For Patients & Families



All childhood cancers are rare, but there are several Very Rare Tumors (VRT). 

On this EXPeRT website, we will discuss the following:  

  • Types of very rare tumors in children 
  • Causes of very rare tumors in children 
  • Treatment for very rare tumors in children 
  • Coping with very rare tumors 

Nowadays, more children than ever survive childhood cancer. There have been new diagnostic methods and better drugs and treatments available for children and adolescents. However, it remains devastating to hear that your child has cancer, and at times you can feel overwhelmed. It is even more difficult for parents and doctors when a child has a VRT, as there seems to be little information available to help with decisions about the most appropriate treatment, or for predicting the future. There are many healthcare professionals and organizations to help you go through this difficult time, and this website can give you information and support. 

VRT could be considered as an “orphan” disease for many reasons:  

  • There has been a paucity of clinical and biological details and many pediatric oncologists/surgeons may be unaware of the treatment options available 
  • No specific clinical or scientific organizations have been established so far to support their clinical management and related research 
  • It is very difficult to conduct clinical trials on them, and this makes it hard to develop evidence-based treatment guidelines, so their treatments are usually individualized 
  • Dedicated financial resources have been limited. 

Understanding more about the VRT your child has and the treatments that may be used can often help parents to cope. We hope you will find here useful and helpful information.  

Of course, your child’s physician will give you more detailed information. If you have any question, it’s important to ask the doctor or nurse who knows your child’s particular situation. 


Types of Very Rare Tumors in children 

Very rare tumors in children make up less than 1 in 30 of all childhood cancers. They can be broadly grouped as: 

  • Very rare tumours affecting mostly children (e.g. pancreatoblastoma, malignant rhabdoid tumour and pleuropulmonary blastoma) 
  • Very rare tumours usually affecting adults (e.g. epithelial cancer of the digestive system, thyroid cancer and adrenal cortical carcinoma) 
  • Very rare tumours in the head and neck area (e.g. nasopharyngeal carcinoma, salivary glands tumors) 
  • Very rare endocrine tumours (e.g. pheochromocytoma, paraganglioma, neuroendocrine tumors of the lung or of the digestive system) 
  • Very rare brain tumours (e.g. meningioma) 
  • Very rare skin tumours (e.g. melanoma) 


Causes of Very Rare Tumors in children 

The causes of most VRT in children are unknown. But if other family members have had particular types of cancer, this may sometimes suggest that there is an inherited faulty gene in the family. In such a situation, your child’s specialist will talk to you about it and propose you to see a genetisist. 


Treatment for Very Rare Tumors in children 

In most cases, VRT in children are treated with the same treatments like those used for other childhood cancers. They include surgery, chemotherapy and radiotherapy. Your child may need a combination of these methods. Sometimes, however, the treatment for childhood VRT is the same as in adults with this particular type of cancer. 



Usually an operation is done to remove all or as much of the tumor, as possible. Other treatments, such as chemotherapy or radiotherapy, may be given after or sometimes before surgery. They may also be used if an operation isn’t possible. 



Chemotherapy is giving your child anti-cancer (cytotoxic) drugs to destroy cancer or tumor cells in the body. Chemotherapy may be used to lower the risk of tumor regrowth (relapse) or to treat tumors that have spread to other parts of the body. It is usually given as injections and drips (infusions) into a vein. Sometimes, chemotherapy is also in a form of tablets and is given orally.  



Radiotherapy uses high-energy rays to destroy the cancer cells, while doing as little harm as possible to normal cells. It can be used for some types of childhood cancer to treat the cancer cells that may have been left behind after surgery or to shrink cancer. 


Coping with a Very Rare Tumor 

It’s always a difficult time for parents when a child has a rare disease. We will work to prepare information that could help you to understand the tumor and what this diagnosis means for your child and your family 


  • Adrenocortical tumors subsection 
  • Pancreatoblastoma subsection 
  • Pleuropulmonary Blastoma subsection 
  • Salivary Gland tumors subsection 
  • Sex Cord Stromal tumors subsection 
  • Thymic carcinoma subsection 
  • Thyroid carcinoma subsection