FREQUENT SUBCLINICAL MACULAR CHANGES IN COMBINED BRAF/MEK INHIBITION WITH HIGH-DOSE HYDROXYCHLOROQUINE AS TREATMENT FOR ADVANCED METASTATIC BRAF MUTANT MELANOMA: Preliminary Results From a Phase I/II Clinical Treatment Trial

Retina. 2019 Mar;39(3):502-513. doi: 10.1097/IAE.0000000000002027.

Abstract

Purpose: To assess the potential ocular toxicity of a combined BRAF inhibition (BRAFi) + MEK inhibition (MEKi) + hydroxychloroquine (HCQ) regime used to treat metastatic BRAF mutant melanoma.

Methods: Patients with stage IV metastatic melanoma and BRAF V600E mutations (n = 11, 31-68 years of age) were included. Treatment was with oral dabrafenib, 150 mg bid, trametinib, 2 mg/day, and HCQ, 400 mg to 600 mg bid. An ophthalmic examination, spectral domain optical coherence tomography, near-infrared and short-wavelength fundus autofluorescence, and static perimetry were performed at baseline, 1 month, and q/6 months after treatment.

Results: There were no clinically significant ocular events; there was no ocular inflammation. The only medication-related change was a separation of the photoreceptor outer segment tip from the apical retinal pigment epithelium that could be traced from the fovea to the perifoveal retina noted in 9/11 (82%) of the patients. There were no changes in retinal pigment epithelium melanization or lipofuscin content by near-infrared fundus autofluorescence and short-wavelength fundus autofluorescence, respectively. There were no inner retinal or outer nuclear layer changes. Visual acuities and sensitivities were unchanged.

Conclusion: BRAFi (trametinib) + MEKi (dabrafenib) + HCQ causes very frequent, subclinical separation of the photoreceptor outer segment from the apical retinal pigment epithelium without inner retinal changes or signs of inflammation. The changes suggest interference with the maintenance of the outer retinal barrier and/or phagocytic/pump functions of the retinal pigment epithelium by effective MEK inhibition.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / adverse effects*
  • Antineoplastic Agents / therapeutic use
  • Enzyme Inhibitors / adverse effects*
  • Enzyme Inhibitors / therapeutic use
  • Female
  • Humans
  • Hydroxychloroquine / adverse effects*
  • Hydroxychloroquine / therapeutic use
  • Imidazoles / adverse effects*
  • Imidazoles / therapeutic use
  • MAP Kinase Kinase 1 / antagonists & inhibitors
  • Macula Lutea / pathology*
  • Male
  • Melanoma / drug therapy*
  • Melanoma / genetics
  • Middle Aged
  • Oximes / adverse effects*
  • Oximes / therapeutic use
  • Photoreceptor Cells / pathology
  • Protein Kinase Inhibitors / adverse effects*
  • Protein Kinase Inhibitors / therapeutic use
  • Proto-Oncogene Proteins B-raf / antagonists & inhibitors
  • Pyridones / adverse effects*
  • Pyridones / therapeutic use
  • Pyrimidinones / adverse effects*
  • Pyrimidinones / therapeutic use
  • Retinal Diseases* / chemically induced
  • Retinal Diseases* / pathology
  • Retinal Pigment Epithelium / pathology

Substances

  • Antineoplastic Agents
  • Enzyme Inhibitors
  • Imidazoles
  • Oximes
  • Protein Kinase Inhibitors
  • Pyridones
  • Pyrimidinones
  • trametinib
  • Hydroxychloroquine
  • Proto-Oncogene Proteins B-raf
  • MAP Kinase Kinase 1
  • dabrafenib