A Heart Valve That Grows Along With a Child Could Reduce Invasive Surgeries

Clinical trials have started for the first prosthetic pulmonary valve replacement that is specifically designed for pediatric patients and can expand over time inside a child’s anatomy. 

Instead of having invasive replacement surgeries every few years, as is the practice now, a child can have the valve fitted to their individual body size and, if needed, adjusted for size through a minimally-invasive transcatheter balloon dilation procedure to help maintain blood flow. They potentially wouldn’t need another replacement procedure until adulthood.

The device, known as the Autus Valve, was invented at Boston Children’s. It was first implanted in a young patient at Nationwide Children’s Hospital in late 2021 as part of a collaborative clinical study involving Boston Children’s, Nationwide Children’s, and New York-Presbyterian/Columbia University Irving Medical Center. “It’s exciting and incredibly motivating that we’re at the stage where we can actually see the device helping patients,” says the device’s inventor, Sophie-Charlotte Hofferberth, MD. 

Boston Children’s cardiac catheterization team has already made other significant advancements in transcatheter valve replacement technology, and the Autus Valve continues that pace of innovation, according to Christopher Baird, MD, director of the hospital’s Congenital Heart Valve Program.

Mimicking the design of human venous valves
An estimated 110,000 children in the U.S. have congenital pulmonary valve disease, which impedes the flow of blood between the heart and lungs. Pulmonary valves that are narrowed or leaking and can’t be treated effectively with a catheter are usually replaced with a prosthetic valve. But existing prosthetic pulmonary valves are adult-sized and fixed in diameter, usually requiring a child to have these devices replaced as they grow.

Hofferberth was tasked with creating a valve that could accommodate the growth of a child when she joined the research laboratory of Pedro J. del Nido, MD, chief of Boston Children’s Cardiac Surgery Department, in 2016.

Commercially available prosthetic heart valves have three leaflets that serve as flaps to control blood flow, mirroring the tri-leaflet structure of the human aortic valve. A device that mimicked the bi-leaflet function of a venous valve in leg veins showed promise to Hofferberth and colleagues. That’s because a venous valve’s two elastic flaps have the ideal geometry to maintain proper closure and one-way blood flow even when veins in the leg expand in diameter. Believing this process could also work in the heart, Hofferberth studied the geometric profile of the human venous valve and used this as the basis for creating a valve prototype.

Designing a size-adjustable valve that will last

Hofferberth and her team collaborated with Elazer Edelman, MD, PhD, of the Massachusetts Institute of Technology. They implanted prototypes of the replacement pulmonary valve in growing lambs, and those studies showed the device can be fitted and then expanded in sync with the growth of heart anatomy. The valve also maintained the control of blood flow — without stretching and compromising the device’s frame or material. The device’s two leaflets are made of a polymer with a long track record of use as a pediatric pulmonary valve leaflet. “That gave us a lot of confidence, using a well-known synthetic material that has proven long-term results in children,” she says.

Doctors can adjust the valve diameter to match a patient’s heart anatomy before it is implanted. Once the device is implanted, cardiac catheterization specialists can use a catheter balloon to expand the valve if it becomes too small after a child’s growth spurt. The doctors use an echocardiogram to assess the valve’s integrity and how well it is controlling the flow of blood.

“If a valve expansion is needed after the device is implanted, we anticipate a child would recover from the procedure within a few hours,” says Hofferberth, who is no longer with Boston Children’s.

Aiming for children’s long-term heart health

Boston Children’s is now holding FDA-approved early clinical studies on the valve’s effectiveness in children ages 2 to 11, along with Nationwide Children’s and New-York Presbyterian/Columbia University Irving Medical Center. Since the initial patient, several other children received Autus Valve implants; Boston Children’s had its first implantation procedure in late December.

Hofferberth intends to follow the initial study with a larger clinical trial, with the goal of seeking FDA approval to make the device commercially available.

“There is a huge need for better solutions for children with valve disease,” Hofferberth says. “A pulmonary valve that can be adjusted for size could give young patients a bridge through childhood and have a huge impact on their long-term quality of life.”

Originally Posted at Boston Children’s Hospital on January 4, 2023 by Albert McKeon.

Congenital Heart Defect Awareness Week-February 7th-14th

I recently saw some moms being discouraged when it comes to their efforts for Congenital Heart Defect Awareness.  Any effort you make has an impact, whether you realize it or not. Your effort may be something as life changing as sharing your experience with a mom who does not yet know her unborn child has a heart defect. My favorite example of this was with our local Kids With Heart support group.  We had a family hayride at a local ranch. The driver of our wagon was a pregnant lady. Her baby was not due for another three months. This was back before the days of cardiac ultrasounds as routine.  After the hayride through the woods, we invited her back to the cabin to have dinner with all the “heart kids” and their families.  She got to witness all of the “heart kids, at various stages of their surgeries, running around, having fun, with their siblings, hear the conversations between the moms, dads, and both.  It turned out that when her baby was born 3 months later, she was diagnosed with several heart defects.  This mom has told me many times how much it helped her to deal with this devastating news because she had been at the hayride.  While I could share several other positive stories, this one is my favorite. This is how I came up with our title for our Kids With Heart brochure: EVERYONE SOMEWHERE KNOWS SOMEONE AFFECTED BY CONGENITAL HEART DEFECTS!

Being involved in Congenital Heart Defect Awareness efforts since 1991, I can tell you that CHDs are much more known than they were when we started on this road. There was no such thing as an IEP for kids with CHDs, there was no such thing as special services for the heart kids, in school or sports or anything else. The first recognized Congenital Heart Defect Day was February 14, 1999(?)- pretty sure that was the year. It was a national proclamation effort that was started by a mom, Jean Imperati, and aided by a lot of different support groups, parents, and at that time, the few CHD organizations. Now, we have Congenital Heart Defect Awareness Week!  Yes, it gets frustrating but seeing people like Jimmy Kimmel come out and talk about his child’s chd just goes to show you how far we have come. Over the years, CHD affected many famous celebrities but they never wanted anyone to know. The fact that it took Shaun White this long for him to come forward to talk about his heart defects should be an indication of progress. Not only that but the multitude of non-profits dedicated to congenital heart defects is now overwhelming as well as the advocacy work that is being done. In addition, there is a huge variety of congenital heart defect awareness clothes, pins, etc.  The very first Congenital Heart Defect Awareness Ribbon Pins were made by hand by Gabrielle Harlow, a mom from Maryland, and was then modified so it could be mass produced for Kids with Heart National Assn for Children’s Heart Disorders, Inc. Gabrielle also started the Congenital Heart Defect Awareness Quilt Project under the Kids with Heart National Assn for Children’s Heart Disorders, Inc umbrella and is now her own Non-Profit organization.

Back in the 1990s and early 2000s, the American Heart Assn wanted nothing to do with congenital heart defects. It has been in the last 5-10 yrs that they have gotten involved at all with heart defect research. All of this has been accomplished by parents like you who got involved on a small scale to make a BIG DIFFERENCE! Remember, there is strength in numbers and to quote a favorite phrase: United we Stand, Divided we Fall!!! Please do not give up!!!!



FDA Approves First in the World Device to Treat Patients with Congenital Heart Disease

New implant device provides less invasive option to treat pulmonary valve regurgitation for patients with a native or surgically-repaired right ventricular outflow tract

Today, the U.S. Food and Drug Administration approved the first in the world non-surgical heart valve to treat pediatric and adult patients with a native or surgically-repaired right ventricular outflow tract (RVOT), the part of the heart that carries blood out of the right ventricle to the lungs. The device is designed for patients who have severe pulmonary valve regurgitation (blood leaking backward into the right lower chamber of the heart), a condition that often results from congenital heart disease. The device, called the Harmony Transcatheter Pulmonary Valve (TPV) System, is intended to improve blood flow to the lungs in patients with severe pulmonary valve regurgitation without open-heart surgery, which is the current standard of care. The use of the Harmony valve may delay the time before a patient needs additional open-heart surgery. It can also potentially reduce the total number of open-heart surgeries required over an individual’s lifetime.

“The Harmony TPV provides a new treatment option for adult and pediatric patients with certain types of congenital heart disease. It offers a less-invasive treatment alternative to open-heart surgery to patients with a leaky native or surgically-repaired RVOT and may help patients improve their quality of life and return to their normal activities more quickly, thus fulfilling an unmet clinical need of many patients with congenital heart disease,” said Bram Zuckerman, M.D., director of the Office of Cardiovascular Devices in the FDA’s Center for Devices and Radiological Health.

Congenital heart defects (CHDs) are conditions that are present at birth and can affect the structure of a baby’s heart and the way it works. They are the most common type of birth defect, affecting about 40,000 babies born each year. It is estimated that over two million infants, children, adolescents and adults are living with CHDs in the U.S. Patients with CHDs often require heart procedures early in life to help improve blood flow to the lungs. After having one of these procedures, the patient may or may not have a working pulmonary valve, which could lead to pulmonary regurgitation. Severe pulmonary valve regurgitation may be corrected through open-heart surgery to place a right ventricle-pulmonary artery conduit or an artificial valve.

During the implantation procedure of a Harmony valve, a thin, hollow tube (catheter) with a collapsed Harmony valve on the end is inserted through a vein in the groin or in the neck and into the right side of the heart, and then into the RVOT where it is placed into position. The valve is then released from the catheter; it expands on its own, and anchors to the RVOT. Once the new valve is in place, it opens and closes like a door to force the blood to flow in the correct direction.

The FDA assessed the safety and effectiveness of the Harmony TPV device through a prospective, non-randomized, multi-center clinical study. During the study, physicians implanted the device in a total of 70 patients. All patients were scheduled for follow-up examinations at the start of the study, at implant procedure, discharge, and post implant at one month, six months, and annually through five years. The follow-up has been extended to 10 years as part of the post-approval study. The primary safety endpoint was no procedure- or device-related death within 30 days following the implant, which 100% of patients attained. The primary effectiveness endpoint was percentage of patients with no additional surgical or interventional procedures related to the device and acceptable heart blood flow function at six months. Among patients with evaluable echocardiography data, 89.2% of them achieved the primary effectiveness endpoint.

Adverse events observed during the clinical study included irregular or abnormal heart rhythms (23.9%, including 14.1% ventricular tachycardia), leakage around the valve (8.5%, including 1.4% major leakage), minor bleeding (7.0%), narrowing of the pulmonary valve (4.2%), and movement of the implant (4.2%).

The Harmony TPV device is contraindicated for patients with an infection in the heart or elsewhere; patients who cannot tolerate blood thinning medicines; or patients who have sensitivity to Nitinol (titanium or nickel).

The Harmony TPV device was granted Breakthrough Device Designation for the treatment of pediatric and adult patients with severe pulmonary valve regurgitation. Breakthrough Device Designation is a process designed to expedite the development and review of devices that may provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions.

The device was also part of the U.S.-Japan Medical Device Harmonization by Doing Collaboration, which seeks to promote timely access to innovative devices in both the U.S. and Japan through greater collaboration and communication among regulatory, academic and industry stakeholders across all stages of product development, and by developing practical solutions to shared real-world challenges.

The Harmony TPV device was approved using the Premarket Approval (PMA) pathway. Premarket approval is the most stringent type of device marketing application required by the FDA and is based on a determination by the FDA that the PMA application contains sufficient valid scientific evidence to provide reasonable assurance that the device is safe and effective for its intended use(s).

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Originally posted March 21st, 2021, Press Release by the FDA at

Artificial heart valve can grow with kids, prevent repeat surgeries

Originally posted at

Current artificial heart valves are fixed in size, meaning children need to get larger ones as they grow, but a new design could eliminate the need for continued replacement.

ByHealthDay News

A new design means children could keep the same artificial heart valve until adulthood, and it could also benefit adults with heart valve defects. Photo by Semevent/Pixabay

An expandable artificial heart valve could save children with congenital heart disease from repeated open heart surgeries as they grow up, researchers report.

Current artificial heart valves are fixed in size, meaning children need to get larger ones as they grow. Children who receive their first artificial valve before age 2 will require up to five open-heart operations before they become adults.

This new design means children could keep the same artificial heart valve until adulthood, and it could also benefit adults with heart valve defects, according to the Boston Children’s Hospital team.

Current artificial heart valves have three leaflets, tiny flaps that provide a one-way inlet or outlet for blood to keep it flowing in the right direction. This new valve has two leaflets and as a patient grows it can be expanded through a minimally invasive balloon catheter procedure.

Lab tests, computer simulations and extensive testing in animals show that the new artificial valve works across a wide range of sizes, and remains functional when expanded, according to a study published online Feb. 19 in the journal Science Translational Medicine.

“We hope to bring this new device into clinical testing fairly rapidly,” said senior author Dr. Pedro del Nido, chair of cardiovascular surgery at Boston Children’s.

“If our preclinical results hold up in human testing, this could transform the field,” del Nido added in a hospital news release.

Research in animals doesn’t always pan out in humans. But a human clinical trial could begin within one or two years, according to the researchers.

The investigators said their design encourages good blood flow through the valve, which may reduce the risk of blood clot formation that often occurs with existing artificial valves.

In tests with sheep, there was no evidence of blood clot formation over 10 weeks of observation, even without the use of blood-thinning medications typically given to patients with artificial heart valves.

Each year, more than 330,000 children worldwide are born with a heart valve defect, and millions of others develop rheumatic heart disease requiring valve replacement.

CHD Awareness

Many heart parents are quite new to the CHD world but we would just like to let you know how far CHD awareness has actually come.

In 1999, Jeanne Imperati spearheaded the first ever effort for a CHD awareness day. It was February 14th and there were moms from everywhere going to their governors to get their states to proclaim that day as CHD Awareness Day in their state. It was truly a United Effort.

Today, February 7th thru February 14th is the official Congenital Heart Defect Awareness Week!  This level of awareness, this week of dedication, would not exist if it weren’t for their efforts.  Remember, if you are doing anything special in honor of this historic event, please share your pictures and ideas.  We invite you to share with us by email, with, or via facebook at Kids With Heart Facebook.

Please spread the word so that no family ever has to feel alone again.
Thank you so much for continuing to grow awareness.


Kids with Heart is pleased to announce it’s third annual benefit event!  Come join us at Jackson’s Point in Seymour Wisconsin, with a motorcycle ride, raffles, food, and live music!

Ride registration starts at 10 am, $15 a bike, $5 for a second rider.  Ride goes 11 approximately 2:30, with 50/50 raffles on the ride.  Then it’s back to Jackson’s Point.

Where we start the after party (No admission, open to everyone), including live music from the Wilson Huff Band, and bucket raffles.  Food and drinks available, check out Jackson’s Point’s amazing menu, and support our sponsor Hinterland Beer, by having a cold one.  Raffle drawings will begin at approximately 4:15.

For further details, or to follow the event and see a list of sponsors, visit our event page at: Kids With Heart 3rd Annual Benefit