Efficacy, Retention and Tolerability of Everolimus in Patients with Tuberous Sclerosis Complex: A Survey-Based Study on Patients' Perspectives.
Adolescent
Adult
Aged
Child
Child, Preschool
Everolimus
/ adverse effects
Fatigue
/ chemically induced
Female
Germany
/ epidemiology
Humans
Immunosuppressive Agents
/ adverse effects
Infant
Male
Medication Adherence
Middle Aged
Patient Preference
Surveys and Questionnaires
Treatment Outcome
Tuberous Sclerosis
/ diagnosis
Young Adult
Journal
CNS drugs
ISSN: 1179-1934
Titre abrégé: CNS Drugs
Pays: New Zealand
ID NLM: 9431220
Informations de publication
Date de publication:
10 2021
10 2021
Historique:
accepted:
21
06
2021
pubmed:
19
7
2021
medline:
17
2
2022
entrez:
18
7
2021
Statut:
ppublish
Résumé
The approval of everolimus (EVE) for the treatment of angiomyolipoma (2013), subependymal giant cell astrocytoma (2013) and drug-refractory epilepsy (2017) in patients with tuberous sclerosis complex (TSC) represents the first disease-modifying treatment option available for this rare and complex genetic disorder. The objective of this study was to analyse the use, efficacy, tolerability and treatment retention of EVE in patients with TSC in Germany from the patient's perspective. A structured cross-age survey was conducted at 26 specialised TSC centres in Germany and by the German TSC patient advocacy group between February and July 2019, enrolling children, adolescents and adult patients with TSC. Of 365 participants, 36.7% (n = 134) reported the current or past intake of EVE, including 31.5% (n = 115) who were taking EVE at study entry. The mean EVE dosage was 6.1 ± 2.9 mg/m From the patients' perspective, EVE is an effective and relatively well-tolerated disease-modifying treatment option for children, adolescents and adults with TSC, associated with a high long-term retention rate that can be individually considered for each patient. Everolimus therapy should ideally be supervised by a centre experienced in the use of mechanistic target of rapamycin inhibitors, and adverse effects should be monitored on a regular basis.
Sections du résumé
BACKGROUND
The approval of everolimus (EVE) for the treatment of angiomyolipoma (2013), subependymal giant cell astrocytoma (2013) and drug-refractory epilepsy (2017) in patients with tuberous sclerosis complex (TSC) represents the first disease-modifying treatment option available for this rare and complex genetic disorder.
OBJECTIVE
The objective of this study was to analyse the use, efficacy, tolerability and treatment retention of EVE in patients with TSC in Germany from the patient's perspective.
METHODS
A structured cross-age survey was conducted at 26 specialised TSC centres in Germany and by the German TSC patient advocacy group between February and July 2019, enrolling children, adolescents and adult patients with TSC.
RESULTS
Of 365 participants, 36.7% (n = 134) reported the current or past intake of EVE, including 31.5% (n = 115) who were taking EVE at study entry. The mean EVE dosage was 6.1 ± 2.9 mg/m
CONCLUSIONS
From the patients' perspective, EVE is an effective and relatively well-tolerated disease-modifying treatment option for children, adolescents and adults with TSC, associated with a high long-term retention rate that can be individually considered for each patient. Everolimus therapy should ideally be supervised by a centre experienced in the use of mechanistic target of rapamycin inhibitors, and adverse effects should be monitored on a regular basis.
Identifiants
pubmed: 34275102
doi: 10.1007/s40263-021-00839-4
pii: 10.1007/s40263-021-00839-4
pmc: PMC8478774
doi:
Substances chimiques
Immunosuppressive Agents
0
Everolimus
9HW64Q8G6G
Banques de données
DRKS
['DRKS00016045']
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1107-1122Informations de copyright
© 2021. The Author(s).
Références
Urology. 2016 Sep;95:80-7
pubmed: 27132503
PLoS Med. 2007 Oct 16;4(10):e296
pubmed: 17941714
Eur J Paediatr Neurol. 2018 May;22(3):427-433
pubmed: 29475820
Epilepsy Behav. 2017 May;70(Pt A):245-252
pubmed: 28457992
J Epilepsy Res. 2018 Dec 31;8(2):74-80
pubmed: 30809500
Seizure. 2019 Jul;69:92-98
pubmed: 31004927
Cardiol Young. 2018 Jul;28(7):903-909
pubmed: 29759095
Lancet Oncol. 2017 Oct;18(10):1411-1422
pubmed: 28838862
J Eur Acad Dermatol Venereol. 2018 Oct;32(10):1796-1803
pubmed: 29569806
Orphanet J Rare Dis. 2018 Sep 21;13(1):168
pubmed: 30241551
Orphanet J Rare Dis. 2018 Feb 20;13(1):34
pubmed: 29458386
J Mark Access Health Policy. 2019 May 22;7(1):1618661
pubmed: 31156762
Epilepsy Behav. 2020 Oct;111:107182
pubmed: 32535369
CNS Drugs. 2021 Sep;35(9):935-963
pubmed: 34145528
Lancet. 2016 Oct 29;388(10056):2153-2163
pubmed: 27613521
Neurol Res Pract. 2021 Jun 28;3(1):35
pubmed: 34176514
Curr Med Res Opin. 2017 Jul;33(7):1277-1282
pubmed: 28358266
Pediatr Ann. 2017 Apr 1;46(4):e166-e171
pubmed: 28414398
Seizure. 2021 Oct;91:287-295
pubmed: 34265583
J Pediatr. 2016 May;172:151-155.e1
pubmed: 26858193
Paediatr Drugs. 2012 Feb 1;14(1):51-60
pubmed: 22136276
Eur J Paediatr Neurol. 2018 Nov;22(6):1066-1073
pubmed: 30005812
Ann Neurol. 2015 Dec;78(6):929-38
pubmed: 26381530
N Engl J Med. 2011 Apr 28;364(17):1595-606
pubmed: 21410393
BMJ Open. 2019 Nov 4;9(11):e030746
pubmed: 31690606
Epilepsia. 2010 Jul;51(7):1236-41
pubmed: 20041940
Epilepsy Behav. 2008 Jul;13(1):178-83
pubmed: 18353732
JAMA Dermatol. 2018 Jul 1;154(7):761-762
pubmed: 29800103
Neurol Clin Pract. 2018 Oct;8(5):412-420
pubmed: 30564495
Front Neurol. 2018 Jul 23;9:569
pubmed: 30083127
Orphanet J Rare Dis. 2021 Jun 2;16(1):250
pubmed: 34078440
Eur J Paediatr Neurol. 2013 Nov;17(6):631-8
pubmed: 23845174
Orphanet J Rare Dis. 2019 May 3;14(1):96
pubmed: 31053163
Expert Rev Clin Pharmacol. 2015;8(6):733-40
pubmed: 26436331
Biostatistics. 2017 Jan;18(1):91-104
pubmed: 27445132
Epilepsia. 2015 Sep;56(9):1388-97
pubmed: 26235849
J Pediatr. 2017 Nov;190:21-26.e7
pubmed: 28888564
Transplant Proc. 2010 Oct;42(8):3050-2
pubmed: 20970607
Pediatr Neurol. 2013 Oct;49(4):243-54
pubmed: 24053982
Pediatr Neurol. 2017 Jul;72:81-85
pubmed: 28511812
Expert Opin Pharmacother. 2020 Dec;21(18):2215-2223
pubmed: 32812825
Epilepsy Behav. 2020 Feb;103(Pt A):106844
pubmed: 31864941
Genet Mol Biol. 2017 Jan-Mar;40(1):69-79
pubmed: 28222202
Ann Med. 2001 Jul;33(5):337-43
pubmed: 11491192
Epilepsia. 2018 Aug;59(8):1549-1556
pubmed: 29943451
Orphanet J Rare Dis. 2017 Feb 15;12(1):35
pubmed: 28202028
Dtsch Arztebl Int. 2018 Jun 8;115(23):393-400
pubmed: 29960607
Epilepsia. 2019 Aug;60(8):1697-1710
pubmed: 31247127
PLoS One. 2016 Jun 28;11(6):e0158476
pubmed: 27351628
Biostatistics. 2014 Jan;15(1):13-6; discussion 39-45
pubmed: 24068247
Ann Pediatr Cardiol. 2019 Jan-Apr;12(1):45-48
pubmed: 30745769
Expert Opin Pharmacother. 2019 Oct;20(14):1755-1765
pubmed: 31264486
Br J Clin Pharmacol. 2016 Dec;82(6):1601-1612
pubmed: 27597136
PLoS One. 2017 Aug 9;12(8):e0180939
pubmed: 28792952
Pediatr Cardiol. 2017 Feb;38(2):394-400
pubmed: 27878332
Eur J Hum Genet. 2005 Jun;13(6):731-41
pubmed: 15798777
Dan Med J. 2019 Dec;66(12):
pubmed: 31791480
Childs Nerv Syst. 2013 Dec;29(12):2301-5
pubmed: 23743818
Orphanet J Rare Dis. 2020 Sep 25;15(1):264
pubmed: 32988393
Prog Urol. 2020 Sep;30(10):500-506
pubmed: 32718886
Epilepsia. 2017 Jul;58(7):1208-1216
pubmed: 28480518
Paediatr Drugs. 2019 Jun;21(3):185-193
pubmed: 31124053
Eur J Paediatr Neurol. 2018 Sep;22(5):738-748
pubmed: 29880258
Orphanet J Rare Dis. 2021 Jun 21;16(1):282
pubmed: 34154622
Medicine (Baltimore). 2017 Mar;96(10):e4562
pubmed: 28272193
Neurology. 2013 Feb 5;80(6):574-80
pubmed: 23325902
Epilepsy Behav. 2012 Apr;23(4):451-7
pubmed: 22381385
Orphanet J Rare Dis. 2020 Jan 21;15(1):23
pubmed: 31964424
Qual Life Res. 1998 Jul;7(5):399-407
pubmed: 9691720
Brain Dev. 2019 Jan;41(1):1-10
pubmed: 30060984
J Am Acad Dermatol. 2015 Nov;73(5):802-8
pubmed: 26365597
Annu Rev Genomics Hum Genet. 2019 Aug 31;20:217-240
pubmed: 31018109
Pharmacogenomics. 2013 Sep;14(12):1517-26
pubmed: 24024901
Epilepsia. 2021 Mar;62(3):785-794
pubmed: 33534134
Expert Rev Clin Pharmacol. 2018 Mar;11(3):309-324
pubmed: 29285947
Int J Clin Oncol. 2021 Jan;26(1):163-168
pubmed: 32990779
Epilepsy Behav. 2004 Dec;5(6):841-6
pubmed: 15582830
Pediatrics. 2011 May;127(5):e1335-7
pubmed: 21464184
Expert Rev Clin Pharmacol. 2016;9(5):637-45
pubmed: 26891946
Orphanet J Rare Dis. 2016 Nov 3;11(1):145
pubmed: 27809914
Epilepsy Behav. 2019 Feb;91:94-98
pubmed: 29941212
Eur J Paediatr Neurol. 2019 May;23(3):392-403
pubmed: 30871879
Epilepsy Behav. 2018 Dec;89:89-93
pubmed: 30390435