June 2023
Volume 64, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2023
A Phase II, prospective, single centre, single arm interventional trial to evaluate the safety and effectiveness of human ex vivo expanded autologous limbal stem cells transplantation for the treatment of unilateral total limbal stem cell deficiency
Author Affiliations & Notes
  • Francisco C Figueiredo
    Ophthalmology & Biosciences Institute, Newcastle University Faculty of Medical Sciences, Newcastle upon Tyne, Newcastle upon Tyne, United Kingdom
    Ophthalmology, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom
  • Oliver Baylis
    Biosciences Institute, Newcastle University Institute of Genetic Medicine, Newcastle upon Tyne, United Kingdom
    Ophthalmology, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom
  • Hardeep Mudhar
    Histopathology, Royal Hallamshire Hospital Histopathology Training, Sheffield, United Kingdom
  • Majlinda Lako
    Biosciences Institute, Newcastle University Institute of Genetic Medicine, Newcastle upon Tyne, United Kingdom
  • Gustavo Figueiredo
    Biosciences Institute, Newcastle University Institute of Genetic Medicine, Newcastle upon Tyne, United Kingdom
    Ophthalmology, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom
  • Footnotes
    Commercial Relationships   Francisco Figueiredo None; Oliver Baylis None; Hardeep Mudhar None; Majlinda Lako None; Gustavo Figueiredo None
  • Footnotes
    Support  MRC UK - G0900879; GRANT ID: 91481
Investigative Ophthalmology & Visual Science June 2023, Vol.64, 2358. doi:
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      Francisco C Figueiredo, Oliver Baylis, Hardeep Mudhar, Majlinda Lako, Gustavo Figueiredo; A Phase II, prospective, single centre, single arm interventional trial to evaluate the safety and effectiveness of human ex vivo expanded autologous limbal stem cells transplantation for the treatment of unilateral total limbal stem cell deficiency. Invest. Ophthalmol. Vis. Sci. 2023;64(8):2358.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Examination of patient outcomes over a 36-month period following autologous ex vivo cultivated limbal epithelial transplant (CLET) in patients with unilateral total limbal stem cell deficiency (LSCD).

Methods : A Phase II, 3-year, prospective, single-centre, non-comparative, interventional case series was conducted in a tertiary hospital in the UK. Consecutive adult patients with acquired and unilateral total LSCD, primarily due to ocular surface burns underwent autologous CLET and were followed up over 36 months. Patients with poor visual acuity and successful transplantation outcomes underwent penetrating keratoplasty (PKP) after 12 months post-CLET. The co-primary endpoint comprised reversal of LSCD at 6 months, corneal surface reconstruction and improvement in ocular pain at 12 months.

Results : Overall, 23 patients received CLET and were included in the treatment (TRT) group. Ocular surface reconstruction, including corneal impression cytology, at 6, 12 and 36 months, revealed LSCD reversal in 23 (100%), 22 (95.7%) and 18 (78.3%) patients respectively. Corneal neovascularisation was reduced from baseline in 20 (87.0%) patients at 6 months and 19 (82.6%) patients at months 12 and 36. Ocular pain scores were significantly reduced from baseline to 36 months (p = 0.02). One serious (perforation of corneal ulcer) and 10 non-serious adverse events (mainly mild) were related to the study procedure.

Conclusions : Autologous CLET demonstrated efficacy and safety in ocular surface reconstruction in LSCD due to ocular burns. Pain scores were also significantly improved. These data support the use of autologous CLET in preparation for PKP to improve visual acuity when required. Given the small number of patients with this rare disease, more patients are needed to further support our results.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.

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