Pediatric vascular malformations program provides state-of-the-art care to children who need it most
Long before Mia Sanchez was born, her doctors at NewYork-Presbyterian/Columbia University Medical Center knew something was profoundly amiss. Her heart pumping furiously, she appeared headed for heart failure. Imaging conducted by the Center for Prenatal Pediatrics revealed the cause: the heart was working overtime to feed blood to a large vascular malformation, a mass of abnormal blood vessels growing in the tiny baby's neck.
With no way to surgically remove the mass before birth, the team needed to somehow reduce the stress on the baby's heart and prevent heart failure. It was unclear whether enough medication would cross the placenta if the mother took oral heart medicine, but the team tried this approach, administering digoxin to the mother when the baby was at about 25 weeks gestation. Fortunately for Mia, the medication eased the strain on her heart, allowing her to grow to full term.
On August 6, 2011, Mia was delivered by elective caesarian section. By this time, the mass of blood vessels gone awry was as large as her head.
According to Angela Kadenhe-Chiweshe, MD, Mia's pediatric surgeon, the baby was in distress at birth and needed mechanical assistance to breathe. The vascular malformation was causing heart failure because of the large amount of turbulent blood flow, which was also affecting her clotting functions. Although it was clearly a threat to the baby's life, the team could not remove the mass just yet. As Dr. Chiweshe explains, "Very large vessels were feeding this mass, and there was such a large volume of blood that immediate removal could have led to intraoperative death (by hemorrhage). We knew we would eventually need to operate, but we had to first make it safe."
To accomplish this, the team first stabilized Mia with steroids and propanolol. These medications helped to stop the growth of the large mass and improved her heart function enough that she could breathe without mechanical assistance. Once the baby had derived maximum benefit from these medications, Philip M. Meyers, MD, FAHA, a neurointerventional radiologist and Co-Director of Neuro–endovascular Services, NewYork-Presbyterian Hospital, plugged the major vessels feeding the mass through coil embolization. "We elected to do this at the time we felt the baby was in the best clinical state possible to withstand the operation," says Dr. Chiweshe. Embolization was successful in cutting the blood supply to the mass by about 75%. Then, while still under anesthesia, Mia was transported to the operating room where the surgical team removed the mass.
The surgery, done by Dr. Chiweshe and June K. Wu, MD, a plastic and reconstructive surgeon who specializes in vascular anomalies, was a success. The mass was safely removed, Mia recovered, and she was soon weaned off the steroid medications. Although her heart is now functioning normally, she will continue to see her pediatric cardiologist for followup. Today she is eating, growing, and is considered fully cured.
Mia's team included expert specialists in maternal-fetal medicine, obstetrics, neonatology, pediatric cardiology, pediatric surgery, pediatric plastic surgery, pediatric dermatology, pediatric hematology, neonatal nursing, basic science research, radiology, and more. "It truly takes a village to properly treat children with a complex condition like Mia's," says Dr. Chiweshe. "This child's outcome is a direct result of the multidisciplinary program created by the Division of Pediatric Surgery and other divisions to treat vascular anomalies."
The pediatric vascular anomalies program was formed as a multidisciplinary group in 1998, in conjunction with an initiative by physician researchers at NYP/Columbia to investigate angiogenesis, the growth of blood vessels, and factors that cause blood vessels to grow abnormally. In particular the researchers were uncovering the role of VEGF, or vascular endothelial growth factor, which is an important regulator of cell growth. Their work on VEGF contributed to the development of the cancer drug Avastin, particularly as this applies to pediatric patients with cancer, and has led to other 'translational' therapies, or therapies translated rapidly from the laboratory to the clinic. Faculty in the Division of Pediatric Surgery understood that the fruits of this research could provide patients with new therapeutic options, and that a multidisciplinary team was needed to provide the best level of care possible.
In Mia's case, molecular therapies targeting an aspect of angiogenesis were not needed because the conventional approaches — medications for heart failure, embolization of the vessels, and surgery — were successful. But for other children, the understanding gleaned from the team’s research has provided molecular-based options that are simply unavailable elsewhere. For example, the lab has recently shown that very common medicines used in small doses, such as an antibiotic related to tetracycline, can treat vascular malformations by directly targeting cells that line abnormal blood vessels.
"Children with vascular malformations can be very difficult to treat," says Dr. Chiweshe. "Very few hospitals have the expertise or resources to treat them, and they often have poor outcomes. We are fortunate here to be able to offer not only expertise in conventional therapies, but also novel treatments as a result of our research program."
To learn more about the treatment of vascular malformations at NewYork-Presbyterian Hospital, please visit: childrensnyp.org/mschony/pediatric-surgery.html or call 212.342.8585.