Understudied Areas in CF Get Extra Attention at NACFC

I could fill a book with all the things I learned at the virtual North American Cystic Fibrosis Conference this year, but I am especially glad that there was a focus on diversity, gender health, and sex-based differences in CF.

Jan. 14, 2021 | 5 min read
Jacqui Sjoberg

I was honored to be able to virtually attend this year's North American Cystic Fibrosis Conference (NACFC). I am an adult with CF and very active in the CF community. I also am a patient advocate for the Therapeutics Development Network's Women's Health Research Working Group and a working member of a couple other research groups. I lend a patient's perspective to help guide and inform clinicians on the gaps in CF -- particularly women's -- care.

This is why I was so excited that this year's theme of NACFC seemed to be the “year of misfit toys.” Don't get me wrong.

This is a very positive direction for CF care overall -- the focus has shifted to new ways of approaching old ideas, bringing the smaller niche and understudied areas of CF care to the forefront, and trying unique ideas to develop better care.

As the CF population lives well into adulthood, we are finding that areas of care that seem glaringly obvious have been understudied because they weren't issues when the average life expectancy was under the age of 18. 

Sessions on gene editing addressed the 7% of the CF population who cannot benefit from modulators. There was a new focus on tailoring care and medications to the individual versus following a set of standard guidelines. I saw a movement to examine understudied pathogens such as nontuberculous mycobacteria and Aspergillus. Presentations on the roles of nutrition and CF-related diabetes in long-term health and how to better manage or detect abnormalities in these areas offered refreshing new takes on old areas of study. It was encouraging to see the psychological toll of CF -- both to the individual and families -- being talked about and made to feel less taboo. Even a current niche -- addressing COVID-19 and its impacts on the CF population -- was brought up during this event.

Excited to watch NACFC sessions online this year!

One other little section that caught my attention was the mention of an upcoming novel study called HERO-2 that will be the first, patient, home-reported outcome study ever. It just shows the innovative ways clinicians are approaching care and how the CF community will lend a hand in guiding better care practices through tracking day-to-day trends. The list of exciting advances on the horizon goes on and on.

I could write a book on all that I learned from this event, but the sessions that I really want to focus on are the sessions around diversity, gender health, and sex-based differences in CF progression and care. CF care teams are recognizing that many patients come to them with issues outside of their normal focus, and being able to openly bring these sensitive topics up during visits and recognize any implicit biases could greatly improve and guide future care. I found the sessions on addressing LGBTQ issues in clinic and reevaluating the representation of minority families to be particularly eye opening. Just being able to state that clinicians are aware of the issues and active studies are being conducted to understand these communities are huge steps in the right direction for quality care.

Females are a well-represented, but very understudied population in the CF community. In fairness, most women did not -- until fairly recently -- have the chance to start families, experience menopause, or worry about cyclic hormonal impacts on their health. So, there is a large gap in the research behind this topic. Not to mention CF doctors do not regularly practice gynecology or obstetrics, so it is not in their wheelhouse. But with most patients using their CF team as their primary care physician, women's health is something that needs to be brought up or a plan needs to be made to source it out. Even something as simple as urinary incontinence is an issue many women with CF suffer through because they are too embarrassed to bring it up, or they simply don't know it's abnormal.

Together with my sons Hunter (11) and Ayden (14).

Studies are being done to find better treatments for this problem, and there is a push to make this line of questioning more mainstream. Now in the age of highly effective modulators such as Trikafta®, safety during pregnancy and efficacy of birth control are front and center for many women with CF.

I love that there are clinicians out there trying to close the information gap and supporting research for better reproductive health in the CF community.

There are websites being built to help women with CF decide which birth control option would be best to use. There are also new studies coming out like MAYFLOWERS that will be a prospective observational study on the effects of pregnancy in women with CF. I am hopeful that women with CF in the future will not have to flounder through uncharted territory like I did for my pregnancies.

As all these topics become more mainstream, I will be more confident to bring them up with my physicians. Based on what I saw through this NACFC event, I think the misfit niches, populations, and studies will become just part of the main conversation very soon.

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This site contains general information about cystic fibrosis, as well as personal insight from the CF community. Opinions and experiences shared by members of our community, including but not limited to people with CF and their families, belong solely to the blog post author and do not represent those of the Cystic Fibrosis Foundation, unless explicitly stated. In addition, the site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.

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Jacqui was diagnosed with cystic fibrosis at birth. She has participated in numerous clinical trials to further advance CF care. Jacqui is serving as a patient advocate on the CF Women's Health Research Working Group and as a clinical research community reviewer. You can find Jacqui in her kitchen, cooking for her husband and two boys or walking along the beach and trails near her home in southern California. She can be reached at entertain365@gmail.com.

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