2206. The global blood donation index: an imperfect measure of transfusion need.
作者: Jeremy W Jacobs.;Imelda Bates.;Claudia S Cohn.;Nabajyoti Choudhury.;Shirley Owusu-Ofori.;Hans Vrielink.;Eshan U Patel.;Silvano Wendel.;Aaron A R Tobian.;Evan M Bloch.
来源: Lancet. 2024年404卷10456期988-990页
The optimum number of units of blood and the associated number of blood donors required to meet a given population's needs remain undetermined globally. Typically, a whole blood donation rate of ten donations per 1000 population, at a minimum, is necessary to meet a country's blood needs. This rate is attributed to a WHO recommendation that 1% of a given country's population should donate blood to ensure a blood supply that is sufficient to meet clinical needs. This often cited metric was first referenced in a 1971 WHO report, yet neither supporting data or references were provided, suggesting that it was flawed at its founding. Regardless, this metric does not provide an accurate or contemporary determination of blood needs, which has ramifications for health service provision and planning, particularly in low-income and lower-middle-income countries. Modelling studies that account for geographical variability in disease burden, health-care infrastructure, and transfusion practices are needed to accurately estimate blood needs. A paucity of data to inform modelling remains a major obstacle in this regard. We discuss the history of the global blood donation index and highlight some factors that should be considered to better understand contemporary blood needs.
2208. Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
作者: Paul Yang.;Laura P Pardon.;Allen C Ho.;Andreas K Lauer.;Dan Yoon.;Shannon E Boye.;Sanford L Boye.;Alejandro J Roman.;Vivian Wu.;Alexandra V Garafalo.;Alexander Sumaroka.;Malgorzata Swider.;Iryna Viarbitskaya.;Tomas S Aleman.;Mark E Pennesi.;Christine N Kay.;Kenji P Fujita.;Artur V Cideciyan.
来源: Lancet. 2024年404卷10456期962-970页
Leber congenital amaurosis 1 (LCA1), caused by mutations in GUCY2D, is a rare inherited retinal disease that typically causes blindness in early childhood. The aim of this study was to evaluate the safety and preliminary efficacy of ascending doses of ATSN-101, a subretinal AAV5 gene therapy for LCA1.
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