Gaylis Franklin D, Emond Bruno, Manceur Ameur M, Tardif-Samson Anabelle, Morrison Laura, Pilon Dominic, Lefebvre Patrick, Ellis Lorie A, Balaji Hiremagalur, Ireland Andrea
1Genesis Healthcare Partners, San Diego, California, USA.
Analysis Group, Inc., Montréal, Québec, Canada.
J Health Econ Outcomes Res. 2024 Oct 28;11(2):109-117. doi: 10.36469/001c.124208. eCollection 2024.
Bacillus Calmette-Guérin (BCG) can reduce recurrence and delay progression among patients with high-risk non-muscle invasive bladder cancer (NMIBC), but is associated with a substantial emotional, physical, and social burden. This study evaluated the adequacy of first-line intravesical BCG treatment among high-risk NMIBC patients in the United States, including the subgroup with carcinoma in situ (CIS) of the bladder. Adults with high-risk NMIBC treated with BCG were selected from de-identified MarketScan® Commercial, Medicare, and Medicaid Databases (1/1/2010-2/28/2021). Adequacy of BCG induction and maintenance was evaluated from the first BCG claim until the end of the patient's observation, using a previously published claims-based algorithm (induction: ≥5 instillations within 70 days; induction and maintenance: ≥7 instillations within 274 days of first instillation) and a definition based on the landmark Southwest Oncology Group (SWOG) trial (induction: ≥5 instillations without gaps >7 days; followed by ≥2 instillations at month 3, 6, and every 6 months thereafter). Proportions of patients with adequate BCG induction and maintenance were reported overall and compared between those with and without CIS. Of 5803 high-risk NMIBC patients treated with first-line BCG (mean age, 67.3 years; 20.6% female), 930 (16.0%) had CIS. After first-line BCG, 56.6% received another treatment. Although 86.9% had adequate BCG induction based on the claims-based algorithm (SWOG, 73.6%), only 41.5% had adequate BCG induction and maintenance (SWOG, 1.6%). Similar trends were observed for patients with and without CIS, with higher adherence to guidelines for patients with CIS (adequate induction using claims-based algorithm: 90.3% vs 86.2%; adequate induction and maintenance: 50.8% vs 39.7%, all < .001). A greater proportion of CIS patients than non-CIS patients had cystectomy (CIS, 14.4%, non-CIS, 8.5%; < .001) after first-line BCG. Among patients with NMIBC treated with first-line intravesical BCG, most received adequate BCG induction but less than half had adequate BCG maintenance. BCG treatment was also inadequate for patients with CIS, with only half of patients receiving adequate BCG maintenance and a higher proportion undergoing cystectomy following first-line BCG. Results emphasize the need for additional treatment options for patients with NMIBC.
卡介苗（BCG）可降低高危非肌层浸润性膀胱癌（NMIBC）患者的复发率并延缓疾病进展，但会给患者带来巨大的情感、身体和社会负担。本研究评估了美国高危NMIBC患者一线膀胱内灌注BCG治疗的充分性，包括膀胱原位癌（CIS）亚组。从去识别化的MarketScan®商业、医疗保险和医疗补助数据库（2010年1月1日至2021年2月28日）中选取接受BCG治疗的高危NMIBC成人患者。使用先前发表的基于索赔的算法（诱导期：70天内≥5次灌注；诱导期和维持期：首次灌注后274天内≥7次灌注）和基于具有里程碑意义的西南肿瘤协作组（SWOG）试验的定义（诱导期：≥5次灌注且间隔不超过7天；随后在第3个月、第6个月及此后每6个月各进行≥2次灌注），从首次BCG索赔记录直至患者观察期结束，评估BCG诱导治疗和维持治疗的充分性。报告了总体上以及伴有和不伴有CIS的患者中BCG诱导治疗和维持治疗充分的患者比例，并进行了比较。在5803例接受一线BCG治疗的高危NMIBC患者中（平均年龄67.3岁；女性占20.6%），930例（16.0%）患有CIS。一线BCG治疗后，56.6%的患者接受了其他治疗。尽管根据基于索赔的算法，86.9%的患者BCG诱导治疗充分（SWOG标准为73.6%），但只有41.5%的患者BCG诱导治疗和维持治疗均充分（SWOG标准为1.6%）。伴有和不伴有CIS的患者呈现相似趋势，CIS患者对指南的依从性更高（基于索赔的算法诱导治疗充分：90.3%对86.2%；诱导治疗和维持治疗充分：50.8%对39.7%，均P＜0.001）。一线BCG治疗后，CIS患者接受膀胱切除术的比例高于非CIS患者（CIS患者为14.4%，非CIS患者为8.5%；P＜0.001）。在接受一线膀胱内灌注BCG治疗的NMIBC患者中，大多数患者BCG诱导治疗充分，但不到一半的患者BCG维持治疗充分。BCG治疗对CIS患者也不充分，只有一半的患者BCG维持治疗充分，且一线BCG治疗后接受膀胱切除术的比例更高。研究结果强调了为NMIBC患者提供更多治疗选择的必要性。
