Impact of highly effective CFTR modulator therapy on digital clubbing in patients with cystic fibrosis.

BACKGROUND: The association of certain disease processes with digital clubbing is well documented. Digital clubbing is often reversible after successful treatment of the underlying pathology, for example, after lung transplantation in patients with cystic fibrosis (CF). We examined the effect of highly effective Cystic Fibrosis Transmembrane Regulator (CFTR) modulators, defined for the purposes of this study as ivacaftor or the combination of ivacaftor, tezacaftor, and elexacaftor (ETI), on digital clubbing. MATERIALS AND METHODS: Clubbing index was measured on plaster of Paris casts of right index fingers obtained from 15 patients with cystic fibrosis, before and after initiation of CFTR modulator therapy. Similar measurements were made on casts for 9 cystic fibrosis patients who underwent lung transplantation. Measurements were made on the most recent casts available before treatment and the first cast available at least 3 months after initiation of treatment. The Wilcoxon signed-rank text was used to detect any significant difference in the pre- and post-treatment casts for each individual. RESULTS: A significant decrease in the clubbing index was found after both lung transplantation and treatment with highly effective CFTR modulator therapy. CONCLUSIONS: These results add to the body of evidence demonstrating the efficacy of highly effective CFTR modulator therapy, the first agents that act directly at the dysfunctional chloride channel responsible for CF. By demonstrating that CFTR modulator therapy is capable of reversing digital clubbing, this study suggests a beneficial effect on lung pathology aside from air flow and gas transfer.

Pediatric Non-Refractive Vision Screening with EyeSwift, PDI Check and Blinq: Non-Refractive Vision Screening with Two Binocular Video Games and Birefringent Scanning.

BACKGROUND: The purpose of this paper is to validate three recent non-refractive devices that target sensory and/or strabismus risk factors for amblyopia. EyeSwift® with eye tracking and PDI Check on autostereoscopic Nintendo 3DS are video games that estimate acuity, binocularity and color. The Rebion blinq assesses foveation by birefringence. METHODS: Students were consented to be screened by each device as if by a school nurse before confirmatory examination with optimized, refined refraction. Results were compared with correlation and Bland Altman plots while screening ability was compared with 2021 AAPOS Guidelines and the strabismus-amblyopia rubric proposed by David Hunter. RESULTS: A total of 77 ethnically diverse students, aged 11±4 (4-19) years had a 77% prevalence of 2021 risk factors. Near visual acuity, inter-eye difference and stereopsis correlated significantly (p<0.01) for the EyeSwift® (r2 0.14, 0.06 and 0.45) and PDI Check (r2 0.23, 0.22 and 0.32). The sensitivity/specificity to target 2021-AAPOS: Hunter-rubric for EyeSwift® was 82%/56%: 74%/62%, for PDI Check was 75/64%: 69%/69% and for blinq 75/48%: 82%/39%. Sensitivity/specificity for the seven color-deficient students was 86%/84% for EyeSwift® and 100%/81% for PDI Check. Screening time was 96±19 s for PDI Check and 375±102 s for EyeSwift but less than 25 s for blinq. CONCLUSION: Reliable near visual acuity, stereo and color testing were obtained from the two binocular and touch screen devices often with minimal coaching of the student. The blinq rapidly gives an estimate of whether both eyes are steadily fixing, and the ProVersion predicted 100% which eye is deviated or amblyopic. Non-refractive validation metrics were beneficial.