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Experts including Dr. Barbara Haber and Dr. Elizabeth Rand provided detailed updates on Biliary Atresia at the annual BA Education Day. CHOP's Biliary Atresia Clinical Care Program has been caring for children with the liver disorder since the 1970s.
Dr. Barbara Haber: The current concepts about biliary atresia — you may have seen some of these slides before but that — in the past several decades the interest in biliary atresia has really grown. In part, I would say, some of this has to do with our interest here in being a biliary atresia center. We have a group of people who are interested in research related to biliary atresia, clinical care related to biliary atresia, and transplantation for biliary atresia. And along with that, nationwide, there's something that originally was called the Biliary Atresia Research Consortium that is now called ChiLDREN, and the goal there is to have a group of physicians around the country think about what needs to be done and do whatever they can to make outcomes better.
I'll start out with a very brief history. It used to be about ten slides, now it's about three. Is that the first description of biliary atresia in the western world is attributed to Dr. John Thompson who presented detailed description of 50 cases. And prior to that there were just sporadic case reports. In his first series of cases in 1891, he drew out each biliary tree. And I'm sure that for some of the parents they will say, "That's my child." In some cases you can see that there's a gallbladder and everything else is missing. In other cases there's atresia, and it's somehow not connected to the intestine. And he drew out each case. And he even goes as far as some things that look like this, which is a choledochal cyst that can happen with biliary atresia.
From there, nothing more happened for the next hundred years, and there was no change in science or clinical care, and it was considered a fatal disease by the age of 2 years. Until this man came along, Dr. Kasai, who lived a long life but died last December. Dr. Kasai invented this operation, and he actually worked on it while at — spending a year or two, I believe it was, during his training at CHOP he worked with C. Everett Koop. And what he invented is called the Kasai hepatoportoenterostomy also known as HPE. And here is the liver. Here is the stomach. And what he does is he takes the intestine, cuts it there, and brings up a portion directly up to the liver. And that substitutes for the drainage system. So the child's gallbladder and all that shriveled material that isn't flowing is taken out and replaced with a piece of intestine.
Moving on to what causes biliary atresia. As you could see, the pictures were very varied, and we really don't know. It seems like each case is very different. In the way we look at complex diseases, like biliary atresia, is that we often think that there's a number of different factors. That there could be genetic factors, maybe something in the — a toxin in the environment, some sort of immune issue, and some sort of viral issue. Thank you.
It's likely to be a combination of each of those factors. I put this in — it wasn't going to be part of my talk, but it's back by popular demand because somebody specifically asked, "Are we going to hear about the sheep again?" So — not in detail this year — but I can give you a brief background and, if you recall, in New South Wales, Australia, originally there were two different reports, one in 1964, and one in 1988, where there were groups of lamb or cows, all living within proximity to this dam, who gave birth to offspring with biliary atresia. And what was unique about that time in history is that there had been a severe drought, and the pregnant animals grazed on land that had been previously submerged by the dam, around the dam.
Recently, if you knew about Australia, there was another severe, severe drought and again it happened. And you can't quite see it, but there are — this is a little lamb with biliary atresia along with this little frail guy also has biliary atresia. And after observing this, one of our researchers here managed to get this plant, which is called — I can't pronounce it. Dysphania, which is a very unusual plant native to that area, and they're working now on breaking down the compounds and testing it out in an animal. And the animal that they're testing it in right now is a zebrafish, which has a very short life span. And it looks like they got it down to a group of compounds, 20 to 50 that might trigger biliary atresia in certain circumstances. It's very encouraging. But it's not the whole story, guaranteed.
So what is biliary atresia?
Biliary atresia is a progressive, meaning that it keeps on moving up the biliary tree, fibro-obliterative process of the external biliary tree. It's defined by a characteristic biopsy and obstruction on the cholangiogram. It only starts in the perinatal period. You can't get it at any other time in life. It's something that shows up within the first couple of months after birth. And because it is progressive, we've had some children who, on their first biopsy it's not clear, or on their first DISIDA it is not clear. And you just have to go back and think it still could be and repeat some of those tests.
The typical presentation is that the child's jaundice. They looked healthy prior to starting to look yellow. It started early in life and the bilirubin is generally greater than 20 percent of the total. In 90 percent of the cases, the gallbladder is shriveled and in 20-30 percent it's associated with some anomalies. There's a term used these days called B-A-S-M, called Biliary Atresia Splenic Malformation. And in those kids they either have asplenia, polysplenia, malrotation, midline liver, and a number of other things.
The stools, and I always like this picture, and this is pictures of poop. And not used in this country, but acholic stools, those pale stools that people talk about are not always as obvious as people think. Numbers 1, 2, and 3 are the pale types of stools that you might see in biliary atresia. Whereas, 4, 5, and 6 are what typical baby poop looks like. I'm sure you guys all remember, know more about that than I do, hopefully, these days.
The evaluation is the ultrasound, DISIDA, liver biopsy, cholangiogram. In each of those steps, here we typically do all the steps, and with that we feel that we're very accurate. But it's important to move quickly from the first test to the last test. Not that you have to be reckless, but the goal is to get somebody to surgery as soon as you can without being wrong. You don't want to do surgery on somebody who doesn't need it.
When I talked about what B-A-S-M looks like, this is a picture where you can see the liver's in the midline and you either have too many things that would normally be on the right side of your body or too many things that would normally be on the left side of your body. So, if normally left has two lobes of the lung and your right has three lobes of the lung, if you have no spleen, you'll have three lobes of your lung on both sides. And, if you have multiple spleens, you'll have two lobes of your lung on both sides. And it's obviously biliary atresia with some sort of developmental anomaly. And that's why that grass that you munch on can't be the whole story.