Clinical Response to PI3K-α Inhibition in a Cohort of Children and Adults With
Klippel Trenaunay
PIK3CA-related overgrowth spectrum
alpelisib
therapy
vascular malformation
Journal
Journal of vascular anomalies
ISSN: 2690-2702
Titre abrégé: J Vasc Anom (Phila)
Pays: United States
ID NLM: 9918265901206676
Informations de publication
Date de publication:
Mar 2022
Mar 2022
Historique:
entrez:
26
10
2022
pubmed:
27
10
2022
medline:
27
10
2022
Statut:
ppublish
Résumé
The goal of this report is to describe, through a series of 5 cases, the clinical response and safety of alpelisib (BYL719) use in children and adults with We reviewed clinical records of 5 patients from October 2019 through September 2021 followed by the pediatric hematology and multidisciplinary vascular anomalies teams at the Monroe Carell Jr. Children's Hospital at Vanderbilt (MCJCHV). All patients carried a clinical or genetic diagnosis of PROS and were treated with alpelisib provided by a Novartis managed access program. We highlight improvement in reported symptoms, objective overgrowth measurements, and quality of life to varying degrees in all patients. We note dose-dependent hyperglycemia and gastrointestinal side effects in 2 of the 5 patients. No patients experienced any serious side effects. This case series reports on the real-world use of PI3K-α inhibition in the management of PROS. Ongoing clinical trials will provide efficacy and safety data as these drugs become more widely used in patients with vascular anomalies and syndromes secondary to somatic
Identifiants
pubmed: 36284525
doi: 10.1097/jova.0000000000000038
pmc: PMC9592062
mid: NIHMS1842971
pii:
doi:
Types de publication
Journal Article
Langues
eng
Subventions
Organisme : NIGMS NIH HHS
ID : T32 GM007569
Pays : United States
Déclaration de conflit d'intérêts
The authors declare that they have no funding and conflicts of interest with regard to the content of this report.
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