PMID- 36716766
OWN - NLM
STAT- MEDLINE
DCOM- 20230524
LR  - 20230524
IS  - 1525-1470 (Electronic)
IS  - 0736-8046 (Linking)
VI  - 40
IP  - 3
DP  - 2023 May-Jun
TI  - The use of mTOR inhibitors for the treatment of kaposiform hemangioendothelioma. 
      A systematic review.
PG  - 440-445
LID - 10.1111/pde.15262 [doi]
AB  - BACKGROUND: Kaposiform hemangioendothelioma (KHE) is a locally aggressive and 
      potentially lethal vascular tumor of infancy. Current consensus recommendations 
      include the use of vincristine and/or systemic steroids as first-line treatment. 
      Mammalian target of rapamycin (mTOR) inhibitors represent a promising therapy for 
      patients with KHE. The goal of our study is to critically assess the existing 
      literature on outcomes of patients with KHE treated with mTOR inhibitors. 
      METHODS: We conducted a literature search from 1 January 2000, to 30 April 2022. 
      Articles reporting outcomes of patients treated with mTOR inhibitors for KHE were 
      included. Descriptive statistics were used to describe and summarize the outcomes 
      of the treatment. RESULTS: We included 327 patients with a mean age at diagnosis 
      of 9.1 months (SD +/- 9). Patients were treated with an mTOR inhibitor for a mean 
      of 15.2 months (SD +/- 4.1). A total of 315 (96.3%) patients had positive outcomes 
      including improvement of the tumor size, symptoms and/or laboratory parameters in 
      227 (85%) and complete remission in 38 (12%). Seven (2%) patients did not respond 
      to treatment and seven (2%) died of sepsis (4), Kasabach-Merritt phenomenon 
      complications (1), cardiac and liver failure due to ductus arteriosus (1), or 
      metastatic disease (1). CONCLUSION: This systematic review supports the efficacy 
      and safety of mTOR inhibitors for KHE. Their use resulted in positive outcomes in 
      terms of decreased symptoms, reduction in tumor size and improvement in 
      biochemical parameters with a mortality rate of 2%. According to these findings, 
      we suggest revised consensus treatment guidelines for KHE with mTOR inhibitors 
      potentially considered first-line therapy.
CI  - (c) 2023 Wiley Periodicals LLC.
FAU - Maza-Morales, Mariana
AU  - Maza-Morales M
AD  - Dermatology Department, National Institute of Pediatrics, Mexico City, Mexico.
FAU - Valdes-Loperena, Sofia
AU  - Valdes-Loperena S
AUID- ORCID: 0000-0002-5035-2181
AD  - Dermatology Department, National Institute of Pediatrics, Mexico City, Mexico.
FAU - Duran-McKinster, Lourdes Carola
AU  - Duran-McKinster LC
AUID- ORCID: 0000-0003-4977-4958
AD  - Dermatology Department, National Institute of Pediatrics, Mexico City, Mexico.
FAU - Garcia-Romero, Maria Teresa
AU  - Garcia-Romero MT
AUID- ORCID: 0000-0002-1408-8109
AD  - Dermatology Department, National Institute of Pediatrics, Mexico City, Mexico.
LA  - eng
PT  - Journal Article
PT  - Systematic Review
DEP - 20230130
PL  - United States
TA  - Pediatr Dermatol
JT  - Pediatric dermatology
JID - 8406799
RN  - W36ZG6FT64 (Sirolimus)
RN  - 0 (MTOR Inhibitors)
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
RN  - EC 2.7.1.1 (MTOR protein, human)
RN  - Kaposiform Hemangioendothelioma
SB  - IM
MH  - Humans
MH  - Infant
MH  - *Kasabach-Merritt Syndrome/diagnosis
MH  - Sirolimus/therapeutic use
MH  - MTOR Inhibitors
MH  - *Hemangioendothelioma/diagnosis
MH  - *Sarcoma, Kaposi/complications
MH  - TOR Serine-Threonine Kinases/therapeutic use
OTO - NOTNLM
OT  - hemangioendothelioma
OT  - therapy-systemic
OT  - vascular tumors
EDAT- 2023/01/31 06:00
MHDA- 2023/05/24 06:42
CRDT- 2023/01/30 19:02
PHST- 2022/09/13 00:00 [received]
PHST- 2023/01/06 00:00 [accepted]
PHST- 2023/05/24 06:42 [medline]
PHST- 2023/01/31 06:00 [pubmed]
PHST- 2023/01/30 19:02 [entrez]
AID - 10.1111/pde.15262 [doi]
PST - ppublish
SO  - Pediatr Dermatol. 2023 May-Jun;40(3):440-445. doi: 10.1111/pde.15262. Epub 2023 
      Jan 30.