Stem cell therapy for inherited retinal diseases: a systematic review and meta-analysis

Xiaodong Chen; Ningda Xu; Jiarui Li; Mingwei Zhao; Lvzhen Huang

doi:10.1186/s13287-023-03526-x

Stem cell therapy for inherited retinal diseases: a systematic review and meta-analysis

Stem Cell Res Ther. 2023 Oct 5;14(1):286. doi: 10.1186/s13287-023-03526-x.

Authors

Xiaodong Chen^{1

2

3}, Ningda Xu^{1

2

3}, Jiarui Li^{1

2

3}, Mingwei Zhao^{1

2

3}, Lvzhen Huang^{4

5

6}

Affiliations

¹ Department of Ophthalmology, Peking University People's Hospital, Eye Diseases and Optometry Institute, Beijing, China.
² Beijing Key Laboratory of Diagnosis and Therapy of Retinal and Choroid Diseases, Beijing, China.
³ College of Optometry, Peking University Health Science Center, Beijing, China.
⁴ Department of Ophthalmology, Peking University People's Hospital, Eye Diseases and Optometry Institute, Beijing, China. drlvzhenhuang@sina.com.
⁵ Beijing Key Laboratory of Diagnosis and Therapy of Retinal and Choroid Diseases, Beijing, China. drlvzhenhuang@sina.com.
⁶ College of Optometry, Peking University Health Science Center, Beijing, China. drlvzhenhuang@sina.com.

Abstract

Purpose: Stem cell therapy is a promising therapeutic approach for inherited retinal diseases (IRDs). This study aims to quantitatively examine the effectiveness and safety of stem cell therapy for patients with IRDs, including retinitis pigmentosa and Stargardt disease (STGD).

Methods: We searched PubMed, EMBASE, Web of Science, Cochrane Library databases, and the ClinicalTrials.gov website. The latest retrieval time was August 20, 2023. The primary outcomes were rates and mean difference (MD) of best-corrected visual acuity (BCVA) improvement. Subgroup analyses were conducted according to administration routes and stem cell types. This study was registered with PROSPERO (CRD42022349271).

Results: Twenty-one prospective studies, involving 496 eyes (404 RP and 92 STGD) of 382 patients (306 RP and 76 STGD), were included in this study. For RP, the rate of BCVA improvement was 49% and 30% at 6 months and 12 months, respectively, and the BCVA was significantly improved in the operative eyes at 6 months post-treatment (MD = - 0.12 logMAR, 95% CI .17 to - 0.06 logMAR; P < 0.001), while there was no significant difference at 12 months post-treatment (MD = -0.06 logMAR; 95% CI - 0.13 to 0.01 logMAR; P = 0.10). For STGD, the rate of BCVA improvement was 60% and 55% at 6 months and 12 months, respectively, and the BCVA was significantly improved in the operative eyes at 6 months (MD = - 0.14 logMAR, 95% CI - 0.22 to - 0.07 logMAR; P = 0.0002) and 12 months (MD = - 0.17 logMAR, 95% CI - 0.29 to - 0.04 logMAR; P = 0.01). Subgroup analyses showed suprachoroidal space injection of stem cells may be more efficient for RP. Eleven treated-related ocular adverse events from three studies and no related systemic adverse events were reported.

Conclusions: This study suggests stem cell therapy may be effective and safe for patients with RP or STGD. The long-term vision improvement may be limited for RP patients. Suprachoroidal space injection of stem cells may be a promising administration route for RP patients. Limited by the low grade of evidence, large sample size randomized clinical trials are required in the future.

Keywords: Inherited retinal diseases (IRD); Meta-analysis; Retinitis pigmentosa (RP); Stargardt disease (STGD); Stem cell.

Publication types

Meta-Analysis
Systematic Review
Review
Research Support, Non-U.S. Gov't

MeSH terms

Cell- and Tissue-Based Therapy
Choroidal Effusions*
Humans
Prospective Studies
Retinitis Pigmentosa* / genetics
Retinitis Pigmentosa* / therapy
Visual Acuity