| Title: |
Angiomyolipoma rebound tumor growth after discontinuation of everolimus in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis |
| Authors: |
Bissler J. J.; Nonomura N.; Budde K.; Zonnenberg B. A.; Fischereder M.; Voi M.; Louveau A. -L.; Herbst F.; Bebin E. M.; Curatolo P.; Zonta A.; Belousova E. |
| Contributors: |
Bissler, Jj; Nonomura, N; Budde, K; Zonnenberg, Ba; Fischereder, M; Voi, M; Louveau, A-; Herbst, F; Bebin, Em; Curatolo, P; Zonta, A; Belousova, E |
| Publisher Information: |
PUBLIC LIBRARY SCIENCE |
| Publication Year: |
2018 |
| Collection: |
Universitá degli Studi di Roma "Tor Vergata": ART - Archivio Istituzionale della Ricerca |
| Subject Terms: |
Adult; Angiomyolipoma; Antineoplastic Agent; Disease Progression; Everolimu; Female; Human; Kidney Neoplasm; Lymphangioleiomyomatosi; Male; Middle Aged; Tuberous Sclerosi; Withholding Treatment; Young Adult; Settore MED/39 - NEUROPSICHIATRIA INFANTILE |
| Description: |
IntroductionThe EXIST-2 (NCT00790400) study demonstrated the superiority of everolimus over placebo for the treatment of renal angiomyolipomas associated with tuberous sclerosis complex (TSC) or sporadic lymphangioleiomyomatosis (LAM). This post hoc analysis of EXIST-2 study aimed to assess angiomyolipoma tumor behavior among patients who submitted to continued radiographic examination following discontinuation of everolimus in the noninterventional follow-up phase.MethodsFor patients who discontinued everolimus at the completion of extension phase for reasons other than angiomyolipoma progression, a single CT/MRI scan of the kidney was collected after 1 year of treatment discontinuation. Changes from baseline and from the time of everolimus discontinuation in the sum of volumes of target angiomyolipoma lesions were assessed in the non-interventional follow-up phase (data cutoff date, November 6, 2015).ResultsOf the 112 patients who received >= 1 dose of everolimus and discontinued treatment by the end of extension phase, 34 (30.4%) were eligible for participation in the non-interventional follow-up phase. Sixteen of 34 patients were evaluable for angiomyolipoma tumor behavior as they had at least one valid efficacy assessment (i.e. kidney CT/MRI scan) after everolimus discontinuation. During the non-interventional follow-up phase, compared with baseline, two patients (12.5%) experienced angiomyolipoma progression (angiomyolipoma-related bleeding [n = 1], increased kidney volume [n = 1]). Five patients out of 16 (31.3%) experienced angiomyolipoma progression when compared with the angiomyolipoma tumor assessment at everolimus discontinuation. The median (range) percentage change in angiomyolipoma tumor volume (cm 3) from baseline was -70.56 (-88.30; -49.64) at time of everolimus discontinuation (n = 11), and -50.55 (-79.40; -23.16) at week 48 (n = 7) after discontinuation of everolimus. One patient death was reported due to angiomyolipoma hemorrhage.ConclusionsAngiomyolipoma lesions displayed an increase in ... |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/pmid/30192751; info:eu-repo/semantics/altIdentifier/wos/WOS:000444093600009; volume:13; issue:9; firstpage:e0201005; journal:PLOS ONE; https://hdl.handle.net/2108/212756 |
| DOI: |
10.1371/journal.pone.0201005 |
| Availability: |
https://hdl.handle.net/2108/212756; https://doi.org/10.1371/journal.pone.0201005 |
| Accession Number: |
edsbas.B2C54671 |
| Database: |
BASE |