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Trametinib for Refractory Chylous Effusions and Systemic Complications in Children with Noonan Syndrome.
Nakano, Taizo A; Rankin, Alexander W; Annam, Aparna; Kulungowski, Ann M; McCallen, Leslie M; Hill, Lauren R; Chatfield, Kathryn C.
Afiliação
  • Nakano TA; Vascular Anomalies Center, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO; Center for Cancer and Blood Disorders, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO.
  • Rankin AW; Center for Cancer and Blood Disorders, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO.
  • Annam A; Vascular Anomalies Center, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO; Department of Pediatric Radiology, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO.
  • Kulungowski AM; Vascular Anomalies Center, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO; Department of Pediatric Surgery, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO.
  • McCallen LM; Vascular Anomalies Center, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO; Department of Pediatrics, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO.
  • Hill LR; Vascular Anomalies Center, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO.
  • Chatfield KC; Vascular Anomalies Center, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO; Department of Pediatrics, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO. Electronic address: Kathryn.chatfield@cuanschutz.edu.
J Pediatr ; 248: 81-88.e1, 2022 09.
Article em En | MEDLINE | ID: mdl-35605646
OBJECTIVE: To evaluate the effect of the RAS-MAPK pathway inhibitor trametinib on medically refractory chylous effusions in 3 hospitalized patients with Noonan syndrome. STUDY DESIGN: Pharmacologic MEK1/2 inhibition has been used to treat conditions associated with Noonan syndrome, given that activation of RAS-MAPK pathway variants leads to downstream MEK activation. We describe our experience with 3 patients with Noonan syndrome (owing to variants in 3 distinct genes) and refractory chylous effusions treated successfully with MEK inhibition. A monitoring protocol was established to standardize medication dosing and monitoring of outcome measures. RESULTS: Subjects demonstrated improvement in lymphatic leak with additional findings of improved growth and normalization of cardiac and hematologic measurements. Trametinib was administered safely, with only moderate skin irritation in 1 subject. CONCLUSIONS: Improvements in a variety of quantifiable measurements highlight the potential utility of MEK1/2 inhibition in patients with Noonan syndrome and life-threatening lymphatic disease. Larger, prospective studies are needed to confirm efficacy and assess long-term safety.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Antineoplásicos / Síndrome de Noonan Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Revista: J Pediatr Ano de publicação: 2022 Tipo de documento: Article País de publicação: Estados Unidos
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Antineoplásicos / Síndrome de Noonan Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Revista: J Pediatr Ano de publicação: 2022 Tipo de documento: Article País de publicação: Estados Unidos