Creating an automated contemporaneous cohort in sickle cell anemia to predict survival after disease-modifying therapy.
Creating an automated contemporaneous cohort in sickle cell anemia to predict survival after disease-modifying therapy.
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DOI：
10.1182/bloodadvances.2022008692
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发表时间：
2023-08-08
期刊：
影响因子：
7.5
通讯作者：
Debaun, Michael R.
中科院分区：
文献类型：
--
作者：
Cronin, Robert M.;Wuichet, Kristin;Ghafuri, Djamila L.;Hodges, Brock;Chopra, Maya;He, Jing;Niu, Xinnan;Kassim, Adetola A.;Wilkerson, Karina;Rodeghier, Mark;Debaun, Michael R.
为了估计生存率，对患有SCA的儿童和成人进行自动同期队列研究是可行和有效的。与不进行疾病改善治疗相比，羟基脲治疗至少1年可提高SCA成人患者的生存率。美国食品和药物管理局要求对接受实验性基因治疗或基因编辑临床试验的参与者进行同期对照，以比较临床结果。然而，建立一个罕见疾病的同期队列需要多个人小时。在镰状细胞病的单一转诊中心，我们测试了假设，我们可以创建一个自动的镰状细胞性贫血（SCA）儿童和成人同期队列来预测死亡率。数据于2004年1月1日至2021年4月30日期间获得。我们确定了419名SCA患者，他们接受了一致的医疗护理，定义为连续随访100年至0.5年，无随访间隔100年至3.0年。中位年龄为10.2岁（IQR， 1-24岁），中位随访时间为7.4年（IQR, 3.6-13.5年），47例死亡。共有98%（277名儿童中有274名）的儿童在18岁时仍然存活，其中34.3%（274名儿童中有94名）的儿童被随访至成年。成人的中位生存年龄为49.3岁。治疗组相互排斥，并按等级顺序排列：造血干细胞移植（n = 22）； >定期输血至少2年（n = 56）； >羟基脲至少1年（n = 243）； >无疾病改善治疗（n = 98）。羟基脲治疗组与未治疗组相比，死亡率风险显著降低（风险比= 0.38,P = 0.016），但定期输血组与未治疗组相比，死亡率风险无统计学差异（风险比= 0.71,P = 0.440）。可以生成一个自动同步SCA队列来估计患有SCA的儿童和成人的死亡率。
To estimate survival, an automated contemporaneous cohort of children and adults with SCA is feasible and efficient. Hydroxyurea therapy for at least 1 year is associated with increased survival in adults with SCA compared with no disease-modifying therapy. The Food and Drug Administration requires contemporaneous controls to compare clinical outcomes for participants receiving experimental gene therapy or gene editing clinical trials. However, developing a contemporaneous cohort of rare diseases requires multiple person-hours. In a single referral center for sickle cell disease, we tested the hypothesis that we could create an automated contemporaneous cohort of children and adults with sickle cell anemia (SCA) to predict mortality. Data were obtained between 1 January 2004 and 30 April 2021. We identified 419 individuals with SCA with consistent medical care defined as followed continuously for >0.5 years with no visit gaps >3.0 years. The median age was 10.2 years (IQR, 1-24 years), with a median follow-up of 7.4 years (IQR, 3.6-13.5 years) and 47 deaths. A total of 98% (274 of 277) of the children remained alive at 18 years of age, and 34.3% (94 of 274) of those children were followed into adulthood. For adults, the median age of survival was 49.3 years. Treatment groups were mutually exclusive and in a hierarchical order: hematopoietic stem cell transplant (n = 22)>regular blood transfusion for at least 2 years (n = 56)>hydroxyurea for at least 1 year (n = 243)>no disease-modifying therapy (n = 98). Compared to those receiving no disease-modifying treatment, those treated with hydroxyurea therapy had a significantly lower hazard of mortality (hazard ratio = 0.38; P = 0.016), but no statistical difference for those receiving regular blood transfusions compared to no disease-modifying therapy (hazard ratio = 0.71; P = 0.440). An automated contemporaneous SCA cohort can be generated to estimate mortality in children and adults with SCA.
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影响因子：
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作者：
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