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Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients
Mucous membrane pemphigoid (MMP) is a heterogeneous group of rare, chronic, subepithelial autoimmune blistering diseases (AIBDs) with predominant involvement of mucous membranes that can be sight-threatening and life-threatening. Rituximab (RTX) has demonstrated its efficacy in severe MMP refractory...
Autores principales: | , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Frontiers Media S.A.
2022
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9281543/ https://www.ncbi.nlm.nih.gov/pubmed/35844526 http://dx.doi.org/10.3389/fimmu.2022.915205 |
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author | Bohelay, Gérôme Alexandre, Marina Le Roux-Villet, Christelle Sitbon, Ishaï Doan, Serge Soued, Isaac Shourick, Jason Rousset, Laurie Mellottee, Benoît Heller, Michel Lièvre, Nicole Zumelzu, Coralie Morin, Florence Grootenboer-Mignot, Sabine Gabison, Eric Caux, Frédéric Prost-Squarcioni, Catherine Musette, Philippe |
author_facet | Bohelay, Gérôme Alexandre, Marina Le Roux-Villet, Christelle Sitbon, Ishaï Doan, Serge Soued, Isaac Shourick, Jason Rousset, Laurie Mellottee, Benoît Heller, Michel Lièvre, Nicole Zumelzu, Coralie Morin, Florence Grootenboer-Mignot, Sabine Gabison, Eric Caux, Frédéric Prost-Squarcioni, Catherine Musette, Philippe |
author_sort | Bohelay, Gérôme |
collection | PubMed |
description | Mucous membrane pemphigoid (MMP) is a heterogeneous group of rare, chronic, subepithelial autoimmune blistering diseases (AIBDs) with predominant involvement of mucous membranes that can be sight-threatening and life-threatening. Rituximab (RTX) has demonstrated its efficacy in severe MMP refractory to conventional immunosuppressants in small series that differed in RTX scheme, concomitant therapies, and outcome definitions. In a meta-analysis involving 112 patients with MMP treated with RTX, complete remission (CR) was reported in 70.5% of cases. Herein, we report the largest retrospective monocentric study on RTX efficacy in a series of 109 severe and/or refractory patients with MMP treated with RTX with a median follow-up period of 51.4 months. RTX was administered in association with immunomodulatory drugs (dapsone, salazopyrine) without any other systemic immunosuppressant in 104 patients. The RTX schedule comprised two injections (1 g, 2 weeks apart), repeated every 6 months until CR or failure, with a unique consolidation injection (1 g) after CR. The median survival times to disease control and to CR were 7.1 months and 12.2 months, respectively. The median number of RTX cycles required to achieve CR in 85.3% of patients was two. The larynx was the lesional site that took the longest time to achieve disease control. One year after RTX weaning, CR off RTX was obtained in 68.7% of cases. CR off RTX with only minimum doses of immunomodulatory drugs was achieved in 22.0% of patients. Further, 10.1% of patients were partial responders and 4.6% were non-responders to RTX. Relapse occurred in 38.7% of cases, of whom 91.7% had achieved CR again at the last follow-up. In MMP, CR was achieved in a longer time and after more rituximab cycles than in pemphigus, especially for patients with MMP with anti-type VII collagen reactivity. RTX with concomitant immunomodulatory drugs was not responsible for an unusual proportion of adverse events. This large study confirms that RTX is an effective therapy in patients with severe and/or refractory MMP, corroborating previous findings regarding the effects of RTX on AIBDs such as pemphigus. |
format | Online Article Text |
id | pubmed-9281543 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-92815432022-07-15 Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients Bohelay, Gérôme Alexandre, Marina Le Roux-Villet, Christelle Sitbon, Ishaï Doan, Serge Soued, Isaac Shourick, Jason Rousset, Laurie Mellottee, Benoît Heller, Michel Lièvre, Nicole Zumelzu, Coralie Morin, Florence Grootenboer-Mignot, Sabine Gabison, Eric Caux, Frédéric Prost-Squarcioni, Catherine Musette, Philippe Front Immunol Immunology Mucous membrane pemphigoid (MMP) is a heterogeneous group of rare, chronic, subepithelial autoimmune blistering diseases (AIBDs) with predominant involvement of mucous membranes that can be sight-threatening and life-threatening. Rituximab (RTX) has demonstrated its efficacy in severe MMP refractory to conventional immunosuppressants in small series that differed in RTX scheme, concomitant therapies, and outcome definitions. In a meta-analysis involving 112 patients with MMP treated with RTX, complete remission (CR) was reported in 70.5% of cases. Herein, we report the largest retrospective monocentric study on RTX efficacy in a series of 109 severe and/or refractory patients with MMP treated with RTX with a median follow-up period of 51.4 months. RTX was administered in association with immunomodulatory drugs (dapsone, salazopyrine) without any other systemic immunosuppressant in 104 patients. The RTX schedule comprised two injections (1 g, 2 weeks apart), repeated every 6 months until CR or failure, with a unique consolidation injection (1 g) after CR. The median survival times to disease control and to CR were 7.1 months and 12.2 months, respectively. The median number of RTX cycles required to achieve CR in 85.3% of patients was two. The larynx was the lesional site that took the longest time to achieve disease control. One year after RTX weaning, CR off RTX was obtained in 68.7% of cases. CR off RTX with only minimum doses of immunomodulatory drugs was achieved in 22.0% of patients. Further, 10.1% of patients were partial responders and 4.6% were non-responders to RTX. Relapse occurred in 38.7% of cases, of whom 91.7% had achieved CR again at the last follow-up. In MMP, CR was achieved in a longer time and after more rituximab cycles than in pemphigus, especially for patients with MMP with anti-type VII collagen reactivity. RTX with concomitant immunomodulatory drugs was not responsible for an unusual proportion of adverse events. This large study confirms that RTX is an effective therapy in patients with severe and/or refractory MMP, corroborating previous findings regarding the effects of RTX on AIBDs such as pemphigus. Frontiers Media S.A. 2022-06-30 /pmc/articles/PMC9281543/ /pubmed/35844526 http://dx.doi.org/10.3389/fimmu.2022.915205 Text en Copyright © 2022 Bohelay, Alexandre, Le Roux-Villet, Sitbon, Doan, Soued, Shourick, Rousset, Mellottee, Heller, Lièvre, Zumelzu, Morin, Grootenboer-Mignot, Gabison, Caux, Prost-Squarcioni and Musette https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Immunology Bohelay, Gérôme Alexandre, Marina Le Roux-Villet, Christelle Sitbon, Ishaï Doan, Serge Soued, Isaac Shourick, Jason Rousset, Laurie Mellottee, Benoît Heller, Michel Lièvre, Nicole Zumelzu, Coralie Morin, Florence Grootenboer-Mignot, Sabine Gabison, Eric Caux, Frédéric Prost-Squarcioni, Catherine Musette, Philippe Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients |
title | Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients |
title_full | Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients |
title_fullStr | Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients |
title_full_unstemmed | Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients |
title_short | Rituximab Therapy for Mucous Membrane Pemphigoid: A Retrospective Monocentric Study With Long-Term Follow-Up in 109 Patients |
title_sort | rituximab therapy for mucous membrane pemphigoid: a retrospective monocentric study with long-term follow-up in 109 patients |
topic | Immunology |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9281543/ https://www.ncbi.nlm.nih.gov/pubmed/35844526 http://dx.doi.org/10.3389/fimmu.2022.915205 |
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