Outcomes of older patients with primary central nervous system lymphoma treated in routine clinical practice in the UK: methotrexate dose intensity correlates with response and survival.

Data on older patients with primary central nervous system lymphoma (PCNSL) are scarce. Comorbidities and performance status frequently compromise outcomes in this group. Medical records for consecutive patients ≥65 years (n = 244) with PCNSL diagnosed 2012-2017 from 14 UK centres were retrospectively reviewed.

Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65.

The authors originally published this article under the incorrect license type; this has now been corrected and is published under the CC-BY license.

Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65.

Leber congenital amaurosis is a group of inherited retinal dystrophies that cause severe sight impairment in childhood; RPE65-deficiency causes impaired rod photoreceptor function from birth and progressive impairment of cone photoreceptor function associated with retinal degeneration. In animal models of RPE65 deficiency, subretinal injection of recombinant adeno-associated virus (AAV) 2/2 vectors carrying RPE65 cDNA improves rod photoreceptor function, and intervention at an early stage of disease provides sustained benefit by protecting cone photoreceptors against retinal degeneration. In affected humans, administration of these vectors has resulted to date in relatively modest improvements in photoreceptor function, even when retinal degeneration is comparatively mild, and the duration of benefit is limited by progressive retinal degeneration.

Does left ventricular diastolic dysfunction progress through stages? Insights from a community heart failure study.

Unlabelled: We performed a retrospective pilot study on a group of symptomatic patients attending our community heart failure clinic with left ventricular diastolic dysfunction (LVDD), rising or elevated LV end diastolic pressure, elevated brain natriuretic peptide (BNP), but with no clinical or radiographic evidence of heart failure; a group we hypothesised may be in the pre-HFPEF stage.

Methods: Those with LVEF >45% and LV diastolic dysfunction were included and divided into two groups: E/e' <15 and E/e' ≥15 corresponding with rising and raised LVEDP, respectively. Clinical events (deaths and hospital admissions) were compared at 1year and were grouped into all-cause events or cardiovascular events.