PMID- 33754454
OWN - NLM
STAT- MEDLINE
DCOM- 20220121
LR  - 20220121
IS  - 1545-5017 (Electronic)
IS  - 1545-5009 (Linking)
VI  - 68
IP  - 6
DP  - 2021 Jun
TI  - Long-term outcomes of pediatric and young adult patients receiving radiotherapy 
      for nonmalignant vascular anomalies.
PG  - e28955
LID - 10.1002/pbc.28955 [doi]
AB  - BACKGROUND: Nonmalignant vascular anomalies (VA) comprise a heterogeneous 
      spectrum of conditions characterized by aberrant growth or development of blood 
      and/or lymphatic vessels and can cause significant morbidity. Little is known 
      about outcomes after radiotherapy in pediatric and young adult patients with 
      nonmalignant VA. METHODS: Thirty patients who were diagnosed with nonmalignant VA 
      and treated with radiotherapy prior to 2017 and before the age of 30 were 
      identified. Clinical and treatment characteristics and outcomes were recorded. 
      RESULTS: Median age at first radiotherapy was 15 years (range 0.02-27). Median 
      follow-up from completion of first radiotherapy was 9.8 years (range 0.02-67.4). 
      Lymphatic malformations (33%), kaposiform hemangioendothelioma (17%), and venous 
      malformations (17%) were the most common diagnoses. The most common indication 
      for first radiotherapy was progression despite standard therapy and/or urgent 
      palliation for symptoms (57%). After first radiotherapy, 14 patients (47%) had a 
      complete response or partial response, defined as decrease in size of treated 
      lesion or symptomatic improvement. After first radiotherapy, 27 (90%) required 
      additional treatment for progression or recurrence. Long-term complications 
      included telangiectasias, fibrosis, xerophthalmia, radiation pneumonitis, ovarian 
      failure, and central hypothyroidism. No patient developed secondary malignancies. 
      At last follow-up, three patients (10%) were without evidence of disease, 26 
      (87%) with disease, and one died of complications (3.3%). CONCLUSIONS: A small 
      group of pediatric and young adult patients with nonmalignant, high-risk VA 
      experienced clinical benefit from radiotherapy with expected toxicity; however, 
      most experienced progression. Prospective studies are needed to characterize 
      indications for radiotherapy in VA refractory to medical therapy, including 
      targeted inhibitors.
CI  - (c) 2021 Wiley Periodicals LLC.
FAU - Liu, Kevin X
AU  - Liu KX
AUID- ORCID: 0000-0003-2071-5137
AD  - Department of Radiation Oncology, Brigham and Women's Hospital, Dana-Farber 
      Cancer Institute, Boston Children's Hospital, Harvard Medical School, Boston, 
      Massachusetts, USA.
FAU - Lamba, Nayan
AU  - Lamba N
AD  - Department of Radiation Oncology, Brigham and Women's Hospital, Dana-Farber 
      Cancer Institute, Boston Children's Hospital, Harvard Medical School, Boston, 
      Massachusetts, USA.
FAU - Marcus, Karen J
AU  - Marcus KJ
AD  - Department of Radiation Oncology, Brigham and Women's Hospital, Dana-Farber 
      Cancer Institute, Boston Children's Hospital, Harvard Medical School, Boston, 
      Massachusetts, USA.
FAU - Sandler, Eric S
AU  - Sandler ES
AD  - Department of Pediatric Oncology, Nemours Children's Health Systems, 
      Jacksonville, Florida, USA.
FAU - Gold, Stuart H
AU  - Gold SH
AD  - Division of Pediatric Hematology/Oncology, University of North Carolina, Chapel 
      Hill, North Carolina, USA.
FAU - Margolin, Judith F
AU  - Margolin JF
AD  - Cancer and Hematology Centers, Baylor College of Medicine and Texas Children's 
      Hospital, Houston, Texas, USA.
FAU - Haas-Kogan, Daphne A
AU  - Haas-Kogan DA
AD  - Department of Radiation Oncology, Brigham and Women's Hospital, Dana-Farber 
      Cancer Institute, Boston Children's Hospital, Harvard Medical School, Boston, 
      Massachusetts, USA.
FAU - Adams, Denise M
AU  - Adams DM
AUID- ORCID: 0000-0002-2892-5369
AD  - Department of Hematology/Oncology, Vascular Anomalies Center, Boston Children's 
      Hospital, Boston, Massachusetts, USA.
AD  - Department of Oncology, Children's Hospital of Philadelphia, Philadelphia, 
      Pennsylvania, USA.
LA  - eng
PT  - Journal Article
DEP - 20210323
PL  - United States
TA  - Pediatr Blood Cancer
JT  - Pediatric blood & cancer
JID - 101186624
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Child
MH  - Child, Preschool
MH  - Hemangioendothelioma
MH  - Humans
MH  - Infant
MH  - Infant, Newborn
MH  - Kasabach-Merritt Syndrome
MH  - Lymphatic Abnormalities
MH  - *Radiotherapy
MH  - Retrospective Studies
MH  - Sarcoma, Kaposi
MH  - Vascular Malformations
MH  - Young Adult
OTO - NOTNLM
OT  - Gorham Stout disease
OT  - kaposiform hemangioendothelioma
OT  - lymphatic malformation
OT  - palliation
OT  - venous malformation
EDAT- 2021/03/24 06:00
MHDA- 2022/01/22 06:00
CRDT- 2021/03/23 07:15
PHST- 2021/01/18 00:00 [revised]
PHST- 2020/11/16 00:00 [received]
PHST- 2021/01/28 00:00 [accepted]
PHST- 2021/03/24 06:00 [pubmed]
PHST- 2022/01/22 06:00 [medline]
PHST- 2021/03/23 07:15 [entrez]
AID - 10.1002/pbc.28955 [doi]
PST - ppublish
SO  - Pediatr Blood Cancer. 2021 Jun;68(6):e28955. doi: 10.1002/pbc.28955. Epub 2021 
      Mar 23.