EUTOS Registry Work plan  · 

Work plan for subproject Registry

Study type

The registry will collect strictly observational data only, which will be recorded retrospectively and prospectively from all kinds of healthcare settings across Europe. The predominant study design will be that of a cohort study, eventually with comparator groups (depending on the analyses, for example, we may compare the outcome or treatment of patients with low, intermediate or high risk, or compare groups with different cytogenetic characteristics at baseline).

The aim is to study the epidemiology of CML and to gain insight into the ‘real world’ treatment of patients with CML, and considerable care will be taken to provide representative samples of patients. Bearing in mind the differing healthcare systems in Europe, plans are being formulated in close collaboration with the national CML study groups, to facilitate collection of data from CML patients in routine care across Europe.

Registry population

To reduce bias, data will be collected from three different groups of patients:

In-study: Patients from national study groups enrolled in prospective studies, taking imatinib frontline

• Will include ~1900 patients enrolled in on-going studies of imatinib-based regimens frontline
• Patients will have been diagnosed between 2002 and 2006
• High quality and quantity of data are expected, due to the rigorous study protocols enforced by the respective Study Groups
• Patients in this group will have substantial follow-up data (4–8 years in 2010)

Out-study: Patients already registered in existing databases, irrespective of frontline treatment

• Will include ~1560 patients registered in already existing or planned databases of national/regional study groups or reference centers
• Patients will have been diagnosed between 2002 and 2006
• Out-study patients will serve as a control group for the in-study patients (the selection bias is different)
• Creation of the out-study group also allows some countries with no in-study patients to contribute to the Registry
• Patients in this group will have substantial follow-up data (4–8 years in 2010)

Prospective: Newly diagnosed patients not previously included in registries or clinical studies

• Will include ~1700 patients newly diagnosed from 2009 onwards
• As much as possible, this part of the registry will be population-based
• Study groups should cover either a whole country or a region of a country, not exceeding 10 million people (details to be negotiated with study groups or with reference centers)

Patient Eligibility

Patients must be newly diagnosed with Ph+ or BCR-ABL positive CML, and aged ≥ 18 years, to be eligible for the registry.

Core Data Set (In- and Out-Study Patients)

A core data set for ‘prospective’ group of patients is still in development. The finalized core data set for ‘in-study’ and ‘out-study’ patients will be made available through the ‘Documents’ section of the EUTOS for CML Registry website.

Key baseline data correspond to criteria of eligibility for including a case in the registry, and are therefore mandatory; these baseline data include essential anagraphical variables, hematologic, cytogenetic and molecular data confirming the diagnosis, the phase of the disease, and the prognostic scores. If lacking or incomplete, specific queries will be sent to the responsible investigator.

Other baseline variables as well as follow-up data are also very important but their absence should not preclude the registration of a patient, as this would introduce a selection bias into the registry. However, these ‘non-mandatory’ variables will contribute greatly to improving the general quality of the information retrieved and, consequently, the quality and the scientific level of the registry. For this reason, they could be the object of specific queries.

Scientific Committees

Data Management and Quality Control

The data will be collected and quality controlled by the national data collection centers and then forwarded electronically, in English, at regular intervals to the Central Data Center in Munich. There will be a variety of electronic options for forwarding the data, including internet, CD, etc. Data will be checked for completeness and plausibility at the Central Data Center.

Data Confidentiality Issues and Ethics

The registry will use pseudonymized data only. Thus the identity of patients will only be known by the attending physician, if not specified in a different manner by national regulations. In any case the data held at the Central Data Center in Munich will be handled in strict compliance with the German law; the data collected and transferred by the various ELN participants will be handled in strict compliance with the data confidentiality laws of the various member states.

The registry is strictly observational in nature, and no diagnostic or therapeutic intervention will be caused by the registry.

Author: Tim Ryder

Created by: D.Goekbuget , generated 2007/12/19 , last changed: 2008/08/21