The open door policy – An important and under-recognized activity of the adult CF center

By Anna Sze Tai and Sue Morey

Improvement in survival in cystic fibrosis (CF) has led to a substantial increase in numbers of the adults with CF past few decades [12]. In many parts of the world, the number of adults now exceeds the numbers of children with CF [12]. Adults with CF face the daily challenge in balancing education, work and family commitments with their disease management. Patients often need to contact the Adult CF specialist team for advice on disease management and coordination of care around their work and family time-table. Current ECFS guideline recommends patients have ready access to the CF center for telephone advice for emergency management or consultation [34]. The ‘open-door’ policy of adult CF centers is an important component of ambulatory care in adults with CF, ensuring a timely and expert-level response to patient queries. Management of unscheduled patient-initiated contacts is an important and often under-recognized function of the adult CF team. There is a paucity of data on the nature and frequency of these unscheduled contacts and their potential impact (and demands) on the organization of the CF team.

In this issue of the Journal of Cystic Fibrosis, Burnet et al. reported the results of a two-staged prospective study of unplanned patient-initiated contacts in a single adult CF center (n = 410 patients) [5]. During the first stage, all patient-initiated contacts were prospectively recorded over a four-month period. Four physicians and three nurses prospectively documented how and why patients contacted the team and the time spent responding to these requests. During the second stage, contacts generated from a subset of 212 patients were prospectively recorded to explore the association between contact frequency and disease severity.

During the first stage, 259/410 (65%) patients initiated 1067 contacts over 4 months, averaging 13.3 contacts per day. Average time spent addressing patient contacts by all participants combined was 8 h and 7 min per work week. This was likely an underestimate given not all team members (e.g. allied health specialists) who might be contacted by patients participated, and data collection was limited to working hours during weekdays. Nevertheless, the result confirmed that managing unscheduled contacts added a significant workload to the CF team. Factors associated with greater contacts (≥5 contacts/year) included female sex and greater disease severity, reflected in severe airflow obstruction (FEV1 ≤ 30% predicted), ≥ 5 physician visits/year and ≥ 1 hospital admission/year.

The frequency of unscheduled contact in adults with CF was markedly higher than that observed amongst high-risk COPD patients, reflecting the greater healthcare demand of adults with CF [6]. Interestingly, only <20% of requests were related to medical issues (e.g. exacerbations), whereas the bulk of requests were related to daily management issues (e.g. script renewal), intravenous antibiotic requests, administrative queries and non-urgent miscellaneous matters. This perhaps reflects that the adult CF center has generally provided comprehensive, primary up to expert-level tertiary care to their adults with CF. The adult CF center can be viewed by the patient as ‘the one-stop shop’ for both their CF and non-CF related healthcare needs. Patients may not be aware of the need for a family physician for their non-CF related healthcare needs, including travel vaccinations, women health issues, age-specific screening (e.g. mammograms, monitoring for hypertension).

This is the first study which demonstrates the significant burden of unplanned contacts on the organization of the adult CF centre. The strength of the study is the prospective design which enables differentiation between planned and unplanned contacts. There was a comprehensive recording of response time in the clear majority of patient contacts (>95%) by the team members as they carry out their day-to-day services within a large CF centre which is an impressive undertaking. The key limitation is the single centre nature of the study which limits its comparability to other centres in other health settings internationally. Nevertheless, Burnet et al. has clearly revealed the burden of unscheduled contacts and highlighted the need for more efficient management strategies. Firstly, development of an effective centralised triaging system (e.g. collective ‘CF Team’ email inbox and phone line) managed by experienced CF staff who prioritise and streamline all incoming requests to appropriate team members can maximize efficiency of the CF team. Secondly, involvement of administrative staff to assist with relevant non-medical requests (e.g. clinic rescheduling, etc) will lighten the burden on the clinical team. Thirdly, ultilisation of new telecommunication and health informatics technologies can potentially improve communication and data transparency. Telehealth technologies (e.g. Smartphone apps, Skype or WhatsApp) have already shown significant promise in improving patient access to care and early treatment in CF, particularly in setting where distance from centre to the patients is considerable and pop-in clinics are likely to be impractical [[7][8][9]]. Some of these telehealth technologies can potentially serve as communication platforms to provide education resources on commonly-asked questions.

To continue reading please visit: https://www.sciencedirect.com/science/article/pii/S1569199318306982

Pushing Through the Fear – Guest Blog By Andrea Eisenman

By Andrea Eisenman

So many fears, where do I begin. Let’s start with my impending trip to Seattle from NYC. I like to travel but it gets complicated. How much room in my suitcase do I have to pack my myriad of machines and meds? And how much will I forget, despite my thorough list? I learned I had to put obvious things on my list like a hairbrush after I forgot that a few times. But when it is easily purchased at a drug store, no biggy. When it is my immune suppressants or a nebulizer, that is harder to replace.

I now have a lot more machinery to tote around when I leave home. I have my CPAP, my percussor and my inhalation machine and a facial steamer for my sinuses, plus my Neti pot for nasal lavage. These things become cumbersome and traveling light is not an option, I have to check my bag. So, planning is key for several days prior to take off. I am in that phase now. Packing it all. I bring enough meds for twice my travel time. My last trip to Seattle happened during 9/11. I could not fly home for a week. Luckily, I had an extra 10 days of medications to cover me.

My dad asked if I was up to the flight, it is a longer one than I have taken in many years. My answer is, I don’t know. I am fearful as I know I have lymphedema and even though I wear compression tights when I fly, it is less than comfy and I will swell in my upper body. I do have a compression machine for upper body swelling but it is way too big to bring. Will I be ok not using it for a few days? I am hoping the answer is yes. But because I do not know these things for certain, I have anxiety. And I worry I might get sick either from the flight or anytime during my trip. I do wear a mask in flight and try to stay as hydrated as possible in order to keep well. And of course, I will wipe down the area near my seat with cleaning wipes.

But in order to live a life, I have to take some risks. I had wanted to go to Seattle for a few years. It is therapeutic to get away once in a while and I had not traveled too far from home while my mom was alive. I wanted to be near enough if she needed me. I no longer have that worry. And maybe I used that as an excuse so I am now pushing myself to go on this trip. I know I can be resourceful and my doctors are only a phone call away if I get sick. There is a great CF center there and my friend is sensitive to my CF needs. When we were in college together she gave my CPT when I let her.

I find that when I push myself beyond my fears, I feel triumphant and am happy that I conquered them. Sometimes one has to get out of their comfort zone, even if it means wearing horribly tight pantyhose for six hours on a flight! I know it will be worth it and I can bond with my friend. I will feel like I accomplished something worthwhile. Maybe my next trip will be to Europe.

Cystic Fibrosis Podcast 195 Summiting Mount Everest and Denali with Nick Talbot

In the latest CF Podcast, Nick Talbot shares his incredibly unique journey with cystic fibrosis. From being diagnosed at the age of 13 to his latest climb – a trek up Denali, the highest point in North America – he constantly challenges himself to reach for his dreams. Nick stresses the importance of pushing one’s own personal limits and never being scared to fail – because failure simply means you tried.
Tune in to learn more about Nick and his cystic fibrosis story.

This video podcast was made possible through an unrestricted educational grant from Novartis to the Boomer Esiason Foundation.

Apply Now for Our LMK Scholarship! Due October 15th

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. Spring 2019 applications due on October 15th, 2018. 

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.

Airway Clearance Vests Fail to Show Measurable Short-term Lung Benefits in Study

A note from CF Roundtable: Please do not stop using your Vest or other HFCWO device because of this impractical study. There are important differences in this study that make it not applicable to CF and therefore, not meaningful for us. First, healthy volunteers without CF were enrolled. Too many adults with CF have experienced significant benefits with these HFCWO devices, not to mention the preventive benefits. To imply these devices worsen lung function when used by a healthy nonCF person vs one with CF – with the usual accompanying inflammation, mucus +/- bronchiectasis, etc – is not practical. Second, these healthy subjects tested all 4 HFCWO devices in one day. Even when I repeat spirometry in one day, my lung function numbers most often decline over time, even with rest periods in between. The researchers tested lung function with a rest period of 15 minutes on these healthy individuals after use of the HFCWO device then moved on to the next device. Very impractical and again – not applicable to use by individuals with CF.

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By Iqra Mumal

A clinical study into high-frequency chest wall oscillation vests — assessing their short-term impact on standard measures of lung function before and during use — challenges the view that these devices work through airflow bias in the lungs, the process responsible for mucus movement when breathing.

Findings, using established tests that include forced vital capacity (FVC), forced expiratory volume (FEV1), and forced expiratory flow (FEF25%-75%), suggest “that the concept of HFCWO vest-induced cephalad airflow bias is not supported by standard spirometry measurements,” researchers concluded. “None of the vest groups showed statistically significant increased airflow in the lungs.” Continue reading Airway Clearance Vests Fail to Show Measurable Short-term Lung Benefits in Study

Mutations in Genes Regulating Digestion Prevalent in CF Patients with Pancreatitis

By Vijaya Iyer

In addition to mutations in the CFTR gene, cystic fibrosis (CF) patients with pancreatitis also have a high prevalence of mutations in genes regulating pancreatic function, according to researchers.

Conducted by a research team in Italy, the study, “Trans-heterozygosity for mutations enhances the risk of recurrent/chronic pancreatitis in patients with Cystic Fibrosis,” was published in the journal Molecular Medicine Continue reading Mutations in Genes Regulating Digestion Prevalent in CF Patients with Pancreatitis

Public Voting is Open for Thriving Student Awards

Earlier this year, 40 AbbVie CF Scholars were awarded $3,000 based on their academic excellence, creativity and community involvement/extracurricular activities.

The public is now invited to vote for students to help determine this year’s Thriving Undergraduate and Thriving Graduate Students.

Now in its 26th year, the AbbVie CF Scholarship program has awarded more than $3.2 million to deserving students living with CF.

There are two easy ways to vote*:
  • Visit www.AbbVieCFScholarship.com and follow the instructions
  • Text “VOTE[ID]” (i.e. VOTE1) to 31901 using the personalized ID code provided for each specific scholar

*Please note: Individuals can place only one vote for a Thriving Undergraduate and one vote for a Thriving Graduate Student either online or via text message.

AbbVie, a research-based global biopharmaceutical company, today announced its annual call for the public to vote for students living with cystic fibrosis (CF), an inherited chronic disease that affects the lungs and digestive system, as part of its 2018 Thriving Student Scholarship. Scholarships totaling $25,000 each – the Thriving Undergraduate Scholarship and the Thriving Graduate Scholarship – will be awarded to two deserving students.

From today through September 19 at 11:00 AM EDT, the public is encouraged to visit the AbbVie CF Scholarship website (www.AbbVieCFScholarship.com) to learn more about this year’s AbbVie CF Scholars and review their online profiles to cast a vote to help determine the 2018 Thriving Undergraduate and Thriving Graduate Students. The public can vote online or via text message by texting the student’s personalized ID code to “31901” during the two-week voting period.

“I have always dreamt of becoming a child psychologist, and thanks to the AbbVie CF Thriving Graduate Scholarship, I have been able to focus more on my studies and less on the stresses that come with paying for my education,” said Taylor MacLean, 2017 AbbVie CF Scholar and Thriving Graduate Student. “I want to let other students living with CF know that despite the challenges, achieving your goals is possible, and I’m grateful that the AbbVie CF Scholarship, along with my family and care team, has allowed me to do so. Being acknowledged for my work and determination has helped me realize my full potential.”

Earlier this year, AbbVie selected 40 undergraduate and graduate students living with CF to each receive a $3,000 AbbVie CF Scholarship for use during the 2018-2019 academic year based on their academic excellence, creativity and community involvement/extracurricular activities. These AbbVie CF Scholars now have the opportunity to earn the title of 2018 Thriving Undergraduate or Graduate Student along with an additional $22,000 in scholarship funding for use toward their dreams of higher education. The Thriving Undergraduate and Graduate Students will be determined based on a combination of their academic achievements, community involvement/extracurricular activities, essay and creative presentation scores, as well as the number of public votes cast.

“Students living with CF inspire us every day. They are true fighters who don’t let their diagnosis hold them back from making a difference – whether it’s in the classroom or in their community,” said John Duffey, vice president, U.S. Specialty, AbbVie. “AbbVie has been proud to show our commitment to students with CF and their families by carrying on the AbbVie CF Scholarship tradition for more than 25 years.”

During the voting period, the public can show their support for the AbbVie CF Scholars by tweeting the link below. AbbVie will announce the 2018 Thriving Undergraduate and Graduate Students in October.

Click to Tweet: Students living with #cysticfibrosis need your help! Cast your vote to help award two $25K scholarships and determine the 2018 AbbVie CF Scholarship Thriving Undergraduate and Graduate Students https://bit.ly/TnPPq1

About Cystic Fibrosis

Cystic fibrosis (CF) is an inherited chronic disease that affects the lungs and digestive system of those living with this condition.1 In patients with CF, a thick, sticky mucus is produced in certain organs throughout the body, most commonly the lungs and digestive system. The mucus build-up in the lungs can cause difficulty breathing and may lead to life-threatening lung infections. In the digestive system, the thick mucus may prevent proper food digestion, potentially leading to malabsorption and malnutrition.

About the AbbVie CF Scholarship

The AbbVie CF Scholarship was established 26 years ago in recognition of the financial burdens many families touched by CF face and to acknowledge the achievements of students with CF. Since its inception, the scholarship program has awarded over $3.2 million in scholarships to over 1,000 students. The AbbVie CF Scholarship is part of AbbVie’s ongoing commitment to the CF community, which is comprised of more than 30,000 people in the United States. As of 2016, more than half of the CF population are 18 years or older.1

It is not necessary for scholarship applicants to have taken, currently take, or intend to take in the future, any medicine or product marketed by AbbVie, and this is not a consideration in the selection criteria. More information about the AbbVie CF Scholarship criteria and application can be found at www.AbbVieCFScholarship.com.

About AbbVie

AbbVie is a global, research and development-based biopharmaceutical company committed to developing innovative advanced therapies for some of the world’s most complex and critical conditions. The company’s mission is to use its expertise, dedicated people and unique approach to innovation to markedly improve treatments across four primary therapeutic areas: immunology, oncology, virology and neuroscience. In more than 75 countries, AbbVie employees are working every day to advance health solutions for people around the world. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook or LinkedIn.

References

  1. Cystic Fibrosis Foundation. About Cystic Fibrosis. Diagnosis and Genetics. Available at:https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/. Accessed September 2018.

 

Machine learning to help cystic fibrosis decision-making

By James Hayes

New research claims to have demonstrated that machine learning techniques can predict with a 35% improvement in accuracy – in comparison to existing statistical methods – whether a cystic fibrosis patient should be referred for a lung transplant.

The research, led by Professor Mihaela van der Schaar of the Alan Turing Institute at the University of Oxford, has been generated through a partnership between The Alan Turing Institute and charity the Cystic Fibrosis Trust. Continue reading Machine learning to help cystic fibrosis decision-making

Join in BreatheCon 2018!!

Calling all adults with CF!

Join me at #BreatheCon 2018! Step back from your day-to-day routine and connect with other adults with #cysticfibrosis to discuss topics that are unique to #lifewithCF. Created by and for people with CF, BreatheCon is a two-day virtual event with keynote panels, group chats, fun activities, and small-group video breakouts.
https://cff.swoogo.com/BreatheCon2018

CF Foundation | BreatheCon 2018

Registration is open for BreatheCon! Step away from your day-to-day routine and connect with other adults living with cystic fibrosis to discuss topics that are unique to life with CF. Created by and for people with CF, BreatheCon is a two-day virtual event with keynote panels, group chats, fun activities, and small-group video breakouts.Register today: https://on.cff.org/BreatheCon2018

Posted by Cystic Fibrosis Foundation on Tuesday, August 28, 2018

Patient-reported outcomes: Time for a new approach?

By Janice Abbott

Patient-reported outcome (PRO) measurement (e.g. health-related quality of life questionnaires, symptom diaries) can provide a standardized, valid and reliable way of gaining the patients’ perspective on ‘how they are’ or the benefits and limitations of a specific intervention. The insights that patients have concerning their health are important given that aspects of patient-reported quality of life are independent predictors of survival in cystic fibrosis (CF) [1]. Regulatory authorities require the inclusion of PROs in clinical trials as an additional outcome parameter and PRO information is becoming important in labelling claims. It is noteworthy that the top 10 research questions, reached by global consensus of patient and healthcare providers, all require the inclusion of CF-specific PROs to achieve meaningful answers [2]. This represents a significant paradigm shift but capturing data that matters to patients, families and clinicians is challenging. Two of the persistent challenges in CF PRO measurement are a) the development and use of technologies to enable efficient administration, accurate scoring, and the correct interpretation of data and b) being able to accurately measure PROs (or parental proxy assessment) across the entire CF lifespan. These important issues are considered by two papers in this issue of the Journal of Cystic Fibrosis [34].

PRO measurement largely remains a research endeavour with little uptake in clinical practice. Administering, scoring and interpreting PROs in a busy clinic is difficult. It requires staff time and expertise and the results are not instantly accessible to steer a discussion with the patient or to aid clinical decision making. Paper-based data collection suffers from missing, unreadable data that is prone to scoring/mathematical error. The development of electronic PRO (ePRO) technologies is immensely important in clinical practice and for endpoint assessment in clinical trials. It is a cost-saving, patient-friendly approach to PRO assessment: data collection can occur in clinic, the patient’s home, workplace or school. Results can be added to a patient’s electronic medical file, alerts triggered by problematic scores and clinicians can track patient/parent-reported symptom/event data over time. Importantly, electronic data capture enhances the integrity and accuracy of the data, makes it logistically easier to collect repeated assessments (daily or at several points over a trial), and is preferred over paper-based data collection by the US Food and Drug Administration (FDA).

There is growing evidence that paper and electronic versions of PROs typically provide comparable data but this requires psychometric evaluation if transferring an original paper-based questionnaire to an electronic mode of administration. Solé and colleagues have demonstrated measurement equivalence with paper and electronic administrations of the Cystic Fibrosis Questionnaire-Revised (CFQ-R teen/adult version) [3]. The e-CFQ-R web version is linked to an online database that can be adapted for any electronic devise (smartphone, tablet, computer). Immediately the patient completes the questionnaire, results are sent to the healthcare team and the data are saved in a centralized, protected database. Real-time patient-reported data are available to the clinician as an adjunct to clinical data. Access to the English and Spanish versions are by independent web addresses provided in the paper. Ultimately, the integration of PRO data within electronic care records as developed by Peckham et al. [5], or in CF patient registries would enable efficient patient care and longitudinal research endeavours.

There is a lack of PROs that can be used as endpoints in early intervention studies in CF. Such instruments are time-consuming and painstakingly difficult to develop so the research of Edwards et al. reporting on the initial development of a CF-specific, parent-reported instrument for children 0–11 years is welcome [4]. The need for an effective way of data collection is also considered. The instrument takes the form of an electronic (web-based data capture), observational sign/symptom diary containing 17 respiratory and activity signs that parents report the presence or absence of. Results suggest that children aged 7 to 11 years are best reporting for themselves, therefore observational reporting by parents should focus on young children aged 0 to 6 years. Considerable evaluation has yet to determine the final instrument but the development of the scale follows FDA guidance enabling its acceptance as a clinical trial endpoint in infants and young children with CF.

Over the last twenty years we have learned a great deal about measuring patient-reported outcomes in CF, and there are many pitfalls when employing PROs in CF trials [6]. They are typically secondary endpoints and the trial is not powered on them, often making it difficult to draw valid inferences about treatments. However, there are trials that have collected patient-reported respiratory symptom data as the primary endpoint [78], employing the only CFQ-R subscale that has been approved by the FDA for use as an endpoint. Scientific, regulatory and pragmatic factors are driving the shift towards ePRO data collection. The development of ePROs is not trivial, yet they are fast becoming the ‘gold standard’ for PRO data capture in clinical trials. The challenge now is to develop CF-specific, lifespan PROs, utilising new technologies that can deliver real-time, high-quality PRO information. They also need to be acceptable to the regulatory bodies to aid their decisions on cost-effectiveness and ensure the appropriate commissioning of new medicines to improve the lives of people with CF and their families.

Original article with references here.