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Real-life use of mTOR inhibitor-based therapy in adults with autoimmune cytopenia highlights strong efficacy in relapsing/refractory multi-lineage autoimmune cytopenia

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Abstract

Data on mTOR inhibitors (mTORi) in autoimmune cytopenia (AIC), in adults are scarce. We retrospectively analysed 30 cases of refractory or relapsing AIC treated with an mTORi-based therapy. Eleven warm autoimmune hemolytic anaemia, 10 autoimmune thrombocytopenia, 6 acquired pure red cell aplasia, 3 autoimmune neutropenia were included. Twenty were multilineage AIC (67%) and 21 were secondary AIC (70%). mTORi were associated with other therapies in 23 AIC (77%). Twenty-two AIC (73%) responded to mTORi-based therapy: 5 reached a partial response (17%) and 17 a complete response (57%). Survival without unfavourable outcome (failure, requirement of a new therapy, or death) was longer in multilineage AIC compared to single-lineage AIC (p = 0.049) with a median event-free survival of 48 versus 12 months. Median event-free survival was 48 months in secondary AIC and 33 months in primary AIC (p = 0.79). mTORi were discontinued in 4 patients (15%) for safety reasons and in 3 patients for patient’s choice (12%). In conclusion, mTORi could be considered as an alternative or an add-on therapy in refractory or relapsing AIC in adult patients, especially in multilineage AIC.

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Data availability

The data that support the findings of this study are available from the corresponding author upon reasonable request.

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Authors and Affiliations

  1. Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France

    Boris Sorin, Jehane Fadlallah, Margaux Garzaro, Rémi Bertinchamp, David Boutboul, Eric Oksenhendler, Claire Fieschi, Marion Malphettes & Lionel Galicier

  2. Department of Biostatistics and Medical Information, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France

    Julien Vigneron

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  1. Boris Sorin
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Contributions

BS, MM and LG wrote the manuscript. BS, MG, RB, DB, JF, CF, EO, MM and LG contributed to patient recruitment and management. BS, JV, MM and LG extracted and analysed the data. BS performed statistical analyses. MM and LG supervised the project. All the authors reviewed and approved the final manuscript.

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Correspondence to Boris Sorin.

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In conformity with the principles of the Helsinki declaration and according to the French Jardé law, written consent of the patients is not required in France for retrospective and observational non-interventional study.

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The authors declare they have no competing interests to declare.

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Sorin, B., Fadlallah, J., Garzaro, M. et al. Real-life use of mTOR inhibitor-based therapy in adults with autoimmune cytopenia highlights strong efficacy in relapsing/refractory multi-lineage autoimmune cytopenia. Ann Hematol 102, 2059–2068 (2023). https://doi.org/10.1007/s00277-023-05340-0

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