CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Estimating the proportion of transplant-eligible patients in diffuse large b-cell lymphoma (DLBCL): Insights from German hospital billing data.
Estimating the proportion of transplant-eligible patients in diffuse large b-cell lymphoma (DLBCL): Insights from German hospital billing data.
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本分析介绍了一种利用真实世界住院账单数据,近似估计在 2L 背景下拟接受 HD-ASCT 的 DLBCL 患者中移植资格分布的方法。结果对广泛假设的移植合格组与不合格组之间的平衡提出了质疑,表明可能存在移植合格病例比例略高的倾向,这些病例可能在临床上适合接受 HD-ASCT。
根据指南对弥漫性大B细胞淋巴瘤(DLBCL)二线及以后(2L+)治疗的建议,需区分适合移植和不适合移植的患者。适合移植的患者通常接受挽救性免疫化疗,随后进行大剂量化疗和自体干细胞移植(HD-ASCT)或CAR-T 细胞治疗。尽管文献提示适合移植与不适合移植的患者分布为50-50,但来自德国的真实世界证据尚缺乏。本研究旨在利用用于HD-ASCT的国家住院结算数据,估算适合移植和不适合移植患者的比例,以评估通常假设分布的有效性。
分析了由医院薪酬系统研究所(InEK)提供的2024年全德国医院住院账单数据。以主要诊断为“C83.3”(DLBCL)的病例定义基础人群。应用基于指南的一线治愈率(60%[最低]-70%[最高]),估算2L人群规模。通过挽救性免疫化疗(R-ICE、R-DHAP、R-GDP)的OPS编码识别适合移植的病例。适合移植的比例通过将适合移植的病例数除以估算的2L人群计算得出。使用2022年和2023年的数据进行敏感性分析,以德国真实世界索赔数据验证2L复发/难治率假设,并评估polatuzumab vedotin相关编码偏倚的潜在影响。
基础人群包括32,851例DLBCL病例。基于治愈率假设,估计的2L人群规模范围为9,855例(最低)至13,140例(最高)。根据编码标准,共识别出7,523例接受了挽救性免疫化疗。这对应于拟接受HD-ASCT的适合移植患者比例为57%(最低)至76%(最高)。敏感性分析得出的适合移植比例范围为40%至54%(2022年)和52%至69%(2023年),应用德国真实世界索赔数据中的复发/难治率时为52%,排除polatuzumab vedotin相关病例时为34%至46%。
Inpatient billing data from all German hospitals in 2024, provided by the Institute for the Hospital Remuneration System (InEK), were analyzed. Cases with the main diagnosis "C83.3" (DLBCL) defined the base population. Applying guideline-based first-line cure rates (60% [minimum]-70% [maximum]), the 2L population size was estimated. Transplant-eligible cases were identified via OPS codes for salvage immunochemotherapies (R-ICE, R-DHAP, R-GDP). The transplant-eligible proportion was calculated by dividing the number of transplant-eligible cases by the estimated 2L population. Sensitivity analyses were performed using data from 2022 and 2023, validating the 2L relapse/refractoriness rate assumption against German real-world claims data, and assessing potential impact of polatuzumab vedotin-related coding bias.
The base population included 32,851 DLBCL cases. Based on cure rate assumptions, the estimated 2L population size ranged from 9,855 (minimum) to 13,140 (maximum). A total of 7,523 cases were identified to have received salvage immunochemotherapy based on coding criteria. This corresponds to a transplant-eligible proportion of patients intended for HD-ASCT of 57% (minimum) and 76% (maximum). Sensitivity analyses yielded a transplant-eligible proportion range of 40% to 54% (2022) and 52% to 69% (2023), 52% when applying the relapse/refractoriness rate from German real-world claims data, and 34% to 46% when excluding polatuzumab vedotin-related cases.
This analysis introduces a method for approximating estimating the distribution of transplant eligibility among DLBCL patients intended for HD-ASCT in the 2L setting using real-world inpatient billing data. The results question the widely assumed balance between transplant-eligible and -ineligible groups, indicating a possible tendency toward a slightly higher proportion of transplant-eligible cases which may be clinically eligible for HD-ASCT.
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