Chronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 330
- 试验地点
- 135
- 主要终点
- Prevalence, type, and severity of chronic health conditions (CHC)
研究概览
简要总结
This study attempts to learn more about the health of persons with Down syndrome after treatment for acute leukemia. Children with Down syndrome are at increased risk for side effects during treatment for acute leukemia, but it is unclear of their risk for long-term effects of cancer treatment. By learning more about the factors that may contribute to chronic health conditions and long-term effects after treatment for leukemia in persons with Down syndrome, clinical practice guidelines for survivorship care can be developed to help improve their quality-of-life.
详细描述
PRIMARY OBJECTIVE:
I. To determine the prevalence, type, and severity of chronic health conditions (CHC) in survivors of Down syndrome-associated acute leukemia (DS-AL), and to compare CHC with frequency-matched DS individuals that have no cancer history.
SECONDARY OBJECTIVES:
I. To characterize post-treatment clinical outcomes of DS-AL by prospective, in-person assessment.
II. To determine the prevalence and severity of parent-reported adverse neuropsychological (NP) outcomes in survivors of DS-AL, compared with frequency-matched DS individuals with no cancer history.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- 6 Years 至 39 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients age >= 6 and < 40 years at the time of enrollment
- •A diagnosis of Down syndrome is required, and may include any of the three recognized types: trisomy 21 resulting from chromosomal nondisjunction (most common), translocation (the patient has 46 chromosomes, but all or part of an additional copy of chromosome 21 is attached to another chromosome), or mosaicism (trisomy 21 that is present in only a fraction of cells)
- •All patients must be DS-AL survivors (acute lymphoblastic leukemia [ALL] or acute myeloid leukemia [AML])
- •Note 1: Myeloid leukemia of Down syndrome (ML-DS) is included in the AML category above. Per the World Health Organization (WHO) definition of ML-DS, this diagnosis encompasses both myelodysplastic syndrome (MDS) and overt AML. Also, note that survivors of relapsed disease are eligible, so long as the patient otherwise meets eligibility criteria, i.e., treatment for relapse was completed at least 36 calendar months prior to enrollment and did not include stem cell transplant
- •Note 2: A diagnosis of transient abnormal myelopoiesis (TAM), also known as transient myeloproliferative disease (TMD), is not alone sufficient for inclusion in this study
- •Patients must have been treated for ALL or AML
- •Note: History of COG therapeutic trial participation is not required. As a reminder ML-DS would be included under the AML category here above
- •All cancer treatment (oral or intravenous) must have been completed at least 36 calendar months prior to enrollment
- •Patients must have a life expectancy of > 1 year
- •Patient and parent of subject must be either English or Spanish speaking. At least one parent or guardian must be able to read and write in English or Spanish
- •Note: Parents or guardians are responsible for completing all questionnaires, even in the case of subjects that are >= 18 years old
- •All patients and/or their parents or legal guardians must sign a written informed consent
- •All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
排除标准
- •Patients with history of hematopoietic stem cell transplant (HSCT) are excluded
- •Note: Patients with previous chimeric antigen receptor T-cell (CAR T-cell) therapy, and other cellular cancer therapies can participate, as long as all other eligibility criteria are satisfied
- •Patients with a history of cancers prior to their ALL or AML diagnosis are excluded. Patients that developed a subsequent malignant neoplasm following their ALL or AML diagnosis are also excluded
- •Note: Prior history of transient abnormal myelopoiesis is allowed, but is not sufficient for eligibility
- •Patients whose parents or guardians are unable to complete the required forms are excluded
研究组 & 干预措施
Observational (biospecimen collection, clinical evaluation)
Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.
干预措施: Biospecimen Collection (Procedure)
Observational (biospecimen collection, clinical evaluation)
Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.
干预措施: Clinical Evaluation (Other)
Observational (biospecimen collection, clinical evaluation)
Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.
干预措施: Neurocognitive Assessment (Other)
Observational (biospecimen collection, clinical evaluation)
Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.
干预措施: Questionnaire Administration (Other)
Observational (biospecimen collection, clinical evaluation)
Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.
干预措施: Survey Administration (Other)
结局指标
主要结局
Prevalence, type, and severity of chronic health conditions (CHC)
时间窗: Up to study completion
Summary statistics will be used to characterize the study populations on CHC outcomes. Quantitative data (number of comorbidities) will be summarized using descriptive statistics and correlational techniques. Will use pooled logistic regression to estimate overall response, 95% confidence interval (CI), and p-values for association of acute leukemia (AL) diagnosis with medical record-verified CHC, agnostic of time to CHC. Will use stratified Cox models to refine associations of AL diagnosis with CHC based on time to CHC incidence. Within each age interval, will estimate the hazard ratio, 95% CI, and p-values to report time-dependent effects of AL diagnosis on CHC.
次要结局
- Prevalence and severity of parent-proxy neuropsychological (NP) outcomes(Up to study completion)
- Health-related quality of life (HRQOL)(Up to study completion)
- Post-treatment clinical outcomes(Up to study completion)
- Clinical risk determinants of CHC, NP, and clinical outcomes(Up to study completion)
- Well-annotated cohort of Down syndrome phenotyping acute leukemia study (DS-PALS) survivors and associated biobank(Up to study completion)