Archives for August 2010

So we ended up back in the ER today.

It all began last night when Hannah was sounded really junky in her trach.   On a normal day, we maybe suction her about 4 or 5 times in a 24-hour period (she is great at coughing out what is in her trachea).   Last night, I had to suction her at least 15 times within a few-hour period.  I stayed up late keeping an eye on her, and around 2 am she had woken up.  At around 3:30 am, I finally had to wake Daddy up to take over because I was just beyond exhausted.

After unsuccessfully trying to get her back to sleep, at 4 am he ended up taking her downstairs to the family room to just let her relax on him.  She napped for about 45 minutes more, but she still was incredibly junky and needed a lot of suctioning.

Our favorite nurse was with us today.  She came around 9 am.  Daddy had found a free place to take the kids (Spring Preserve for those locals), so they had taken off.  I was staying home with Hannah and our nurse because I had some studying to do.   Our nurse finally got Hannah to sleep around 10 am.    She slept quite peacefully, and we both commented on the lack of suctioning that we had to do this time.

Around 11:45 am, Hannah woke herself up with a harsh junky cough attack, and it required a lot of suctioning to get the gook out of her lungs.  I mean, a lot.  We both noticed that Hannah was having difficulty catching her breath after she was cleared out, and so I had placed a call to our pediatrician.  She was out of the office today, and the earliest we could see her resident was 3:30 pm.

Hannah was starting to have more respiratory distress, so I made the decision to head straight to the ER (our nurse agreed).   Like the three previous times we had been there, we were taken immediately into triage and given a room.

My biggest fear was that her pneumonia had come back.  After all, she is most susceptible to getting pneumonia again within 6 months of a previous bout, and we were only a month or so out of the hospital.

They did chest xrays, blood work (the only abnormality was her WBCs which were 18,800 which means infection), and they took a urine sample and trach culture.  The only results not available were the trach culture, which is going to take a few days.  They also had given her Tylenol because her fever hit 102.9 from 101.1 just an hour earlier in triage.

The good news is it is not pneumonia!  Her lungs looked good.  It is likely tracheitis (infection of the trachea), which kids with trachs are apparently more susceptible to.  The doctor (who recognized us, as she was the same ER doc when Hannah had her cellulitis) decided to give her about two hours worth of IV fluids and IV Rocephin before discharging us.   They gave us a prescription of augmentin b.i.d. for the infection.  This is an antibiotic that covers a lot of different bugs, so hopefully when we get the culture and sensitivities back from her trach sample, it will be covered with the augmentin.

I was so nervous that Hannah would have to be admitted again.  We had never made it to the ER discharge office since we had been to Vegas.  So when she said “No admittance this time,” my heart jumped for joy!

It is going to be another long night, as Hannah still is really junky as she sleeps.  It is about midnight now, and she has had to be suctioned almost 10 times in a 3-hour period.  My hope is to stay awake until around 4 am again and then Daddy is going to take over (he went to be early tonight).  I don’t feel comfortable with both of us sleeping with her being this congested.

What I would give for night nursing tonight….  but that is another story for another day…

** By Chris Hempel

Great news for the pediatric rare disease community came out late last week — rare disease advocates please get this out on your blogs!

Senators Sam Brownback (R-KS), Sherrod Brown (D-OH), and Al Franken (D-MN) are supporting the bipartisan bill S. 3697, the “Creating Hope Act of 2010.” Nancy Goodman, Executive Director of Kids v Cancer, is the person leading the charge on S. 3697 and a priority review voucher system for pediatric rare diseases.

In 2009, Nancy lost her son Jacob to a rare pediatric cancer called medulloblastoma. She is an inspiration to all in the rare disease community!

The Creating Hope Act of 2010 builds upon the Food and Drug Administration Amendments Act of 2007, often called the “treat and trade” program, which established a priority review voucher program for drugs or biologics targeting neglected tropical diseases. At the time this bill was passed, rare childhood diseases were excluded.

he Creating Hope Act of 2010 will encourage the creation of new drugs for underserved children like Addi and Cassi who suffer from serious and life threatening medical conditions by providing a priority review voucher (PRV) as an incentive to pharmaceutical companies who develop drugs for rare pediatric diseases like Niemann Pick Type C.

This is exactly the type of novel incentive system I have been asking for that could fast track cyclodextrin research. For example, with a PRV system in place, I could get a company like Johnson and Johnson to actually take on Niemann Pick Type C disease research and help me make a cyclodextrin drug for Niemann Pick Type C kids.  In turn, Johnson and Johnson could receive a priority review voucher that gives them priority FDA review of another application that would otherwise be reviewed under FDA’s standard review clock.

This priority review voucher could be used for a blockbuster drug that a company would want want to bring to market and receiving priority review could mean millions of dollars to a Pharma or biotech company.  This is why they would be willing to invest in Niemann Pick Type C research and cyclodextrin and help our small community bring a potentially life saving compound to market for kids like Addi and Cassi.

Since I already have an orphan drug application filed and approved with the FDA, having a priority review voucher system in place potentially makes Niemann Pick Type C an attract investment risk by Pharmas or BioTechs.

Priority reviews vouchers for pediatric rare diseases are a  win-win for everyone!  We need to rally the rare disease community to fight for the passing of S. 3697, Creating Hope Act 2010 bill.

Below are some key provisions of the S. 3697, Creating Hope Act 2010 bill:

• Extension to pediatric rare diseases: This legislation includes rare pediatric disease within the scope of the program. This category encompasses any disease that is “rare” within the meaning of the Orphan Drug Act (affects less than 200,000 people, or the cost of development would exceed revenue) is recognized in the medical community as affecting a pediatric population and is a new drug that has not received FDA approval for an adult indication
• Closing a loophole: This legislation would prevent companies from receiving a voucher for tropical disease products that they already market in other countries. This change will ensure that the program rewards only innovative treatments
• Unlimited transferability of vouchers: A voucher may now be transferred unlimited times provided that the transferee, in each instance of transfer, notifies the FDA of the change in ownership. This change enables drug companies to maximize the value of the voucher in the marketplace
• Optional upfront priority review designation process: Under the current law, sponsors do not know whether their new drug application will qualify for a voucher until the time of FDA approval. The proposed legislation permits sponsors of both tropical disease drugs and rare pediatric disease drugs to seek a designation that the new drug would qualify for a voucher, should it be approved, even before they submit their new drug application.
• Adds Chagas disease to the list of neglected tropical diseases: Chagas disease is responsible for more deaths in Central and South America than every other parasite-borne disease, including malaria. Yet, despite its profound impact, research and development of new treatments is severely underfunded. The addition of Chagas to the list of eligible diseases fulfills the intent of the original authors.
• Reporting and marketing requirements: The Creating Hope Act requires that the sponsor submit a statement of good faith intent to market the eligible drug, as well as a report describing the demand and distribution of the ultimate product.

Article Source

Sure, it sounds good, doesn’t it?   Maybe if I say it enough, “we are getting into a rhythm,” that maybe we will eventually get there…

Hannah is doing really well.  I would say she is about 75% back to where she was pre-hospitalization.   Well, if you separate the desire and ability to play with her toys, she is at about 95%!  Girl LOVES her toys.

Now that we are weaning down the Keppra (finally weaned off her midday dose), we are noticing more choreathetoid movements, especially at night.  We are going to keep an eye on it for the next few days, and if it still gets worse at night, then I’m going to call her neuro and discuss it with her.  We have 3 more weeks before we are off the Keppra, and perhaps this “third line of defense” drug is actually doing a lot more work on her movements than we thought.

Getting Hannah into the Katie Beckett Program so we can get on Medicaid has been a nightmare.  I dropped off the forms about 5 weeks ago.  First, they screwed up by putting the application under MY name and MY social security number.  Then, they sent it to the wrong office and under a different program that Hannah does not qualify for.   Also, all the additional paperwork that we had walked into the “wrong office” that they accepted had disappeared.  A few days ago, I got a call from one of the supervisors who is going to “hand walk” it to the right office and department and put it under Hannah’s info.    We’ll see how that works out.

We are also changing our nursing schedule.   In an effort to become more of a family unit, we are making it so there is less time a nurse is here when the kids are here.   We will have nursing during the day while the kids are in school and after they get home from school so we can have time for homework and playdates, but we will have dinner alone and all together.   So instead of Monday thru Sunday, 12 pm to 8 pm, we are going to go to the following schedule:

• Monday thru Wednesday, 8 am to 6 pm
• Thursday, 12 pm to 8 pm (Hannah has her Cerezyme every other Thursday morning)
• Friday, 8 am to 8 pm
• Saturday, 3 pm to 9 pm
• Sunday, no nurse

As for us?  House in Texas still is on the market, Daddy is still unemployed, finances are stressful, counting the days until school starts because the kids are bored and at each other’s throats…. but we are making it through!

Hannah is still well on her way on the road to recovery.

Feeding-wise, we have finally got her feeds up to 170 mL/hour (a bit less than 6 ounces).   She was at 200 mL/hour before her bout with the rotavirus, but we ended up having to go back to 40 mL/hour continuous feeds just to get her stomach used to food again.

We are hoping to be able to get her to bolus feeding in the next few weeks which would cut down each meal (5 a day) to 20 minutes instead of 90 minutes or so!   Before this hospitalization, she would down an 8- to 10-ounce bottle of formula/babyfood in 5 to 10 minutes!   So if we could get 8 ounces down in about 20 minutes, wow, would that be wonderful 🙂

Before we left Texas, after her trach was put in, she failed her swallow study.   We were told it was likely due to the fact she had no coordinated swallow or suck reflex because of the choreathetoid movements.  In the notes of the swallow study results, I found that the speech pathologist recommended that we can try thicker foods like pudding and baby foods, those with a distinct color, and see how she tolerates that once we felt she was ready to try.  The reason for the distinct color was so that we could see if she aspirated it, as she would likely cough it back up into her HME, and we would be able to see the coloring as we did when OT first tried it back in Texas.

Since her swallow and suck reflex has tremendously improved in the past month, we decided to give baby food a shot today.  We bought a two-pack of Stage 2 peaches (orange) and a 2-pack of berry banana (purple).

We were confronted with the pre-hospitalization problem that we had where she would not take food by spoon (except for gerber yogurt).  But we decided to try anyway.     Our nurse was here when we tried it as an expert eye looking for signs that she may aspirate the food.

It took just a couple of little spoonfuls, but she LOVED it.  Amazingly, she was opening her mouth for each bite!  We fed her a whole entire plastic tub of baby food BY SPOON without argument!  TWICE!  We tried the berry banana around lunch time and the peaches around dinner time.  Both times, she took it by mouth.

Even more amazingly… she put her hand out to ask for more!   She never communicated “more” to us for anything, but after almost every bite, she would put her arm out as if she was trying to grab the spoon for more.    As soon as we put another bite in her mouth with the spoon, she sucked it all down.

We checked for aspiration in her HME dozens of times, just out of paranoia.  But there was not one instance of aspiration today!  Of course, this does not mean we are out of the woods with this aspiration issue, but I am feeling so much more confident now that we can likely continue with thicker foods.  We don’t plan to try juice or formula again until we get another swallow study at the NIH in Sept or Oct, but thicker foods is a great start — especially is she is willing to take it by spoon!

Makes me wonder — did she forget she had an oral aversion to being fed by spoon before all of this happened?  Or perhaps she just loved the taste in her mouth so much, after having nothing in there for almost 3 months, that she was willing to go for it?

Doesn’t matter the reason — she ate baby food today.  By mouth.  Yeah, baby!