A Prospective Registry Study on Biological Disease Profile, Intervention Type and Clinical Outcome in Patients With Myeloid Neoplasms
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,000
- 试验地点
- 1
- 主要终点
- median overall survival (mOS)
研究概览
简要总结
The Myeloid Neoplasms Biology and Outcome Project (MyBOP) aims to establish a registry study for patients with myeloid neoplasms. It integrates clinical data, biological samples, socio-demographic information, long-term follow-up and patient reported outcomes in a structured manner for scientific purposes.
The ultimate benefits are:
- Improvement of evidence-based clinical management of patients with myeloid neoplasms through better understanding of the course of disease and prognostic and predictive parameters
- Direct access to new and personalized treatment approaches through recruitment into clinical studies based on the myeloid neoplasms study platform
- Quality assurance of participating centers by evaluating and comparing clinical outcomes and side effects of the MyBOP patients with published data.
详细描述
During recent years, considerable progress has been made in deciphering the molecular genetic and epigenetic basis of myeloid neoplasms and in defining new diagnostic and prognostic as well as predictive markers. Myeloid neoplasms are categorized according to the current WHO Classification of Tumors of Haematopoietic and Lymphoid Tissues based on the revision of 2016 [1]. This includes Myeloproliferative neoplasms (MPN), Mastocytosis, Myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK2, Myelodysplastic/myeloproliferative neoplasms (MDS/MPN), Myelodysplastic syndromes (MDS), Myeloid neoplasms with germ line predisposition, Acute myeloid leukemia (AML) and related neoplasms (i.e. Myeloid sarcoma and Myeloid proliferations related to Down syndrome), Blastic plasmacytoid dendritic cell neoplasm and Acute leukemia of ambiguous lineage (Table 1).
A growing number of recurring genetic changes are recognized in the current WHO 2016 classification of myeloid neoplasms [2] and additional molecularly defined subgroups as well as new entities are expected to be included in future versions. Furthermore, novel therapies are now available and being developed, which target specific genetic lesions, and several surface antigens are being explored as targets for immunotherapy-based treatment strategies, e.g. CAR-T-cell therapy [3].
Although the WHO 2016 classification represents an enormous progress in terms of reliability, validity and objectivity, there are still huge diagnostic uncertainties left [4-18] and the field of targeted therapy [19-25] in myeloid neoplasms is just at its beginning. Furthermore, clonal evolution and transition from one entity to another is a clinically relevant issue [26-30].
Thus, key areas of interest are:
- Systematic collection and evaluation of comprehensive clinical information from patients with myeloid neoplasm, including morphomolecular disease subtype, as well as drug treatments, radiation therapy, surgical procedures and long-term follow-up data
- Systematic collection and evaluation of comprehensive biological specimens and information from patients with myeloid neoplasms, including data on the genomic, transcriptomic, epigenomic and proteomic "landscapes" as well as expression of surface antigens of myeloid disease subtypes, to identify novel prognostic and predictive parameters as well as entry points for targeted therapeutic interventions
- Regular assessment of patient reported outcomes
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 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
- •Suspected or proven diagnosis of Myeloid Neoplasms according to the WHO Classification of Tumors of Haematopoietic and Lymphoid Tissues
- •Age ≥18 years
- •Ability to understand the nature and individual consequences of the registry
- •Written informed consent
- •Subjects who are physically or mentally capable of giving consent
排除标准
- •Severe neurological or psychiatric disorder interfering with the ability to give written informed consent
结局指标
主要结局
median overall survival (mOS)
时间窗: 5 years
Time period of survival from date of diagnosis of myeloid neoplasy
overall survival (mOS)
时间窗: 10 years
Time period of survival from date of diagnosis of myeloid neoplasy
progression free survival (PFS)
时间窗: 5 years
Time period of progression survival from date of diagnosis of myeloid neoplasy
event free survival (EFS)
时间窗: 5 years
Time period of event free survival from date of diagnosis of myeloid neoplasy
次要结局
- Questionnaire for physical, cognitive and emotional aspects of cancer-related fatigue QLQ-FA12(5 years)
- Questionnaire for the health- related quality of life QLQ-C30(5 years)
- Questionnaire for anxiety and depression PHQ-4(5 years)
- Functional Assessment of Cancer Therapy Fact-Cog(5 years)
- The Pittsburgh Sleep Quality Index PSQI(5 years)
研究者
Prof. Dr. Richard F Schlenk
Head of NCT trials center and Clinical Trials Office Hematology/Oncology
University Hospital Heidelberg