Diary November & December 2001

Saffy bloated due to steroids and IV fluids. Doesn't she look like Alfred Hitchcock?

Early Signs

Shortly after Saffy was born in August 2001, a red birthmark appeared on her side.  It was a hemangioma, a benign vascular tumor, that is relatively common on the skin of infants--about 1 in 100 full term babies and about 1 in 4 premature babies get them.  Even though five more hemangiomas appeared on her skin (on her head, arm, hand, and foot), we were told not to worry about them.  We were informed that they would grow in size for several months and would then start shrinking and be gone in a few years.  They are very common, and usually only require attention if they interfere with the eyes or nose or such.  We didn't give them much thought.

When Saffy was about two months old, she began to have trouble with feeding.  She wasn't eating as much as before and her weight gain slowed.  (See Saffy's Growth for daily weight.)  She began to vomit when she ate too much, and her abdomen appeared to be distended.  She was prescribed medication for reflux; but, after a few more doctor's appointments and no improvement, our pediatrician recommended she be hospitalized (in late November).

Diagnosis

At the hospital, the pediatrician discovered that Saffy's liver was enlarged.  It was pressing against her stomach and other organs, which was why she was having problems eating.  An ultrasound revealed numerous lesions in her liver, which was nearly twice normal size.  Her doctors suspected two possibilities: either she had cancerous tumors or hemangiomas of the liver (hepatic infantile hemangioendothelioma).  After a CT scan, they confirmed the lesions were hemangioendothelioma.  A biopsy was discussed but was overruled due to the danger of excessive bleeding since hemangiomas are a mass of living blood vessels.

While hemangiomas are rarer in internal organs, some people have them and don't even know it (when the lesions are small and not growing).  A typical case that requires attention usually means a few growths (five or six is common); however, Saffy has 40 or 50 in her liver.  This is extremely rare, and some of Saffy's doctors even described her condition as unique.

Saffy's enlarged liver is still functioning, but the large size is interfering with other organs.  If the growth went unchecked, she would eventually be unable to eat and her lungs could even collapse.

Besides the dangers of the hemangiomas growing, they also pose a risk to her heart.  A hemangioma is a mass of living blood vessels, and the tumors are making her heart work extra hard to move around more blood.

Treatment Options

We discussed treatment options with several doctors.  Basically, there are five options (in order of severity):

• steroids

• Interferons

• angiogenesis inhibitors (experimental)

• embolization

• liver transplant

The least invasive option is steroids, which can stop the growth and/or shrink the hemangiomas.  We've been told varying information about the success rate of steroids, but it appears they work in only about 30% of patients.  Interferons work in a similar way but have more side effects and take much longer.

The experimental procedure involving angiogenesis inhibitors is being researched by Dr. Judah Folkman from Boston Children's Hospital at Harvard Medical School.  A urine sample was sent to Boston to see if Saffy is eligible for this procedure.

Embolization is a surgical procedure to cut off the blood vessels supplying the hemangiomas.  This can involve multiple operations to try to locate all the vessels supplying blood to the tumors.

Finally, the most invasive option is a liver transplant.  This would most likely be a living relative donor transplant, where a matching relative (such as Daddy or Mommy) would donate a lobe of their liver.  Saffy's new liver would grow along with her.

It would probably be better to treat Saffy medically (the first three options) as opposed to surgically (the last two).  A liver transplant would mean she would need to take anti-rejection drugs for the rest of her life.  Each option has varying degrees of success.  The good news is that the chances of successfully treating her condition have improved significantly in the last few years.  To quote Dr. Folkman's lecture, "the mortality from liver hemangioma, which was up to 40% prior to 1990, is now down to approximately 3%."

Treatment Decision

One difficulty is that Saffy's many doctors don't always agree on the best course of action.  At one point, she was even scheduled to undergo surgery (biopsy and embolizations), but this was later changed again to give the medical options a chance.

The current plan is give the steroids a chance.  While the success rate isn't that high, it is the least invasive option.  Even if the steroids work, they might just slow or stop the growth as opposed to shrinking the tumors, so other procedures may be necessary anyway.  Unfortunately, it takes a few weeks to see if the steroids have an effect, but Saffy is stable and able to be treated at home.

Saffy's enlarged liver is pressing against her other organs, but she is able to eat in moderation.  Her heart is working harder than it normally should (to supply the hemangiomas with blood), but she is not yet in danger.  Blood tests revealed her to be anemic, so she received a blood transfusion while in the hospital.

Saffy started on intravenous steroids and then switched to oral steroids, so she could continue her treatment at home.  She was discharged after spending a week in the hospital.

Other Cases

Saffy's condition is rare, but we've been lucky to find other parents on the Internet who faced the same situation.  Jeff Compagner put together a very informative web site at www.MariasLiver.com about his daughter Maria and other children who faced the same condition.

In 1996, Maria was diagnosed with hepatic hemangioendothelioma at two months of age (sound familiar?).  She was treated with steroids, Interferons, chemotherapy, and five embolizations.  After a year in the hospital, she received a liver transplant.  Maria is now five years of age and on a waiting list for a second transplant.

We have been in communication with Jeff as well as Julie Tovar and Matthew Burger about their son James and Crystal Haydt about her daughter Adrianne.  Since Jeff published his web site, he has learned about three other children with this case.  Of those, two have been successfully treated with chemotherapy without the need for a transplant.

Update Early December

After a few weeks on steroids, Saffy is doing well but it is still too early to tell if her treatment is working.  Based on physical exams and ultrasounds, her Hematologist/Oncologist does not believe her liver has grown larger.  Her next exam is scheduled for mid-December; and, hopefully, we'll have a better idea if the steroids are having an effect.

Update Mid-December

Unfortunately, Saffy has not responded to the steroid treatment.  She will continue for another two weeks, but our doctor has little hope that it will be successful.  She is stable and eating well, but has not gained weight in the last month.  Blood tests revealed she was anemic again, so she was admitted to the hospital for a day and a half for another blood transfusion and some tests.

We are currently discussing further treatment options with our doctors.  While it wasn't mentioned above, chemotherapy appears to be a promising option and will probably be our next choice.  It has been used successfully in similar cases and has a relatively short treatment regimen.  Alternatively, Interferons require 7-10 months to work and have some serious side effects.  (Not that chemotherapy is without risks.)

We met with the liver transplant specialist again, but still hope that option won't be necessary.

New Updates

Dec 29 2001 News: Unfortunately, Saffy is back in the hospital as of yesterday.  She had trouble feeding and has not gained weight in six weeks.  The next steps to rectify that problem as well as treat her liver condition are uncertain at this point.  We're not sure how long Saffy will be in the hospital but will add more information later.

Dec 31 2001 News: Saffy took a turn for the worse today and is now in the Pediatric Intensive Care Unit (PICU).  To add to her other woes, she went into respiratory failure this morning and is now on a ventilator.  All her problems appear to result from her internal hemangioendothelioma, so we're fairly certain we will accelerate plans for chemotherapy.

www.usticke.org Saffy Liver Condition Home

Previous Saffy Liver Condition Diary - Next Saffy Liver Condition Diary