METHODS: Age, sex, and visual acuity were recorded and spectral domain OCT and ultra-wide-field images of the macula and retina were reviewed in a consecutive series of 74 adults with sickle cell disease.
RESULTS: The median age was 37 years (range 19-73 years) and 36 cases (48.6%) were male. SCM was present in at least 1 eye of 40 cases (54.1%) or in 67 of all eyes (42.3%). SCM prevalence was 54.8%, 62.5%, and 25% for the HbSS, HbSC, and HbS/BThal or other genotypes, respectively. SCM was observed in 41 (39.4%) of the eyes with PSR stages 0, 1, and 2, and in 21 (51.2%) of the eyes with PSR stages 3, 4, and 5, respectively. Mild visual impairment or worse was present in 3 eyes (4.8%) with SCM but this was secondary to other pathology.
CONCLUSION: SCM is a frequent finding in the eyes of adults with sickle cell disease. The prevalence is similar for the HbSS and HbSC genotypes and is not related to the PSR stage. High-contrast distance visual acuity is typically preserved.
DESIGN: Recommendations from a working group of international experts in macular degeneration outcomes registry development and patient advocates, facilitated by the International Consortium for Health Outcomes Measurement (ICHOM).
METHODS: Modified Delphi technique, supported by structured teleconferences, followed by online surveys to drive consensus decisions. Potential outcomes were identified through literature review of outcomes collected in existing registries and reported in major clinical trials. Outcomes were refined by the working group and selected based on impact on patients, relationship to good clinical care, and feasibility of measurement in routine clinical practice.
RESULTS: Standardized measurement of the following outcomes is recommended: visual functioning and quality of life (distance visual acuity, mobility and independence, emotional well-being, reading and accessing information); number of treatments; complications of treatment; and disease control. Proposed data collection sources include administrative data, clinical data during routine clinical visits, and patient-reported sources annually. Recording the following clinical characteristics is recommended to enable risk adjustment: age; sex; ethnicity; smoking status; baseline visual acuity in both eyes; type of macular degeneration; presence of geographic atrophy, subretinal fibrosis, or pigment epithelial detachment; previous macular degeneration treatment; ocular comorbidities.
CONCLUSIONS: The recommended minimum outcomes and pragmatic reporting standards should enable standardized, meaningful assessments and comparisons of macular degeneration treatment outcomes. Adoption could accelerate global improvements in standardized data gathering and reporting of patient-centered outcomes. This can facilitate informed decisions by patients and health care providers, plus allow long-term monitoring of aggregate data, ultimately improving understanding of disease progression and treatment responses.