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A systematic review of cellular therapies for the treatment of limbal stem cell deficiency affecting one or both eyes

Lookup NU author(s): Professor Francisco FigueiredoORCiD, Dr Jeffry Hogg, Arthur Okonkwo, Dr Gustavo Figueiredo, Professor Majlinda LakoORCiD


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© 2021 Elsevier Inc.Purpose: This systematic review (SR) assessed the efficacy, safety and cost-effectiveness of cell-based therapy to manage limbal stem cell deficiency (LSCD), a sight-threatening orphan condition most frequently associated with severe chemical or thermal burns. LSCD has historically been treated by transplanting limbal tissue. In 1997, a new treatment, cultured limbal epithelial autografts, was described for unilateral LSCD. In cases of bilateral disease cultured autologous oral mucosa stem cells have been used. The relative efficacy of different cultured tissue procedures is unknown. Methods: A protocol was registered with PROSPERO (CRD42017081117). Searches were conducted in 14 databases and 6 conference websites. Two reviewers independently selected studies, conducted data extraction and assessed risk of bias. One reviewer extracted individual patient data (IPD); a second checked extracted data. Data were assessed to determine the feasibility of statistical analysis, with Bayesian synthesis used to estimate improvement achieved by different treatments. Results: Fifty-two studies were eligible for inclusion (1113 eyes); 41 studies (716 eyes) reported IPD. No evidence was identified on cost-effectiveness. This SR was unable to confirm that any of the types of ex vivo cultured stem cell transplants identified for LSCD treatment were statistically superior when assessed against the outcomes of interest. Conclusions: We believe this SR is the first to include IPD analysis of LSCD data. There is no evidence for the superiority of any method of limbal stem cell transplant. Confirmation of the safety and efficacy of this treatment modality is challenging due to heterogeneity within and between the studies identified. Therefore, recommendations for future research are proposed.

Publication metadata

Author(s): Figueiredo FC, Glanville JM, Arber M, Carr E, Rydevik G, Hogg J, Okonkwo A, Figueiredo G, Lako M, Whiter F, Wilson K

Publication type: Review

Publication status: Published

Journal: Ocular Surface

Year: 2021

Volume: 20

Pages: 48-61

Print publication date: 01/04/2021

Online publication date: 04/01/2021

Acceptance date: 28/12/2020

ISSN (print): 1542-0124

ISSN (electronic): 1937-5913

Publisher: Elsevier Inc.


DOI: 10.1016/j.jtos.2020.12.008