Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa
| العنوان: | Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa |
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| المؤلفون: | Acharaporn Dambua, Areerat Khorchai, Adisak Trinavarat, Aekkachai Tuekprakhon, Phitchapa Pongpaksupasin, Patcharaporn Boonchu, Mongkol Uiprasertkul, Chonlada Yodtup, Panya Luksanapruksa, La-ongsri Atchaneeyasakul, Monchai Ruangchainikom, Somchai Sangkitporn, Aulia Rahmi Pawestri, Visit Vamvanij, Siripakorn Sangkitporn |
| المصدر: | Stem Cell Research & Therapy Stem Cell Research & Therapy, Vol 12, Iss 1, Pp 1-15 (2021) |
| بيانات النشر: | BioMed Central, 2021. |
| سنة النشر: | 2021 |
| مصطلحات موضوعية: | 0301 basic medicine, medicine.medical_specialty, medicine.medical_treatment, Retinitis pigmentosa (RP), Medicine (miscellaneous), Phases of clinical research, Disease, Mesenchymal Stem Cell Transplantation, Biochemistry, Genetics and Molecular Biology (miscellaneous), Transplantation, Autologous, Retina, lcsh:Biochemistry, 03 medical and health sciences, 0302 clinical medicine, Inherited retinal diseases, Internal medicine, Retinitis pigmentosa, medicine, Humans, lcsh:QD415-436, Adverse effect, Mesenchymal stem cell, lcsh:R5-920, Stem cell therapy, business.industry, Research, Mesenchymal Stem Cells, Cell Biology, Stem-cell therapy, medicine.disease, Transplantation, 030104 developmental biology, Intravitreal Injections, 030221 ophthalmology & optometry, Phase I clinical trial, Molecular Medicine, Stem cell, business, lcsh:Medicine (General), Retinitis Pigmentosa |
| الوصف: | 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 ClinicalTrials.gov, NCT01531348. Registered on February 10, 2012 |
| اللغة: | English |
| تدمد: | 1757-6512 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::934e497b8f2b4ea2efba87fbba2a9395 http://europepmc.org/articles/PMC7796606 |
| Rights: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....934e497b8f2b4ea2efba87fbba2a9395 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 17576512 |
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