Background: Limited cost-effectiveness studies have evaluated the long-term efficacy of subcutaneous immunotherapy (SCIT).

Objective: This study's objective was to analyze the cost-effectiveness of house dust mite-specific SCIT (HDM-SCIT), including post-SCIT efficacy, in allergic rhinitis (AR) patients.

Methods: In this retrospective study, the post-treatment period ranged from 1 year to 13 years. The costs included the direct cost of HDM-SCIT, and the actual AR treatment cost estimated by the patients at different follow-up stages.

Results: The median annual cost of AR treatment before, during, and after HDMSCIT was 1,000, 6,000, and 300 RMB/year, respectively. A moderate negative correlation was found between the post-SCIT period and the CUR of SCIT with longterm efficacy (r = -0.545, p < 0.001). The combined long-term efficacy CUR of SCIT within 5 years (including 5 years) post-SCIT was significantly higher than that in the pre-SCIT period. However, the subgroups that had completed SCIT for 6 years or longer showed no difference. For HDM-SCIT in China, the direct ICER (18,269 RMB/QALY) and actual ICER (19,549 RMB/QALY) were both lower than the average per capita gross domestic product (GDP) from 2005 to 2020 (41,469 RMB/year).

Conclusion: HDM-SCIT is highly cost-effective in treating AR in China. The addition of SCIT to pharmacotherapy can further improve the quality of life in patients with AR. For patients who gained long-term efficacy of HDM-SCIT for 6 years or more, the application of SCIT would not increase the actual costs of AR treatment.

Background: Stem cell transplantation is a promising therapy for degenerative retinal diseases, including age-related macular degeneration (AMD), retinitis pigmentosa (RP), and Stargardt's disease (STGD). This study quantitatively evaluates long-term outcomes of different stem cell treatments.

Methods: A systematic review and outcome prediction analysis were conducted using data published before September 1, 2024. The primary outcome was the change in best-corrected visual acuity (BCVA), expressed as logarithm of the minimum angle of resolution (LogMAR), adjusted for baseline BCVA. Predictions were disease-specific for dry AMD, wet AMD, RP, and STGD, refined through regression analysis while ensuring data standardization across studies.

Results: A meta-analysis of 43 studies (666 eyes) revealed varying BCVA improvements at 6 months post-treatment. For dry AMD, adipose-derived mesenchymal stem cells (ADMSCs) showed the best improvement (0.65, 95% CI: 0.57-0.72 logMAR), exceeding that of human embryonic stem cells (hESCs, 0.49, 95% CI: 0.33-0.65 logMAR). For wet AMD, hESCs improved BCVA by 0.45 logMAR (95% CI: 0.28-0.61 logMAR). For RP, Wharton's jelly-derived mesenchymal stem cells (WJMSCs) achieved the highest improvement (0.50, 95% CI: 0.28-0.73 logMAR), while umbilical cord-derived mesenchymal stem cells (UCMSCs, 0.39, 95% CI: 0.31-0.46 logMAR) showed moderate effects. For STGD, ADMSCs were most effective (0.53, 95% CI: 0.42-0.64 logMAR).

Conclusions: Stem cell therapy demonstrates significant potential for treating AMD, RP, and STGD, with ADMSCs showing superior benefits in dry AMD and STGD, hESCs being more effective in wet AMD, and WJMSCs outperforming other therapies in RP. These comparative insights highlight disease-specific advantages of different stem cell sources, informing future clinical applications.

Background: Age-related macular degeneration (AMD) and diabetic retinopathy (DR) are two prevalent retinal neovascular diseases globally. Although observational studies have suggested a link between micronutrients and both AMD and DR, the causal relationship remains unconfirmed.

Methods: The causal estimation was performed using the Mendelian randomization (MR) method. Single-nucleotide polymorphisms (SNPs) for circulating micronutrient levels were sourced from published genome-wide association studies (GWAS). Data for AMD and DR GWAS were obtained from the FinnGen consortium. The inverse variance weighted (IVW) method served as the primary MR analysis, with sensitivity analyses conducted to validate the MR hypothesis. Additionally, a reverse MR analysis was performed to assess the potential influence of reverse causality in our MR study.

Results: The MR analysis using the IVW method indicated that magnesium is associated with a reduced risk of AMD (OR: 0.687, 95% CI: 0.512-0.921, p = 0.012) and wet AMD (OR: 0.640, 95% CI: 0.433-0.944, p = 0.025). Furthermore, vitamin B6 is linked to a decreased risk of DR (OR: 0.754, 95% CI: 0.574-0.990, p = 0.042). The reverse MR analysis did not detect any reverse causality.

Conclusions: This study found that genetically determined serum magnesium is associated with a reduced risk of AMD, and genetically determined serum vitamin B6 is associated with a decreased risk of DR. These findings suggest that magnesium and vitamin B6 could be potential therapeutic targets for both AMD and DR, respectively.

Background: Neurotrophic keratopathy (NK) is a rare degenerative corneal disease characterized by reduced corneal sensitivity and impaired epithelial healing, frequently resulting in persistent epithelial defects and corneal ulcers. Conventional therapies provide symptomatic relief but do not restore corneal innervation, highlighting a substantial unmet clinical need.

Objective: To evaluate the efficacy and safety of cenegermin, a recombinant human nerve growth factor, in patients with stage 2 and stage 3 NK.

Methods: This prospective, single-arm, open-label clinical trial enrolled eight patients who received cenegermin (20 mcg/mL) administered six times daily for 8 weeks, with a 48-week follow-up period. Corneal ulcer area, corneal sensitivity, and nerve parameters were assessed throughout the study.

Results: By week 8, 75% of patients achieved corneal healing, increasing to 100% by week 56. Ulcer area significantly decreased (-5.32 mm² at week 8), central corneal sensitivity improved by 22.5 mm, and main nerve density increased by 3.1 mm/mm² at week 8. The most frequently reported adverse events were mild eye pain, foreign body sensation, and tearing.

Conclusions: Cenegermin demonstrated substantial efficacy and a favorable safety profile in patients with advanced NK. It was effective, safe, and well tolerated, presenting a promising therapeutic option for NK.