Frequently Asked Questions

How is IWILFIN different from high-risk neuroblastoma up-front treatment?

IWILFIN is a prescription medicine used for the maintenance treatment of children with high-risk neuroblastoma.   IWILFIN is used after your child has achieved remission through the completion of upfront therapy, such as chemotherapy and immunotherapy.

What is maintenance therapy, and why is it important?

Maintenance therapy refers to a treatment strategy used in certain diseases, particularly cancer, to prolong the period of remission or control the disease after the initial treatment. The purpose of maintenance therapy is to prevent relapse, reduce the risk of disease progression, or manage residual cancer cells.

Who is IWILFIN for? 

IWILFIN is for children with high-risk neuroblastoma who have achieved remission through the completion of multiagent, multimodality therapy (such as chemotherapy, stem cell transplant, and immunotherapy).

How does IWILFIN work? 

IWILFIN limits an enzyme called ornithine decarboxylase, or ODC. ODC helps create polyamines. These amino acids are key to cell growth and survival and play an important part in forming neuroblastoma tumors. IWILFIN limits how much ODC the body activates and slowing ODC activity lowers polyamine levels. Fewer polyamines cut down on the growth of cancer cells and the formation of new tumors and can reduce the risk of relapse.

How effective is IWILFIN? 

In clinical studies, the risk of high-risk neuroblastoma relapse was reduced by 52% for patients taking IWILFIN when compared to patients not taking IWILFIN. 

Is IWILFIN chemotherapy?

IWILFIN is not a type of chemotherapy.  It’s a prescription medicine that acts as a targeted therapy to disrupt the polyamine pathway responsible for tumor development and growth. 

How is IWILFIN given?

IWILFIN is an oral pill that can be taken at home. For patients who are unable to swallow whole tablets, IWILFIN tablets may be chewed or crushed and mixed with two tablespoons of pudding. 

How long is the course of IWILFIN treatment?

Based on your child’s weight, your doctor will prescribe from 1 to 4 IWILFIN tablets, which will be taken twice a day for two years. Adjustments to dosage based on your child’s weight, particularly for younger growing children, should be conducted every three months from the initiation of therapy and as necessary for the duration of therapy.

Are there any dietary or lifestyle changes necessary while on treatment? 

IWILFIN is an oral tablet taken at home twice daily, and there are no specific dietary restrictions for IWILFIN treatment. 

What are the side effects of IWILFIN? 

The most common side effects of IWILFIN are hearing loss, anemia, vomiting, and dehydration. 12% of patients who received IWILFIN reported new or worsening hearing loss, which was sometimes reversible by adjusting IWILFIN dosage.  Hearing assessments should also be performed prior to initiation of therapy and at 6-month intervals, or as clinically indicated, to monitor for potential hearing loss.

How are IWILFIN side effects managed?  

Please report all side effects to your doctor. Your doctor might choose to pause treatment or reduce your IWILFIN dose if you are experiencing certain side effects. If treatment is paused, your doctor may recommend restarting IWILFIN at a lower dose and slowly increasing it until you can tolerate it. You should never adjust your dose without speaking to a healthcare provider.

How can I access IWILFIN? 

Once your doctor submits your IWILFIN prescription to IWILFIN Cares, a dedicated team of specialists will help you understand the process of getting your medication.  IWILFIN is distributed by a specialty pharmacy and conveniently shipped to your doorstep.

What is IWILFIN Cares? 

IWILFIN Cares is a customer service program specifically designed to support your IWILFIN treatment journey. IWILFIN Cares Specialists will work with you and your healthcare team to figure out insurance coverage and eligibility for financial support programs. IWILFIN Cares also offers education and resources to help you start and stay on therapy.

Will my insurance cover IWILFIN treatment? 

Coverage for IWILFIN will vary by insurance plan. An IWILFIN Cares Specialist can help determine your insurance coverage and confirm your eligibility for the financial support programs available to you. You can reach an IWILFIN Cares Specialist by calling 1-866-750-8796, option 6, Monday through Friday (9 AM to 6 PM ET).

Are there any other names for IWILFIN? 

The established name for IWILFIN is eflornithine or difluoromethylornithine, also known as DFMO. 

Advocacy & Support Groups

Childhood Neuroblastoma Cancer Foundation

The Children’s Neuroblastoma Cancer Foundation (CNCF) is comprised of families just like yours. It was created in 2000 to support the patients and families affected by neuroblastoma and to help find a cure. 

Candlelight’s Childhood Cancer Family Alliance

Candlelighters Childhood Cancer Family Alliance – founded by families of children with cancer FOR families of children with cancer; to be a light in the darkness.

We are there each and every day providing emotional, educational, and practical support to families of children with cancer.

End Kids Cancer Foundation

The mission of the End Kids Cancer Foundation is to provide financial support for clinical research and clinical trials that will cure neuroblastoma (NB), with an emphasis on non-toxic treatments. Our narrow focus on neuroblastoma is designed to increase the success of finding a cure.