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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.
Sorin, Boris; Fadlallah, Jehane; Garzaro, Margaux; Vigneron, Julien; Bertinchamp, Rémi; Boutboul, David; Oksenhendler, Eric; Fieschi, Claire; Malphettes, Marion; Galicier, Lionel.
Afiliación
  • Sorin B; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France. boris.sorin@institutimagine.org.
  • Fadlallah J; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Garzaro M; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Vigneron J; Department of Biostatistics and Medical Information, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Bertinchamp R; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Boutboul D; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Oksenhendler E; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Fieschi C; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Malphettes M; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
  • Galicier L; Department of Clinical Immunology, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Université de Paris, 1 Avenue Claude Vellefaux, 75010, Paris, France.
Ann Hematol ; 102(8): 2059-2068, 2023 Aug.
Article en En | MEDLINE | ID: mdl-37386347
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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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Trombocitopenia / Anemia Hemolítica Autoinmune Tipo de estudio: Observational_studies / Risk_factors_studies Límite: Adult / Humans Idioma: En Revista: Ann Hematol Asunto de la revista: HEMATOLOGIA Año: 2023 Tipo del documento: Article País de afiliación: Francia

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Trombocitopenia / Anemia Hemolítica Autoinmune Tipo de estudio: Observational_studies / Risk_factors_studies Límite: Adult / Humans Idioma: En Revista: Ann Hematol Asunto de la revista: HEMATOLOGIA Año: 2023 Tipo del documento: Article País de afiliación: Francia