A Real-World Observational Study on Patient-Reported Outcomes in Allogeneic Stem Cell Transplantation (Allo-SCT) and CAR-T Therapy
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 162
- 主要终点
- To assess and compare short-term patient-reported outcomes [PROs (symptom burden and QoL impact)] between patients receiving allo-SCT and CAR-T therapy using the HM-PRO questionnaire (Impact Scale, Symptoms Scale) from admission to hospital discharge
研究概览
简要总结
3.1. Overview Prospective, multicenter, observational cohort study comparing short-term PROs measured with the HM-PRO between two exposure groups: patients undergoing allogeneic stem cell transplantation (allo-SCT) and patients receiving CAR-T cell therapy. Patients will be enrolled at hospital admission for the index inpatient procedure and followed through the inpatient stay (admission → discharge). The study is non-randomized and designed to describe trajectories of symptoms and HRQoL and to estimate the between-group difference in deterioration of HM-PRO scores (primary estimand: mean difference in change score, CAR-T vs allo-SCT).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Diagnosed with a hematologic malignancy (e.g., leukemia, lymphoma, multiple myeloma)
- •Undergoing either allogeneic SCT or CAR-T therapy
- •Able and willing to provide written informed consent
- •Sufficient Italian proficiency to complete the HM-PRO
- •Signed informed consent
排除标准
- •Cognitive or physical impairments that preclude the ability to complete questionnaires
- •Estimated life expectancy < 7 days at the time of admission
结局指标
主要结局
To assess and compare short-term patient-reported outcomes [PROs (symptom burden and QoL impact)] between patients receiving allo-SCT and CAR-T therapy using the HM-PRO questionnaire (Impact Scale, Symptoms Scale) from admission to hospital discharge
时间窗: 3 MONTHS
The primary outcome aims to evaluate the impact of CAR-T therapy and allogeneic transplantation on patients' quality of life using a specific questionnaire (HM-PRO), which assesses PROs (quality of life and symptoms) in hematologic patients, with the goal of determining whether there are differences between the two patient groups. In the HM-PRO questionnaire values range from 0 to 100. Lower values correspond to a better quality of life and a lower symptom burden.
次要结局
- To describe PRO trajectories during hospitalization(3 MONTHS)
- To assess differences in PROs based on disease type (e.g., Leukemia, lymphoma, myeloma)(3 MONTHS)
- To assess differences in PROs based on disease status (remission vs progression)(3 MONTHS)
- To assess differences in PROs based on treatment line (first-line vs multiple lines)(3 MONTHS)
- To validate the use of the HM-PRO in the context of inpatient care for advanced therapies.(3 MONTHS)