Division of Hematology & Oncology, Department of Medicine, University of Illinois at Chicago, Chicago, Illinois, United States of America.
Department of Pharmacy Practice, College of Pharmacy, University of Illinois at Chicago, Chicago, Illinois, United States of America.
PLoS One. 2020 Feb 26;15(2):e0229710. doi: 10.1371/journal.pone.0229710. eCollection 2020.
Patients with sickle cell disease (SCD) have access to fewer health care resources and therapies compared to other diseases, which contributes to increased morbidity and health care utilization. We compared health care utilization (inpatient hospital days, emergency care visits) and health care-related costs between SCD adults that underwent hematopoietic stem cell transplantation (HSCT) using a nonmyeloblative conditioning regimen versus those referred for HSCT but did not proceed due to lack of an HLA-matched sibling donor, denial by insurance, red blood cell antibodies to the potential donor, or declining further evaluation. Between 8/2011 and 4/2016, 83 SCD patients were referred for allogeneic HSCT and 16 underwent the procedure. The HSCT and non-HSCT groups were similar by age, sex, prior SCD-related therapy and complications. Compared to pre HSCT, significantly fewer inpatient hospital days (median of 1 versus 22 days, P = 0.003) and emergency care visits (median of 1 versus 4 visits, P = 0.04) were observed by the 2nd year post-HSCT. Similar results were observed in comparison to the standard-of-care group (median of 1 versus 12 hospital days, P = 0.002; median of 1 versus 3 emergency visits, P = 0.03). Lower health care costs were observed by the 2nd year post-HSCT (median of $16,281 versus $64,634 pre-HSCT (P = 0.01) and versus $54,082 in the standard-of-care group (P = 0.05). A median reduction of -$20,833/patient/year (IQR, -$67,078-+$4,442/patient/year) in health care costs compared to pre-HSCT was observed in the 2nd year post-HSCT. In conclusion, allogeneic HSCT leads to improvements in health care utilization and costs compared to standard-of-care therapy in high-risk SCD adults.
与其他疾病相比，镰状细胞病（SCD）患者获得的医疗资源和治疗方法较少，这导致发病率和医疗保健利用率增加。我们比较了接受非清髓性预处理方案造血干细胞移植（HSCT）的 SCD 成人与因缺乏 HLA 匹配的同胞供体、保险拒绝、潜在供体的红细胞抗体或拒绝进一步评估而未进行 HSCT 的患者的医疗保健利用（住院天数、急诊就诊）和与医疗保健相关的费用。在 2011 年 8 月至 2016 年 4 月期间，有 83 名 SCD 患者被转介接受异基因 HSCT，其中 16 名患者接受了该手术。HSCT 和非-HSCT 组在年龄、性别、既往 SCD 相关治疗和并发症方面相似。与 HSCT 前相比，HSCT 后第 2 年的住院天数（中位数为 1 天与 22 天，P = 0.003）和急诊就诊次数（中位数为 1 次与 4 次，P = 0.04）明显减少。与标准治疗组相比，也观察到类似的结果（中位数为 1 天与 12 天住院天数，P = 0.002；中位数为 1 次与 3 次急诊就诊，P = 0.03）。HSCT 后第 2 年观察到医疗保健费用降低（中位数为 16281 美元与 HSCT 前的 64634 美元（P = 0.01）和标准治疗组的 54082 美元（P = 0.05）。与 HSCT 前相比，HSCT 后第 2 年观察到每位患者每年的医疗保健费用中位数降低了-20833 美元（IQR，-67078 美元至+4442 美元/患者/年）。与标准治疗相比，高危 SCD 成人接受异基因 HSCT 可改善医疗保健利用率和成本。
