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Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world

To investigate the effectiveness and safety of sirolimus in childhood-onset systemic lupus erythematosus in a real world. This is a retrospective real world clinical study. All childhood-onset systemic lupus erythematosus patients treated with sirolimus in Children’s Hospital of Hebei Province China...

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Autores principales: Zhang, Lei, Cui, Jie-Yuan, Zhang, Lin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9678567/
https://www.ncbi.nlm.nih.gov/pubmed/36401486
http://dx.doi.org/10.1097/MD.0000000000031551
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author Zhang, Lei
Cui, Jie-Yuan
Zhang, Lin
author_facet Zhang, Lei
Cui, Jie-Yuan
Zhang, Lin
author_sort Zhang, Lei
collection PubMed
description To investigate the effectiveness and safety of sirolimus in childhood-onset systemic lupus erythematosus in a real world. This is a retrospective real world clinical study. All childhood-onset systemic lupus erythematosus patients treated with sirolimus in Children’s Hospital of Hebei Province China were analyzed. They were treated with sirolimus and followed up regularly. The patients had systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score, levels of antidouble-stranded DNA antibody, complement components C3 and C4, 24-hour proteinuria and corticosteroid reduction were recorded at baseline and at 6, 12, and 18 months. Adverse events were also collected. Thirty-two patients were enrolled in the study. SLEDAI-2K were improved on all time-points (P < .05). Complement levels increased and the levels of antidouble-stranded DNA antibody decreased during treatment. The mean dose of prednisone tapered and achieved significant reduction after 12 months therapy (15.4 ± 5.8 mg/d to 4.8 ± 2.1 mg/d; P < .05). Sirolimus was well tolerated and only 5 patients (15.6%) experienced adverse events, all of which were classified as infections (2 bacterial infection and 3 viral infections). No deaths, severe infusion reactions, or hypersensitivity reactions were found. Sirolimus use was associated with a decrease in disease activity and ability to tolerate tapering of oral glucocorticoid dose with a favorable risk–benefit profile.
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spelling pubmed-96785672022-11-22 Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world Zhang, Lei Cui, Jie-Yuan Zhang, Lin Medicine (Baltimore) 3600 To investigate the effectiveness and safety of sirolimus in childhood-onset systemic lupus erythematosus in a real world. This is a retrospective real world clinical study. All childhood-onset systemic lupus erythematosus patients treated with sirolimus in Children’s Hospital of Hebei Province China were analyzed. They were treated with sirolimus and followed up regularly. The patients had systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score, levels of antidouble-stranded DNA antibody, complement components C3 and C4, 24-hour proteinuria and corticosteroid reduction were recorded at baseline and at 6, 12, and 18 months. Adverse events were also collected. Thirty-two patients were enrolled in the study. SLEDAI-2K were improved on all time-points (P < .05). Complement levels increased and the levels of antidouble-stranded DNA antibody decreased during treatment. The mean dose of prednisone tapered and achieved significant reduction after 12 months therapy (15.4 ± 5.8 mg/d to 4.8 ± 2.1 mg/d; P < .05). Sirolimus was well tolerated and only 5 patients (15.6%) experienced adverse events, all of which were classified as infections (2 bacterial infection and 3 viral infections). No deaths, severe infusion reactions, or hypersensitivity reactions were found. Sirolimus use was associated with a decrease in disease activity and ability to tolerate tapering of oral glucocorticoid dose with a favorable risk–benefit profile. Lippincott Williams & Wilkins 2022-11-18 /pmc/articles/PMC9678567/ /pubmed/36401486 http://dx.doi.org/10.1097/MD.0000000000031551 Text en Copyright © 2022 the Author(s). Published by Wolters Kluwer Health, Inc. https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the Creative Commons Attribution License 4.0 (CCBY) (https://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle 3600
Zhang, Lei
Cui, Jie-Yuan
Zhang, Lin
Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world
title Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world
title_full Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world
title_fullStr Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world
title_full_unstemmed Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world
title_short Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world
title_sort clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world
topic 3600
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9678567/
https://www.ncbi.nlm.nih.gov/pubmed/36401486
http://dx.doi.org/10.1097/MD.0000000000031551
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