FAQ

FCF’S FAQ’S FOLLOW: THE ANSWERS ARE FROM OUR OWN RESEARCH AND EXPERIENCE, AND DO NOT NECESSARILY REPRESENT PROVEN SCIENTIFIC FACT.  FCF DOES NOT RECOMMEND DOCTORS OR PROMOTE ANY SPECIFIC FORMS OF TREATMENT. FCF ONLY PROVIDES INFORMATION FOR THE PATIENT COMMUNITY TO SHARE WITH THEIR PERSONAL PHYSICIANS.

How is fibrolamellar diagnosed?

Fibrolamellar is difficult to diagnose, primarily because its symptoms are attributed to more common causes. For example, many patients complain of general abdominal pain and fatigue.  For most patients with fibrolamellar, the diagnosis is made after a CT-scan or MRI of the abdomen is performed. Imaging studies will often reveal a large liver mass.  The diagnosis of fibrolamellar is then confirmed by a biopsy or after surgery to remove the mass.

What treatments are there for fibrolamellar?*

  1. Surgery –  to “debulk” the tumor and remove any disease visible to the eye of the surgeon. The only treatments with a track record of curing patients are resection or transplant (if possible.)
  2. Chemotherapy – In certain patients chemo can provide partial or complete responses. The only chemo with published results is the combination of 5fu+interferon. In light of the dearth of evidence supporting the use of any particular treatment doctors will often try a wide variety of treatments on a case by case basis.
  3. Local non-surgical treatment:
     a) cryoablation(freezing the tumor)
     b) radio frequency ablation( killing the tumor with heat)
     c) external beam radiation
     d) embolization-with or w/o chemo(for liver only, blood flow to one side of the liver is blocked and sometimes chemo is infused directly into the liver)
     e) nanoknife (irreversible electroporation), this involves the killing of tumors by making holes in them through the application of electrical current
     f) percutaneous hepatic perfusion- the liver’s blood supply is temporarily disconnected from the body’s circulation and chemo is circulated throughout the liver for a short time.
     g) y90 microspheres- small radioactive beads are introduced into the blood vessels that feed the tumor and irradiate them from within
  4. Immunotherapy:
    a) Checkpoint inhibitors – PD1 inhibitors such as Keytruda (pembrolizumab) or Opdiva(nivolumab) may be beneficial alone or in combination with other checkpoint inhibitors, or local treatment such as radiation or cryoablation
    b) CTLA4 inhibitors such as Yervoy (ipilimumab)

The above list may not be complete.

Click here for a very complete overview of treatments for fibrolamellar written by one of our medical advisors, Dr. Choti, on medscape.com 

Are there any clinical trials for fibrolamellar patients?

NCI CLINICAL TRIAL

The National Cancer Institute has announced the launch of a Molecular Analyses for Therapy Choice (MATCH) Clinical Trial, exploring the effectiveness of targeted cancer therapy based on genetic mutation rather than type of cancer. There is a Rare Cancer arm to this trial, so Fibrolamellar patients should be eligible to participate.  Please click THIS LINK below for details and talk to your doctor to see if this is right for you.

Note: while at this point only adults 18 and over may participate, a pediatric version for those under 18 is in development for early 2016. To keep track of developments in the pediatric version check HERE

CASI PHARMACEUTICALS CLINICAL TRIAL

Casi Pharmaceuticals is sponsoring a Phase 2 clinical trial which is currently recruiting patients. The purpose of the study is to determine whether once-daily dosing with ENMD-2076 will be a safe and effective treatment in patients with fibrolamellar. Safety will be measured by looking at the adverse events that may happen and the efficacy will look at the progression of the disease over time.  The primary objective of the trial is to determine the 6-month progression free survival (PFS6) rate when patients with advanced fibrolamellar carcinoma (FLC) are treated with daily oral ENMD 2076.  A detailed explanation of this trial can be found here.  The study chair for this trial is Dr. Ghassan Abou Alfa, an oncologist at Memorial Sloan-Kettering Cancer Center, who is one of FCF’s medical and scientific advisors.

TRANSPLANT/IMMUNOTHERAPY TRIAL

FCF and the Johns Hopkins University School of Medicine are sponsoring a trial of sequential partial liver transplantation followed by bone marrow transplantation from the same living related donor. This treatment applies to patients whose cancer remains confined to the liver but is too widespread to be removed by surgery or treated by a liver transplant from a deceased donor. The purpose of this combined treatment is to reduce the risk of the cancer coming back after the liver transplant   The bone marrow transplant may reduce the risk of the cancer coming back in two ways.  First, patients who have combined bone marrow and solid organ transplants may be able to get off all anti-rejection drugs, which inhibit the immune system from destroying cancer cells.  Second, the donor’s bone marrow contains cells of the immune system, which can attack any cancer cells that remain after the liver transplant. This trial is actively recruiting patients.  More information is available through clinicaltrials.gov

What are the causes of fibrolamellar?

It is not presently known. Some suggest an environmental link, yet here is no firm evidence pointing to this cause. Recent research has shown a distinct mutation (chimera) present in all those with fibrolamellar. Research is continuing to determine if this chimera plays a role in a patient developing fibrolamellar.

How aggressive should follow-up CT scans be, balancing between early diagnosis of recurring disease and radiation exposure?

A CT scan has about 500x the radiation of an xray. MRI’s are magnetic and have no known adverse effects. Each patient’s doctor should determine which is the best scan to use at varying points in time as different scans yield different information.

What doctors are familiar with fibrolamellar?Samreen Raza, M.D., from left, Claire Verschraegen, M.D., Michael Boggs, class of 2013.

Click here for a listing of doctors – surgeons, oncologists, radiologists – whom we know to have treated fibrolamellar. Contact one of the comprehensive cancer centers in the United States for additional names if your area is not listed.

What is the survival rate for fibrolamellar?

Reported survival rates range from 7 to 40 percent five years after diagnosis. Complete surgical removal of the tumor improves this survival rate. Combined with early detection, complete removal of the tumor can also decrease the rate of recurrence.

Patients whose tumors are not removed surgically have an average survival of 12 months, while those who had tumors completely removed survived for an average of 9 years. The Foundation is aware of fibrolamellar patients who remain NED (no evidence of disease) for as long as 20 years after complete removal of the tumor and before any spread of disesase.