Home-CALM Home-based Treatment With Intravenous Cytarabine for Acute Myeloid Leukemia: Feasibility, Health Economics, Barriers and Facilitators - a National Multicenter Study in Denmark
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 80
- 主要终点
- Rates of retention to standard protocol in patients receiving home-based treatment.
研究概览
简要总结
The goal of this observational multicenter feasibility and implementation study is to evaluate home-based treatment with intravenous Cytarabine for patients with acute myeloid leukemia in Denmark. The study aims to generate research-based evidence on feasibility, safety, patient impact, health economic costs, and implementation of home-based intravenous chemotherapy treatment.
The main questions it aims to answer are:
1. What is the existing evidence on home-based chemotherapy treatment for patients with hematological malignancies, including feasibility, safety, symptoms, quality of life, and cost-effectiveness?
2. Is home-based treatment with intravenous Cytarabine feasible and safe for patients with acute myeloid leukemia in Denmark?
3. What are the costs, patient-reported outcomes, and caregiver roles related to home-based Cytarabine treatment?
4. What are the key barriers and facilitators experienced by patients, caregivers, and healthcare professionals during home-based Cytarabine treatment? Which national strategies and recommendations can support the implementation, scalability, sustainability, and equitable access to home-based intravenous Cytarabine treatment across healthcare settings in Denmark?
详细描述
Introduction Acute myeloid leukemia (AML) is an aggressive hematological malignancy characterized by rapid proliferation of malignant blasts in the bone marrow, leading to suppression of normal hematopoiesis and bone marrow failure. Curative treatment typically involves intensive chemotherapy, often including intravenous Cytarabine (IV-Cytarabine), administered either as intermittent or continuous infusions. Due to the need for prolonged infusion and close clinical monitoring, patients are commonly treated during hospitalization or through repeated outpatient visits. This places a considerable burden on patients, caregivers, and healthcare systems, and may increase risks associated with prolonged hospital stays, including infections, reduced mobility, impaired nutrition, and decreased quality of life. New delivery models, including portable programmable infusion pumps, make it possible for selected patients to receive IV-Cytarabine at home.
Home-based hematological care is increasingly recognized as a patient-centered model that may reduce hospital visits, support daily living, improve psychosocial well-being, and potentially lower risks related to hospitalization. Previous Danish studies have shown that home-based chemotherapy can be feasible, safe, and acceptable to patients. However, current evidence remains limited, and no national or international guidelines exist to support systematic implementation of home-based hematological treatment. Important gaps remain regarding organizational models, safety, feasibility, health economic costs, patient-reported outcomes, caregiver involvement, and long-term sustainability. Preliminary findings from the Treat@Home program indicate that home-based treatment is already used in Denmark, but access is unequal, data registration is inconsistent, and the evidence base is limited. The HOME-CALM study therefore aims to generate robust evidence on home-based IV-Cytarabine for patients with AML in Denmark. This knowledge will support the development of national recommendations and sustainable, equitable care pathways for home-based intravenous chemotherapy.
Hypothesis This study is based on the following assumptions and hypotheses.
Study 2 Home-based IV-Cytarabine treatment for patients with AML is feasible and safe when compared to inpatient or outpatient treatment settings, reducing the burden on healthcare systems and supporting everyday lives.
Study 3 Key barriers to home-based IV-Cytarabine treatment include lack of patient and caregiver education, low level of health literacy, and challenges in coordination with HCPs across sectors, while facilitators include effective communication, support systems, and tailored treatment plans.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 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
- •PATIENT (Study 2+3)
- •Age ≥ 18 years
- •Can provide informed consent.
- •Participants that have been diagnosed with AML, to receive IV-Cytarabine
- •CAREGIVER (Study 2+3)
- •Age ≥18 years
- •Can provide informed consent.
- •HEALTHCARE PROFESSIONALS (Study 3)
- •Employed as Physician or Registered Nurse
- •Work experience with home-based treatment for patients with AML for minimum 1 year.
排除标准
- •(Patient/Caregiver):
- •Inability to read, write and/or speak Danish or English
- •Otherwise deemed unethical to participate due to mental disability
结局指标
主要结局
Rates of retention to standard protocol in patients receiving home-based treatment.
时间窗: Through study completion, an average of 20 weeks per patient.
Retention will be measured as the proportion of patients who remain in the standard home-based treatment protocol until completion of the planned treatment course. The retention rate will be calculated as: Number of patients who complete the planned home-based treatment protocol / Number of patients who initiate home-based treatment × 100. Non-retention is defined as any deviation from the site-specified standard home-based treatment protocol resulting in temporary or permanent interruption of home-based treatment and transition to hospital-based treatment, either temporarily or permanently.
Number and proportion of treatment doses administered at home, administered at the hospital, diverted from home to hospital, or not administered
时间窗: From date of inclusion, up to 20 weeks
Treatment delivery will be assessed by recording the location of administration for each planned treatment dose via EHJ. Doses will be categorized as: * Administered at home as planned * Administered at the hospital as planned * Diverted from home to hospital, defined as a dose planned for home administration but administered at the hospital * Not administered/missed doses The number and proportion of doses in each category will be calculated relative to the total number of planned treatment doses.
Non-adherence to treatment protocol
时间窗: From date of inclusion, up to 20 weeks
Non-adherence will be assessed as any deviation from the planned treatment or the site-specific eligibility and delivery criteria for home-based treatment. For each deviation, the primary reason will be recorded and categorized as: Clinical Logistical Technical Patient-related Organizational Other Deviations may result in a planned home-based dose being administered at the hospital, temporary or permanent interruption of home-based treatment, permanent transition to hospital-based treatment, or non-administration of a planned dose. The number and proportion of deviations within each category will be reported.
Rate of transition from home-based to hospital-based treatment
时间窗: From date of inclusion, up to 20 weeks
The number and proportion of patients who transition from home-based treatment to hospital-based treatment/vice versa, during the treatment course. Transitions will be classified as temporary or permanent and recorded according to the reason for transition. Also, the number and proportion of nights spent at home during treatment and the number and proportion of treatment cycles completed without hospital admission or hospital visits will be registered as well. Planned hospital admissions or visits specified in the site-specific treatment protocol will not be included in this outcome.
Total healthcare-sector costs associated with home-based IV-cytarabine treatment
时间窗: Through study completion, an average of 24 weeks
Total healthcare-sector costs will be estimated for each participant based on healthcare resource use during the treatment period. Costs will include implementation activities, healthcare professional time, training, equipment and materials, home-based treatment administrations, virtual contacts, inpatient and outpatient visits, hospital admissions, and unscheduled healthcare contacts. Resource use will be estimated, based on observations and informal expert interviews. Costs will bereported in US Dollars.
Incidence and characteristics of selected adverse events during home-based IV-cytarabine treatment
时间窗: Through study completion, an average of 20 weeks per patient
Safety will be assessed by the number and proportion of participants experiencing selected adverse events, classified and graded according to the Common Terminology Criteria for Adverse Events, version 6.0 (CTCAE v6.0). For each event, the following information will be recorded: * Date of onset and resolution * CTCAE term and grade * Description of the event * Treatment location at the time of the event * Hospital admission and discharge dates * Oral or intravenous antibiotic treatment * Impact on home-based treatment, including interruption, delay, or transition to hospital-based treatment * Cause of treatment interruption or delay * Mitigation measures implemented and their effectiveness * Clinical outcome of the event Relevant laboratory results, including hemoglobin, leukocyte count, neutrophil count, platelet count, and C-reactive protein, will be recorded in relation to the event.
Patient and caregiver acceptability
时间窗: At the end of Cycle 1, 2, 3 & 4 (each cycle is 5-10 days)
Acceptability of home-based chemotherapy will be assessed using an eight-item questionnaire based on the Theoretical Framework of Acceptability (TFA). The questionnaire evaluates patients' and caregivers' perceptions, satisfaction, and emotional responses related to home-based chemotherapy. Each item is rated on a 5-point Likert scale: 1 ("Not at all"), 2 ("Not really"), 3 ("No opinion"), 4 ("Somewhat"), and 5 ("Very much"). Higher scores indicate greater acceptability of home-based chemotherapy. If the patient is not receiving home-based chemotherapy treatment, acceptability data will not be collected of that specific treatment cycle.
Patient and caregiver time
时间窗: At the end of Cycle 1, 2, 3 & 4 (each cycle is 5-10 days)
Non-healthcare resource use among patients and caregivers will be assessed by recording treatment-related transportation via a self-developed questionnaire. For patients and caregivers, data pertaining to treatment-related time, including preparation for treatment visits, attendance at the treatment, waiting time, and time spent on treatment at-home, will be collected
Patient and caregiver transportation
时间窗: At the end of Cycle 1, 2, 3 & 4 (each cycle is 5-10 days)
Non-healthcare resource use among patients and caregivers will be assessed by recording treatment-related transportation via a self-developed questionnaire. For patients and caregivers, transportation mode, number of treatment-related journeys, travel time, including public transportation, private vehicle use, patient transport services, and parking, will be recorded. Special for caregivers, data pertaining assistance with home-based treatment, and other informal care or treatment-related support will be collected.
Patient and caregiver informal care
时间窗: At the end of Cycle 1, 2, 3 & 4 (each cycle is 5-10 days)
For caregivers, time spent on activities pertaining to informal care or treatment-related support at-home will be collected.
次要结局
- Change in health-related quality of life assessed using the EORTC QLQ-C30(Baseline (Day 1) and at the end of Cycle 2 & 4 (each cycle is 5-10 days))
- Change in patient health literacy assessed using the Health Literacy Questionnaire(Baseline (Day 1) and at the end of Cycle 2 & 4 (each cycle is 5-10 days))
- Change in caregiver well-being assessed using the Caregiver Roles and Responsibilities Scale(Baseline (Day 1) and at the end of Cycle 2 & 4 (each cycle is 5-10 days))