A retrospective study based on data collected from Memorial Sloan-Kettering Cancer Center, New York shows high efficacy and low toxicity of intravitreal Evomela formulation for retinoblastoma . Evomela is an intravenous formulation currently with no indication of intravitreal use. The center switched in November 2016 from the propylene glycol containing melphalan composition (Alkeran, Apopharma USA Inc.) to Captisol(R) stabilized melphalan (Evomela, Acrotech Biopharma LLC) with the hope that it would decrease retinal toxicity while retaining efficacy for treatment of intraocular retinoblastoma. The study compares the degree of retinal toxicity and overall ocular survival between formulations in a large, single-center retrospective analysis.
Retinal toxicity was measured using electroretinogram (ERG) and compared using a multivariate analysis of 338 injections in 101 eyes of 96 patients.
No differences were found in retinal toxicity and ocular survival between 30 μg intravitreal injections of Alkeran or Evomela for intraocular retinoblastoma. Given the increased stability of Evomela, intravitreal treatment could be expanded to centers without the ability to supply Alkeran due to its shorter safety window (which is only 1 hour).
The study shows that SBECD is safe to use in this rare application route. The center could not demonstrate superior toxicity profile of Evomela over the solvent-based traditional composition, but the higher stability profile of the cyclodextrin-based form definitely pays-off.
Widening of the applicability of cyclodextrins by adding a new administration route is always reflecting the triumph of the founding fathers of CD chemistry who believed that CDs just find their use in every field and every way.
 Hsieh T, Liao A, Francis JH, et al. Comparison of efficacy and toxicity of intravitreal melphalan formulations for retinoblastoma. PLoS One. 2020;15(7):e0235016. doi:10.1371/journal.pone.0235016
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