THERAPI E CELLULAIRE ET SCLERODERMIE

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1 THERAPI E CELLULAIRE ET SCLERODERMIE Pr Dominique Farge, Hôpital St Louis, INSERM U 796, Université Paris 7 Denis Diderot, ADWP EBMT Chair The European Group for Blood and Marrow Transplantation

2 CORTICOIDES CSH : Moelle Osseuse, CSHP, CSM, Sang Cordon Remise à zéro de la Réponse Immune: TOLERANCE AZATHIOPRINE SAL CYCLO A MTX NEORAL GREFFE..iv CYCLO po, iv Anti TNF, Anti-CD2, Anti-Blys MMF 25 MSC FORMES SEVERES ou RAPIDEMENT EVOLUTIVES : Sclérodermie Systémique: survie 5 ans 3% 4% Lupus Erythémateux: survie 1 yrs /IRT: 7 / 5% 9 / 35% Sclérose en Plaques: DMD IFN, copaxone, natalizumab, mitoxantrone, fingolimod, cladribine Maladie de Crohn: DMD 5-ASA, steroids, Aza, Metho infliximab, Adalimumab, natalizumab DID type I précoce non cétoacidotique: Insulinothérapie à vie 21

3 GM? pour traiter MAI severe chez homme IKEHARA PNAS 1985 dysfonction celllules T<= involution thymique souris AI + an cellules CHS V BEKKUM Best Pract Res : premier succès du tt par allo GM chez souris LED Meilleurs résultats avec fragments MO foetale: cellules stromales? CSM?

4 Haematopoietic stem cell transplantation (HSCT) in severe auto-immune diseases: updated guidelines written on behalf of the EBMT ADWP and PDWP J Snowden, R Saccardi, M Allez, S Ardizzone, R Arnold, R Cervera, C Denton, JM van Laar, M Labopin, G Mancardi, R Martin, JJMoore, J Passweg, C Peters, M Rabusin, M Rovira,, D Farge BMT 211on line, free access Per Ljungmann BMT 29 Niveau II = au moins 1 essai clinique de bonne qualité non randomisé: cohorte ou étude analytique cas controle ( de préférence > 1 centre), séries avec suivi répété temps MAI Donneur Appar Donneur non apparenté Donneur non apparenté Autologue bonne concordance SEP D/III GNR/III GNR/III CO / II SSc D/III GNR/III GNR/III CO /II SLE LED D/III GNR/III GNR/III CO / II Crohn s GNR/III GNR/III GNR/III CO / II PR GNR/III GNR/III GNR/III CO/II VAscularite GNR/III GNR/III GNR/III CO/II Polymyositis- Dermatomyosite GNR/III GNR/III GNR/III CO/II CIPD GNR/III GNR/III GNR/III CO/II Cytopenie CO/II D/III GNR/III CO/II DT1 GNR/III GNR/III GNR/III D/III RCD Type II GNR/III GNR/III GNR/III D/III Special report Blood and marrow stem cell transplants in auto-immune disease: a consensus report written on behalf of the EULAR and the EBMT A Tyndall and A Gratwohl BMT 1997

5 Nombre de Greffes CSHP: 1357 (1254 autologues) Centres /Pays 215/31 Durée Suivi 2.9 ans (<1-24) MULTIPLE SCLEROSIS 476 CONNECTIVE TISSUE D. SSc SLE 12 PM-DM 18 Sjogren 3 Antiphosph. syndrome 3 Other/Unknown 15 ARTHRITIS 163 Rheumatoid arthritis 8 Juvenile chronic arthritis : - Systemic JIA 49 - Other JIA 18 - Polyarticular JIA 1 Psoriatic arthritis 3 Other 4 INFLAMMATORY BOWEL Crohn's disease Ulcerative colitis Other 4 7 HAEMATOLOGICAL 8 ITP 26 Evan s 19 AIHA 18 Other 17 VASCULITIS 41 Wegener s 1 Behcet s 8 Takayasu 2 Microscopic poly. nodosa 3 Classical poly. nodosa 1 Churg-Strauss 2 Other/Unknown 14 OTHER NEUROLOGICAL 38 Myasthenia gravis Other/Unknown 5 33 INSULIN DEPENDANT DIABETES 1 OTHER/UNKNOWN/MISSING 29

6 Nombre de Greffes CSH par an: Registre EBMT *Données en cours pour * Italy Germany United Kingdom France Netherlands... Spain Sweden Australia Czech Republic Greece China Russia Belgium Hungary Poland Israel

7 Nombre de Greffes CSH par an: Registre EBMT *

8 Survie Globale 3 ans (n= 9) Survie sans Progression à 3 ans (n=9) 1,,8,6, % RA (n = 89) % MS (n = 345) % SLE (n = 85) % JIA (n = 65) % HIC (n=37) 8 + 3% SSc (n = 137) 1,,8,6, % SSc (n = 137) % MS (n = 345) % SLE (n = 85) % JIA (n = 65) % HIC (n=37) % RA (n = 89),2,2, , Effet centre TRM, OS, PFS : lié à activité (n > 13); TRM : %

9 Systemic Sclerosis: prevalence 7 5 / Million SKIN THICKNESS joint contractures, GI, lung, heart, kidney Intermediate Diffuse Cutaneous Late Limited Cutaneous Intermediate pulmopnary hypertension malabsorption Late N risk factors Total no. of pts Nb / RR of deaths Fransen EUSTAR 21 FVC DLCO Nanini Athr Res Ther 28

10 RESULTATS CLINICO-PATHOLOGIQUES: Régression Fibrose cutanée et pulmonaire + Améliorations fonctionnelles Performance statut FVC 15 * 7 * * V C Year year 1 DLCO % 2% 4% 6% 8% 1% Percentage Farge et al BJH 2, Ann Rheum 24, Vonk et al Ann Rheum 28 3,5 3 2,5 2 1,5 1,5 Verrecchia F, O Verola Rheumatology 27 DP DRS DRM DRP Aschwanden Daikeler et al ARD 28 Launay D J Rheumatol 29 scanner + CVF DLCO

11 DETERMINANTS DE LA REPONSE APRES HSCT Baraut J Autoimmunity Review 21 CD19+ et CD2+ et AC anti SCL7 (r =.27, p<.5) Lymphopenie T CD4+, CD4+CD45RA+: favorable SSc TREC /CRP: rp<.1, TREC / CD19+: rp< O.OO1 ( PR, SEP) Farge Arthr Rheum 25

12 ASTIS: Pts rapidly progressive or severe SSc (n = 156 ) 4 yrs + skin score 15 (-51) + involvement heart/lung/kidney 2 yrs + skin score 2 + ESR>25mm/1 st hr and/or Hb<11 gr/dl Immunoablation + AST = 1. Mobilisation CYC4 g/m 2,G-CSF 1 m g/kg 2. Leukapheresis /CD34-selection 3. Conditioning C YC 2 mg/kg, ATG 7.5 mg/kg Reinfusion CD34+ cells Standard-therapy 12x monthly 2 i.v. pulse CYC 75 mg/m EFS = survival minus persistent major organ failure (heart, lung, kidney) Exclusion criteria: PHT > 5 mmhg, DLCO < 4%, creat.cl. < 4 ml/min. LVEF < 45%; uncontrolled arhythmia; cardiac tamponade infection, etc. previous treatment with CYCLO: >5 gr iv, >3 mths po

13 [si1] Accrual ASTIS trial Accrual per centre number of patients /22/21 3/22/22 3/22/23 3/22/24 3/22/25 3/22/26 date 3/22/27 3/22/28 3/22/29 Paris Leiden Nijmegen Florence Amsterdam Basel Herne-Trier Tubingen Strasbourg Leeds Vienna Marseille Grenoble Freiburg Toulouse Wurzburg Bordeaux Clerm Ferr Ferrara Frankfurt Leuven Lille Montreal Montpellier Thessaloniki Milan Middlesbrough 156 SSc: 79 SCT+77 controles (27 centres) France: 49; Netherlands: 54 Allemagne: 2; Italie: 16 Suisse 7, GB: 5, Austriche:3, Belgique 1, Can1 1Grece: 1 RESEARCH COLLABORATION AGREEMENT N VAL / 211/ /1 version 12 dec BETWEEN: ASSISTANCE PUBLIQUE HOPITAUX DE PARIS, AND EUROPEAN GROUP FOR BLOOD AND MARROW TRANSPLANTATION

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16 RECONSTITUTION IMMUNE YES WE CAN INDUCE RESET OF TOLERANCE Radbruch A Ann Rheum Dis 24 Hugle T Daikeler T Hematological 21

17 Apres greffe : renouvellement répertoire immunologique Muraro, Douek 26 immunophenotyping, TREC (Thymic output), CDR3 spectratyping / nucleotide sequencing Type I : remplacement du repertoire mature T/B memoire par cellules naïve, non-pathogèniques Type II :remise à zéro Réponse Immune nb et / ou fonction des cellules regulatrices Naïve Memoire Senescentes T regulatory cells Foxp3 naive B cells after HSCT CD4 + CD25 high FoxP3 regulatory T cells CD8 + FoxP3 suppressive function Sclerose en Plaques Herman Blood 25 (mice) P Murao J. Exp. Med. 25 (n = 7) Sclerodermie Systemique Farge et al Arthritis Rhum 28 (n = 7) DID précoce Yanting W 28 Biochem. Biophys. Res. Commun. Polyarthrite Rheumatoide de Kleer I Blood 26 Lupus Erythemateux Systemique Alexander Blood 29, Zhang Blood 29 (n = 15)

18 A new Bone Marrow Transplantation Method for Stem cell Disorders PERFUSION METHOD + INTRA BONE IKEHARA S Ann N Y Acad Sci 29; 1173: 774 A new concept for AD disorders

19 Remerciements «Thérapie cellulaire et MAI» Revised guidelines EBMT hand book SFGM Agence Biomédecine RCP multidisciplinaire recommend autologous HSCT for dcssc ideally in the context of a multicenter clinical trial but it may be considered as treatment for selected patients with early diffuse SSc with a modified Rodnan skin score 15 plus major organ involvement in respiratory, cardiovascular, or renal systems. Comprehensive cardiopulmonary screening and pre-transplant evaluation of heart, lung, kidney and gastrointestinal function is critically important to exclude patients at high risk of TRM. E Gluckman, M Labopin, M Badoglio et EBMT Autoimmune Diseases WP Equipe St Louis UG4, INSERM U 796 CICBT AFS, AFL+, GFRS, DRCD AP-HP EULAR, EUROLUPUS, DIABETIC ASSOCIATION, ECTRIMS, ECCO

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