PMID- 31930696
OWN - NLM
STAT- MEDLINE
DCOM- 20200626
LR  - 20200626
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 67
IP  - 4
DP  - 2020 Apr
TI  - Topical sirolimus for the treatment of cutaneous manifestations of vascular 
      anomalies: A case series.
PG  - e28088
LID - 10.1002/pbc.28088 [doi]
AB  - BACKGROUND: Vascular anomalies (VA), characterized by the abnormal development or 
      growth of blood and/or lymphatic vessels, encompasses a spectrum of conditions 
      with a range of symptoms and complications. VA are frequently associated with 
      cutaneous complications that can cause significant morbidity. Systemic sirolimus 
      has previously been shown to be effective in the treatment of complicated VA. 
      There are limited studies to date on the use of topical sirolimus for the 
      treatment of cutaneous manifestations of VA. METHODS: Retrospective review of 
      medical records of pediatric patients with VA treated with topical sirolimus at a 
      single quaternary pediatric institution. Response was determined by clinical 
      subjective and objective measures of improvement. RESULTS: Twenty-three patients 
      with cutaneous VA manifestations were treated with topical sirolimus. Median age 
      was 14 (range 4-27 years). The main indication for treatment was complication of 
      lymphatic blebbing (82%, n = 19) including lymphatic fluid leakage, bleeding, 
      pain, pruritus, swelling, or recurrent infection. Treatment course ranged from 
      109 to 1424 days with median of 622 days. No major side effects were reported. 
      Eighty-six percent of patients (n = 20) had subjective or objective improvement 
      of cutaneous lesions. Lymphatic blebbing complications improved in 90% (n = 17) 
      of individuals. Eighty-two percent (n = 14) of patients not receiving concurrent 
      systemic sirolimus demonstrated improvement with topical therapy. One patient 
      electively stopped treatment due to pruritus and burning sensation. CONCLUSION: 
      Topical sirolimus appears to be a beneficial therapy for lymphatic blebbing 
      associated with lymphatic malformations or mixed malformations with a lymphatic 
      component, although benefit in other VA remains unclear. Topical sirolimus was 
      well-tolerated with minimal side effects.
CI  - (c) 2020 Wiley Periodicals, Inc.
FAU - Badia, Priscila
AU  - Badia P
AUID- ORCID: 0000-0002-7916-9022
AD  - Division of Hematology, Hemangioma and Vascular Malformation Center, Cincinnati 
      Children's Hospital Medical Center, Cincinnati, Ohio.
FAU - Ricci, Kiersten
AU  - Ricci K
AUID- ORCID: 0000-0002-7253-5632
AD  - Division of Hematology, Hemangioma and Vascular Malformation Center, Cincinnati 
      Children's Hospital Medical Center, Cincinnati, Ohio.
AD  - Department of Pediatrics, University of Cincinnati College of Medicine, 
      Cincinnati, Ohio.
FAU - Gurria, Juan P
AU  - Gurria JP
AD  - Division of Pediatric Surgery, Cincinnati Children's Hospital Medical Center, 
      Cincinnati, Ohio.
FAU - Dasgupta, Roshni
AU  - Dasgupta R
AD  - Department of Pediatrics, University of Cincinnati College of Medicine, 
      Cincinnati, Ohio.
AD  - Division of Pediatric Surgery, Cincinnati Children's Hospital Medical Center, 
      Cincinnati, Ohio.
FAU - Patel, Manish
AU  - Patel M
AD  - Department of Pediatrics, University of Cincinnati College of Medicine, 
      Cincinnati, Ohio.
AD  - Division of Interventional Radiology, Cincinnati Children's Hospital Medical 
      Center, Cincinnati, Ohio.
FAU - Hammill, Adrienne
AU  - Hammill A
AUID- ORCID: 0000-0003-0820-4924
AD  - Division of Hematology, Hemangioma and Vascular Malformation Center, Cincinnati 
      Children's Hospital Medical Center, Cincinnati, Ohio.
AD  - Department of Pediatrics, University of Cincinnati College of Medicine, 
      Cincinnati, Ohio.
LA  - eng
PT  - Journal Article
DEP - 20200113
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
RN  - 0 (Immunosuppressive Agents)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Administration, Topical
MH  - Adolescent
MH  - Adult
MH  - Child
MH  - Child, Preschool
MH  - Female
MH  - Humans
MH  - Immunosuppressive Agents/*administration & dosage
MH  - Lymphatic Abnormalities/complications/drug therapy
MH  - Male
MH  - Retrospective Studies
MH  - Sirolimus/*administration & dosage
MH  - Skin Diseases/*drug therapy/*etiology
MH  - Vascular Malformations/*complications/*drug therapy
MH  - Young Adult
OTO - NOTNLM
OT  - blebs
OT  - lymphatic
OT  - sirolimus
OT  - topical
OT  - vascular malformations
EDAT- 2020/01/14 06:00
MHDA- 2020/06/27 06:00
CRDT- 2020/01/14 06:00
PHST- 2019/06/26 00:00 [received]
PHST- 2019/10/16 00:00 [revised]
PHST- 2019/10/25 00:00 [accepted]
PHST- 2020/01/14 06:00 [pubmed]
PHST- 2020/06/27 06:00 [medline]
PHST- 2020/01/14 06:00 [entrez]
AID - 10.1002/pbc.28088 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2020 Apr;67(4):e28088. doi: 10.1002/pbc.28088. Epub 2020 
      Jan 13.