Clinical trial NCT05074316: The Myeloid Neoplasms Biology and Outcome Project
Official title: A Prospective Registry Study on Biological Disease Profile, Intervention Type and Clinical Outcome in Patients With Myeloid Neoplasms
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myeloid Neoplasm
Keywords: outcome project
registry
Lead sponsor: University Hospital Heidelberg
Sponsor class: OTHER
First posted: 2021-10-12
Enrollment: 1000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: the patient cohort will be selected from the University Hospital Heidelberg.

Brief summary: 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:

1. Improvement of evidence-based clinical management of patients with myeloid neoplasms through better understanding of the course of disease and prognostic and predictive parameters
2. Direct access to new and personalized treatment approaches through recruitment into clinical studies based on the myeloid neoplasms study platform
3. Quality assurance of participating centers by evaluating and comparing clinical outcomes and side effects of the MyBOP patients with published data.

Detailed description: 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

The above challenges are ideally met by a registry study with a sufficient population size in order to answer relevant questions in rare cancer entities. The aim is to set up a registry study that covers systematic and comprehensive clinical data acquisition. In addition, the banking of tumor and germline samples from patients with myeloid neoplasms is intended by all patients. This resource will spur patient-oriented investigations into relationships between clinical and biological parameters in myeloid neoplasms and lay the groundwork for novel, molecular mechanism- and immunotherapy-based treatment approaches in poorly understood and difficult-to-treat subsets.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

Severe neurological or psychiatric disorder interfering with the ability to give written informed consent

Primary outcomes:
measure: median overall survival (mOS)
timeFrame: 5 years
measure: overall survival (mOS)
timeFrame: 10 years
measure: event free survival (EFS)
timeFrame: 5 years
measure: progression free survival (PFS)
timeFrame: 5 years
Secondary outcomes:
measure: Questionnaire for the health- related quality of life QLQ-C30
timeFrame: 5 years
measure: Questionnaire for physical, cognitive and emotional aspects of cancer-related fatigue QLQ-FA12
timeFrame: 5 years
measure: Questionnaire for anxiety and depression PHQ-4
timeFrame: 5 years
measure: Functional Assessment of Cancer Therapy Fact-Cog
timeFrame: 5 years
measure: The Pittsburgh Sleep Quality Index PSQI
timeFrame: 5 years
Other outcomes:
Locations:
city: Heidelberg
country: Germany
facility: UHHeidelberg
state: Baden-Württemberg
Study officials:
affiliation: University Hospital Heidelberg, Department of Internal Medicine V, German Cancer Research Center
name: Richard F Schlenk, M.D.
role: PRINCIPAL_INVESTIGATOR
References: