Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China
Abstract Purpose To evaluate the efficacy and safety of everolimus and sirolimus in patients with tuberous sclerosis complex-associated angiomyolipomas (TSC-AML). Materials and methods We performed a multi-institutional retrospective study of TSC-AML patients treated with oral everolimus 10 mg or si...
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| Format: | Article |
| Language: | English |
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BMC
2021-07-01
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| Series: | Orphanet Journal of Rare Diseases |
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| Online Access: | https://doi.org/10.1186/s13023-021-01932-z |
| _version_ | 1818724121182208000 |
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| author | Cong Luo Yu-Shi Zhang Ming-Xin Zhang Min-Feng Chen Yuan Li Lin Qi Han-Zhong Li Xiong-Bin Zu Yi Cai |
| author_facet | Cong Luo Yu-Shi Zhang Ming-Xin Zhang Min-Feng Chen Yuan Li Lin Qi Han-Zhong Li Xiong-Bin Zu Yi Cai |
| author_sort | Cong Luo |
| collection | DOAJ |
| description | Abstract Purpose To evaluate the efficacy and safety of everolimus and sirolimus in patients with tuberous sclerosis complex-associated angiomyolipomas (TSC-AML). Materials and methods We performed a multi-institutional retrospective study of TSC-AML patients treated with oral everolimus 10 mg or sirolimus 2 mg per day for at least 3 months. Angiomyolipoma volume was estimated using orthogonal measurements by MRI or CT. Adverse events (AEs) were assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events. All analyses were performed using SPSS 19.0 software. Results Response rates were high in both groups. With the prolonged medication durations, the therapeutic efficacy of both agents became more significant. The TSC-AML volume reduction after 6 and 12 months was more pronounced in patients with everolimus than those with sirolimus. More than half of the patients treated with everolimus had ≥ 50% reduction, and approximately 80% of them had ≥ 30% reduction, which was higher than that in patients treated with sirolimus. Regarding safety, there was no significant difference in the incidence of AEs between the two groups. Conclusions Both everolimus and sirolimus are excellent therapeutic options for TSC-AML. However, everolimus has a better therapeutic efficacy than sirolimus, particularly in reducing TSC-AML volume. Everolimus is therefore recommended as the first choice of therapy for TSC-AML. |
| first_indexed | 2024-12-17T21:21:22Z |
| format | Article |
| id | doaj.art-1917415098e34c62b6f7ec0b2ab93b14 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-12-17T21:21:22Z |
| publishDate | 2021-07-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-1917415098e34c62b6f7ec0b2ab93b142022-12-21T21:32:12ZengBMCOrphanet Journal of Rare Diseases1750-11722021-07-011611810.1186/s13023-021-01932-zEverolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in ChinaCong Luo0Yu-Shi Zhang1Ming-Xin Zhang2Min-Feng Chen3Yuan Li4Lin Qi5Han-Zhong Li6Xiong-Bin Zu7Yi Cai8Department of Urology, Xiangya Hospital, Central South UniversityDepartment of Urology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeDepartment of Urology, The Affiliated Hospital of Qingdao UniversityDepartment of Urology, Xiangya Hospital, Central South UniversityDepartment of Urology, Xiangya Hospital, Central South UniversityDepartment of Urology, Xiangya Hospital, Central South UniversityDepartment of Urology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeDepartment of Urology, Xiangya Hospital, Central South UniversityDepartment of Urology, Xiangya Hospital, Central South UniversityAbstract Purpose To evaluate the efficacy and safety of everolimus and sirolimus in patients with tuberous sclerosis complex-associated angiomyolipomas (TSC-AML). Materials and methods We performed a multi-institutional retrospective study of TSC-AML patients treated with oral everolimus 10 mg or sirolimus 2 mg per day for at least 3 months. Angiomyolipoma volume was estimated using orthogonal measurements by MRI or CT. Adverse events (AEs) were assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events. All analyses were performed using SPSS 19.0 software. Results Response rates were high in both groups. With the prolonged medication durations, the therapeutic efficacy of both agents became more significant. The TSC-AML volume reduction after 6 and 12 months was more pronounced in patients with everolimus than those with sirolimus. More than half of the patients treated with everolimus had ≥ 50% reduction, and approximately 80% of them had ≥ 30% reduction, which was higher than that in patients treated with sirolimus. Regarding safety, there was no significant difference in the incidence of AEs between the two groups. Conclusions Both everolimus and sirolimus are excellent therapeutic options for TSC-AML. However, everolimus has a better therapeutic efficacy than sirolimus, particularly in reducing TSC-AML volume. Everolimus is therefore recommended as the first choice of therapy for TSC-AML.https://doi.org/10.1186/s13023-021-01932-zTuberous sclerosis complexEverolimusSirolimusTreatment outcomeAdverse events |
| spellingShingle | Cong Luo Yu-Shi Zhang Ming-Xin Zhang Min-Feng Chen Yuan Li Lin Qi Han-Zhong Li Xiong-Bin Zu Yi Cai Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China Orphanet Journal of Rare Diseases Tuberous sclerosis complex Everolimus Sirolimus Treatment outcome Adverse events |
| title | Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China |
| title_full | Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China |
| title_fullStr | Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China |
| title_full_unstemmed | Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China |
| title_short | Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China |
| title_sort | everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex a multi institutional retrospective study in china |
| topic | Tuberous sclerosis complex Everolimus Sirolimus Treatment outcome Adverse events |
| url | https://doi.org/10.1186/s13023-021-01932-z |
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