{
  "abstract": "Pediatric low-grade gliomas (pLGGs) involving the hypothalamic-pituitary region frequently result in endocrine dysfunction, particularly growth hormone deficiency (GHD), due to tumor location and treatment-related effects. Growth hormone replacement therapy (GHRT) is generally considered after a period of disease stability, whereas its use during active oncologic therapy remains controversial, including during BRAF inhibitor (BRAFi) treatment. We describe two pediatric patients with severe GHD who received GHRT during ongoing vemurafenib, a BRAFi, for progressive chiasmatic-hypothalamic pLGG. The first patient, a girl diagnosed in infancy with desmoplastic infantile ganglioglioma, developed severe GHD after biopsy, chemotherapy, partial tumor debulking, and targeted therapy, together with multiple additional endocrine deficiencies. The second patient, a boy with pilocytic astrocytoma, was diagnosed with severe GHD after multimodal treatment, including partial and subtotal surgical resections, chemotherapy, radiotherapy, and targeted therapy. In both cases, low-dose GHRT was initiated after multidisciplinary assessment, documented radiological stability before GHRT initiation, and family counseling regarding the absence of direct safety data during active BRAFi therapy. Treatment was titrated over two months, with regular IGF-1 monitoring and close endocrine and oncologic surveillance. During approximately six years of concomitant GHRT and vemurafenib exposure, growth stabilized and lipid parameters improved, whereas catch-up growth remained limited and bone outcomes were complex, with increased bone mineral content but persistent or worsening lumbar DXA-derived impairment. No tumor progression or secondary malignancies were detected during the reported follow-up. These two cases add to the limited evidence on GHRT administration during BRAFi therapy by documenting prolonged concomitant GHRT and vemurafenib exposure in children with severe confirmed GHD and previously progressive chiasmatic-hypothalamic pLGG. However, the limited sample size and observational nature of this report preclude conclusions about safety or efficacy. Larger prospective studies are needed to define which patients may benefit and under which monitoring conditions GHRT could be considered during BRAFi therapy.",
  "abstract_shown": true,
  "affiliations": [
    "Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa",
    "Neuro-Oncology Unit, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto Giannina Gaslini",
    "Pediatric Gastroenterology and Endoscopy Unit, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto Giannina Gaslini",
    "Department of Woman and Child, ASST-Settelaghi, University of Insubria",
    "Neuroradiology Unit, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto Giannina Gaslini",
    "Pharmacy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto Giannina Gaslini",
    "Pediatric Clinic, Endocrinology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Giannina Gaslini"
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 11,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Marta Panciroli"
    },
    {
      "affs": [
        1
      ],
      "name": "Antonio Verrico"
    },
    {
      "affs": [
        2
      ],
      "name": "Marco Crocco"
    },
    {
      "affs": [
        0
      ],
      "name": "Sara Cencioni"
    },
    {
      "affs": [
        0,
        3
      ],
      "name": "Chiara Morreale"
    },
    {
      "affs": [
        4
      ],
      "name": "Antonia Ramaglia"
    },
    {
      "affs": [
        5
      ],
      "name": "Valentina Iurilli"
    },
    {
      "affs": [
        1
      ],
      "name": "Gianluca Piccolo"
    },
    {
      "affs": [
        1
      ],
      "name": "Claudia Milanaccio"
    },
    {
      "affs": [
        0,
        6
      ],
      "name": "Mohamad Maghnie"
    },
    {
      "affs": [
        0,
        6
      ],
      "name": "Natascia Di Iorgi"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/1p5zr0t01vs78.bib",
  "cited_by": 0,
  "doi": "10.3389/fendo.2026.1940850",
  "fulltext_url": "https://europepmc.org/articles/PMC13686916",
  "id": "1p5zr0t01vs78",
  "issns": [
    "1664-2392"
  ],
  "landing_url": "https://www.frontiersin.org/articles/10.3389/fendo.2026.1940850/full",
  "language": "eng",
  "licence": "CC-BY-4.0",
  "licence_label": "CC BY 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "openalex",
      "2712|Growth Hormone and Insulin-like Growth Factors"
    ]
  ],
  "openalex": "W7221032024",
  "pdf_url": "https://europepmc.org/articles/PMC13686916?pdf=render",
  "pmcid": "PMC13686916",
  "pmid": "42922835",
  "published": "2026-10-08",
  "publisher": "Frontiers Media SA",
  "ris": "https://research.draconitic.com/paper/1p5zr0t01vs78.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:20:20Z",
      "native_id": "10.3389/fendo.2026.1940850",
      "source": "crossref",
      "url": "https://www.frontiersin.org/articles/10.3389/fendo.2026.1940850/full"
    },
    {
      "first_seen": "2026-10-30T12:20:36Z",
      "native_id": "MED:42922835",
      "source": "europepmc",
      "url": "https://europepmc.org/article/MED/42922835"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2712|Growth Hormone and Insulin-like Growth Factors",
      "subfield": "endocrinology-metabolism"
    }
  ],
  "title": "Case Report: Case series: growth hormone replacement for severe growth hormone deficiency during BRAF inhibitor therapy in pediatric low-grade glioma: long-term follow-up",
  "title_text": "Case Report: Case series: growth hormone replacement for severe growth hormone deficiency during BRAF inhibitor therapy in pediatric low-grade glioma: long-term follow-up",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:20:36Z",
  "url": "https://research.draconitic.com/paper/1p5zr0t01vs78",
  "venue": "Frontiers in Endocrinology",
  "versions": []
}