Retrospective study of rapamycin or rapalog 0·1% cream for facial angiofibromas in tuberous sclerosis complex: evaluation of treatment effectiveness and cost

2018 ◽  
Vol 179 (1) ◽  
pp. 208-209 ◽  
Author(s):  
S. Norrenberg ◽  
M. Masconi ◽  
M. Karamanou ◽  
P. Meylan ◽  
R. Golaz ◽  
...  
Epilepsia ◽  
2021 ◽  
Vol 62 (3) ◽  
pp. 785-794
Author(s):  
Jakob Stockinger ◽  
Adam Strzelczyk ◽  
Andrea Nemecek ◽  
Michal Cicanic ◽  
Frank Bösebeck ◽  
...  

2021 ◽  
Vol 85 (3) ◽  
pp. AB51
Author(s):  
Pablo Cobo Rodríguez ◽  
Pablo Boixeda De Miquel ◽  
Iván Checa Recio ◽  
Jaime Martínez Mariscal ◽  
Raquel Cabana Navia ◽  
...  

2021 ◽  
Author(s):  
luo cong ◽  
Yu-shi Zhang ◽  
Ming-Xin Zhang ◽  
Min-Feng Chen ◽  
Yuan Li ◽  
...  

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 or sirolimus. 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 There were high response rates in both two groups. With the prolonged medication time, the therapeutic efficacy of two agents became more significant. Everolimus had a more significant TSC-AML volume reduction after 6 and 12 months than sirolimus. More than 1/2 of everolimus patients had ≥ 50% reduction and approximately 80% of them had ≥ 30% reduction, higher than that of the sirolimus patients. Regarding safety, there was no significant difference in the incidence of AEs between the two groups. And the most common AE was oral mucositis. Conclusions Both everolimus and sirolimus are excellent therapeutic choices for TSC-AML. However, everolimus had a better therapeutic efficacy than sirolimus, especially in reducing TSC-AML volume. Therefore, everolimus is recommended as the first choice.


2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Cong Luo ◽  
Yu-Shi Zhang ◽  
Ming-Xin Zhang ◽  
Min-Feng Chen ◽  
Yuan Li ◽  
...  

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.


2017 ◽  
Vol 48 (S 01) ◽  
pp. S1-S45
Author(s):  
G. Wiegand ◽  
T. Polster ◽  
C. Hertzberg ◽  
A. Wiemer-Kruel ◽  
J. French ◽  
...  

2017 ◽  
Vol 48 (S 01) ◽  
pp. S1-S45
Author(s):  
T. Stapper ◽  
D. Valcheva ◽  
T. Höll ◽  
T. Rosenbaum

2006 ◽  
Vol 37 (03) ◽  
Author(s):  
C Krahn-Peper ◽  
IEB Tuxhorn ◽  
K Ahlbory ◽  
F Behne ◽  
H Pannek

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