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New devices and techniques
Original research
Efficacy of a prolonged stability melphalan formulation for intra-arterial treatment of retinoblastoma
  1. Jubran H Jubran1,
  2. Hanna Luong2,
  3. Anant Naik1,
  4. Visish M Srinivasan1,
  5. Aparna Ramasubramanian2,
  6. Amanda Li3,
  7. Lea Scherschinski1,
  8. Michael J Feldman4,
  9. Felipe C Albuquerque1,
  10. Todd A Abruzzo1,2,5,6
  1. 1 Department of Neurosurgery, Barrow Neurological Institute, Phoenix, Arizona, USA
  2. 2 Department of Pediatric Ophthalmology, Retinoblastoma Division, Phoenix Children's Hospital, Phoenix, Arizona, USA
  3. 3 Department of Pharmacy, Phoenix Children's Hospital, Phoenix, Arizona, USA
  4. 4 Department of Neurosurgery, Vanderbilt University Medical Center, Nashville, Tennessee, USA
  5. 5 Department of Radiology, Phoenix Children's Hospital, Phoenix, AZ, USA
  6. 6 University of Arizona College of Medicine - Phoenix, Phoenix, AZ, USA
  1. Correspondence to Dr Todd A Abruzzo, Department of Neurosurgery, Barrow Neurological Institute, Phoenix, AZ 85006, USA; tabruzzo{at}phoenixchildrens.com

Abstract

Background Melphalan, which is poorly soluble at room temperature, is widely used for the treatment of retinoblastoma by selective ophthalmic artery infusion. Evomela, a propylene glycol-free formulation of melphalan with improved solubility and stability, has recently been used as an alternative.

To compare the safety and efficacy of Evomela with standard-formulation melphalan (SFM) in the treatment of retinoblastoma by selective ophthalmic artery infusion.

Methods We performed a retrospective case–control study of patients with retinoblastoma undergoing selective ophthalmic artery infusion with SFM or Evomela at a single institution. Cycle-specific percent tumor regression (CSPTR) was estimated by comparing photos obtained during pretreatment examination under anesthesia (EUA) with those obtained during post-treatment EUA 3–4 weeks later. CSPTR, ocular salvage rates, complication rates, operation times (unadjusted and adjusted for difficulty of ophthalmic artery catheterization), and intraprocedural dose expiration rates were compared between Evomela- and SFM-treated groups. Univariate and multivariate analyses were performed.

Results Ninety-seven operations (melphalan: 45; Evomela: 52) for 23 patients with 27 retinoblastomas were studied. The ocular salvage rate was 79% in the SFM-treated group and 69% in the Evomela-treated group. Multivariate regression controlling for tumor grade, patient age, and treatment history revealed no significant differences in ocular salvage rate, CSPTR, complication rates, or operation times. Although the dose expiration rate was higher for the SFM-treated group, the difference was not statistically significant. Notably, there were no ocular or cerebral ischemic complications.

Conclusion Evomela has non-inferior safety and efficacy relative to SFM when used for the treatment of retinoblastoma by selective ophthalmic artery infusion.

  • orbit
  • pediatrics
  • tumor
  • intervention
  • drug

Data availability statement

Data are available upon reasonable request.

https://doi.org/10.1136/jnis-2023-020170

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    Data availability statement

    Data are available upon reasonable request.

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    Footnotes

    • X @visishs

    • Contributors JHJ was responsible for the idea conception, data collection, data analysis, manuscript preparation, and all critical edits and reviews. HL, AN, and LS were responsible for data collection, data analysis, and manuscript preparation. All other authors were responsible for manuscript preparation including drafting and critical manuscript analysis/review. FCA and TAA were the senior authors on the manuscript and treated the patients who were studied in this manuscript. TA was responsible for the overall content as the guarantor.

    • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

    • Disclaimer The drugs described in the manuscript are currently not under Food and Drug Administration approval for the therapies studied.

    • Competing interests FCA is presently on the editorial board of the Journal of Neurointerventional Surgery.

    • Provenance and peer review Not commissioned; externally peer reviewed.

    • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.