Congenital heart disease is present at birth and affects how blood flows through the heart. Treating these structural problems depends on the type and severity of the defect, and the goal is to help children and adults live healthy, active lives despite their heart condition.

How Treatment Helps Children and Adults with Heart Defects

When a baby is born with a structural problem in the heart, doctors focus on helping that child grow up as healthy as possible. The main goals of treating congenital heart disease include improving blood flow through the heart, preventing complications, and supporting the body’s organs to receive enough oxygen. Treatment decisions are made based on the type of defect, how severe it is, and the age of the patient.^[1]

Some heart defects are mild and may never need treatment at all. Children with these simple defects often live normal lives with regular checkups to monitor their heart health. However, more complex defects can be life-threatening and require immediate action, sometimes within the first days or weeks of life. Healthcare providers can detect many heart defects before birth using special ultrasound examinations, or shortly after birth through newborn screening tests.^[8]

Modern medicine has changed the outlook for children born with heart defects dramatically. Advances in diagnosis and treatment now allow most of these children to survive into adulthood. This means that more adults than ever before are living with congenital heart disease and need ongoing medical care throughout their lives. Treatment approaches include regular monitoring, medications, procedures performed through thin tubes called catheters, and open-heart surgery.^[1][7]

Standard Treatment Approaches

The type of treatment recommended for congenital heart disease depends entirely on the specific problem in the heart. Many simple defects, such as small holes in the heart walls, can improve or even close on their own over time. For these cases, doctors recommend a “watchful waiting” approach, with regular checkups using imaging tests like echocardiograms (ultrasound pictures of the heart). These follow-up visits might happen every couple of years for very minor defects.^[13]

Medications

For certain types of heart defects, medicine plays an important role. One specific condition called patent ductus arteriosus (PDA) involves a blood vessel that normally closes after birth but remains open. This vessel connected the lung artery to the main body artery before the baby was born. When it stays open, extra blood flows to the lungs, making the heart work harder.^[4]

Doctors can use medications to help close this vessel. The medicines most commonly used are indomethacin and a special form of ibuprofen. These drugs trigger the open vessel to tighten and eventually close. Acetaminophen is sometimes used as well. These medications work particularly well in premature babies who have this condition.^[14][15]

Other medications may be prescribed to manage symptoms or complications related to heart defects. Diuretics, also known as water tablets, help remove extra fluid from the body, making it easier to breathe. Some children receive digoxin, a medicine that slows down the heartbeat and makes the heart pump more strongly. These medications don’t fix the structural problem, but they help the heart work better while doctors monitor the condition or prepare for other treatments.^[15]

Catheter-Based Procedures

Many heart defects can now be repaired without open-heart surgery, thanks to procedures performed through blood vessels. During cardiac catheterization, doctors insert a thin, flexible tube into a blood vessel in the groin or neck and carefully guide it to the heart. This approach is less invasive than surgery and allows children to recover much faster.^[14]

Through this catheter, doctors can close holes in the heart by placing special closure devices. These devices are mounted on a balloon catheter and positioned precisely where needed. Once in place, they become a permanent part of the heart wall over time. This technique is now used to close about 98% of patent ductus arteriosus cases and is also effective for closing certain types of holes between the upper chambers of the heart, called atrial septal defects (ASD).^[16]

Catheter procedures can also widen narrowed valves or blood vessels. In a procedure called balloon valvuloplasty, doctors inflate a balloon at the site of a narrowed valve to stretch it open. This improves blood flow without the need for major surgery. The advantages of these catheter techniques are remarkable: patients often go home the next day with minimal recovery time, compared to several days in the hospital after open-heart surgery.^[15][16]

Surgical Repair

When catheter procedures are not suitable or when defects are severe, heart surgery becomes necessary. In open-heart surgery, a surgeon makes a cut in the chest to directly access the heart and repair the structural problem. Surgery may be needed to close large holes in the heart walls, fix or replace abnormal valves, widen narrowed blood vessels, or correct complex problems involving multiple defects.^[14]

For babies born with critical heart defects—the most serious type—surgery is usually required within the first year of life, sometimes within days or weeks of birth. About one in four babies born with a heart defect has a critical defect that needs this urgent treatment.^[8]

Different surgical techniques exist depending on the defect. For example, when the main artery leaving the heart (the aorta) is too narrow—a condition called coarctation of the aorta—surgeons may remove the narrowed section and reconnect the healthy parts. They might also insert a metal tube called a stent to hold the vessel open, or use blood vessels from other parts of the body to create a bypass around the blockage.^[15]

Valve problems often require surgical repair or replacement. If a valve cannot be repaired, surgeons can replace it with a new one. Replacement valves can be made from animal tissue, human tissue, or metal. The choice depends on the patient’s age and specific condition. Sometimes, surgeons use a special technique where they move the patient’s own pulmonary valve to replace the damaged aortic valve, and then place a donor valve where the pulmonary valve was. This specialized procedure is called the Ross procedure.^[15]

Recovery from open-heart surgery typically requires several days in the hospital and a longer period of healing at home compared to catheter procedures. Despite being more invasive, surgery remains the best option for many complex heart defects and can provide excellent long-term results when performed by experienced pediatric heart surgeons.^[14]

Treatment in Clinical Trials

While standard treatments for congenital heart disease have improved dramatically over the years, researchers continue to explore new approaches to make treatment even safer and more effective. Clinical trials test innovative methods and devices that may one day become part of routine care. These studies follow careful phases to ensure safety and effectiveness before new treatments are widely available.

Understanding Clinical Trial Phases

Clinical trials for congenital heart disease treatments typically progress through several phases. Phase I trials focus on safety, testing new procedures or devices on a small number of patients to understand any risks. Phase II trials examine whether the treatment actually works and how well it performs in ideal conditions. Phase III trials compare the new treatment directly with current standard approaches to see if it offers advantages. Finally, Phase IV trials continue to monitor treatments after they’ve been approved, watching for long-term effects or rare complications.^[29]

Parents considering clinical trials for their children, or adults with congenital heart disease considering trials for themselves, should discuss thoroughly with their doctors whether participation might be appropriate. Clinical trials offer access to cutting-edge treatments but also involve uncertainties since these approaches are still being studied.

Innovative Devices and Techniques

Research into congenital heart disease treatment continues to advance catheter-based methods as alternatives to open-heart surgery. Scientists are developing new types of closure devices for different kinds of heart defects. For example, newer devices are being tested to close holes between the lower chambers of the heart (ventricular septal defects) that were previously too complex for catheter treatment. These innovative devices are designed to be implanted through blood vessels and, once in place, become integrated into the heart tissue.^[16]

Researchers are also improving balloon and stent technologies. Newer stents can expand as a child grows, potentially reducing the need for multiple procedures as the child ages. Some experimental stents are made from materials that dissolve over time once the blood vessel has healed and stabilized, eliminating the need for permanent metal implants.

Imaging technology plays a crucial role in advancing treatment. Clinical trials are testing new three-dimensional imaging techniques that allow doctors to see heart defects in greater detail before and during procedures. Better imaging helps surgeons and interventional cardiologists plan more precisely and perform procedures with greater accuracy.

Eligibility and Participation

Clinical trials for congenital heart disease are conducted at specialized medical centers, often at major children’s hospitals and academic medical centers. Trials may be available in various locations including the United States, Europe, and other regions with advanced pediatric cardiac care programs.

Eligibility for clinical trials depends on many factors, including the specific type of heart defect, the patient’s age, whether previous treatments have been attempted, and the presence of other medical conditions. Some trials focus on specific defects such as atrial septal defects or patent ductus arteriosus, while others examine approaches for complex conditions. Doctors can help families find appropriate clinical trials and determine whether a patient might qualify for participation.

Most common treatment methods

• Watchful waiting with monitoring
  • Regular checkups with echocardiograms for minor defects that may improve on their own
  • Follow-up visits ranging from every few years to multiple times per year depending on severity
  • Monitoring for potential complications like abnormal heart rhythms or heart failure
• Medications
  • Indomethacin or ibuprofen to close patent ductus arteriosus in premature infants
  • Diuretics (water tablets) to remove excess fluid and ease breathing
  • Digoxin to slow heart rate and strengthen heart contractions
  • Acetaminophen sometimes used to help close patent ductus arteriosus
• Catheter-based procedures
  • Cardiac catheterization to insert closure devices for holes in heart walls
  • Balloon valvuloplasty to widen narrowed heart valves
  • Device closure for atrial septal defects and patent ductus arteriosus
  • Stent placement to open narrowed blood vessels
  • Typically performed as outpatient or one-day hospital admission with minimal recovery time
• Open-heart surgery
  • Surgical closure of large holes in heart walls (ventricular or atrial septal defects)
  • Valve repair or replacement using animal tissue, human tissue, or metal valves
  • Ross procedure for aortic valve replacement using patient’s own pulmonary valve
  • Repair or reconstruction of narrowed aorta (coarctation repair)
  • Complex repairs for critical defects requiring surgery in infancy
  • Hospital stay of several days required with longer recovery period at home

Living with Congenital Heart Disease

Children and adults who have congenital heart disease need ongoing care throughout their lives, even after successful treatment. Many people with heart defects are not cured, even when their defect has been surgically repaired. The outlook has improved dramatically, and today more than 2 million people in the United States are living with congenital heart disease. With proper care, most can live healthy, active lives.^[19][20]

Regular Follow-Up Care

Receiving routine follow-up care is one of the most important aspects of living with a heart defect. Children should see a pediatric cardiologist regularly, and as they transition to adulthood, they need to establish care with a specialist who treats adults with congenital heart disease. Some adults require care from physicians who specifically focus on adult congenital heart disease, as these specialists understand the unique long-term complications that can develop.^[19][23]

The frequency of checkups varies based on the complexity of the heart defect. Someone with a simple defect might need appointments every other year, while someone with a complex defect or frequent health problems may need to be seen multiple times per year. Most people need at least one annual visit with a heart specialist. These regular appointments allow doctors to monitor heart function, check for complications like abnormal heart rhythms or heart failure, and adjust medications as needed.^[23]

People with heart defects can develop other health problems over time, depending on their specific defect, the number of defects they have, how severe the defect is, and whether they receive appropriate medical care. Regular monitoring helps catch these problems early when they’re easier to manage.^[20]

Preventing Infections

Some people with congenital heart disease have a higher risk of developing endocarditis, a serious infection of the heart’s inner lining. To prevent this, some patients need to take antibiotics before certain dental procedures or surgeries. Not everyone with a heart defect requires these preventive antibiotics, so it’s important to discuss your individual risk with your doctor. Good oral hygiene is essential for everyone with a heart defect—this means brushing and flossing teeth daily and visiting the dentist regularly for cleanings.^[22]

Avoiding common infections is also important. People with heart defects should stay up to date on vaccinations, including annual flu shots and COVID-19 vaccines. These infections can be more serious for people with heart conditions, so prevention is key.^[19]

Physical Activity and Exercise

Contrary to old beliefs, most people with congenital heart disease can and should be physically active. Regular physical activity helps the heart become stronger, improves overall fitness, and reduces risk factors like high blood pressure. Exercise can help children and adults with heart defects maintain a healthy weight and improve their quality of life.^[19][21]

In the past, doctors used to limit physical activity for children with heart defects. Unfortunately, children who began inactive lifestyles often developed problems with weight gain. Today, doctors recognize that physical activity is beneficial for most people with heart defects, although some may need to restrict the type or intensity of exercise depending on their specific condition.^[21]

The amount and kind of physical activity that is safe depends on the type of heart defect and how severe it is. Some people with mild defects have no restrictions at all, while others with more complex conditions may need to avoid certain competitive sports or very strenuous activities. It’s essential to talk with your doctor about which activities are safe and appropriate. Even with restrictions, staying active within safe limits is important for health.^[22]

Heart-Healthy Lifestyle

Making healthy lifestyle choices supports heart health and overall wellbeing. Eating heart-healthy foods is important for everyone but especially for people with heart conditions. A heart-healthy diet includes plenty of vegetables, fruits, whole grains, nuts, beans, lean meat, and fish. It’s wise to limit alcohol, sodium (salt), and added sugars. These eating habits help control blood pressure, maintain a healthy weight, and reduce strain on the heart.^[19]

Smoking is particularly harmful for people with congenital heart disease and should be avoided completely. Even exposure to secondhand smoke can be dangerous. If you smoke, quitting is one of the best things you can do for your heart health.

Managing other health conditions is also crucial. People with heart defects who develop high blood pressure, high cholesterol, or diabetes need to work with their doctors to keep these conditions well-controlled. Taking medications exactly as prescribed is essential—never stop or change heart medications without talking to your doctor first.^[19]

Special Considerations for Women

Women of childbearing age with congenital heart disease need to discuss birth control options with their doctors. Some forms of birth control may be safer than others depending on the type of heart defect. It’s important to choose a method that provides effective pregnancy prevention while posing the lowest risk to heart health.^[22]

Pregnancy requires special planning for women with congenital heart disease. Some women face increased health risks during pregnancy, depending on their specific heart condition. It’s essential to talk with both a cardiologist and an obstetrician before becoming pregnant to understand the potential risks and ensure proper care throughout pregnancy. There is also a possibility that a heart defect could be passed on to a child, though the risk varies depending on the specific defect.^[22]

Emotional and Mental Health

Living with a chronic health condition like congenital heart disease can affect emotions and mental health. It’s normal to feel worried, sad, or overwhelmed at times. If you or your child feels sad or hopeless much of the time, or experiences significant worry and anxiety, it’s important to talk with a doctor. Treatment with counseling and medication can help manage these feelings and improve quality of life.^[19]

Support groups connecting people who have similar experiences can also be valuable. Many adults and families find it helpful to talk with others who understand what it’s like to live with a heart defect.