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
The 15% mortality rate in our patients is slightly higher than that reported in developed countries. Delayed diagnosis and treatment is the main cause of avoidable deaths. Improving education of both clinicians and parents would increase survival rates in this potentially fatal disease.
Purpose:To compare the efficacy in pupil dilatation between a mixture containing 0.75% tropicamide and 2.5% phenylephrine and the alternate application of 1% tropicamide and 10% phenylephrine eye drops.Material and Methods:Patients requiring pupil dilatation were randomized to receive one drop of the mixture every 10 min for four times or our standard application of one drop of 1% tropicamide alternating with one drop of 10% phenylephrine every 10 min for two cycles. Pupil size was measured under bright light with the pupil gauge before, and every 5 min after initial application for 40 min. Application of the drops was discontinued once the pupil diameter reached 7 mm. Blood pressure and pulse rate were monitored every 15 min.Results:Of 40 patients (age 57.3±10.9 years, range 35-70 years), 22 were randomized into the mixture group and 18 into the alternate drug group. Baseline pupil sizes were 1.7±0.5 mm in the mixture group and 1.8±0.4 mm in the alternate drug group. The pupils were successfully dilated to 7 mm within 40 min in 17 patients of the mixture group compared to seven patients in the alternate drug group (P=0.004, Log Rank test). The mean pupil sizes at 40 min were 6.6±0.8 and 6.0±0.9 mm in the mixture and alternate drug groups respectively (P=0.050, t-test). Blood pressure and pulse rate were stable and similar in both groups.Conclusions:The mixture of 0.75% tropicamide and 2.5% phenylephrine is superior to our standard application of 1% tropicamide alternating with 10% phenylephrine. It provides faster and more successful pupil dilatation within 40 min.
In recent times, laser treatment combined with intravitreal bevacizumab (Avastin) has been used rarely as early investigations to treat patients with aggressive posterior retinopathy of prematurity (ROP). We describe choroidal ruptures that were documented at 10 weeks after this combined treatment in a preterm infant born at 28 weeks gestation with a birth weight of 1190 g. Aggressive posterior ROP was diagnosed at the age of 6 weeks. Initial treatment included bilateral laser ablation, followed immediately by intravitreal bevacizumab (IVB, 0.75 mg) in the right eye. On day 5, a remarkable regression of plus sign and fibrovascular proliferation was noted only in the right eye. At this point, additional laser treatment combined with IVB was given in the left eye, which dramatically resolved plus disease and neovascularization. At 10 weeks after IVB in the left eye, two sites of choroidal ruptures were revealed along the posterior edges of laser scars. There was no evidence of choroidal neovascularization associated with the choroidal ruptures. Choroidal rupture may occur shortly after laser ablation plus IVB treatment for aggressive posterior ROP. This occurrence requires recognition to determine whether it is actually related to the treatment. Long-term follow-up is important for understanding the nature and progression of this potentially serious complication.
Ocular application of 2% PVP-I was tolerable. This measure successfully relieved ocular discomfort from EKC in 3 quarters of the study group within a week. A randomized, controlled trial is required to verify the benefit of this measure.
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