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Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study

BACKGROUND: To evaluate the efficacy, safety and health economics of sequential everolimus in treating angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC). METHODS: In this prospective cohort study, patients met the inclusion criteria received standard or sequential treatment accor...

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Autores principales: Gu, Liangyou, Peng, Cheng, Zhang, Fan, Fang, Cunjin, Guo, Gang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8201725/
https://www.ncbi.nlm.nih.gov/pubmed/34127034
http://dx.doi.org/10.1186/s13023-021-01913-2
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author Gu, Liangyou
Peng, Cheng
Zhang, Fan
Fang, Cunjin
Guo, Gang
author_facet Gu, Liangyou
Peng, Cheng
Zhang, Fan
Fang, Cunjin
Guo, Gang
author_sort Gu, Liangyou
collection PubMed
description BACKGROUND: To evaluate the efficacy, safety and health economics of sequential everolimus in treating angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC). METHODS: In this prospective cohort study, patients met the inclusion criteria received standard or sequential treatment according to their willingness. All patients received an initial dose of everolimus (10 mg oral, once a day) for 3 months. The standard treatment group maintained 10 mg QD for 12 months, while the sequential treatment group reduced the dose to 5 mg QD from the 4th month. The efficacy, serum everolimus concentration and safety were evaluated at 1, 3, 6, 9 and 12 months after treatment. The primary efficacy endpoint was the proportion of patients with confirmed angiomyolipoma response of at least a 50% reduction in the total volume of target AML relative to baseline. RESULTS: Between June 1, 2016 and June 1, 2017, a total of 53 patients were included. Twenty-three patients received standard treatment, 30 patients received sequential treatment. At 1, 3, 6, 9 and 12 months after treatment, the proportion of patients whose total target tumor volume decreased by ≥ 50% from baseline was 39.1% versus 36.7%, 43.5% versus 56.7%, 47.8% versus 50%, 47.8% versus 60% and 47.8% versus 23.3% respectively (P > 0.05 for all). The overall response rate of skin lesions in the two groups was 40.4%, and the response rates of skin lesions at different times were similar for two groups (P > 0.05 for all). Major adverse effects (AEs) included mouth ulceration, hypertriglyceridemia, hypercholesterolemia, menstrual disorders. There was no significant difference between the two groups in the incidence of AEs at 3 months after treatment. The incidence of overall and grade 3/4 AEs at 12 months after treatment were significantly lower in the sequential treatment group. The average direct cost of the two groups in 12 months was $15,466 and $11,120, respectively. CONCLUSIONS: Compared to standard treatment, sequential treatment was equally effective, with a lower incidence of adverse events and a lower direct cost, suggesting that it may be an alternative treatment for AML associated with TSC.
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spelling pubmed-82017252021-06-16 Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study Gu, Liangyou Peng, Cheng Zhang, Fan Fang, Cunjin Guo, Gang Orphanet J Rare Dis Research BACKGROUND: To evaluate the efficacy, safety and health economics of sequential everolimus in treating angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC). METHODS: In this prospective cohort study, patients met the inclusion criteria received standard or sequential treatment according to their willingness. All patients received an initial dose of everolimus (10 mg oral, once a day) for 3 months. The standard treatment group maintained 10 mg QD for 12 months, while the sequential treatment group reduced the dose to 5 mg QD from the 4th month. The efficacy, serum everolimus concentration and safety were evaluated at 1, 3, 6, 9 and 12 months after treatment. The primary efficacy endpoint was the proportion of patients with confirmed angiomyolipoma response of at least a 50% reduction in the total volume of target AML relative to baseline. RESULTS: Between June 1, 2016 and June 1, 2017, a total of 53 patients were included. Twenty-three patients received standard treatment, 30 patients received sequential treatment. At 1, 3, 6, 9 and 12 months after treatment, the proportion of patients whose total target tumor volume decreased by ≥ 50% from baseline was 39.1% versus 36.7%, 43.5% versus 56.7%, 47.8% versus 50%, 47.8% versus 60% and 47.8% versus 23.3% respectively (P > 0.05 for all). The overall response rate of skin lesions in the two groups was 40.4%, and the response rates of skin lesions at different times were similar for two groups (P > 0.05 for all). Major adverse effects (AEs) included mouth ulceration, hypertriglyceridemia, hypercholesterolemia, menstrual disorders. There was no significant difference between the two groups in the incidence of AEs at 3 months after treatment. The incidence of overall and grade 3/4 AEs at 12 months after treatment were significantly lower in the sequential treatment group. The average direct cost of the two groups in 12 months was $15,466 and $11,120, respectively. CONCLUSIONS: Compared to standard treatment, sequential treatment was equally effective, with a lower incidence of adverse events and a lower direct cost, suggesting that it may be an alternative treatment for AML associated with TSC. BioMed Central 2021-06-14 /pmc/articles/PMC8201725/ /pubmed/34127034 http://dx.doi.org/10.1186/s13023-021-01913-2 Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Research
Gu, Liangyou
Peng, Cheng
Zhang, Fan
Fang, Cunjin
Guo, Gang
Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study
title Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study
title_full Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study
title_fullStr Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study
title_full_unstemmed Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study
title_short Sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study
title_sort sequential everolimus for angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study
topic Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8201725/
https://www.ncbi.nlm.nih.gov/pubmed/34127034
http://dx.doi.org/10.1186/s13023-021-01913-2
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