LEAHRN (Late Effects After High-Risk Neuroblastoma) Study
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 376
- 试验地点
- 195
- 主要终点
- Pediatric Quality of Life (PedsQL) score
研究概览
简要总结
This research trial studies late effects after treatment in patients with previously diagnosed high-risk neuroblastoma. Studying late effects after treatment may help to decide which treatments for high-risk neuroblastoma are better tolerated with less side effects over time.
详细描述
PRIMARY OBJECTIVES:
I. To estimate the prevalence of organ dysfunction, subsequent malignant neoplasm (SMN), growth impairment, abnormal pubertal development, and neurobehavioral dysfunction in a large cohort of representative 5-year survivors of high-risk neuroblastoma treated with modern therapy.
II. To identify the demographic, clinical and treatment-related risk factors associated with increased risk of organ dysfunction, SMN, growth impairment, abnormal pubertal development and neurobehavioral dysfunction in long-term survivors of high-risk neuroblastoma.
III. To explore the impact of new biologic therapies and diagnostics including immunotherapy, immunocytokines, isotretinoin (cis-retinoic acid) and iobenguane I-131 (131 I-MIBG) on the risk of late effects.
IV. To determine the impact of impaired organ function, physical growth, pubertal development, and neurobehavioral function on health-related quality of life (HRQOL) in long-term survivors of high-risk neuroblastoma.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 5 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have been enrolled on COG neuroblastoma biology study ANBL00B1
- •Patient must have been diagnosed with high-risk neuroblastoma per ANBL00B1 definition
- •Patient must have been diagnosed on or after January 1, 2000
- •At least 5 years must have elapsed since diagnosis
- •Patients must have been treated for high-risk neuroblastoma
- •Note: patients may have had any therapy for high-risk neuroblastoma, including second line or non-established therapies (for example in the setting of less than optimal initial response or concerns for high risk of relapse); patients may have received therapy for refractory or relapsed neuroblastoma, or treatment for an SMN; however all cytotoxic anti-neuroblastoma therapy should have been administered >= 2 years of the enrollment date; SMN therapy may be completed or ongoing at the time of enrollment
排除标准
- •Patients must not be currently receiving active anti-neuroblastoma cytotoxic chemotherapy
- •Patients must not have received anti-neuroblastoma cytotoxic chemotherapy within the last two years
- •Note: cytotoxic therapies include (but are not limited to) chemotherapy (platinum agents, alkylators, anthracyclines, topoisomerases, vinca alkaloids, other cytotoxic chemotherapy), any kind of transplant, MIBG therapy, and/or radiation therapy
- •Non-cytotoxic (biologic/targeted/differentiating/other) therapies are permitted at the time of enrollment; for example, patients receiving oral differentiating agents, antiangiogenic therapy, immune modulators, holistic therapies, difluoromethylornithine (DMFO), other minimal residual disease (MRD) therapies/relapse-prevention therapies are eligible
- •Patients with current active neuroblastoma relapse are ineligible
研究组 & 干预措施
Observational (specimen collection)
Patients undergo collection of blood and urine samples on day 1. Patients also undergo clinical assessments, laboratory, radiographic, and other ancillary studies on day 1.
干预措施: Quality-of-Life Assessment (Other)
Observational (specimen collection)
Patients undergo collection of blood and urine samples on day 1. Patients also undergo clinical assessments, laboratory, radiographic, and other ancillary studies on day 1.
干预措施: Cytology Specimen Collection Procedure (Other)
Observational (specimen collection)
Patients undergo collection of blood and urine samples on day 1. Patients also undergo clinical assessments, laboratory, radiographic, and other ancillary studies on day 1.
干预措施: Laboratory Biomarker Analysis (Other)
结局指标
主要结局
Pediatric Quality of Life (PedsQL) score
时间窗: Up to 3 years
PedsQL score will be reported descriptively (mean, standard deviation). The proportion of patients with impaired physical growth, delayed pubertal development, chronic disease, impaired executive functioning, and impaired social functioning will also be reported.
Prevalence of specific late effects
时间窗: Up to 3 years
Late effects of interest are organ dysfunction, subsequent malignant neoplasms (SMN), growth impairment, abnormal pubertal development, and neurobehavioral dysfunction. Prevalence will be calculated as the number of patients with late effects divided by the number with known status of that endpoint.
Risk factors of late effects
时间窗: Up to 3 years
Risk factors of interest include sex, race, ethnicity, current age, length of follow up, MYCN status, stage, primary site, age at diagnosis, total anthracycline dose (doxorubicin equivalents), cyclophosphamide dose equivalent categories, total platinum exposure (dose), topotecan exposure (yes \[Y\]/no \[N\]), cis-retinoic acid exposure (Y/N), GD-2/cytokine exposure (Y/N), radiation (Y/N), abdominal RT (Y/N), radiation to metastatic sites (Y/N), number of transplants, number of meta-iodobenzylguanidine (MIBG) scans, and therapeutic MIBG (Y/N). Will be reported as the number of patients with the risk factor divided by the number with known status of that risk factor for categorical variables and descriptively (mean, standard deviation) for continuous variables.
次要结局
- Collection and storage of blood samples(Up to 3 years)