PMID- 34127034
OWN - NLM
STAT- MEDLINE
DCOM- 20210629
LR  - 20210629
IS  - 1750-1172 (Electronic)
IS  - 1750-1172 (Linking)
VI  - 16
IP  - 1
DP  - 2021 Jun 14
TI  - Sequential everolimus for angiomyolipoma associated with tuberous sclerosis 
      complex: a prospective cohort study.
PG  - 277
LID - 10.1186/s13023-021-01913-2 [doi]
LID - 277
AB  - 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.
FAU - Gu, Liangyou
AU  - Gu L
AD  - Department of Urology, the Third Medical Centre, Chinese PLA General Hospital, 69 
      Yong Ding Road, Beijing, 100039, China.
FAU - Peng, Cheng
AU  - Peng C
AD  - Department of Urology, the Third Medical Centre, Chinese PLA General Hospital, 69 
      Yong Ding Road, Beijing, 100039, China.
FAU - Zhang, Fan
AU  - Zhang F
AD  - Department of Urology, the Third Medical Centre, Chinese PLA General Hospital, 69 
      Yong Ding Road, Beijing, 100039, China.
FAU - Fang, Cunjin
AU  - Fang C
AD  - School of Pharmacy, Capital Medical University, Beijing, China.
FAU - Guo, Gang
AU  - Guo G
AUID- ORCID: 0000-0002-4823-8152
AD  - Department of Urology, the Third Medical Centre, Chinese PLA General Hospital, 69 
      Yong Ding Road, Beijing, 100039, China. greenguo@sina.com.
LA  - eng
PT  - Journal Article
DEP - 20210614
PL  - England
TA  - Orphanet J Rare Dis
JT  - Orphanet journal of rare diseases
JID - 101266602
RN  - 0 (Antineoplastic Agents)
RN  - 9HW64Q8G6G (Everolimus)
SB  - IM
MH  - *Angiomyolipoma/drug therapy
MH  - *Antineoplastic Agents/therapeutic use
MH  - Everolimus/therapeutic use
MH  - Humans
MH  - *Kidney Neoplasms/drug therapy
MH  - Prospective Studies
MH  - *Tuberous Sclerosis/drug therapy
PMC - PMC8201725
OTO - NOTNLM
OT  - Angiomyolipoma
OT  - Everolimus
OT  - Mammalian target of rapamyoin
OT  - Mutation
OT  - Tuberous sclerosis complex
COIS- The authors declare that they have no competing interests.
EDAT- 2021/06/16 06:00
MHDA- 2021/06/30 06:00
PMCR- 2021/06/14
CRDT- 2021/06/15 05:52
PHST- 2021/02/08 00:00 [received]
PHST- 2021/06/07 00:00 [accepted]
PHST- 2021/06/15 05:52 [entrez]
PHST- 2021/06/16 06:00 [pubmed]
PHST- 2021/06/30 06:00 [medline]
PHST- 2021/06/14 00:00 [pmc-release]
AID - 10.1186/s13023-021-01913-2 [pii]
AID - 1913 [pii]
AID - 10.1186/s13023-021-01913-2 [doi]
PST - epublish
SO  - Orphanet J Rare Dis. 2021 Jun 14;16(1):277. doi: 10.1186/s13023-021-01913-2.