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Autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease adult patients in France: analysis of the long-term outcome from the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)

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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.

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Acknowledgments

The authors would like to thank all physicians and professionals who participated in this study:

Mathieu Allez (Paris Saint Louis)

Pauline Belenotti (Marseille)

Denis Caillot (Dijon)

Frederick Christy (Marseille)

Catherine Cordonnier (Créteil)

Audrey Cras (Paris Saint Louis)

Eric Deconinck (Besancon)

Elisabeth Diot (Tours)

Guillaume Drugmanne (Brest)

Nathalie Hugon (Poitiers)

Pauline Lansiaux (Paris Saint Louis)

Thierry Le Gall (Brest)

Xavier Mariette (KB)

Mauricette Michalet (Lyon)

Marc Michel (Créteil)

Luc Mouthon (Cochin)

Vivane Queyrel (Nice)

Danielle Ragonneau (Dijon)

Myriam Renault (Lyon)

Pascal Roblot (Poitiers)

Florence Sarrot-Reynauld (Grenoble)

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Authors and Affiliations

  1. Département de Médecine Interne et Immunologie Clinique, APHP, Hôpital La Pitié Salpêtrière, 47-83 boulevard de l’hôpital, 75013, Paris, France

    Perrine Guillaume-Jugnot

  2. Université Pierre et Marie Curie, Paris, France

    Perrine Guillaume-Jugnot & Stéphanie Nguyen

  3. EBMT Paris study office, Saint Antoine Hospital, 184, rue du Faubourg Saint Antoine, 75012, Paris, France

    Manuela Badoglio

  4. Université Pierre et Marie Curie, INSERM UMR 938, Paris, France

    Manuela Badoglio & Myriam Labopin

  5. EBMT Paris study office, Department of Haematology, Saint Antoine Hospital, 184, rue du Faubourg Saint Antoine, 75012, Paris, France

    Myriam Labopin

  6. Service de Médecine Interne et Hématologie, CHRU Lille, Hôpital Claude Huriez, rue Michel Polonovski, 59037, Lille, France

    Louis Terriou

  7. Service des Maladies du sang, CHRU Lille, Hôpital Claude Huriez, rue Michel Polonovski, 59037, Lille, France

    Ibrahim Yakoub-Agha

  8. Service d’Immunologie Clinique, VIH et Médecine Interne, CHRU Strasbourg, Nouvel Hôpital Civil, 1 place de l’hôpital, 67091, Strasbourg Cedex, France

    Thierry Martin

  9. Service d’Hématologie, Hôpital de Hautepierre, Hôpitaux Universitaires de Strasbourg (HUS), 1 avenue Molière, 67000, Strasbourg, France

    Bruno Lioure

  10. Service d’Hématologie, APHP, Hôpital Saint Antoine, 184, rue du Faubourg Saint Antoine, 75012, Paris, France

    Zora Marjanovic

  11. Institut Paoli-Calmettes, CLCC Institut Paoli-Calmettes, 232 boulevard de Sainte-Marguerite, 13273, Marseille Cedex 9, France

    Didier Blaise

  12. Service Hématologie Clinique, APHP, Hôpital La Pitié Salpêtrière, 47-83 boulevard de l’hôpital, 75013, Paris, France

    Stéphanie Nguyen

  13. Service de Médecine Interne, CHU Toulouse, Hôpital Purpan, 1 place du Docteur Baylac, 31059, Toulouse, France

    Gregory Pugnet

  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

    Anne Huynh

  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

    Christophe Deligny

  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

    Christophe Seinturier

  17. Service d’Hématologie, Pôle cancer et Maladies du Sang, CHU Grenoble Alpes CS10217, 38043, Grenoble Cedex 9, France

    Frédéric Garban

  18. Département de Médecine Interne, APHM, Hôpital de la Timone, 264 rue Saint Pierre, 13385, Marseille Cedex 05, France

    Laure Swiader

  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

    Jacques-Olivier Bay

  20. Service d’Hématologie, APHP, Hôpital d’Avicenne, 125 rue de Stalingrad, 93000, Bobigny, France

    Thorsten Braun

  21. Service d’Hématologie et Greffe de Moelle, APHP, Hôpital St-Louis, 1 avenue Claude Vellefaux, 75010, Paris, France

    Régis Peffault de Latour

  22. Université Denis Diderot, Paris, France

    Régis Peffault de Latour & Dominique Farge

  23. Service d’Hématologie, CHRU Nancy, Hôpital Brabois, Vandoeuvre les Nancy, France

    Marie Thérèse Rubio

  24. IMoPA, CNRS UMR 7365, Université de Lorraine, Vandoeuvre les Nancy, France

    Marie Thérèse Rubio

  25. 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

  26. Centre de Référence des Maladies auto-immunes systémiques Rares d’Ile-de-France (site constitutif), Filière FAI2R, SFGM-TC, Lille, France

    Dominique Farge

  27. Département de Médecine, Université McGill, Montreal, QC, Canada

    Dominique Farge

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  1. Perrine Guillaume-Jugnot
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  2. Manuela Badoglio
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  3. Myriam Labopin
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  6. Thierry Martin
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  9. Didier Blaise
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  10. Stéphanie Nguyen
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  11. Gregory Pugnet
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  12. Anne Huynh
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  13. Christophe Deligny
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  14. Christophe Seinturier
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  17. Jacques-Olivier Bay
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  18. Thorsten Braun
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  19. Régis Peffault de Latour
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  20. Marie Thérèse Rubio
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  21. Dominique Farge
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Corresponding author

Correspondence to Dominique Farge.

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This study was performed in accordance with the ethical guidelines of the 1975 Declaration of Helsinki. Informed consent was obtained from all patients before AHSCT.

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Guillaume-Jugnot, P., Badoglio, M., Labopin, M. et al. Autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease adult patients in France: analysis of the long-term outcome from the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC). Clin Rheumatol 38, 1501–1511 (2019). https://doi.org/10.1007/s10067-019-04435-2

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