A little bit of Congressional Help, Please

We have been getting scary news (and here)from around the world of TPN over the last few weeks: some of the important ingredients in TPN and the supplies that go with it are in critically short supply, as in there is NONE to be found anywhere. This isn't such a big deal for Ellie right now since she is still on just D10 overnight, but if she should need to go back on TPN, there are key elements that would need to be rationed or omitted from her mixture like calcium, magnesium and other trace elements that typical people get through their complete diets.

The cause of this is uncertain but two primary reasons have been suggested: one, that there aren't enough raw materials available to make the additives and two, that these very important drugs just aren't profitable for the drug companies and they have made a cold business decision to stop making the drugs or have slowed the production down.

If they were making widgets or car parts, this would be one thing, but we are talking about really important things that will have real consequences for thousands of people around the country including typical people who are in the hospital, need antibiotics or chemotherapy.

So what is there to do about this other than worry and hope?

One simple thing that we can all do is let our representatives in Washington know about the issue and the very real consequences of letting this problem continue. There are things that even a gridlocked Congress can do to urge and nudge the FDA and the drug companies to fix this problem but Congress won't act unless they hear from their constituents, who vote and are vocal.

A simple letter to your member of Congress and Senators still has power. Find them here, and write them a simple letter telling them who you are, how you or your child are or will be affected by drug shortages and ask for their help. These people have connections and do solve problems for voters every day. Ask them to help and also ask them to consider sponsoring the bills that have been introduced to increase oversight over this emerging problem. Also, if your Congressman is on this list or or Senator is on this list, they sit on an important committee or subcommittee and could be even more influential in getting answers and solving this mess.

For all of the kids and adults with short bowel and the range of other conditions that require the more than 150 medications that are currently in short supply, we thank you for your help.

Ellie got the go-ahead this week to go off of TPN.

Again.

Yes, starting with next week’s shipment, she will only be getting fluids without nutritional additives and without Omegaven.

ONE MORE TIME!!!(click on me) listen while you read: NO MORE TPN!!!

Two and a half years ago she went off of TPN for the first time. She was growing enough in both in height and weight for her team to think she could stop her TPN(total parenteral nutrition, IV nutrition). She was off of TPN for 6 months that time.

Ellie's 5th Birthday Party (she covers her ears when people sing)

Before her surgery to lose her ostomy, we put her back on to get her ready and fatten her up for surgery and keep her weight up during the recovery time while her reconnected bowel learned how to behave. That strategy worked and she did very well and for the next 2 plus years. But, she has done well....ON TPN.

The problem is that TPN even with Omegaven, is hard on her body. We trust that her liver is protected but we never know for sure. Because of this we have been trying and trying for 2 years to get her off again. We have been trying different things on and off to wean her from the calories and fats that come with TPN. She has been growing on and off for over 2 years. But not enough and not consistently. Which sucked. She would put on weight and then a round of bacterial overgrowth would kick her back down to more days of TPN.

This Wednesday we went in for clinic with Dr. Lo (the TPN guru at Children’s) and Ellie had gained enough weight and grown enough for the team to feel that it was time for her to come offof IV nutrition again. She will still be on IV hydration 7 nights a week, but no IV nutrition.

Those of you that have met Ellie have seen how she runs from place to place, swims all summer, dances from room to room and lives life on her toes. The heat of summer is not the time to decrease her IV hydration.

So we are now in a two month trial period away from the complex land of TPN. As with all things short gut we understand that TPN may come back to Ellie if she needs it, but for now this significant step is great and makes us think about the terrifying possibility of a night without the whirr of an IV pump coming through the monitor.

We have a busy summer planned for Ellie. Complete with a Lego camp with 100,000 Legos. We have discovered that soccer is probably not a good option for Ellie. We signed Ellie up for soccer every Saturday afternoon. And after 2 sessions we agreed that soccer is not the sport for Ellie-Here we work all day everyday to get her to gain weight and then we sign her up for something that is just one giant calorie burn. We would watch her run around for over an hour struggling to keep up with the other kids, and then it occurred to us...

WHAT WERE WE THINKING?????

So no more soccer.

For now Ellie will continue to dance ballet, go to school (Kindergarten starts in September), take swimming lessons (at the Y!) and be a very typical 5 year old. And well, I am pretty happy about that. In the future, I think that she will rock T-ball as a left field daisy picker.

About her brother Will: He is an eating machine! He is eating, growing in to a tank of a baby. I was watching him chew the other day and realized that what I was watching took us 6+ months of feeding therapy with Ellie. It was a thing of beauty. I was mesmerized:

He has also decided that crawling is for sissies. I honestly think he started pulling himself to standing and cruising because we told him that Ellie started cruising at 7 months. A Month behind his sister Will started pulling himself up to standing and walking. Bring on the crash helmet.

This is an article that I wrote for Complex Child Magazine back in April. We are working on a new post as well.

Current Information on Omegaven
by Abby Brogan

My daughter Ellie was born in April of 2006. She came a month early, but over all my pregnancy was normal and she was initially doing very well. Things began to change after her first hour. The next 24 hours were something I wouldn’t wish on anyone. It ended with both of us in a new hospital, her recovering from exploratory abdominal surgery, and the very nice surgeon explaining to us that Ellie was born missing 90% of her small bowel and 30% of her colon. After she fully explained what Ellie’s GI system looked like, she started talking about what Ellie’s life was going to be like and what the future could be like. It was a very scary and grim conversation.

Ellie as a baby before Omegaven

Starting TPN

Ellie’s GI tract was not able to absorb enough nutrients to sustain life, so she was placed on IV nutrition support, also called Total Parenteral Nutrition. Total Parenteral Nutrition (TPN) is made up of two main parts. The first part contains all the fluids, vitamins, minerals, amino acids, and medications. The second half of the mixture is the lipid, consisting of a fatty acid that has been broken down enough to be absorbed through the blood stream, and blended into an emulsion that keeps the fats from separating from the fluids. TPN enables an individual to live and grow even though he or she cannot eat.

However, the down side of TPN is that for most individuals on it for an extended period of time, particularly children under two years of age, it will also cause a build-up of fat in the liver causing damage. For these individuals, there is a race to rehabilitate the GI tract and get off TPN before the liver becomes too damaged. If they cannot get off TPN fast enough, they often will be listed for a liver transplant and, if needed, other organs as well. For those ineligible for transplant, liver damage can lead to death.


What is Omegaven?

In 2001, Children’s Hospital Boston started doing research on TPN to find out what part of TPN was causing liver damage. Mark Puder, MD, a surgeon, and Kathleen Gura, PharmD, a pharmacist, collaborated on the research with other doctors at Children's Hospital Boston. Through studies done on mice, they discovered that it was the lipid portion of the TPN that was causing the damage. But the fats contained in lipids are essential for the body, so they needed to find an alternative.

Dr. Gura had previously used Omegaven, a lipid emulsion derived from fish oil that contains omega-3 fatty acids, to treat a patient with a soy allergy and severe fatty acid deficiency. From this experience she suggested they try Omegaven in their trials.

The results were very promising.

In an article written by Children’s Hospital Boston, Dr. Puder explained the results that showed that parenteral nutrition without the fat makes the liver fatty, and by adding the Intralipid, it becomes even more so.¹ "That fat becomes more or less a fuel that burns the liver, causing it to become inflamed....But the Omegaven is an anti-inflammatory lipid that prevents fat build-up in the liver. So if there's no fuel—fat—there's no fire—injury."¹

The results were so promising that on September 26, 2004, the team at Boston Children’s Hospital started the first child on Omegaven. They saw improvements in one week, and a month later the child’s liver function tests were good enough that he was taken off of the transplant list. In most children, during the first month or so the direct bili (liver function test) goes up and then comes down. It is known as the Omegaven roller coaster. But, during this time you get to watch your child turn pink again.

Ellie's bili chart

Some hospitals are using Omegaven in combination with regular vegetable oil-based lipids such as Intralipid. Dr. Gura commented on this, stating, “Based on published papers, it appears that children with PN liver injury do better with Omegaven alone (not mixed with Intralipid). The Diamond paper from Canada actually shows this, although they still like to do the combination until the child fails and then they switch them to plain Omegaven.”²

To date Children’s has treated 166 children with Omegaven and administered over 100,000 doses. Omegaven has helped almost all 166 children, but the team has learned that the faster these children get on Omegaven, the better they do. Using it as a last resort or rescue is not always successful. The damage to the liver is already done.


How to Get Omegaven

Omegaven has not been approved by the FDA to be used broadly. Because of this, it has a Compassionate Use Only status, meaning that it can only be used if it will save the child’s life. To show that the child needs Omegaven, he must have failing liver function tests, including a direct bilirubin number greater than 2 mg/dL, indicating liver damage called cholestasis.

In the beginning, patients had to transfer to Boston to get Omegaven therapy. Now, thanks to the passionate outreach by Puder and Gura to the medical community, it has been used by almost every free-standing children’s hospital in the United States. Physicians can also apply to be able to administer Omegaven themselves, and the Boston Omegaven team has gone so far as to publish their therapy instructions with the Oley Foundation, a support group for those who rely on TPN.

Each year, more and more insurance companies are paying for Omegaven enabling patients to be treated locally and not requiring them to travel to Boston. The FDA now allows billing of insurance companies upon request. Approval, however, isn’t mandatory, and insurance companies are not required to pay for it. For a list of the hospitals that have used Omegaven and the insurance companies that have covered it, visit the Short Gut Wiki.

Making Omegaven available to everyone and covered by insurance will continue to be an issue until it is approved by the FDA for use in all people receiving TPN. Currently, Fresenius Kabi AG, the manufacturer of Omegaven, is working with Children’s Hospital Boston to get the necessary data for a new drug application submission to the FDA. The process to get a drug approved in the US is lengthy. There was hope in the beginning that additional animal testing would not have to be done or could be avoided; however, these tests were ultimately required and are currently underway.

Children’s Hospital Boston has not seen any adverse effects of the use of Omegaven, but they are monitoring all their children with long-term blood work and other follow up examinations.


Success!

The child that received the first dose as an infant in 2004 is still on TPN and Omegaven at age seven, goes to school and has shown no adverse effects from the TPN that likely would have destroyed his liver just a few years ago.

A recent picture of Ellie

My daughter Ellie was the 23rd child to receive Omegaven, and has now been on it for almost five years. She is doing well, growing and thriving. Both my husband and are so thankful for Omegaven, and the doctors that brought it to our attention. I will not let myself imagine where she would be without it.


Thanks to Mark Puder, MD, and Kathleen Gura, PharmD, for their personal assistance in providing information for this article.

Abby lives in the Boston area with her husband and daughter Ellie. Ellie was born in 2006, missing 90% of her small bowel and 30% of her colon due to Jejunal Atrisia. Ellie is diagnosed as having Short Bowel Syndrome. Ellie has had multiple surgeries since birth and is on tube feeds and Home Parenteral Nutrition (HPEN). Abby and her family have faced the many challenges that come along with having a child with tubes, yet lead a remarkably active lifestyle. Abby is also a consumer advocate for ThriveRx. Please feel free to contact Abby at
1-774-270-3425 or via email at abrogan@ThriveRx.net.

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¹http://www.childrenshospital.org/dream/dream_fall06/fishing_for_the_right_solution.html See also http://pediatrics.aappublications.org/cgi/content/full/118/1/e197
²Personal communication with Dr. Gura. The study she cites is Diamond IR, Sterescu A, Pencharz PB, Kim JH, Wales PW. Changing the paradigm: omegaven for the treatment of liver failure in pediatric short bowel syndrome. J Pediatr Gastroenterol Nutr. 2009;48(2):209-15.

Trouble for Omegaven in Maryland

Gib just learned that there is a bill before the Maryland legislature that will prohibit the manufacture, distribution or sale of any products made from Atlantic Menhaden, the tiny smelly fish that we have been told is the primary source for Omegaven's omega-3 oils. HB1142, as it is known, is scheduled for a hearing on March 16. It is very vague but could mean the end of omega-3 supplements, medications, possible research in Maryland unless they can be shown to be derived from non-Menhaden sources.

http://house.state.md.us/2011rs/bills/hb/hb1142f.pdf

We are not fans of the commercial menhaden industry and believe their fishing should come under more stringent control to look out for the oceans and the Chesapeake Bay, but this approach could have real consequences for people, both old and young in Maryland and beyond.

If you would like to have your opinion heard on this, especially if you live in Maryland, letters can be addressed to:

ENVIRONMENTAL MATTERS COMMITTEE
Delegate Maggie McIntosh, Chair
Delegate James E. Malone Jr., Vice Chair

Room 250, House Office Building, Annapolis, MD 21401-1991

Not Heavy Enough

We had clinic today with Dr. Lo's TPN clinic. Ellie had only gained just a tiny bit and had grown an inch since last Nov. The problem is in December she got sick, got admitted, had really bad SBBO and lost a ton of weight. So the fact that she is up from November's weight is good news to us. But when they did the body mass evaluation her numbers were down. So she is growing but needs more calories. They want us to get those calories enterally not through more TPN. So we are going to push more elecare by mouth and turn up the overnight enteral pump. We are also going to try going up in calories per ounce of formula.

They also talked about prepping to get her off of TPN/hydration fluids. To do this we are decreasing the amount of every overnight hydration bag from 1200mls to 1000mls. We have clinic again in 2 weeks and if she is doing well we will decrease more, if not then we go back up. First they said they wanted to leave every night at 1200mls and then do one night at 600mls. The idea of that made me gulp. So we compromised with a decrease to 1000mls.

All in all, they were happy with how she was doing, just not as impressed as I was. So now we try new stuff and hope for a big weight gain over the next few weeks. We're in for big fun now!!!

Think Heavy & Remembering the Beginning.

Ellie has been doing really well lately. She is eating like a horse and we have been able to keep better control over her bouts of overgrowth. From what we learned in the last Maximize Health webinar, protein is key for Ellie. Protein is digested in your Jejunum and the only small bowel that Ellie has is her Jejunum. So we have kicked it up a notch in the protein department. We have started offering, low fat ground turkey, low fat ground beef, poached chicken and some vegetarian options. Eggs apparently at a perfect protein and Ellie is loving her eggs right now. She has been very happy with our diet modifications.

So, we are keeping our fingers crossed that at our TPN clinic tomorrow we will hear good news. We always tell Ellie to think heavy thoughts before she steps on the scale at clinic. And tomorrow she better be thinking HEAVY. We are only on 3 nights of TPN and we want OFFFFFFFFFFFFFF!

Ellie has already come off of TPN once. When she was 2 and half years old she was off for 6 months. Then we did the reconnection surgery and now almost 2 years later we are hoping to get off again. Keep your fingers crossed for us!!

Getting this close to getting off of TPN has made us think about when we went on. I have also been talking to some parents that are right at the beginning of this roller coaster ride. I have forgotten how scary and uncertain things were after Ellie was born. William has been very easy and it has only pointed out to us how hard things really were. When Ellie was born there was very little information out there about Short Bowel, TPN or Omegaven. Many times we felt like we were flying blind. We did get hope when Ellie was a month old, a family that used to live in AZ send us a picture of their son that was short bowel. He looked fantastic. It made us realize that how things were now was not how they were going to end up.

Now there are many resources out there for parents and adults with short bowel. Over the next few weeks I am going to update the links we post on this blog to make sure that all the resources are listed. I also want to update our blog list. Most of the kidos listed are around Ellie's age, give or take a year. I would love to add some blogs/caringbridge sites for more kids and even some for adults. If you have any suggestions for resources or would like your blog added please either leave a comment on here or email me abbybrogan@hotmail.com.

The next topic for Maximize Health is fats in the short bowel diet and how to avoid fatty acid deficiency. This will be very important if we get off of TPN!!

Nutrition for Short Gut

We have struggled with Ellie's nutrition for a while and found that there were very few resources out there to help. It is a balancing act of getting her to eat and gettine her off of TPN,...good diet=less TPN.

We have a good nutritionist at Boston Children's but we only see her once every 2 months or if something is wrong. We have a great nutritionist through ThriveRx but what I have always wanted was a book or guide that we could keep on hand. Something that we can also share with our family and Ellie's nurse to help them understand the intricacies of Ellie's diet.

Finally we got what we needed. ThriveRx has created an online short bowel diet guide called Maximize Health. Each month they release a new topic, so far they have covered Diet overview, hydration, Carbohydrates and the next module is going to be on protein. The educational modules are available on line and then they host a webinar on the second Tuesday of each month led by the author of the modules. She gives a better explanation of the content of the modules and then at the end she answers any questions that you might have.

For us the Carbohydrate module was very informative as this is 80% of Ellie's diet. It changed the way we looked at her food and made us be much more aware of what she is eating. I am very excited about the module on protein since we know this is very important for Ellie but we are always struggling to get enough in to her.

The best part about this education program is that it is free to everyone. If you are interested just visit ThriveRx.net You have to register for the program, this will give you access to all the past modules, and recordings of past webinars and get you an invitation to upcoming webinars.

If you have questions about this email me at abbybrogan@hotmail.com.

They are also hosting a webinar next week on TPN stability called. TPN Compatibility and Stability: What’s the big deal? it s on February 4th @ 12:00 noon EST.

Presenter: Sheila Pedapati, Pharm.D.

She will talk about:

• Verification of IV compounds and supplies
• Patient counseling regarding medications, supplies, and pump use
• TPN compounding and different types of pumps
• Working closely with nurse liaisons to optimally manage patient care

IF you are interested in this one email: info@thriverx.net.

2010 A YEAR IN REVIEW

Again we have let too much time pass between posts. Posting once in 6 months is not OK. Rather than try to catch up with a quick update. I am just going to do a review of the year.

2010 A YEAR IN REVIEW

We started the year with the huge bummer of Gus getting diagnosed with cancer. Then moved on to the excitement of Ellie’s first solid poops. In mid January we had a new refrigerator delivered and installed. The next morning we woke up to a flooded basement, and flood damaged kitchen. This led to a nice little kitchen and basement renovation that we have always wanted to do, Thanks Sears!!. During all this we also found out I was finally pregnant with baby number 2. I was rocked by ragging morning sickness, but I would have put up with anything, we were so excited to be pregnant again. I think this made loosing Gus in April a bit easier to deal with.

We struggled with hydration for Ellie through the end of the spring into the beginning of summer. We finally got things under control just in time for the Oley conference in NY. This kicked off a great summer with Ellie taking to swimming like a fish. We spent a week on the Cape with my family.

Ellie was on the beach and in the water everyday. We managed to get our routine for covering her dressing down so well that she went the whole week on the beach, and in the water, without her dressing ever getting wet. She had such a blast she has been talking about going back ever since.

The summer ended with Ellie starting school again. She started five days a week and we struggled all fall to keep her in all 5 days. She stays home if anyone in her class or the class next to hers has a stomach bug or if she is feeling punky. We work with her school to create a letter that goes out to all the parents explaining Ellie’s issues and why following the school’s sick policy is so important to her. This worked well last year and so far has worked well this year. The best part is it really helped other parents be able to us about Ellie.

We spent the fall apple picking, going to local farms, exploring Boston and enjoying the cooler weather.

I loved the last part of my pregnancy and loved making it all the way to my due date. My pregnancy with William was much easier and I felt much better thought out the whole thing. This just made us realize how sick Ellie was in utero and how much of a toll that took on me. William was a breeze in comparison.

At the center of the hedge Maze

William was born via C-section because I went into labor but never progressed. We called the ball a bit earlier with William than we did with Ellie and that made my recovery much faster with William.

Ellie was a giraffe for Halloween and managed to cover most of the neighborhood with her friends.

She doesn't like chocolate so that made the candy easier to deal with. And after the first few days she forgot that she had candy and that made it easier for Gib and I to finish it off.

I took the rest of the year off from work and enjoyed the rest of the fall and holiday season at home with my kids. I am still getting used to saying “kids”.

Through out the fall we struggled with Ellie’s short bowel bacterial overgrowth (SBBO) , so much so that the intervals between outbreaks got to be shorter than 1 week. We made our rounds through different drugs trying out Flagyl, Vanco and Cipro without really making any progress. We got to the point where we were giving her an extra 1000 mls of hydration fluids each day and still not keeping up with what she was loosing through her loose stools. This finally came to a head in December when we lost control of Ellie’s hydration and had to have her admitted to Children’s.

She spent 5 days in the hospital getting hydrated, doing bowel prep and getting a scope of her bowels to see if we could see anything that was wrong. We were also able to sample some of the fluids that they found in her bowels and culture it see what bacteria we were dealing with. They typed it out and then tested different antibiotics on it and found that Bactrum was the new antibiotic that we should try.

We did a week of Bactrum right before Christmas and it finally stopped the cycle of SBBO that we had been seeing. We also finally started probiotics (VSL3) after talking to another family that has been using them for years. Our team at Children’s was against this decision but they understood why we wanted to try them. And so far so good, it has been just under a month since we saw any signs of SBBO. (knock on wood now)

Christmas was great, we even got some snow to make it a true white Christmas.

We spent the week following Christmas and New Years in Maine with Gib’s family. We had a blast and Ellie loved seeing everyone up there.

So, now we have started the New Year. Things are good so far. In comparison to last year it has been an uneventful year so far. We did make the decision to take Ellie out of school for two days a week; we are hoping this will give her a chance to recover from her fall. William also started part time daycare last week. I think that was harder on me than it was on him.

All in all William has been so easy and laid back that if we could afford it I would be pregnant again. It has been great for Gib and I to have the chance to be typical parents. There is nothing like a very high maintenance baby to make a typical baby fell like a cake walk. Although we did have to remind our pediatrician to treat us like first time parents because we have no clue what to do with a baby that is under 8 months old and at home. All the typical kid stuff throws us for a loop. Things like… eating and pooping, those are things we didn’t have to deal with in Ellie’s first few months.

Today we are spending the day inside watching a true Nor’easter dump close to 2 feet of snow on our house. We have been out playing in it once already today and will head out again as soon as our mittens dry out. We promise to make more of an effort to keep this blog up to date this year. I know you have heard that before, but this time we really, really promise!