At the risk of sounding sappy, this was the first cardiology meeting in my 13 years as a medical blogger when a trial result brought me to tears. It happened not once but twice at the American Heart Association 2017 Scientific Sessions with the ACCELERATOR-2 and the HOPE-Duchenne presentations. A third presentation brought on an almost unstoppable urge to run up and high-five the presenters. I apologized to my colleague, Susan Jeffrey (executive news editor of theheart.org | Medscape Cardiology) for my temporary insanity. "It's okay," she said. "If you lose your humanity, you lose your ability to empathize."
When I was a child, our family made a weekly 18-mile trek to the nearby town of Glasgow, KY to buy groceries and attend church. From the back seat of my father’s Chevy, I would daydream and stare at the beautiful Kentucky pasturelands as cattle and fence posts flew by. In front of a small meager house covered in makeshift roofing material and in terrible disrepair were two young brothers, parked side by side in wheelchairs. Despite their young age, their beards reached mid-chest and their hair fell to their shoulders, a dishevelment that hinted at their profound disability and a lack of resources for daily necessities.
"I feel so badly for those boys," my mother would often say.
A few years later, I noticed there was only one lone boy who sat quietly in the same old wheelchair. And a few years after that, the yard was empty and soon thereafter, the house was gone. Both of those young men succumbed to the terrible disease Duchenne muscular dystrophy before the age of 30. As an adult, I have better insight into their disease process, and it’s a sure bet it was devastating to their family's emotional and economic survival.
The HOPE-Duchenne trial looked at the safety of the orphan drug CAP-1002 (Capricor), allogeneic cardiosphere-derived cells from donated heart muscle. The investigators undertook multivessel coronary artery infusion of these cells in the hope of reducing dystrophy-related myocardial scar compared with usual care. The study met its safety end point so trials will go forward. CAP-1002 also reduced gadolinium uptake in patients with advanced disease—an unusual finding for a trial on Duchenne muscular dystrophy. The potential benefits of this treatment include a halt in left ventricular function decline but there were hints at sidestream improvements in skeletal-muscle function as well.
When the results were presented, I thought of my patient who recently lost his grandchild to the disease and how the grieving process affected his own cardiovascular health. And I thought of the two boys whose wheelchairs were often parked on a hillside in rural Kentucky in the 1970s. I wished they all could have been on a different time line.
American STEMI patients fall into the "haves" and the "have-nots" in terms of ready access to primary PCI. In the ACCELERATOR-2 trial, nine of 12 regions in the US that had previously suffered from fragmented transport patterns reduced their door-to-balloon times and halved mortality rates after a regional education intervention. Congestive heart failure rates declined as well.
The eastern portion of my home state of Kentucky was included in this trial, a geographic location that, thanks to 20/20 and Diane Sawyer, is known all over the world for its poverty and cardiovascular-death rates. Years have passed since two rural hospitals in Kentucky were forced to battle for the right to provide PCI without surgery on site. That 6-year roller-coaster ride was punctuated with promises of support that disappeared out of fear of lost revenue.
The walls of the Kentucky hospital association board room must still echo with our emotional and impassioned pleas for support for a pilot project, a ridiculous requirement to become the 38th state in the Union to get the nod. After presentations to the state cabinet and other branches of the legislature and conversations with three different governors we finally won the right to save lives. In the pressroom during the abstract presentation at AHA 2017, it struck me that not one single person within earshot knew what it took for places like eastern Kentucky to be able to participate in ACCELERATOR-2. Yes, that moment was poignant and heartening.
Finally, for the high-five moment: For so many years studies have failed to demonstrate much benefit for the treatment of HFpEF. I've always argued that we really didn't know who or what we were studying. Often, we invited people to participate in trials if they were simply "short of breath." We used indirect measures of diastology on echo and chest X-rays that are difficult if not impossible to interpret in the obese. We didn't know if we were really studying COPD, poor conditioning, or asthma, much less heart failure.
Finally, REDUCE LAP-HF was presented in its beautifully intricate yet simplistic design where pulmonary capillary wedge pressures (PCWPs) were measured both at rest and with exercise. A transcatheter interatrial shunt device was compared with a sham procedure for reduction in exercise PCWP. It was a beautiful thing indeed to understand what we are studying and therefore many studies can stand on the bones of this trial design. Larger trials are needed to see what the downstream consequences or benefits of atrial shunting will be in this study population.
I do not apologize for allowing these three presentations to unmask the tenderest parts of a heart I've had to harden. As some of you know, the politics of medicine caused me to give up a practice and a partner I cherish and move to another state. Like all physicians who truly care, especially those of us who carry wounds from battles fought in the trenches of medicine, our hearts are happiest when we hear about things that will heal others. Today, I admit there were tears of joy for a change, and I was not ashamed.
Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape
Cite this: HOPE-Duchenne and Help at Last - Medscape - Nov 20, 2017.