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.
Panciroli M, Verrico A, et al. • Frontiers in endocrinology • 2026
In two children with severe growth hormone deficiency and progressive chiasmatic-hypothalamic pediatric low-grade glioma, about six years of concomitant low-dose growth hormone replacement and vemurafenib was followed by growth stabilization and improved lipids, without tumor progression or secondary malignancies, but the small observational report precludes conclusions about safety or efficacy.
Key Findings
Results
Two pediatric patients with severe GHD and progressive chiasmatic-hypothalamic pLGG received growth hormone replacement therapy during ongoing vemurafenib (a BRAF inhibitor).
Patient 1 was a girl diagnosed in infancy with desmoplastic infantile ganglioglioma who developed severe GHD after biopsy, chemotherapy, partial tumor debulking, and targeted therapy, along with multiple additional endocrine deficiencies.
Patient 2 was a boy with pilocytic astrocytoma diagnosed with severe GHD after partial and subtotal resections, chemotherapy, radiotherapy, and targeted therapy.
Concomitant GHRT and vemurafenib exposure lasted approximately six years.
Methods
GHRT was started at low dose after multidisciplinary assessment, documented radiological stability, and family counseling about the lack of direct safety data during BRAFi therapy.
Treatment was titrated over two months.
IGF-1 was monitored regularly, with close endocrine and oncologic surveillance.
Families were counseled that no direct safety data exist for GHRT during active BRAFi therapy.
Results
No tumor progression or secondary malignancies were detected during follow-up of concomitant GHRT and vemurafenib.
Both patients had previously progressive chiasmatic-hypothalamic pLGG.
Radiological stability was documented before GHRT initiation.
Follow-up covered approximately six years of concomitant exposure.
Results
Growth stabilized during concomitant GHRT and vemurafenib, but catch-up growth remained limited.
Growth stabilization was reported across approximately six years of concomitant therapy.
Catch-up growth was limited in both patients.
Results
Lipid parameters improved during GHRT.
Improvement in lipid parameters was observed during the roughly six-year concomitant treatment period.
Results
Bone outcomes were complex: bone mineral content increased, but lumbar DXA-derived impairment persisted or worsened.
Increased bone mineral content was reported.
Lumbar DXA-derived impairment was persistent or worsening.
Conclusions
The authors state that the small, observational nature of the report precludes conclusions on safety or efficacy and call for larger prospective studies.
Only two patients were reported.
The cases add to limited evidence on GHRT during BRAFi therapy by documenting prolonged concomitant exposure in children with confirmed severe GHD.
Prospective studies are needed to define which patients may benefit and under what monitoring conditions.
What This Means
Brain tumors called low-grade gliomas near the hypothalamus and pituitary in children often damage hormone production, including growth hormone. Growth hormone treatment is usually given only after a tumor has been stable for a while, and it is unclear whether it is appropriate while a child is taking targeted drugs such as vemurafenib, a BRAF inhibitor. This report describes two children with severe growth hormone deficiency who received low-dose growth hormone while on vemurafenib.
Over about six years of taking both treatments, the children's growth stabilized and their blood fat (lipid) levels improved. However, they did not catch up much in height, and bone results were mixed: bone mineral content rose, but a lumbar spine bone density measure stayed impaired or worsened. No tumor progression or new cancers were found during follow-up. Both children were started on growth hormone only after specialists reviewed their cases, scans showed stable tumors, and families were told that no direct safety data existed.
This research suggests that growth hormone use during BRAF inhibitor therapy may be possible in carefully selected and closely monitored children, but with only two patients and no comparison group, it cannot show that the combination is safe or effective. Larger prospective studies are needed to determine who might benefit.
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Panciroli M, Verrico A, Crocco M, Cencioni S, Morreale C, Ramaglia A, et al.. (2026). 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.. Frontiers in endocrinology. https://doi.org/10.3389/fendo.2026.1940850