International-CML-Ped Study

OBJECTIVES

The study’s primary objectives are as follows:

• Description of the characteristics of patients with CML under the age of 18 years old
• Description of main therapeutic strategies
• Identifying prognostic risk factors and surrogate markers to predict treatment response
• Defining prognostic scoring systems in the pediatric CML population, in order to optimize treatment options
• Description of treatment side effects and long term complications, especially referring to tyrosine kinase inhibitors (TKIs), regarding growth, organ functions and development
• The development of an international therapeutic platform with homogenous treatment approach within the BFM study group, but also within the broader scientific community (best clinical practice guidelines)
• The improvement of treatment outcomes in pediatric CML

DESIGN

The study is not an interventional clinical study but a retrospective and prospective observational registry, aiming to update modern treatment recommendations and to form a current best practice guidance, available for use from all participating countries.

METHODS
The study is strictly observational. Retrospective and prospective data will be collected from patient flow charts and/or existing databases. A steering committee (SC) will promote the international study of CML in children and adolescents. The SC will include representatives of each country involved in the study. For each objective of the study, there will be a working party (WP), which will be open to all interested participants in the study. Data will be provided by clinicians and biologists involved in pediatric haematology. The data base of the study will be set up in the Clinical Investigation Center (CIC INSERM 802) of the University Hospital of Poitiers (France) which will be the International Central Data Center (ICDC) of the study in charge of and centralizing the data. The study is international. A National Coordinating Center and a leader will be identified in each participating country. The National Coordinating Center will be in charge of collecting data for his country. Each leader of the National Coordinating Center will be in charge of registering the patients and sending the data to the ICDC (CIC INSERM 802, Poitiers, France).

Ενεργή ένταξη ασθενών στο iCML Ped Registry Όλοι οι ασθενείς ηλικίας κάτω των 18 ετών με νεοδιαγνωσθείσα Philadelphia θετική (BCR/ABL+) ΧΜΛ είναι επιλέξιμοι ανεξάρτητα από τη φάση της νόσου, τον τύπο της θεραπείας και την ένταξη ή όχι σε κλινική μελέτη.