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:

  • Imatinib 
  • Sunitinib 
  • Regorafenib 

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.) 

PLease CLICK ON a country to find out about treatments and EUROPEAN REFERENCE NETWORKS (ERN) CENTERS OF EXCELLENCE IN THIS COUNTRY.
Austria1
Belgium3
Bulgaria0
Croatia0
Cyprus0
Czech Republic2
Denmark1
Estonia0
Finland3
France7
Germany10
Greece0
Hungary1
Ireland0
Italy9
Latvia1
Lithuania1
Luxembourg0
Malta0
Poland4
Portugal2
Romania0
Slovakia0
Slovenia1
Spain4
Sweden2
The Netherlands2
United Kingdom3

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:

Patient organisations

 

Other useful links and information