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Risk stratification is used in paediatric haematology-oncology to adapt the intensity of patients’
treatment to their own individual risk of failure. It is part of standard of care, is based on the extent
of the disease and, increasingly, on tumour biology and response (e.g. in neuroblastoma and
leukaemias) [9] [10] [11]. There has been recent progress in the classification of severalpaediatric
malignancies (such as medulloblastoma, high grade glioma, ependymoma and rhabdomyosarcoma)
based on specific aspects of their biology [12]. This will lead to new “biomarkers” that can be used
in clinical practice to improve risk stratification and to better adapt existing and new treatments
(objective 1) to each patient.
Objective 2:
Precision cancer medicine
To use improved risk classification as well
as biological characteristics of both the
tumour and patient (such as molecular
and immunological factors) to help guide
decisions on which therapies to use.
Strategy
g
To analyse the specific biology (molecular profiling) of both the patient and tumour at
the point of diagnosis and throughout treatment to improve risk stratification for adapted
individual treatment by identifying:
h
Patients with a high probability of cure with standard treatment, who may be
proposed new or reduced interventions to decrease the risk of late effects;
h
Patients with a poor prognosis tumour to whom innovative therapies should be
proposed as early as possible, to increase their probability of cure.
Actions
1. Run prospective clinical trials with innovative design and methods to confirm the use of
biomarkers and algorithms in risk stratification, treatment allocation and disease
monitoring;
2. Enhance the collaboration between specialists such as biostatisticians, clinicians and
biologists;
3. Expand the availability and accessibility of biomarkers for clinical and research use by
setting up a network of the necessary molecular laboratories;
THE SIOPE STRATEGIC PLAN
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Credit University of Nottingham, Children's Brain Tumour Research Centre, United Kingdom