Non-interventional Cohort Study of Patients Previously Untreated or First-generation BTKi Intolerant With Chronic Lymphocytic Leukemia Describing the First-line Use of Acalabrutinib and Its Real-world Outcomes in Spain: the PICAROS Study
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- AstraZeneca
- 入组人数
- 192
- 试验地点
- 47
- 主要终点
- Proportion of patients on acalabrutinib therapy at 24 months after treatment initiation.
研究概览
简要总结
This is a multicenter non-interventional study (NIS) of patients with CLL treated with first-line acalabrutinib according to routine clinical practice in Spain. It included an initial cohort of patients who initiated treat-to-progression acalabrutinib for the first time within the year before the first site initiation visit, both for previously untreated CLL and after switching in first line from another BTK inhibitor due to intolerance in absence of progression (cohort 1). This cohort is complemented with the addition of another regarding the first-line fixed-duration acalabrutinib use with venetoclax in routine practice (cohort 2).
详细描述
This is a multicenter, non-interventional study (NIS) based on ambispective (including retrospective and/or prospective) real-world data collection of patients with CLL who received treatment with acalabrutinib for the first time in Spain.
It included an initial cohort of patients who initiated treat-to-progression acalabrutinib for the first time within the year before the first site initiation visit, both for previously untreated CLL and after switching in first line from another BTK inhibitor due to intolerance in absence of progression (cohort 1). This cohort is complemented with the addition of another regarding the first-line fixed-duration acalabrutinib use with venetoclax in routine practice (cohort 2).
For the cohort 1, the start of acalabrutinib treatment (index date) was prior to the first site initiation visit. For both cohorts 1 and 2, the clinical decision of starting patient on acalabrutinib has to independently occur prior to the patient inclusion into this study. Patients' eligibility for study inclusion is regardless of their current status of acalabrutinib therapy, for example, patients already deceased or discontinued therapy are still eligible to be included into this study. Patient data will be collected both retrospectively and/or prospectively up to approximately 32 months from the first site initiation visit for cohort 1, and approximately 25 months after the inclusion of the last patient in the cohort 2. For patients who received acalabrutinib therapy and have deceased, only retrospective medical chart review will be conducted.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years old at starting acalabrutinib treatment.
- •Diagnosis of CLL.
- •Start of acalabrutinib treatment (index date) as per the CLL SmPC:
- •in treatment-naïve CLL patients or those switching in first-line between first-generation BTK inhibitor to acalabrutinib due to intolerance in absence of progression according to routine clinical practice within the year before the first site initiation visit (cohort 1).
- •in treatment-naïve CLL patients for the fixed-duration treatment combination of acalabrutinib plus venetoclax (with or without obinutuzumab) according to routine clinical practice from the EMA approval (i.e., 2 June 2025) (cohort 2).
- •Decision to administer acalabrutinib must be made and documented prior to inclusion into the study and must follow local clinical practice.
- •Informed consent (for alive patients).
排除标准
- •Enrolled in any clinical trial during acalabrutinib treatment.
- •Patients who are unable to understand the study and its questionnaires due to insufficient knowledge of the Spanish language or their health status.
结局指标
主要结局
Proportion of patients on acalabrutinib therapy at 24 months after treatment initiation.
时间窗: 24 months after treatment initiation.
Proportion of patients on acalabrutinib therapy at 24 months after treatment initiation. In addition to this outcome measured in the overall population, it will also be assessed by the following factors: 1. the reason for treatment initiation (i.e., first-line treatment-naïve patients, and those switching due to intolerance in absence of progression), 2. presence/absence of risk factors (i.e., del17p, TP53 mutation, and unmutated IGHV). 3. cardiovascular comorbidities (yes/no).
Proportion of patients on acalabrutinib therapy at 24 months after treatment initiation (cohort 1).
时间窗: 24 months after treatment initiation (cohort 1).
Proportion of patients on acalabrutinib therapy at 24 months after treatment initiation (cohort 1). In addition to this outcome measured in the overall cohort, it will also be assessed by the following factors: 1. the reason for treatment initiation (i.e., first-line treatment-naïve patients, and those switching due to intolerance in absence of progression), 2. presence/absence of risk factors (i.e., del17p, TP53 mutation, and unmutated IGHV). 3. cardiovascular comorbidities (yes/no).
Real-world overall response rate (rwORR) achieved until the end of the observational period for first-line fixed duration acalabrutinib therapy (cohort 2)
时间窗: From fixed-duration acalabrutinib start to the start date of a subsequent line therapy or study end date (cohort 2), assessed up to 25 months after the inclusion of the last patient in the cohort 2.
rwORR (i.e., the proportion of patients that achieved complete or partial response) achieved until the end of the observational period for first-line fixed-duration acalabrutinib therapy as assessed by the treating physician (cohort 2). Complete response may include complete response, complete response with incomplete marrow recovery, or unconfirmed complete response (marrow biopsy not performed). Partial response may include partial response, or partial response with lymphocytosis. The rwORR will be assessed by the treating physician in routine clinical practice, including the use or not of the 2018 International Workshop on Chronic Lymphocytic Leukemia (iwCLL) criteria. The end of observational period for first-line fixed-duration combinations of acalabrutinib is defined as the start date of a subsequent line therapy or study end date, whichever comes first.
次要结局
- Start dose in mg.(At acalabrutinib start date)
- Treatment duration in months.(From acalabrutinib start to acalabrutinib end, assessed up to 3.5 years of prospective study follow-up.)
- Adverse events that lead to acalabrutinib dose changes, temporary interruptions, or permanent discontinuation. Adverse events that are considered serious during acalabrutinib treatment. Events of clinical interest.(From acalabrutinib start to acalabrutinib end, assessed up to 3.5 years of prospective study follow-up.)
- TTNT (i.e., the time from the date of first dose of acalabrutinib to the first dose of the next treatment for CLL, or death from any cause [i.e. deaths are not censored]).(From the date of first dose of acalabrutinib to the first dose of the next treatment for CLL or death from any cause, whichever came first, assessed up to 3.5 years of prospective study follow-up.)
- OS (i.e., the time from the date of first dose of acalabrutinib to death from any cause).(From the date of first dose of acalabrutinib to death from any cause, whichever came first, assessed up to 3.5 years of prospective study follow-up.)
- Patients with acalabrutinib dose reductions (n, %), temporary interruptions (n, %), and permanent discontinuations (n, %).(From acalabrutinib start to acalabrutinib end, assessed up to 3.5 years of prospective study follow-up.)
- Treatment adherence according to the percentage of days covered (PDC) while receiving acalabrutinib.(From acalabrutinib start to acalabrutinib end, assessed up to 3.5 years of prospective study follow-up.)
- Acalabrutinib±venetoclax doses (cohorts 1 and 2).(At start date and subsequent dose adjustments during acalabrutinib±venetoclax administration assessed up to 32 months of prospective study follow-up in the cohort 1 and 25 months after the inclusion of the last patient in the cohort 2.)
- Patients with acalabrutinib±venetoclax dose adjustments (n, %), temporary interruptions (n, %), and permanent discontinuations (n, %) (cohorts 1 and 2).(From acalabrutinib±venetoclax start to acalabrutinib±venetoclax end, assessed up to 32 months of prospective study follow-up in the cohort 1 and 25 months after the inclusion of the last patient in the cohort 2.)
- Treatment duration in months (cohorts 1 and 2).(From acalabrutinib±venetoclax start to acalabrutinib±venetoclax end, assessed up to 32 months of prospective study follow-up in the cohort 1 and 25 months after the inclusion of the last patient in the cohort 2.)
- Treatment adherence according to the percentage of days covered (PDC) while receiving acalabrutinib (cohort 1).(From acalabrutinib start to acalabrutinib end, assessed up to 32 months of prospective study follow-up (cohort 1).)
- TTNT (i.e., the time from the date of first dose of acalabrutinib to the first dose of the next treatment for CLL, or death from any cause) (cohorts 1 and 2).(From the date of first dose of acalabrutinib to first dose of next CLL treatment or death, assessed up to 32 months of prospective study follow-up in the cohort 1 and 25 months after the inclusion of the last patient in the cohort 2.)
- OS (i.e., the time from the date of first dose of acalabrutinib to death from any cause) (cohorts 1 and 2).(From the date of first dose of acalabrutinib to death, assessed up to 32 months of prospective study follow-up in the cohort 1 and 25 months after the inclusion of the last patient in the cohort 2.)
- Adverse events that lead to acalabrutinib±venetoclax dose changes, temporary interruptions, or permanent discontinuation; that are considered serious during acalabrutinib±venetoclax treatment; and events of clinical interest (cohorts 1 and 2).(From acalabrutinib±venetoclax start to acalabrutinib±venetoxlax end, assessed up to 32 months of prospective study follow-up in the cohort 1 and 25 months after the inclusion of the last patient in the cohort 2.)