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Title: Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa
Authors: Aekkachai Tuekprakhon
Siripakorn Sangkitporn
Adisak Trinavarat
Aulia Rahmi Pawestri
Visit Vamvanij
Monchai Ruangchainikom
Panya Luksanapruksa
Phitchapa Pongpaksupasin
Areerat Khorchai
Acharaporn Dambua
Patcharaporn Boonchu
Chonlada Yodtup
Mongkol Uiprasertkul
Somchai Sangkitporn
La ongsri Atchaneeyasakul
Brawijaya University
Faculty of Medicine Siriraj Hospital, Mahidol University
National Institutes of Health (NIH)
Keywords: Biochemistry, Genetics and Molecular Biology;Medicine
Issue Date: 1-Dec-2021
Citation: Stem Cell Research and Therapy. Vol.12, No.1 (2021)
Abstract: Background: Retinitis pigmentosa (RP) is a progressive inherited retinal disease with great interest for finding effective treatment modalities. Stem cell-based therapy is one of the promising candidates. We aimed to investigate the safety, feasibility, and short-term efficacy of intravitreal injection of bone marrow-derived mesenchymal stem cells (BM-MSCs) in participants with advanced stage RP. Methods: This non-randomized phase I clinical trial enrolled 14 participants, categorized into three groups based on a single dose intravitreal BM-MSC injection of 1 × 106, 5 × 106, or 1 × 107 cells. We evaluated signs of inflammation and other adverse events (AEs). We also assessed the best corrected visual acuity (BCVA), visual field (VF), central subfield thickness (CST), and subjective experiences. Results: During the 12-month period, we noticed several mild and transient AEs. Interestingly, we found statistically significant improvements in the BCVA compared to baseline, although they returned to the baseline at 12 months. The VF and CST were stable, indicating no remarkable disease progression. We followed 12 participants beyond the study period, ranging from 1.5 to 7 years, and observed one severe but manageable AE at year 3. Conclusion: Intravitreal injection of BM-MSCs appears to be safe and potentially effective. All adverse events during the 12-month period required observation without any intervention. For the long-term follow-up, only one participant needed surgical treatment for a serious adverse event and the vision was restored. An enrollment of larger number of participants with less advanced RP and long-term follow-up is required to evaluate the safety and efficacy of this intervention. Trial registration:, NCT01531348. Registered on February 10, 2012.
ISSN: 17576512
Appears in Collections:Scopus 2021

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