Myelodysplastic syndrome, MDS for short, is a group of acquired disorders of the bone marrow that disrupt the formation of blood. In its advanced stage in particular, MDS increases the risk of acute myeloid leukaemia developing.
Juvenile myelomonocytic leukaemia, JMML for short, is a rare form of leukaemia for which the only treatment currently available is a bone marrow or stem cell transplant. The rate of recurrence after transplantation is high, and the chance of recovery is currently just 60%. Five to ten children and adolescents are affected by this disease each year in Switzerland and are eligible to participate in the study.
The main aim of this research project is to improve the diagnosis and cure rates of children and adolescents with MDS and JMML. Blood and bone marrow samples obtained from patients during routine examinations will therefore be sent to regional reference laboratories for analysis. The aim of analysing these samples is to identify cellular and molecular features associated with the different forms of MDS and JMML. In conjunction with the clinical information about the course of the disease and the patient’s condition, this should lead to a better understanding of these rare diseases. This is the basis for developing new forms of therapy in future and giving affected children and adolescents a better chance of recovery.
The University Hospital Freiburg in Germany is responsible for the international implementation of the study as sponsor. The Swiss Paediatric Oncology Group (SPOG) is responsible for performing the study in Switzerland (sponsor representative).
In short
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Information on the diagnosis and the course of MDS (myelodysplastic syndrome), JMML (juvenile myelomonocytic leukaemia) and Noonan syndrome (a genetic disorder) with JMML is being collected and analysed.
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The main aim of this research project is to improve the diagnosis and the chances of recovery.
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The study patients benefit from standardised disease assessments and treatment recommendations. The study may also improve the treatments for future patients.
