What is DFMO therapy for neuroblastoma? 

DFMO (short for difluoromethylornithine), also known as eflornithine or Iwilfin, is an oral medicine that has been studied as a treatment for children with neuroblastoma who are in remission after standard frontline or relapse treatment. 

DFMO is sometimes described as continuation therapy because it can be taken after treatment has finished, with the aim of helping reduce the risk of neuroblastoma returning. 

It comes in tablet form and can be swallowed whole, crushed, or chewed, depending on a child’s needs. 

How does DFMO work? 

DFMO affects pathways involved in cell growth and development. In particular, it interferes with an enzyme that some cancer cells rely on to grow and divide. 

By influencing how cells grow and mature, DFMO may help make it harder for neuroblastoma cells to survive and multiply. It is intended to help maintain remission, rather than treat active disease. 

When is DFMO considered? 

DFMO has been studied in children who: 

• Have completed frontline treatment for high‑risk neuroblastoma and are in remission, and 
• In some cases, have completed treatment for relapsed neuroblastoma 

Whether DFMO is appropriate to explore will depend on a child’s individual circumstances and should always be discussed with the child’s consultant.

What do we know from research so far? 

DFMO has been investigated in clinical trials and through expanded access programmes. 

In December 2023, the United States Food and Drug Administration (FDA) approved DFMO for use in children with high‑risk neuroblastoma who have shown at least a partial response to previous intensive treatments, including anti‑GD2 immunotherapy. As a result, treatment guidelines in the United States now include DFMO as a recommended option for these patients. 

In the UK, the Neuroblastoma Clinical Trials Group and the CCLG Neuroblastoma Special Interest Group recommend that DFMO is considered as an option for children with high‑risk neuroblastoma who are finishing frontline or relapse therapy. They advise that this is discussed with families as part of ongoing care planning. It is important to note that DFMO is still not an approved medicine for neuroblastoma in the UK.

While results to date suggest DFMO may help prolong remission for some children, there are still important uncertainties, including how much benefit individual children may experience and the optimal duration of treatment. 

How can DFMO be accessed? 

Access to DFMO is not yet routine NHS care and may be available through specific programmes, which can change over time. 

We provide the latest information about current access routes on a separate DFMO access page, which is updated as pathways change.  

Your child’s consultant and our Family Support Team can also explain what options are currently available. 

If you have questions about whether DFMO or other maintenance therapies are relevant for your child, or about accessing treatment after standard NHS care, please discuss this with your child's clinical team in the first instance.

Page last updated: July 28, 2026