Xing Shan, Bullano Michael, Hale Sarah, Lokhandwala Tasneem, DeYoung Kathryn, Murty Sharanya
Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA, USA.
Cencora, Conshohocken, PA, USA.
J Med Econ. 2024 Jan-Dec;27(1):1086-1098. doi: 10.1080/13696998.2024.2391663. Epub 2024 Aug 22.
Thrombotic thrombocytopenic purpura (TTP) is an ultra-rare blood disorder, characterized by severe ADAMTS13 deficiency. Affected individuals present with potentially life-threatening acute events and may experience sub-acute and chronic TTP manifestations often resulting in long-term organ damage. Incremental symptom prevalence before, during, and after an acute event as well as healthcare resource utilization (HCRU) and costs during and after an acute event were compared between people with TTP and matched non-TTP controls.
This retrospective, matched study used data from Merative MarketScan Commercial Database and Medicare Supplemental Database (from January 1, 2008, through September 30, 2021) to identify people with TTP (inpatient diagnosis for "thrombotic microangiopathy (TMA)" or "congenital TTP," and ≥1 claim for plasma exchange or infusion). People with TTP were matched (1:2) with non-TTP controls on age, sex, geographic region, index year, and select Elixhauser comorbidities.
255 people with TTP were matched with 510 non-TTP controls. Both cohorts had a mean age of 43.9 years; 71% were female. Overall, more people with TTP reported symptoms compared with non-TTP controls prior to (51% vs 43%), during (99% vs 52%), and after an acute event (85% vs 50%; < 0.05 for all periods). Symptom prevalence decreased following an acute event compared with during an acute event, but remained high-85% of people with TTP experienced symptoms compared with 50% of non-TTP controls. HCRU and mean costs per patient per month were significantly higher in all care settings among people with TTP compared with non-TTP controls ( < 0.05).
Identification of patient populations may have been limited due to coding errors, as the data were obtained from an administrative claims database.
TTP is associated with a substantial symptom burden and increased costs and HCRU during and up to almost a year after acute events, demonstrating the longitudinal burden of this disease.
血栓性血小板减少性紫癜（TTP）是一种极为罕见的血液疾病，其特征为严重的ADAMTS13缺乏。受影响个体出现可能危及生命的急性事件，并且可能经历亚急性和慢性TTP表现，常导致长期器官损害。比较了TTP患者与匹配的非TTP对照在急性事件之前、期间和之后症状发生率的增加情况，以及急性事件期间和之后的医疗资源利用（HCRU）和费用。
这项回顾性匹配研究使用了来自默克多市场扫描商业数据库和医疗保险补充数据库（从2008年1月1日至2021年9月30日）的数据，以识别TTP患者（住院诊断为“血栓性微血管病（TMA）”或“先天性TTP”，且有≥1次血浆置换或输注索赔）。TTP患者在年龄、性别、地理区域、索引年份和选定的埃利克斯豪泽合并症方面与非TTP对照进行匹配（1:2）。
255名TTP患者与510名非TTP对照进行了匹配。两个队列的平均年龄均为43.9岁；71%为女性。总体而言，与非TTP对照相比，更多的TTP患者在急性事件之前（51%对43%）、期间（99%对52%）和之后（85%对50%；所有时期P<0.05）报告有症状。与急性事件期间相比，急性事件后症状发生率有所下降，但仍居高不下——85%的TTP患者有症状，而非TTP对照为50%。与非TTP对照相比，TTP患者在所有护理环境中的HCRU和每位患者每月的平均费用均显著更高（P<0.05）。
由于数据来自行政索赔数据库，编码错误可能限制了患者群体的识别。
TTP与严重的症状负担以及急性事件期间及之后直至近一年的费用增加和HCRU增加相关，表明了这种疾病的长期负担。
