Perrine Guillaume-Jugnot1,2, Manuela Badoglio3,4, Myriam Labopin4,5, Louis Terriou6, Ibrahim Yakoub-Agha7, Thierry Martin8, Bruno Lioure9, Zora Marjanovic10, Didier Blaise11, Stéphanie Nguyen2,12, Gregory Pugnet13, Anne Huynh14, Christophe Deligny15, Christophe Seinturier16, Frédéric Garban17, Laure Swiader18, Jacques-Olivier Bay19, Thorsten Braun20, Régis Peffault de Latour21,22, Marie Thérèse Rubio23,24, Dominique Farge25,26,27,28. 1. Département de Médecine Interne et Immunologie Clinique, APHP, Hôpital La Pitié Salpêtrière, 47-83 boulevard de l'hôpital, 75013, Paris, France. 2. Université Pierre et Marie Curie, Paris, France. 3. EBMT Paris study office, Saint Antoine Hospital, 184, rue du Faubourg Saint Antoine, 75012, Paris, France. 4. Université Pierre et Marie Curie, INSERM UMR 938, Paris, France. 5. EBMT Paris study office, Department of Haematology, Saint Antoine Hospital, 184, rue du Faubourg Saint Antoine, 75012, Paris, France. 6. Service de Médecine Interne et Hématologie, CHRU Lille, Hôpital Claude Huriez, rue Michel Polonovski, 59037, Lille, France. 7. Service des Maladies du sang, CHRU Lille, Hôpital Claude Huriez, rue Michel Polonovski, 59037, Lille, France. 8. Service d'Immunologie Clinique, VIH et Médecine Interne, CHRU Strasbourg, Nouvel Hôpital Civil, 1 place de l'hôpital, 67091, Strasbourg Cedex, France. 9. Service d'Hématologie, Hôpital de Hautepierre, Hôpitaux Universitaires de Strasbourg (HUS), 1 avenue Molière, 67000, Strasbourg, France. 10. Service d'Hématologie, APHP, Hôpital Saint Antoine, 184, rue du Faubourg Saint Antoine, 75012, Paris, France. 11. Institut Paoli-Calmettes, CLCC Institut Paoli-Calmettes, 232 boulevard de Sainte-Marguerite, 13273, Marseille Cedex 9, France. 12. Service Hématologie Clinique, APHP, Hôpital La Pitié Salpêtrière, 47-83 boulevard de l'hôpital, 75013, Paris, France. 13. Service de Médecine Interne, CHU Toulouse, Hôpital Purpan, 1 place du Docteur Baylac, 31059, Toulouse, France. 14. Service d'hématologie, CHU / IUCT Oncopole de Toulouse, Institut Universitaire du Cancer de Toulouse, Oncopole, 1 avenue Irène Joliot-Curie, 31059, Toulouse Cedex 9, France. 15. Service de Rhumatologie et de Médecine Interne, CHU Martinique, Hôpital P. Zobda-Quitman, route de Chateauboeuf, Quartier Le Meynard, CS 90632, 97261, Fort de France, Martinique, France. 16. Service de Médecine Vasculaire, Pôle pluridisciplinaire de Médecine, CHU Grenoble Alpes, Site Nord, Hôpital Couple Enfant, boulevard de la Chantourne, CS10217, 38043, Grenoble Cedex 9, France. 17. Service d'Hématologie, Pôle cancer et Maladies du Sang, CHU Grenoble Alpes CS10217, 38043, Grenoble Cedex 9, France. 18. Département de Médecine Interne, APHM, Hôpital de la Timone, 264 rue Saint Pierre, 13385, Marseille Cedex 05, France. 19. Service de Thérapie Cellulaire et Hématologie Clinique, CHU de Clermont Ferrand, Hôpital d'Estaing, 1 place Lucie Aubrac, 63003, Clermont Ferrand Cedex 1, France. 20. Service d'Hématologie, APHP, Hôpital d'Avicenne, 125 rue de Stalingrad, 93000, Bobigny, France. 21. Service d'Hématologie et Greffe de Moelle, APHP, Hôpital St-Louis, 1 avenue Claude Vellefaux, 75010, Paris, France. 22. Université Denis Diderot, Paris, France. 23. Service d'Hématologie, CHRU Nancy, Hôpital Brabois, Vandoeuvre les Nancy, France. 24. IMoPA, CNRS UMR 7365, Université de Lorraine, Vandoeuvre les Nancy, France. 25. Université Denis Diderot, Paris, France. dominique.farge-bancel@aphp.fr. 26. Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Institut Universitaire d'Hématologie EA3518, APHP, Hôpital St-Louis, 1 avenue Claude Vellefaux, 75010, Paris, France. dominique.farge-bancel@aphp.fr. 27. Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France (site constitutif), Filière FAI2R, SFGM-TC, Lille, France. dominique.farge-bancel@aphp.fr. 28. Département de Médecine, Université McGill, Montreal, QC, Canada. dominique.farge-bancel@aphp.fr.
Abstract
INTRODUCTION: The use of autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease (AD) patients has increased progressively worldwide. We retrospectively analysed the long-term outcome of AHSCT for AD reported to the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC). METHOD: All French AD patients (≥ 18 years at transplant) with a first AHSCT between 1997 and 2013 were included. Primary data were derived from the European Society for Blood and Marrow Transplantation (EBMT) registry, and additional data were obtained through a specific questionnaire designed for the study. Primary end-point was overall survival (OS). Secondary end points were progression-free survival (PFS) and non-relapse mortality (NRM). RESULTS: Ninety-four AD patients were included, of whom 71% suffered from rheumatologic diseases (n = 67, including 56 systemic sclerosis (SSc)), 16% from neurological disease (n = 15, including 14 multiple sclerosis (MS)) and 13% from various other AD (n = 12). After a median (interquartile range, IQR) follow-up of 83 months (38-130), OS at 5 and 10 years were 77% (95% CI 68.5-86.2) and 64% (95% CI 51.7-76.3), and for PFS 51% (95% CI 40.4-61.6) and 44% (95% CI 32.8-55.3), respectively. Overall, NRM was 8.7% (95% CI 4.0-15.5) at day 100, 9.8% (95% CI 4.8-16.9) at 5 years and 13.6% (95% CI 6.9-22.5) at 10 years. CONCLUSIONS: This first SFGM-TC retrospective report shows long-term benefit of AHSCT in AD patients with acceptable toxicity.
INTRODUCTION: The use of autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease (AD) patients has increased progressively worldwide. We retrospectively analysed the long-term outcome of AHSCT for AD reported to the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC). METHOD: All French ADpatients (≥ 18 years at transplant) with a first AHSCT between 1997 and 2013 were included. Primary data were derived from the European Society for Blood and Marrow Transplantation (EBMT) registry, and additional data were obtained through a specific questionnaire designed for the study. Primary end-point was overall survival (OS). Secondary end points were progression-free survival (PFS) and non-relapse mortality (NRM). RESULTS: Ninety-four ADpatients were included, of whom 71% suffered from rheumatologic diseases (n = 67, including 56 systemic sclerosis (SSc)), 16% from neurological disease (n = 15, including 14 multiple sclerosis (MS)) and 13% from various other AD (n = 12). After a median (interquartile range, IQR) follow-up of 83 months (38-130), OS at 5 and 10 years were 77% (95% CI 68.5-86.2) and 64% (95% CI 51.7-76.3), and for PFS 51% (95% CI 40.4-61.6) and 44% (95% CI 32.8-55.3), respectively. Overall, NRM was 8.7% (95% CI 4.0-15.5) at day 100, 9.8% (95% CI 4.8-16.9) at 5 years and 13.6% (95% CI 6.9-22.5) at 10 years. CONCLUSIONS: This first SFGM-TC retrospective report shows long-term benefit of AHSCT in ADpatients with acceptable toxicity.
Authors: Keith M Sullivan; Ellen A Goldmuntz; Lynette Keyes-Elstein; Peter A McSweeney; Ashley Pinckney; Beverly Welch; Maureen D Mayes; Richard A Nash; Leslie J Crofford; Barry Eggleston; Sharon Castina; Linda M Griffith; Julia S Goldstein; Dennis Wallace; Oana Craciunescu; Dinesh Khanna; Rodney J Folz; Jonathan Goldin; E William St Clair; James R Seibold; Kristine Phillips; Shin Mineishi; Robert W Simms; Karen Ballen; Mark H Wener; George E Georges; Shelly Heimfeld; Chitra Hosing; Stephen Forman; Suzanne Kafaja; Richard M Silver; Leroy Griffing; Jan Storek; Sharon LeClercq; Richard Brasington; Mary E Csuka; Christopher Bredeson; Carolyn Keever-Taylor; Robyn T Domsic; M Bashar Kahaleh; Thomas Medsger; Daniel E Furst Journal: N Engl J Med Date: 2018-01-04 Impact factor: 91.245
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Authors: Giovanni L Mancardi; Maria P Sormani; Francesca Gualandi; Albert Saiz; Eric Carreras; Elisa Merelli; Amedea Donelli; Alessandra Lugaresi; Paolo Di Bartolomeo; Maria R Rottoli; Alessandro Rambaldi; Maria P Amato; Luca Massacesi; Massimo Di Gioia; Luisa Vuolo; Daniela Currò; Luca Roccatagliata; Massimo Filippi; Umberto Aguglia; Pasquale Iacopino; Dominique Farge; Riccardo Saccardi Journal: Neurology Date: 2015-02-11 Impact factor: 9.910
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