Pediatric endocrinology

Multidisciplinary Treatment of Patients With Noonan Syndrome: A Consensus Statement

JAMA Netw OpenPublished Consensus statement

Methodological detailsmodified Delphi process, multidisciplinary steering committee, 25-member expert panel
  • Modified Delphi process
  • Multidisciplinary steering committee
  • 25-member expert panel

Recommendations

As issued by the authors

  1. 100% agreement

    Molecular characterization is important for diagnosis, prognosis and treatment decisions

  2. 100% agreement

    A multidisciplinary approach to noonan syndrome (NS) treatment is necessary

  3. 100% agreement

    Transition from pediatric to adult care needs improvement

  4. 100% agreement

    Patients with hypertrophic cardiomyopathy (HCM) are at increased risk

  5. 100% agreement

    Adherence to recombinant human growth hormone (rhGH) therapy should be monitored

  6. 100% agreement

    Partial GH insensitivity should not exclude rhGH therapy

  7. 100% agreement

    Safety concerns regarding rhGH therapy, particularly related to HCM and the risk of malignant neoplasms

Original publication

Multidisciplinary Treatment of Patients With Noonan Syndrome: A Consensus Statement. JAMA Netw Open. Published 1 October 2025. doi:10.1001/jamanetworkopen.2025.37603

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