Paediatric Adolescent Wild-type Syndromic Gastrointestinal Stromal Tumour (PAWS-GIST)
Paediatric/adolescent wild-type syndromic gastrointestinal stromal tumours (PAWS-GIST), differ from adult gastrointestinal stromal tumours (GISTs) in many respects. If the genetic make-up of the tumour cells is examined (mutation analysis), typically no mutations are found in either of the genes which are frequently altered in adult patients (KIT and PDGFRA), and this is referred to as "wild-type GIST".
PAWS-GIST is most prevalent among girls and young women. Typically, the illness starts in the stomach, where several primary tumours can present themselves at the same time. Metastases in the lymph nodes are also more common than in the case of ordinary GIST. PAWS-GIST is extremely rare and the scientific community still has limited knowledge about that type of paediatric cancer. PAWS-GIST is part of the paediatric cancer family.
For more information, please consult our dedicated page on paediatric and adolescent wild-type syndromic GISTs.
cancer family: Paediatric Cancers
Paediatric cancers are malignancies that occur in children and adolescents. The main types of paediatric cancers are leukaemia’s, neuroblastomas, Wilms’ tumours, brain tumours, rhabdomyosarcomas, lymphomas, retinoblastomas, osteosarcomas and Ewing’s sarcomas. These types of cancer account for the second most prevalent cause of death in children.
Approved treatment by the EMA
Currently, no treatment have been approved by the EMA specifically for PAWS-GIST.
In PAWS-GIST patients, adult guidance is being followed. Currently available products:
With varying degree of reimbursement, these treatments are reimbursed in several Member States.
How to use the data
Please note that the data available are only meant to inform you on the availability of a specific treatment in European Union Member States and the United Kingdom. However, the treatment might only be available in specific stages or settings of the disease. Please contact your doctor to discuss any questions you might have.
(The information above was obtained during the year 2018, the latest update took place in February 2020.)
More useful information
Definition of European Reference Networks
European Reference Networks for rare, low prevalence and complex diseases (ERNs) are virtual networks involving healthcare providers across Europe. They aim to facilitate discussion on complex or rare diseases and conditions that require highly specialised treatment, and concentrated knowledge and resources.
For more information on Paediatric/adolescent Wild-type Syndromic Gastrointestinal Stromal Tumour (PAWS-GIST), please visit the following links: