This is lengthy, but loaded with info. Each section has notes that Joel typed during the 30-60 minute presentations. The notes don't represent a summary of the presentation, just things that we wanted to remember or that applied to Elena.Marcus Institute - David Jaquess PhD, Director, Stephanie Powers
1-2% of all children require intervention to achieve adequate body growth
50% of children with CF need some sort of assistance (psychological or physical). There is WIDE range of severity.
Bad experiences & Feeding problems can cause eating difficulty. For example...
1) Choking incidents that causes long lasting fear
2) Disrupted parent-child interaction
3) History of pain or discomfort
4) Taking lots of effort to eat
The above list is for all children, but CF children usually overlap with all of them. This is especially caused by the CF child’s consistent need to eat until over-full. When someone becomes hypersensitive to eating challenges, motor & sensory “defects” develop (like being averse to the way a particular food feels in your mouth).
Pavlov thought applies to CF kids. They associate unpleasant sensations after eating just like the “ringing bell” effect on the dog. If we try to change the stimulus or environment of eating VERY gradual over time, the Pavlov effect would be decreased like the treatment of a phobia.
Completing routines is critical, but when and in what order – maybe not so much.
Punishment does not motivate change to new behaviors. Plus, children may not make the connection anyway. Negative side effects of punishment could be physical aggression by the child or disrupted family interactions.
Positive reinforcement idea: use selective attention by reacting fully to behavior that is desirable and reacting minimally to poor behavior.
Changing your method to positive reinforcement can be very difficult. If the child is used to getting their way by throwing a fit, they will only throw more fits when they see that the fit isn’t working the way it used to. For example, we know that Elena fakes tummy pain, and would be likely to fake more tummy pain in response to positive reinforcement that was causing her to eat more.
But Elena doesn’t “get” the idea of rewards – she just restructures her enjoyment. She recognizes the motivator, and is too smart when she alters her behavior without actually eating more.
The presenter’s examples confirmed that we’ve tried everything. We shouldn’t abandon a strategy after a week, but fine tune it and stick with it.
Modeling is a good way to display positive reinforcement. For example, rewarding Ethan when he reaches goals during mealtime to show Elena how it is supposed to work.
Find things that will motivate her daily, weekly, and the BIG DEAL like
Daily: later bed time, family game, movie rental
Weekly: park, bowling, date night with Daddy
Big Deal: Aquarium, Disney, Show at Fox etc.
We really need to put thought into what can motivate Elena and get a routine for a few weeks.
Use motivators during meal (draw a picture, one line per bite or color in a picture one square color in per bite).
Contact Meghan for some ideas (she has feeding experience with her work)
When to seek help – no progress for 3 months, disruptions getting worse over 3-6 months, nutrition becomes critical (feeding tube discussion), you feel lost.
We should call this doctor and review Elena’s case. He specifically said he would help brainstorm and would not charge and be honest about whether help was needed.
New Therapies in Pipeline – Nael McCarty PhD, Senior CF scientist for Emory Medicine
(Look at these notes in conjunction with the pipeline chart)
CFTR Modulation – Emory will soon be starting Phase 3 trials, this is big stuff and we need to look into Elena’s participation. “These are the most exciting things happening.”
VX770 – For G551D, but might be used in conjunction with other drugs that directly target DelF508. We need to cheer on this one too, and didn’t realize this before.
VX809 – Meant for us!!!!! DelF508. Trials starting at Emory when?
Denufosol – restores airway surface liquid, great progress, applies to all CF mutations. TIGER-2 trials are only open to ages 8 and older.
To prepare Elena for NPD procedures in the future, we should start using neti pots. Other CF parents we’ve talked to encouraged their use anyway.
Manage your expectations – no one knows what drug development programs will fail. We saw the drug pipeline from 8 years ago, and a bunch of them fell off the chart since then. 20% of Phase 1 drugs make it, 50% for Phase 2, 80% for Phase 3.
Even if Elena doesn’t start receiving newly developed treatments for another 10 years, it could still extend her life expectancy to beyond age 50-60.
Carriers of CF (Julie, Joel, etc) have a higher incidence of sinus problems because having just one DelF508 actually does affect the way that CFTR produces protein.
Joel spoke to Nael at a break, and learned more about what he does. Through our conversation I gleaned that the rate of CF research is increasing because the research being done applies to many other areas. Interest among scientists in working with CF related topics is generated because the developments have far reaching repercussions outside the relatively small population of CF patients.
Emory CF Center Update – Dr. Schechter
We should not be upset when Elena is hospitalized for a tune-up. It means she is getting the best care possible. Studies show that aggressive use of antibiotics to treat pulmonary exacerbation produces the best results, and that sort of treatment is done in the hospital. Plus, if she’s hospitalized we could reapply for Medicaid (hope & change).
Periactin (sp?) – the appetite stimulant we need to ask about
Elena’s BMI percentile should be over 50% to give her the best chance for good lung function in the long run. This isn’t just a “theory,” it’s proven. Mean BMI for patients under 20 years old at Emory CF Center is 52 percentile.
New and Better Approches to Airway Clearance – Mary Lester
CF is an awful sneaky disease. Even parents and family fall into the false thought process of “symptoms are low, so everything must be going fine.” But symptoms are always there and building over a lifetime. Consistent airway treatment is a must.
Active Cycle Breathing Treatment – introduced at around Elena’s age, we need to research this
It’s a family disease. If everyone is involved with the treatment, it works better!
Importance of CF Clinical Research – Jeannette Peabody RN
Common reasons why people stay out of research trials…
1) Fear of the unknown
2) Possible risks (study drug side effects, safety, procedures/tests)
3) Study is asking too much of you
4) Time off from work/school
5) Costs (time & travel also)
Clinicaltrials.gov and type in cystic fibrosis
Considering Elena’s existing challenges, doing trials just might be too much. We know what Elena can and can’t tolerate. There has to be some trials out there that could work.
Two girls with CF, one 18 & one 22ish, were part of presentations. They both shared that they were part of the original Pulmozyme trials many years ago. It was emotionally straining to see these young women. Why do they appear well? What is their life like? Why do others we’ve met not do as well? We can’t control CF and have to live with the unknown. All of our efforts and focus cannot save our child. We try to turn my feelings of grief and sadness to feelings of hope.
