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Efficacy of eculizumab in transplantation-associated thrombotic microangiopathy: results of the French nationwide study on behalf of the SFGM-TC and the CNR-MAT

Bone Marrow Transplantation (2024)Cite this article

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Transplant-associated thrombotic microangiopathy (TA-TMA) is a rare [1] but severe complication of hematopoietic stem cell transplantation (HSCT), with a letality of 90% without specific treatment. Several triggering factors have been identified (HLA-mismatch, conditioning regimens, immunosuppressive agents, GVHD or viral infections) [2]; recently, the role of uncontrolled complement alternative pathway (CAP) activation after endothelial injury also raised interest. Elevated concentration of serum complement membrane attack complex C5b9 (sC5b9) is common in TA-TMA patients, and correlates with severity [3].

Subsequently, eculizumab, an anti-C5 monoclonal antibody neutralizing the activation of the terminal pathway of complement, emerged as an alternative treatment of TA-TMA [4]. In 2013, our group reported an hematological response of 50% and overall survival (OS) of 33% in thirteen patients [5]. In 2021, a metaanalysis on 119 patients [6] found a pooled OS of 52%.

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Author notes

  1. These authors contributed equally: Flore Sicre de Fontbrune, Régis Peffault de Latour, Paul Coppo.

Authors and Affiliations

  1. Hematology Department, Saint-Antoine Hospital, APHP, Paris, France

    Marion Peyre & Paul Coppo

  2. Hematology Transplant Unit – French Reference Center for Aplastic Anemia, Saint Louis Hospital, APHP, Paris, France

    Flore Sicre de Fontbrune & Régis Peffault de Latour

  3. Hematology Department, Besançon University Hospital, Besançon, France

    Ana Berceanu & Lise Benjemia

  4. Pediatric Hematology-Immunology and Rheumatology Unit, Necker Hospital, APHP, Paris, France

    Martin Castelle

  5. Hematology Department, Nancy University Hospital, Vandoeuvre-lès-Nancy, France

    Maud D’Aveni

  6. Hematology Department, Necker Hospital, APHP, Paris, France

    Ambroise Marçais

  7. Hematology Department, Grenoble University Hospital, Grenoble, France

    Eleonore Kaphan & Claude-Eric Bulabois

  8. Hematology Department, Montpellier University Hospital, Montpellier, France

    Anne Sirvent

  9. Nice University Hospital, Nice, France

    Pierre-Simon Rohrlich

  10. Hematology Department, Lille University Hospital, Lille, France

    Valerie Coiteux

  11. Hematology Department, Caen University Hospital, Caen, France

    Sylvain Chantepie

  12. Hematology Department, Pitié-Salpêtrière Hospital, APHP, Paris, France

    Stéphanie Nguyen-Quoc

  13. French Reference Center for Thrombotic Microangiopathies, Paris, France

    Paul Coppo

  14. INSERM UMRS1138, Centre de Recherche des Cordeliers, Paris, France

    Paul Coppo

Authors
  1. Marion Peyre
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  2. Flore Sicre de Fontbrune
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  3. Ana Berceanu
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  5. Martin Castelle
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  6. Maud D’Aveni
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  7. Ambroise Marçais
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  8. Eleonore Kaphan
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  9. Claude-Eric Bulabois
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  10. Anne Sirvent
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  11. Pierre-Simon Rohrlich
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  13. Sylvain Chantepie
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  14. Stéphanie Nguyen-Quoc
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  15. Régis Peffault de Latour
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  16. Paul Coppo
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Contributions

Conceptualization and Writing – Original draft preparation: MP, FSDF, and PC; Data curation: MP, FSDF, AB, LB, MC, MD, AM, EK, C-EB, AS, P-SR, VC, SC, SN-Q, RPL, and PC; Formal analysis: MP; Methodology and Supervision: FSDF and PC; Writing – Review & editing: MP, FS, AB, LB, MC, MD, AM, EK, C-EB, AS, P-SR, VC, SC, SN-Q, RPL, and PC.

Corresponding authors

Correspondence to Flore Sicre de Fontbrune or Paul Coppo.

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Competing interests

PC received research grants and is a member of advisory board of Sanofi, Alexion, Takeda, Janssen. FSDF and RPL received honoraria and research support (to Saint-Louis Hospital, Paris) from Alexion, AstraZeneca Rare Disease, Samsung and Sobi.

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Peyre, M., Sicre de Fontbrune, F., Berceanu, A. et al. Efficacy of eculizumab in transplantation-associated thrombotic microangiopathy: results of the French nationwide study on behalf of the SFGM-TC and the CNR-MAT. Bone Marrow Transplant (2024). https://doi.org/10.1038/s41409-024-02279-2

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  • DOI: https://doi.org/10.1038/s41409-024-02279-2

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