Central Data Registry of The European Competence Network on Mastocytosis
Although our knowledge about mastocytosis has increased substantially during the past few years, little is known about its biology, course, incidence and prevalence in various countries. In addition, although prognostic factors and a robust classification are available, little is known about prognostic subgroups and factors predicting the course and morbidity in patients with mastocytosis. One explanation for this lack of knowledge is that mastocytosis is a rare disease and only a few multi-center studies have been conducted so far. In an attempt to address this unmet need, the ECNM has launched a Registry with the aim to include all eligible and consenting patients with mastocytosis in Europe. In a second step, the Registry will invite major U.S. centers interested in mastocytosis.
The primary goal of the ECNM Registry is to collect data from a large number of patients with mastocytosis in Europe over a longer time period (10-20 years) in order to learn more about the incidence, biology, pathogenesis, and course of mastocytosis. The specific long term goal of the project is to improve diagnosis, classification and therapy in these patients, and to define prognostic factors concerning survival, morbidity and treatment responses. The project will also compare outcomes in different patients´ groups defined by origin (country), sex, age and other patient-related factors. Data obtained from the ECNM registry project will be published and will be used to improve management, prognostication and treatment of mastocytosis in daily practice, and to formulate guidelines and recommendations in expert panels and local S.O.P.s.
Specific aims, related to various aspects of the disease, will be formulated in defined ECNM registry subprojects. Each single subproject will be initiated after approval by the Decision Board and after having received advice and input from the Scientific Advisory Board (SAB) of the ECNM. Projects will be discussed and will be approved in Annual Meetings of the ECNM.
In the ECNM Registry, only routine parameters will be collected. “Routine parameter” is defined as a parameter that is regarded as standard according to published guidelines (Valent et al, Eur J Clin Invest, 2007;37:435-453) or/and according to local institutional guidelines. All standard therapy will be documented. Experimental therapy will be documented at the discretion of the local investigator. Neither standard therapy nor experimental therapy will be judged part of the Registry Project, although the outcome will be recorded and may influence morbidity and/or survival. Data on experimental therapy will only be presented (published) after written approval has been obtained from the sponsor and principle investigator (PI). In each case, written informed consent will be obtained from patients before data are included in the ECNM Registry. ECNM Registry Projects (each subproject) will be approved by the local ethics committee (institutional review board) in each participating center.
All data will be collected in a central data base. This data base will be checked periodically by the coordinators. Data will be controlled by the coordinators in order to ensure proper documentation and data quality as well as homogeneity in the dataset, accuracy and precision.
Regulations regulating the distribution, conduct and publications of ECNM Registry projects (data obtained in these projects) are provided in the Contract of the ECNM Registry.
The ECNM registry is rapidly recruiting patients in Europe. The total number of enrolled patients amounted to >4,200 in July 2020.