: 19/26 (61%) at last FU PR: 1/26 (4%)

Deaths: 4x transplant related 1x complication post surgery, in CR No JIA related deaths

In summary

• · Allogeneic HSCT is an alternative option for patients with refractory and/or complicated sJIA;
• · Promising results for ILD;
• · Indications and Timing for transplant;
Future perspectives

• · Consensus Statement on indications and timing for transplant

· Prospective study

• · Standardised transplant recommendations;
• · Consolidate response with biologics;
• · Biomarkers analysis to assess disease response
• · Short and LTFU including joint/quality of life assessments;
• · CAR T cells

Acknowledgements

Teaching Hospital Motol, Prague Petr Sedlacek

Renata Formankova

GOSH

Persis Amrolia Kanchan Rao Robert Chiesa Giovanna Lucchini Khushnuma Mullafironze Harini Rao Archana Rauthan

Rheumatology

Team GOSH

Great North Children's Hospital

Andrew Gennery Mary Slatter Sophie Hambleton Terence Flood Zohreh Nademi Mario Abinun Andrew Cant

Mark Friswell

UCLH

Rachael Hough Ben Carpenter

Vicky Grandage

Cincinnati Children's Hospital Rebecca Marsh Sharat Chandra

University of Children's Hospital, Zurich, Switzerland Tayfun Güngör Ulrike Zeilhofer

Necker Enfants Malades, University Hospital Paris, France Bénédicte Neven Despina Moshous Benjamin Fournier

Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, USA Susan Prockop

Thank you!

EBMT