I was diagnosed with heart failure with preserved ejection fraction (HFpEF) six years ago. I have a vivid memory of that meeting with the cardiologist, who told me that HFpEF is untreatable and that 50% of HFpEF patients die within five years of diagnosis. Because of my history of dealing with two cancers and being an active cancer patient advocate, I quickly asked about clinical trials. That began my journey from trial to trial as I worked with and continue to work with my cardiologist to try to find a treatment for this disease.
I enter clinical trials for two reasons: I unashamedly would like to find a treatment that helps me to survive with a good quality of life and I want to help to find a treatment that will be helpful to others with this diagnosis. So far, I have been in phase 1, 2, 3 and 4 trials, totaling eight trials, some for medications and cardiac rehab, one for a device and one for surgery. At every visit with the cardiologist, I ask about new trials for which I might qualify.
While all of the trials were important to me and, I hope, important to moving the science of care forward, the two that have impacted me most are the CardioMEMs trial and the pericardiotomy trial. The CardioMEMs trial gave my cardiologist daily access to my status and the ability to adjust my medications quickly. It has also given me peace of mind as I live 600 miles from the center of excellence where I receive care.
The pericardiotomy trial has, I believe, been life-changing for me. The surgery was done one year ago. A heart MRI done two weeks ago indicates that my heart is stable. I firmly believe that without the pericardiotomy I would have experienced significant deterioration in my symptoms.
One highlight worth mentioning is that two of the trials included cardiac rehab. This is important because HFpEF patients are not eligible for insurance-covered cardiac rehab. The tragedy of that is that regular exercise is known to be beneficial to those of us with HFpEF.
The length of trial visits varied by the trial. For example, one trial required me to be there for three days a week for three months. Most of the trials require one or two day visits separated by about six weeks. For the pericardiotomy trial, I was hospitalized longer than anticipated because some of my comorbidities acted up. In all of the trials I have been fortunate to be cared for by empathic, highly skilled physicians and staff. Where possible, they did everything they could to facilitate short and infrequent visits while focusing on my well being and staying within the parameters of the trial. They were always available to me by phone and email, always responding promptly.
As far as what was expected of me, I was expected to have read and understood the consent form, understand the trial calendar, adhere to trial requirements and report promptly any adverse events. I understood I could stop participation at any time. I remain impressed by the care I receive and the fact that, when a trial is completed and the report written, I receive a thank you letter for my participation, a brief summary of the results and a copy of the report.
One of the values of participating in clinical trials is underscoring my purpose in life. HFpEF can be so debilitating as I struggle to breathe and to stay physically active that it would be easy to give up purpose to just surviving. Through participating in trials I maintain a positive focus and stay meaningfully engaged even when the trial results are negative. I am not as hopeful that I will be cured as I am hopeful that my trial participation will have benefits for other patients. Negative trials do have meaning and do move the science forward. So, I am not discouraged by participating in negative trials. Rather I am encouraged that we are one step closer to treatment and maybe a cure. That is important.
My message for researchers is that partnering with patients in the development of clinical trials has advantages. My perspective as a patient can increase the likelihood that the trial will be attractive to patients, increasing accrual and retention of participants. Unfortunately, this level of patient engagement is difficult for many researchers to appreciate let alone achieve. Researcher resistance appears to be the biggest barrier to patient engagement in trial development. I hope that we will find a way to encourage patient involvement in the inception and development of trials. National Cancer Institute (NCI) has a program of Specialized Programs of Research Excellence (SPOREs) that engages patients in this way. I would like to see NHLBI develop a Specialized Center of Research Excellence (SCORE) program for cardiovascular research modeled on the SPORE program. This would provide cardiovascular researchers with a roadmap and incentive to meaningful patient engagement not only as trial participants but also as trial developers.