Peek Behind the Paper: a patient perspective and plain language summary publication of the Cincinnati study
In this feature, we take a ‘Peek Behind the Paper’ with Hawken Miller (BioNews, FL, USA), to discuss their recent Plain Language Summary of Publication article summarizing the Cincinnati study – a study that aimed to further understand the differences between using two types of steroid treatment in males with Duchenne muscular dystrophy.
Read the full paper here >>>
Please could you introduce yourself, your organization and provide a brief summary of your career to date?
My name is Hawken Miller. I am a writer for a company called BioNews (FL, US), which publishes over 40 rare and chronic disease-related websites, ranging from multiple sclerosis and cystic fibrosis to muscular dystrophy. I report on human interest stories throughout these communities and the broader rare disease population. Examples include aid to rare disease patients fleeing the war in Ukraine, patients overcoming all odds to start their own companies and pursue their careers, and the experience of doctors and patients with stem cell transplant therapy for multiple sclerosis.
I previously worked for The Washington Post (DC, US), where I focused on content curation for the iOS and Android app, writing for the Post’s Snapchat account and reporting stories relating to the growing video game and e-sports market.
I also interact with CureDuchenne (CA, US), a foundation created by my parents to cure Duchenne muscular dystrophy. I was diagnosed with Duchenne at 5 years old. I attend events for the organization and connect with other families affected by the disease.
Could you please outline what your recent plain language summary of publication article published in the Journal of Comparative Effectiveness Research was about?
The plain language summary compared the differences in mobility and clinical test results for patients who took deflazacort and prednisone, both commonly utilized steroids to reduce inflammation in Duchenne. The article the summary was based on utilized retrospective real-world data from 435 patients.
What were the results of the study and what significance do they hold?
The study showed that patients on deflazacort were ambulatory for longer and had a lower risk of scoliosis. This is significant because it gives doctors and patients a clearer picture of the differences between these two medications. Ultimately, a decision on which treatment to pursue should be made based on a person’s individual case and with support from their doctor.
From the patient perspective, why is real-world evidence in Duchenne muscular dystrophy important?
Real-world evidence is extremely important because not all patients have the ability to enrol in clinical trials and clinical trial sponsors do not have enough space to accommodate everyone. This also allows us to build a broader evidence base for how Duchenne progresses across patients. It can help inform future research as treatments for Duchenne are developed.
What challenges lie ahead for the incorporation of real-world evidence in Duchenne muscular dystrophy studies?
Real-world evidence isn’t always standardized, making it difficult to compare clinical outcomes measured at different hospitals. Additionally, the information utilized in these type of studies should be secure, encrypted and anonymized to protect patient privacy. Even with the challenges, this information is already being collected by clinics and it makes more sense to utilize real-world evidence to better understand the disease on a broader scale rather than an individual level.
What do you see for the future of real-world evidence in Duchenne muscular dystrophy studies?
This summary still leaves more questions than answers. Researchers were unable to, for example, determine the average age of onset of scoliosis patients who took deflazacort. That means more data is needed and clinics should consider, with patient approval, sharing that patient information for future studies.
Data from these studies give scientists a better look into the progression of Duchenne and what variables lead to better or worse clinical outcomes. Real-world evidence when paired with genetic information can help uncover the variability of Duchenne and bring us closer to answering the question: why do some patients have more aggressive forms of the disease and others don’t?
