GETTING TO THE HEART OF MEDICINE
(continued from page 23)
echo screening harm as well as benefit?” To answer this, we engaged
several Ugandan communities in our research, learning from
stakeholders that echo screening was highly acceptable to teachers,
parents and children 10 , but that having a positive screen could lower
quality of life for the children in which it was detected. 11 In close
consultation with key community members, we established peerto-peer
support networks in Uganda—playgroups of a sort—for
children with early RHD. This social support broke the isolation
and fear around a cardiac diagnosis and did indeed normalize
quality of life. 12
However, the second part of the “should” question remained:
“Would screening make a difference?” In other words, once we had
identified a child with early RHD, could we improve that child’s outcome
by providing secondary prophylaxis? Secondary prophylaxis
requires delivering an intramuscular penicillin injection every 28
days, preventing recurrent strep sore throat and further immune
damage to the heart valves. While secondary prophylaxis is the
mainstay for acute rheumatic fever and clinical RHD, the impact
on very early RHD – only diagnosed because of echo screening –
has been unknown.
THE GOAL TRIAL
To answer this question, our research teams designed the GOAL Trial,
which stands for “GwokO Adunu pa Lutino”, or “Protect the Heart
of a Child” in Luo (ClinicalTrials.gov Identifier: NCT03346525). In
summer 2018, I boarded a plane for a more than 24-hour journey
with my (very flexible and understanding) husband, and our three
children, to kick off the study. Alongside a team of more than 30
local and international volunteers, we conducted community-based
echo screening in the schools around Gulu, northern Uganda. We
assessed more than 120,000 children for the presence of early RHD
over three months. Many, like Michael, had disease too advanced to
qualify for the trial. But we enrolled nearly 1,000 students who met
the criteria. Now, in mid-2020, the trial is starting to wind down
and the answer to “can we improve outcomes” is within reach. We
should have our final data analyzed before Christmas. We anticipate
using the results to shape public health policy regarding if and when
echo screening should be used to identify early RHD.
Our hypothesis is that echo is useful in identifying RHD early,
and intervening will improve outcomes before kids like Michael
are sidelined, or worse. In 2013, Michael died from RHD at the
age of 12. Regardless of the study’s outcome, however, we will have
learned much about how to, or how not to, address the enormous
global burden of RHD.
NOT GONE, BUT STARTING TO BE REMEMBERED
The global health agenda has largely neglected RHD since the mid-
20th century. Much of the key research in this area is now 70 years
old. The GOAL trial is one of the only contemporary randomized
controlled trials to be conducted in patients with RHD, which
stands as one of the world’s least-funded conditions relative to
disease burden. 13 And, while RHD may seem a world away from
Kentucky, it continues to affect vulnerable populations in the rural
south, western states and US territories. 14
But there is reason for hope. In 2015, the World Heart Federation,
The Medtronic Foundation and Reach (a global RHD nongovernmental
organization) launched “RHD Action”, a movement
to improve awareness and build resources for countries wishing
to tackle RHD (rhdaction.org). 15 In May 2018, the World Health
24 LOUISVILLE MEDICINE
Assembly, the governing body of the World Health Organization,
passed a global RHD Resolution that now commits governments
to reprioritizing RHD on their health agenda. 16 And recently, the
American Heart Association—the group largely responsible for the
near elimination of ARF in the US in the 1950s—recommitted to
tackling RHD globally in their 2030 Impact Goals. Will the efforts
of these groups make rapid progress, as they did two generations
ago? For the sake of hundreds of thousands of children like Michael
who are living with RHD, I hope so. I hope future generations will
remember this moment as the start of RHD being “gone but not
forgotten” rather than “forgotten but not gone.”
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Dr. Beaton is a pediatric cardiologist at Cincinnati Children’s Hospital. Her primary
outpatient location and home is in Louisville, Kentucky. (non-member)