Antioxidant-Enriched Multivitamin May Decrease Respiratory Illnesses

MedicalResearch.com Interview with:

Scott D Sagel MD PhD
Professor of Pediatrics
University of Colorado School of Medicine
Aurora, Colorado

MedicalResearch.com: What is the background for this study?

Response: Inflammation is an important feature of cystic fibrosis (CF) lung disease and contributes to lung damage and lung function decline in CF. We need safe and effective anti-inflammatory treatments in CF. Anti-oxidant therapy has been an area of promise, but with mixed results in CF.

This clinical trial, conducted at 15 CF centers affiliated with the cystic fibrosis Foundation Therapeutics Development Network, enrolled 73 patients who were 10 years and older (average age 22 years), with pancreatic insufficiency, which causes malabsorption of antioxidants. Subjects were randomized to either a multivitamin containing multiple antioxidants including carotenoids such as beta(β)-carotene, tocopherols (vitamin E), coenzyme Q10 (CoQ10), and selenium or to a control multivitamin without antioxidant enrichment. The antioxidants used in the study were delivered in a capsule specifically designed for individuals with difficulties absorbing fats and proteins, including those with cystic fibrosis.

MedicalResearch.com: What are the main findings?

Response: Antioxidant supplementation was safe and well-tolerated. Supplemental antioxidants increased antioxidant concentrations in the bloodstream in treated subjects and temporarily reduced inflammation in the blood at four weeks but not 16 weeks. Airway inflammation, as measured in sputum, did not change significantly with antioxidant treatment. Importantly, antioxidant treatment appeared to both prolong the time to the first respiratory illness requiring antibiotics and reduce the frequency of respiratory illnesses they experienced.

MedicalResearch.com: What should readers take away from your report?

Response: Taking a specially formulated antioxidant-enriched multivitamin, containing multiple dietary antioxidants, may decrease respiratory illnesses in people with cystic fibrosis. While more research needs to be done to find a treatment that delivers a sustained anti-inflammatory effect, we believe the prolonged time patients had before their first respiratory illness is clinically meaningful. Also, the cost of a dietary antioxidant-enriched multivitamin is relatively modest compared to other currently available therapies that have been proven to reduce pulmonary exacerbations in cystic fibrosis.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: We still don’t know the optimal dosing of these various dietary antioxidants. We also don’t know the added benefit of antioxidant supplementation in the era of CFTR modulator therapy, emerging treatments that get at the basic protein defect in cystic fibrosis.

MedicalResearch.com: Is there anything else you would like to add?

Response: This clinical trial, funded by a grant from the Cystic Fibrosis Foundation, was an investigator-initiated study led by Scott D. Sagel, MD, PhD, a Professor of Pediatrics at Children’s Hospital Colorado and Director of the University of Colorado Cystic Fibrosis Center. It was not an industry initiated or funded trial. Callion Pharma manufactured the antioxidant-enriched and control multivitamins and provided them at no charge for this study.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.

Citation:
Effects of an Antioxidant-enriched Multivitamin in Cystic Fibrosis: Randomized, Controlled, Multicenter Trial
Scott D Sagel , Umer Khan , Raksha Jain , Gavin Graff , Cori L Daines , Jordan M Dunitz , Drucy Borowitz , David M Orenstein , Ibrahim Abdulhamid , Julie Noe , John P Clancy , et al
https://doi.org/10.1164/rccm.201801-0105OC PubMed: 29688760
American Journal of Respiratory and Critical Care Medicine

Published Online: April 24, 2018

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

Original interview article here. 

A Cost-Utility Analysis Of Vertex’s CF Drugs — What It Teaches Us About Trial Design

By Claudia Dall’Osso, Ph.D., Ian Love, Ph.D., and Nuno Antunes, Ph.D., Decision Resources Group (DRG)

Commercial success in the pharmaceutical industry requires that clinical programs, in addition to demonstrating clinical effectiveness, also provide data supporting a drug’s value. The Institute for Clinical and Economic Research (ICER), a U.S.-based cost-effectiveness watchdog, recently released an analysis suggesting that Vertex Pharmaceuticals’ cystic fibrosis (CF) franchise — Kalydeco, Orkambi, and the recently launched Symdeko — while offering meaningful clinical efficacy, would require discounts of approximately 70 percent1 to be cost-effective.

Here, we review ICER’s cost-effectiveness analysis of the Vertex CF franchise to highlight lessons for orphan drug developers related to clinical trial designs and outcome metrics that would facilitate more favorable cost-effectiveness evaluations by stakeholders who employ cost-utility modeling (e.g., ICER, U.K.’s National Institute for Health and Care Excellence [NICE]).

Calculating The Cost-Effectiveness Of Vertex’s Cystic Fibrosis Franchise

In a cost-utility model, health economic analysts strive to calculate the incremental cost to gain an extra quality-adjusted life year (QALY); they estimate a therapy’s impact on the level of utility patients are deriving from their life based on their health status and incorporate these data into a quantitative estimate of QALYs (Figure 1). The goal of the cost-utility analysis is to determine whether a therapeutic intervention changes the QALYs that patients will accumulate over a set time period (e.g., lifetime), and at what added cost. The threshold for what is considered an acceptable incremental cost per QALY varies by stakeholder; ICER typically presents a sensitivity analysis across a range of thresholds (e.g., $50,000 to $500,000 per QALY for an ultra-rare disease like CF).

In our view, data gaps opened the possibility of a potential undervaluation of the Vertex CF transmembrane conductance (CFTR) modulators on several metrics and, ultimately, on overall survival in the context of the ICER model. Vertex’s pivotal clinical trials captured the effect of CFTR modulators on the two organs chiefly affected in CF — the lungs and the pancreas — with outcome metrics for pulmonary function, percent-predicted 1-second forced expiratory volume (ppFEV1), and pulmonary exacerbation rate, as well as pancreatic sufficiency (body weight) (Figure 2). However, data on metrics assessing emerging complications (e.g., CF-dependent diabetes or bacterial infections), impact on use of other medications (e.g., pancreatic enzyme replacement therapy, mucolytics), reduction in healthcare resource utilization, or reduction in disease burden were far more limited, but these attributes were included in ICER’s cost-effectiveness analysis. Lacking clear clinical trial data on the metrics outlined above, health economists relied on arguably conservative assumptions to estimate the impact of the Vertex CFTR modulators on these domains. Because the Vertex CF franchise has a relatively short market history, and the long-term risks/benefits of the drugs are incompletely understood, assumptions to model the long-term impact of these medicines were also necessary.

For instance, to evaluate survival, ICER modeled the impact of CF-related diabetes in its analysis of CF patients’ health status. Owing to the dearth of clinical trial data on CF-related diabetes in the development program for the Vertex drugs, the company’s CFTR modulators were assumed not to impact this outcome (Figure 2). Treatment with the Vertex CFTR modulators was also conservatively assumed to have no long-term impact on weight after an initial increase and, without long-term data, the drugs’ impact on ppFEV1 beyond two years of treatment was modeled as a 50 percent reduction in the rate of ppFEV1 decline.

Notably, several CF experts interviewed by DRG consider it possible that early treatment of newborns could prevent disease development. The potential impact of early treatment with CFTR modulators on disease development and survival was not explored in the ICER analysis; although little data is available to support such an impact of the Vertex drugs, ICER has considered such scenarios largely unsupported in other evaluations (e.g., a cost-effectiveness evaluation of Spark Therapeutics’ Luxturna for the treatment of retinitis pigmentosa).

The translation of clinical trial data to utility is a second area wherein a manufacturer may lose traction in a cost-utility analysis, if the utility calculation isn’t sufficiently comprehensive or if the drug’s data package is insufficient to support its impact on all relevant metrics. In the ICER analysis of the Vertex franchise, health economists used the ppFEV1 metric to derive a utility curve by assigning a level of benefit to a specific ppFEV1 value. Although this is the most straightforward approach, it also results in an assessment of health benefits that relies exclusively on a mechanical respiratory metric, which may not adequately capture the quality of life experienced by patients, especially considering the multi-organ nature of CF. Indeed, at the May 17 presentation of the ICER model, stakeholders from the Cystic Fibrosis Foundation levied this criticism. Furthermore, ICER’s sensitivity analyses showed that changes in the relationship between ppFEV1 and utility could significantly affect the overall cost-effectiveness assessment. Notably, an alternative scenario in which the utility was increased by 5 percent, to account for clinical effects of a drug beyond pulmonary function, led to a 15 percent decrease in the cost-effectiveness ratio.

Similarly, the impact of the Vertex franchise on payer budgets in the ICER model related only to pulmonary supportive care, while other non-pulmonary expenses remained unchanged — an assumption made in the context of available data, but one that may not fully reflect the benefit of the drugs. Furthermore, the CFTR modulators did not impact the burden of supportive care for CF patients in the model, nor did they impact patients’ productivity. Ultimately, suboptimal alignment of clinical trial data with the demands of a comprehensive (e.g., multi-organ) cost-effectiveness model may have diminished the opportunity for the Vertex franchise to perform maximally in this cost-utility analysis.

Key Lessons And Takeaways For Drug Developers

Although clinical outcome data collected by Vertex was sufficient to gain an FDA green light, it was not sufficient to support a comprehensive analysis of cost-effectiveness in this multi-organ disease. As such, assumptions regarding drug impact were necessary in areas not adequately supported by data, opening the possibility for a suboptimal cost-effectiveness evaluation. To support more favorable and data-supported evaluations, developers should design clinical trials with an eye on cost-effectiveness.

  • Prior to initiating clinical trials, manufacturers should consider how a health status model is likely to be designed to assess cost-effectiveness. They should consider enrolling the assistance of academic researchers to understand which metrics may be important in such a model and to aid in the development of a reliable model in an area where none is established. With this analysis in mind, developers should strive to design a clinical program that covers relevant metrics and the durability of a drug’s impact on them. Indeed, an alternative scenario developed by ICER showed that a change in the long-term effectiveness assumption on ppFEV1 would have a profound impact on the final cost-effectiveness assessment; for Kalydeco, assuming no decline in ppFEV1 after the first two years (rather than 50 percent) decreased the incremental cost-effectiveness ratio ($ per QALY) by approximately 35 percent.
  • Developers should work to understand how key clinical metrics in a given disease area are translated into utility. In a disease with an established function, it is prudent to carefully survey the relevant literature. When developing a pioneering treatment, manufacturers should consider investment into the development of a utility curve that accurately accomplishes this, which would likely facilitate a reliable QALY calculation or at least more detailed/specific alternative scenarios and sensitivity analyses.
  • Understand the patient journey and track healthcare resource utilization during a clinical trial to more fully support an accurate assessment of cost of care, as a favorable impact on direct healthcare costs is important to attain widespread reimbursement.
  • Although metrics such as burden of care, caregiver burden, or productivity loss are difficult to rigorously track, they can be immensely valuable in highlighting the favorable indirect effects of disease-modifying drugs beyond the clinical efficacy. Understanding patients’ pain points and, ideally, tracking these metrics when possible (e.g., with real-world data or social media listening analyses) may further strengthen and support conventional metrics from clinical trials.

As market access hurdles intensify, and ICER’s analyses increasingly inform payer policy, anticipating and preparing for cost-utility analyses early in the design of a clinical program will be paramount to support a medicine’s value proposition with U.S. insurers.

Original article found here.

Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

By Jennifer Prince

A marine omega-3 compound comprising a docosahexaenoic acid (DHA) sn1-monoacylglyceride (MAG-DHA) may act as an anti-inflammatory for subjects with cystic fibrosis, according to a new pilot study1 published in the journal Marine Drugs. In the study, MaxSimil (Neptune Wellness Solutions; Laval, QC, Canada) increased omega-3 red blood cell levels, helped moderate the ratio of arachidonic acid (AA) to docosahexaenoic acid, and reduced key inflammatory biomarkers in subjects with cystic fibrosis. Continue reading Omega-3 Compound Reduces Inflammation in Cystic Fibrosis Patients in New Pilot Study

Jerry Cahill’s CF Podcast: Stem Cell Research with Dr. Hans-Willem Snoeck

In this feature of The Path Forward with CF series, Dr. Hans-Willem Snoeck, Professor of Medicine (Microbiology and Immunology) at CUMC, sits down to discuss stem cell research as it relates to CF.

Because lung cells regenerate and repair themselves regularly, researchers believe that – some day – stem cell technology could be a one-time therapy to cure cystic fibrosis. Research is ongoing, but in the meantime, scientists can currently use human pluripotent stem cells to create lung organoids (tiny, 3-D structures that mimic features of a full-sized lung), introduce various mutations, and apply technologies to learn more about those mutations’ characteristics.

This video was originally published on JerryCahill.com

Vertex Employees Donate $1M to CF and Other Communities via Matching Gift Program

By Carolina Henriques

Vertex Pharmaceuticals employees have raised more million $1 million  using  the Vertex Foundation‘s matching gift program in a show of commitment to causes that include the cystic fibrosis (CF) community, a company press release states.

The dollar-for-dollar matching gift program is being run through the nonprofit Vertex Foundation, established by the company in November 2017 as part of it’s charitable giving goal of donating $500 million to qualified nonprofits and other causes worldwide over 10 years.

To date, more than 500 Vertex employees have used the program to support 753 charities around the globe working to advance work in areas that include healthcare, human services, education, and disaster relief.

Vertex’s charitable commitment has four primary goals: supporting CF patients and caregivers worldwide, including enabling access to Vertex’s medicines; helping underserved students and young women with STEAM (science, technology, engineering, arts and math) education; supporting young doctors and scientists; and strengthening and fostering innovation in local communities through health and wellness programs.

“Giving back is in our DNA at Vertex, and our employees have a long history of going the extra mile to improve the lives of patients, students and their neighbors,” Jeffrey Leiden, president, chairman and chief executive officer of Vertex, said in the release. “I’m proud that The Vertex Foundation is able to help extend the impact of our employees’ giving and look forward to seeing the reach of these investments in the causes they care about most.”

Also as part of its 10-year commitment, Vertex awarded $400,000 in scholarships to eligible CF patients and their family members in May as part of its second “All in for CF” scholarship program. In total, 80 scholarships worth $5,000 each were awarded for the upcoming academic year.

Vertex, which specializes in cystic fibrosis, has three approved CF therapies: Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor).

The company is also testing potential triple combination treatments for CF.

For the rest of this article, click here.

How to Beat Brain Fog

By Wendy Caroline

As I sat down to write my column for this week, a massive wave of writer’s block hit me. This has been plaguing me for a while. Great timing, right?!

I started five different column pieces, each of which I would get a solid start on, and then nothing. I stopped understanding where the piece was going and didn’t like any of the words that I was writing. A few years ago, I started developing chronic fatigue. It started slowly, then quickly snowballed. In the past year, it has developed into brain fog. Very frustrating. I love using my brain for writing, reading, problem-solving, and critical thinking. It increasingly feels like cystic fibrosis is taking all these parts of my personality away from me. So here I am today, compiling a list of some tips and tricks that I use to combat brain fog and feel a little more like myself.

1. Don’t force it.

I can’t tell you how many times I have blocked my own brain by trying to force my way through brain fog. “Just do it.” How many times have we heard that? Unfortunately, at least for me, if I “just do it” and try to force myself to work through the brain fog, it just puts up more blocks. Then I get frustrated, and all I can think about is how I can’t think. It just spirals from there.

Cool, so how do you get past brain fog and still make your deadlines (because if you’re forcing it, that’s often why)? Be ahead of the game. I am guilty of this not being a natural instinct. In fact, I’m still learning how to do this. I used to be a procrastinator. I would thrive under the stress. My brain fog does not. Now I start writing and drafting at least a week before.

2. Get inspired.

One of the best ways to get inspired is to look to others. CF News Today columnist Brad Dell’s latest piece about brain fog directly inspired this post. It got me thinking about my own experience with brain fog, and how I have been insanely frustrated by its hold on my life. Find a piece of other people’s lives or the world around you that makes you think about your own. Capture it and go. I find that making a note of these inspirations in a note-taking app on my phone really helps because brain fog causes forgetfulness.

2. Create the mood.

Ditch the phone and whatever other fancy gizmos that cause a distraction. Find the type of environment that makes you feel inspired and ready to work. For me, that’s open space with lots of natural light and structured seating.

Next, put on some music — music that is going to make you focus and productive. My go-to is this Classical Essentials playlist on shuffle. This helps me drown out enough thoughts that I won’t get distracted without drowning out the thoughts that I need to work. Whatever you listen to, just make sure it doesn’t have words.

It’s also important to get your brain in the right mood. My brain fixates on chores and tasks that need doing, so I always make sure to get those done before I sit down to write. Also yoga and meditation, that’s some great stuff.

4. Let it flow.

A friend of mine gave me this very useful tip. Just let the words flow out of you, even if you don’t like them. When I use this technique, I have to write everything my heart desires and then walk away. Don’t reread it right then. You will get frustrated. Instead, write all you can and come back to it after you’ve had a solid break. For me, this is usually the next day. Your rough work can have a really good base but just needs to be re-worked.

I know that these tips and tricks are focused on writing, but I use the general ideas in all aspects of my life. Whether it is work, family, friends, or hobbies, brain fog can affect every aspect of your life. It’s one of the reasons that I haven’t been very active on any of my social media platforms — Instagram, YouTube, Facebook, and even my blog. As my brain fog developed into almost a daily issue, I took a step back and prioritized what I needed to focus on first. I truly believe that as these steps of beating brain fog become a habit, I will be able to handle more. Hopefully, you’ll see me around the internet again real soon.

» Find me on the internet at The Living, Breathing Wendy «

This article was originally published on CF News Today.

This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition.

Cystic Fibrosis Awareness: Sharing A Story & The Facts

Cystic fibrosis is a devastating disease. About 30,000 children and adults have cystic fibrosis (CF) in The United States. While the life expectancy for someone with CF has doubled in the last 30 years, children and adults people still lose their lives to this disease every day. There is no cure yet. It’s time to find it.

How can you help? Use your voice and get involved! Participate in Great Strides walks and other events supporting CF and share the facts on social media and in your community. The Cystic Fibrosis Foundation is working hard to find a cure. Other foundations and organizations help the CF community as well. Like the CF Roundtable which supports adults with CF, there is a foundation that supports children and families living with CF: Claire’s Place Foundation.

Claire’s Place Foundation, founded by Claire Wineland, when she was just 14 years old, is a 501c3 non-profit organization designed to help children and families affected by cystic fibrosis. Claire was born with CF. In her short life, Claire has undergone more than 20 surgeries and has spent months at a time in the hospital.  She survived months in a coma on mechanical ventilation despite the odds and was also a Make-a-Wish recipient. She has 40% of ideal lung function and the doctors have estimated two years left to live. This is not, however, what characterizes or defines her. Claire has dedicated her life work to making a difference in the lives of others living with CF.

Now 21 years old as an adult living with CF, Claire’s Place Foundation is a way for Claire to give back with hope, strength, and joy.  She wants to live with intention and purpose. In her own words, “I know my life will be shorter so every day has meaning.”  She strives to find new ways to help the CF community and other children afflicted with chronic illness. Claire’s Place is making a huge impact through its two programs – The Extended Hospital Stay Fund and the Family Support Program.  These programs are in high demand and the foundation needs your donations to continue assisting young patients in need. You can meet Claire and support her foundation at her next fundraiser, “Glow Ride,” on August 18, 2018 in Hermosa Beach, CA.

To find others ways you can help bring awareness to CF find a local chapter near you.

Get involved. Donate. Come to events.

Antioxidant Supplement Helps Cystic Fibrosis Patients, Study Finds

By Carolina Henriques

A special formulation of an antioxidant-enriched multivitamin helped decrease the frequency of pulmonary exacerbations in patients with cystic fibrosis (CF), according to a new study.

The findings, by researchers at Children’s Hospital Colorado and the University of Colorado School of Medicine, were published in the American Journal of Respiratory and Critical Care Medicine under the title “Effects of an Antioxidant-enriched Multivitamin in Cystic Fibrosis: Randomized, Controlled, Multicenter Trial.”

Inflammation is an important contributor to lung damage and to progressive lung function decline in CF. In the study, researchers looked at the effects of a “cocktail” of multiple antioxidants on inflammation and health outcomes in CF patients.

“Single oral antioxidant formulations have been tested previously in CF with mixed results. However, there had not been a well-designed, randomized controlled trial of an antioxidant ‘cocktail’ that included multiple antioxidants in a single formulation,” Scott Sagel, MD, PhD, said in a University of Colorado news story. Sagel is the study’s first author and a pediatric pulmonologist at Children’s Colorado.

The 16-week study (NCT01859390) was conducted from September 2013 to October 2015 at 15 U.S. CF centers affiliated with the CF Foundation’s Therapeutics Development Network.

The study included 73 pancreatic-insufficient CF patients, 10 years and older (average age 22), who could not adequately absorb important dietary antioxidants such as beta-carotene, coenzyme Q10 (CoQ10), tocopherols (vitamin E), and selenium, which help neutralize inflammation in the body.

Participants received either capsules of antioxidant-enriched multivitamins, or control multivitamins without antioxidant enrichment. The capsules were designed specifically for people with difficulties in absorbing fats and proteins, like CF patients.

The investigational antioxidant-enriched multivitamin supplement, called AquADEKs-2, contains standard amounts of fat-soluble vitamins (A, D, E, K) plus several antioxidants, including beta-carotene, mixed tocopherols, CoQ10, mixed carotenoids (lutein, lycopene and zeaxanthin), and the minerals zinc and selenium.

Sagel and his team found that the antioxidant-enriched multivitamin supplement increased the concentration of antioxidants in the bloodstream, and temporarily reduced inflammation at four weeks, even though these results were not sustained through the end of the 16 weeks of the study.

In addition, antioxidant supplementation was found safe and well-tolerated by study participants.

Researchers also observed that antioxidant treatment appeared to prolong the time to the first pulmonary exacerbation requiring antibiotics, and also to reduce the frequency of pulmonary exacerbations altogether.

For the full article, please visit CF News Today.

Calling All Students! LMK Scholarship deadline June 30th!

CF Roundtable offers the Lauren Melissa Kelly (LMK) Scholarship award each semester, in honor of Lauren Melissa Kelly. The academic scholarships of up to $2500 are awarded to adults with cystic fibrosis who are pursuing career certifications, associates, and bachelor and graduate degrees.

Nancy Wech, Lauren’s mother, established this scholarship in Lauren’s name and memory. In Nancy’s own words:

Lauren Melissa Kelly was an extraordinary woman. Laughing, gregarious, spontaneous, fun, talkative, driven, thoughtful, smart, kind and loving — all descriptive terms for Lauren, who lost her battle with CF late in her senior year at the University of Georgia. In 1992, Lauren was chosen as one of ten Senior Leaders at University of Georgia. She had distinguished herself as a member of the Golden Key Honor Society, Mortar Board, Phi Upsilon Omicron, Gamma Beta Phi, the Tate Society and Delta Gamma sorority.

Lauren felt the most significant opportunities of her college career included participation in the reconstruction and formation of organizations, which will serve the university in the future. She acted as one of the re-founding members of the Phi Kappa Literary Society and was significant in the metamorphosis of the Z Club into the William Tate Society. Her other activities included Greeks Advocating Mature Management of Alcohol (GAMMA) in which she served as Secretary and Special Events Chair. She was also a member of the Women’s Glee Club for more than two years.

In recognition of her academic performance, Lauren’s degree of Bachelor of Science in Family and Consumer Sciences was awarded posthumously. At the time of her death, Lauren was engaged to be married and living off campus in an apartment. She lived life to the fullest!

Walt Disney said, “Don’t cry because it’s over, smile because it happened. It’s not the days in life you remember, it’s the moments.” As Lauren’s mother stated, “I smile because she happened to me. Now, I want you to smile because she has happened to you.”

Please visit our website for application and full scholarship criteria and details. http://www.cfroundtable.com/community-outreach/united-states-adult-cystic-fibrosis-association-scholarship/

Send any questions to scholarships@usacfa.org.

Lessons Learned Through Parenting and CF

Guest Blog By Jeannine Ricci

Back in 2001, when I became a mother, resources on parenting
with CF were practically nonexistent. Thanks to all of the
research advancements, it’s so exciting to now see more and
more people with CF exploring the possibility of parenthood.
Because this topic is becoming more prevalent, I thought it might
be a good time to resurrect an article that I wrote 5 years ago. It
discusses my experience with talking with my children about CF
and the possibility of a shortened life expectancy. Hopefully it will
help other parents as they face this challenging subject.
At the end of the article, I give an update on my daughters
and how their experience with my CF continues to impact their
lives.

***Lessons Learned- Parenting with CF***

My CF has shaped my children’s lives for as long as they can
remember. As toddlers, they developed patience. After many
temper tantrums, they eventually learned that I had to finish my
treatments before we embarked on our day’s adventures. As
preschoolers they learned empathy. They would run to get me
tissues and water if I was having a coughing fit. Their teachers
would comment on the level of empathy they showed their
classmates and how unusual it was at such a young age. During
their school years, they are learning to become more
independent. Just last week, my older daughter surprised me by
making their lunches and helping her sister with her homework
because she knew I was not feeling well.

Since CF had been woven into their everyday lives, I took their
comfort level with my CF for granted. They knew that CF meant
coughing, treatments, and IVs. What they did not know was that
it is a life-threatening disease. I regret having not broached this
subject with them as soon as I felt they were mature enough to
handle this information. Last year my daughter’s teacher called
to tell me that he was concerned about her. She was not herself
at school. She was very distracted and seemed depressed. I
knew immediately what was bothering her. I was on IVs because
of an especially difficult exacerbation. The side effects of the
antibiotics left me on the couch most of the day. It was the
sickest she had ever seen me. We had a long discussion after the
phone call and she opened up to me and told me how she had
read something at the CF walk that stated that the life
expectancy of someone with CF was 37 years old. I was 40. It
truly broke my heart to know that I was not there to answer her
questions and ease her fears when she read this critical piece of
information. She deserved to hear this from me. As I spoke to
her, I tried to give her realistic but hopeful answers. I told her
that I wanted nothing more than to watch her and her sister
grow up and that I hoped to be there for her college graduation,
her wedding, and the birth of her children. I explained that there
are so many new medicines being discovered that would help to
make this a possibility, but I also told her that there are no
guarantees, and that is why I try so hard to keep myself as
healthy as possible.

Just as my children have always incorporated important life
lessons from my illness into their lives, I believe that this
discussion will only further strengthen their character. It will
teach them the value of treating every day as a gift. I hope my
experience encourages other parents with CF to be prepared to
have this pivotal discussion with their children. No matter how
difficult it may seem, it will be worth the peace of mind knowing
that you will be the one delivering this information, ready to help
them cope with their fears.

Here are some guidelines from Lisa C. Greene, a mom of two
children with CF and co-author with Foster Cline, M.D. of the book
Parenting Children with Health Issues (www.PCWHI.com)

• Pivotal parenting moments can take us by surprise, so be
prepared ahead of time. Our answers should be honest, calm,
matter-of-fact, and hopeful. We shouldn’t use terms like “fatal”
or “life-shortening” nor should we make empty promises. Use
terms like “healthier” rather than “healthy,” “more likely to live a
long time” rather than “will live a long time.”

• We need to try our best not to let our own fears and worries
show, both in our words and in our body language. Children pick
up on (and tend to mirror) their parents’ emotional cues,
especially when they are young. If you are having trouble
controlling your own emotions about these tough issues,
counseling might be helpful.

• At some point, we do need to address the issue of life
expectancy. Hopefully, this will be clarified by around the age of
eight (around 3rd grade) depending on the maturity of your
child. One way to address this issue is to ask your child questions
to open up dialogue. Some examples are:
“How much do you know about CF?”
“How are you handling it?”
“Is there anything about CF that worries you?”

With a little awareness and preparation, you can make talking
about these difficult issues a positive experience. Relationships
can grow closer when people go through tough times together.

Update: 5 Years Later
It’s no surprise that this disease has continued to shape my
daughters’ lives over the past 5 years. There’s no denying that my CF, anxiety, and depression have caused many hardships for my family. And it’s
difficult not get swallowed up in the guilt of knowing that there
are times that I’m not able to be the mom that I desire to be, the
mom that they deserve. During these times, I try my best to
focus on the positive ways CF has touched their lives.

The attributes of fortitude and courage were fostered as they
watched my battle with CF progression as it inched its way closer
and closer to the center of my life–our lives–demanding more
attention. And then, in November of 2014, they witnessed hope
being transformed into tangible reality as I swallowed my first
dose of Kalydeco. This new reality has allowed them to more
confidently envision me by their sides in the distant future. They
have embraced this gift with a deep sense of gratitude that can
only be felt when someone has experienced the threat of the
unbearable alternative.

They have watched as this same gratitude has fueled my desire
to help others who are still waiting for their miracle. At the young
ages of 15 and 17, they possess a keen understanding of the
intrinsic value in every life, and that the amount of money in
someone’s bank account or what type of insurance they have
should not be dictating access to these life-saving medications.

They have learned the importance of taking action and
advocating for others, even if your voice is seemingly
overpowered by others. They have both participated in the Cystic
Fibrosis Foundation’s Teen Advocacy Days in Washington D.C.
the last few years, meeting with members of Congress and
stressing the importance of ensuring adequate healthcare
coverage for all. They recognize the gifts both given and received when you touch a person’s life indelibly and both of my daughters have expressed an interest in pursuing a career in the healthcare field. 

I’m so proud of them as I watch them develop into
compassionate, strong, young women, inspired to make their
mark on this world; a mark that undoubtedly would not be so
deep and impactful if it wasn’t for the valuable life lessons they
have gleaned from having someone they love with CF.