Department of Pediatric Hematology/Oncology, ProMedica Russell J. Ebeid Children's Hospital, Toledo, OH, USA.
Department of Women, Child and General and Specialized Surgery, University of Campania "Luigi Vanvitelli", Naples, Italy.
Blood Cells Mol Dis. 2021 Dec;92:102612. doi: 10.1016/j.bcmd.2021.102612. Epub 2021 Sep 21.
Sickle cell disease (SCD) is characterized by frequent, unpredictable pain episodes and other vaso-occlusive crises (VOCs) leading to significant healthcare utilization. VOC frequency is often an endpoint in clinical trials investigating novel therapies for this devastating disease.
The Consortium for the Advancement of Sickle Cell Research (CASiRe) is an international collaboration investigating clinical severity in SCD using a validated questionnaire and medical chart review standardized across four countries (United States, United Kingdom, Italy and Ghana).
This study, focused on pain crisis incidence and healthcare utilization, included 868 patients, equally represented according to age and gender. HgbSS was the most common genotype. Patients from Ghana used the Emergency Room/Day Hospital for pain more frequently (annualized mean 2.01) than patients from other regions (annualized mean 1.56 U.S.; 1.09 U.K.; 0.02 Italy), while U.K. patients were hospitalized for pain more often (annualized mean: U.K. 2.98) than patients in other regions (annualized mean 1.98 U.S.; 1.18 Ghana; Italy 0.54). Italy's hospitalization rate for pain (annualized mean: 0.57) was nearly 20 times greater than its emergency room/day hospital only visits for pain (annualized mean: 0.03). When categorized by genotype and age, similar results were seen.
Geographic differences in pain crisis frequency and healthcare utilization may correlate with variable organization of healthcare systems among countries and should be considered regarding trial design, endpoints, and analysis of results when investigating novel agents for clinical benefit.
镰状细胞病(SCD)的特征是频繁、不可预测的疼痛发作和其他血管阻塞性危象(VOC),导致大量的医疗保健利用。VOC 频率通常是评估新型疗法治疗这种破坏性疾病的临床试验终点。
镰状细胞研究进展联盟(CASiRe)是一个国际合作组织,使用经过验证的问卷和在四个国家(美国、英国、意大利和加纳)标准化的医疗图表审查来研究 SCD 的临床严重程度。
这项侧重于疼痛危机发生率和医疗保健利用的研究纳入了 868 名患者,按照年龄和性别均衡代表。HgbSS 是最常见的基因型。加纳患者因疼痛而使用急诊室/日间医院的频率更高(年化平均值 2.01),而其他地区的患者(年化平均值美国 1.56;英国 1.09;意大利 0.02),而英国患者因疼痛住院的频率更高(年化平均值:英国 2.98),而其他地区的患者(年化平均值美国 1.98;加纳 1.18;意大利 0.54)。意大利因疼痛住院的比率(年化平均值:0.57)几乎是因疼痛急诊室/日间医院就诊的 20 倍(年化平均值:0.03)。按基因型和年龄分类,也观察到类似的结果。
疼痛危机频率和医疗保健利用的地理差异可能与各国医疗保健系统的不同组织方式有关,在研究新型药物的临床获益时,应考虑到试验设计、终点和结果分析。