All Children's Hospital Telethon

Sami

Samantha Bass was the picture of health when she was born in July of 2005.

Twelve days later, she was in intensive care at All Children's, fighting for her life - all because of a severe case of dehydration.

Sami

"Newborns who become dehydrated can sometimes have hypercoagulation," explains pediatric intensivist Perry Everett, MD. "Their blood tends to clot in places that it shouldn't clot, particularly in blood vessels." In Sami's case, the clot lodged in one of the worst possible places - her aorta, the major vessel coming out of the heart that feeds oxygen-rich blood to every organ system in the body.

Sami's clot extended all the way into arteries feeding her kidneys. "Therefore, her kidneys couldn't work," recalls pediatric nephrologist Sharon Perlman, MD. "They couldn't clean her blood. They couldn't make urine. She had pretty severe kidney failure."

Dialysis in newborns is difficult and dangerous. Blood-thinning agents that might dissolve the clot carry added risks in newborns who are already susceptible to bleeding in the brain. When standard treatment options didn't work, attention turned to alternatives more risky for a patient so small.

"Sami was born with no medical problems and this was a tragedy," says pediatric intensivist Michele Smith, MD. "And if there was something that we could provide to change that course, then you do everything you can. Luckily, that meant looking in the literature to find someone else had experienced this and wrote about it."

The article Smith found documented the successful use of an extremely high-risk procedure. Doctors at Johns Hopkins Hospital had guided a catheter through their patient's tiny arteries and into the clot. Once there, they injected a powerful clot-busting drug, tPA.

All Children's pediatric interventional radiologist Kevin Potthast, MD would take on the challenge of threading the catheter through Sammie's tiny arteries, no thicker than a pencil lead. A team of doctors and nurses in intensive care would then keep vigil over Sami, managing the risks of her chemically-thinned blood, while parents Cindi and Vaughn prayed that fragments of the clot wouldn't dislodge and cause catastrophic damage to their baby girl's brain, heart or lungs.

Fast-forward nearly eight years, and Sami is a bright, outgoing big sister to little brother Stone.

All Children's Hospital is Johns Hopkins Medicine's only member hospital outside of the Baltimore/Washington DC metro area.

And one of the world's leading experts in clotting disorders in children has come to St. Petersburg, FL to establish a program to help children like Sami and further research efforts toward new treatment options.

New Program - International Leader

The Pediatric Thrombosis Program at ACH-JHM in St. Petersburg, FL combines the expertise of All Children's Hospital and Johns Hopkins Medicine for children who have developed a blood clot in the veins (venous thrombosis) or arteries (arterial thrombosis). Services extend from inpatient to outpatient care, consultation, and access to the latest research developments in treatment. The program participates in - and even leads - a variety of clinical trials.

Program director Dr. Neil Goldenberg was recruited by ACH-JHM in 2012 from one of the top pediatric blood clotting programs internationally, the Mountain States Regional Hemophilia and Thrombosis Center at Children's Hospital Colorado, to lead the pediatric thrombosis programs at ACH-JHM and the Johns Hopkins Children's Center in Baltimore. The physician team also includes Dr. Irmel Ayala, who has led the All Children's bleeding and clotting disorder clinics at the main hospital in St. Petersburg and its Outpatient Care Center in Tampa for over a decade. The ACH-JHM program provides a variety of services for pediatric thrombosis patients and their families in a comprehensive, multidisciplinary setting.

How Are Blood Clots Treated in Children?

Reports have indicated that the rate of thrombosis in U.S. children has dramatically increased in recent years. While clots are less common in children than adults, the consequences of these clots can have tremendous impact on the growing bodies of young patients. This can also raise concerns about their future participation in sports and active lifestyles. Even though clots often occur in children with other underlying medical conditions, whenever a clot occurs in a child there is a need for expert care. And future risks should not be overlooked.

Evaluation and treatment of children with thrombosis begins with radiologic imaging to establish the extent of the clot and its impact on blood flow to other parts of the body. Doppler ultrasound, CT, or MRI scan may be used, depending on the location and extent of clot. Specialized laboratory testing of a child's blood clotting system is vitally important. Administration of blood thinner medication is often a first response. Follow-up laboratory testing and radiologic imaging to monitor the treatment's effects provide information that is key to decision-making on how long such treatment should continue. After treatment is complete, patients and families need education on measures to prevent further blood clots, including guidance on future situations in which temporary use of blood thinners may be recommended. In each step along the way, the fact that the patient is a growing child makes it essential that care be provided by a devoted team of pediatric experts in blood clotting and related specialties.

Pediatric Focus Is Key

The multidisciplinary thrombosis team at All Children's is focused solely on treating infants, children and teens who have developed a blood clot in the veins or arteries. It also provides evaluation of infants, children, and teens who have a family history of blood clots before 50 years of age, and therefore may be at risk for developing young-onset blood clots themselves.Our pediatric thrombosis doctors have devoted their careers to caring for children who experience a stroke and strive to provide the best possible outcomes for their patients.

Dr. Goldenberg is recognized as one of the world's authorities in the care of children and young adults with thrombosis, and is consulted by hematology colleagues throughout the U.S. to provide expertise in complex patient management. Monthly videoconference meetings run by Dr. Goldenberg draw physicians from such diverse institutions as Tulane, Boston Children's Hospital and Texas Children's Hospital to discuss their most challenging patients with their All Children's - Johns Hopkins peers.

Dr. Goldenberg has a leadership role in several national/international guidelines committees in pediatric thrombosis, and is also helping to lead a national pediatric hospital network effort on preventing blood clots and their complications in hospitalized children. In addition, Dr. Goldenberg is a 2012 recipient of the international Eberhard F. Mammen Excellence in Thrombosis and Haemostasis Award, and is a leader in several national/international clinical trials of pediatric thrombosis.

Because of the rarity of thrombosis in children, few physicians have extensive experience in treating this problem and preventing short-term and long-term consequences. The potential issues can be serious and long-lasting, from bleeding complications of blood thinners to persistent blockage in blood flow, despite a routine course of treatment with blood thinner medication. Second blood clots can develop. Blood vessels themselves can be damaged by the clot. Drs. Goldenberg and Ayala have a combined experience of managing over 1,000 pediatric thrombosis patients in the past 10 years. It's this experience - combined with their involvement in the latest national/international guidelines and clinical research studies - that helps them to quickly determine whether a child is at high versus low risk for those potential long-term complications. An individualized treatment and follow-up plan is then developed for each patient, in order to achieve the best outcomes for each infant, child, and teen.

It Takes a Broader Team

Clots in children can occur almost anywhere - in the veins or arteries of the limbs, brain, lungs, heart, liver, kidneys, or other vital areas of the body. Because of this, a pediatric hematologist (or blood specialist) who specializes in thrombosis often relies on the expertise of a variety of pediatric specialty physicians and health care providers.

When children are first hospitalized with thrombosis, our multidisciplinary care team consists of pediatric intensive care physicians and nurses, pediatric hematologists, pediatric radiologists, pediatric anesthesiologists, pharmacists, child life specialists, social workers, discharge planners, research personnel, and any other specialists that an individual child's condition might warrant.

In the outpatient setting, our multidisciplinary clinic involves Drs. Goldenberg and Ayala as pediatric hematologists with specialized expertise in thrombosis, an anticoagulant ("blood thinner" medication) pharmacist, a pediatric thrombosis nurse and research personnel. Visits are coordinated with other appropriate specialized physicians who are experts on organ systems affected by the blood clot or any underlying chronic medical conditions - for example, pediatric cardiologists, for children with congenital heart disease who have developed blood clots.

Care continues even after a patient goes home, with an expert pediatric physician-pharmacist team focused on adjusting and monitoring dosages of any blood-thinning medications that a teen or younger child might require. Testing essential to making the right medication decisions can be done at All Children's or a variety of outpatient labs. And in either case, our thrombosis program continues to review results and coordinate care with the patient's primary care provider.

After all, getting a patient back home - and to a normal routine - is the ultimate goal.

It's a goal Sami has clearly achieved. Now almost eight years old, she participates in school and softball and all the things one would hope for. Her family knows those hopes were made possible in part through the team in St. Petersburg that saw Sami through those frightening early weeks.

As mom Cindi says, "We wouldn't have Samantha if All Children's wasn't there."



Children's Miracle Network - the alliance of premier hospitals for children Logo - Click to visit the web site All Children's Hospital is a member of Children's Miracle Network Hospitals, which represents children's hospitals across North America. Our Telethon shares the courageous stories of our young patients, who are at the heart of it all. No matter which part of the hour you call, your entire donation stays with All Children's Hospital to benefit children from your community.
Contact Us   |   Telethon Stories   |   Donors   |   Share Your Story   |   Join In!   |   About ACH


©2014 All Children's Hospital - All Rights Reserved  

Link to Telethon Homepage Join us on Facebook, Twitter, or Flickr Visit the News Channel 8 Website Children's Miracle Network Donate Now!