Thoracic Insufficiency Syndrome and Early-Onset Scoliosis: Salma's Story

Families go to extraordinary lengths to get the best care for their children. For some, that means traveling thousands of miles from home.

When 13-year-old Salma was diagnosed with severe scoliosis, her family left their home in the United Arab Emirates and brought her to one of the few places in the world with the expertise to treat her: The Children’s Hospital of Philadelphia.

The Division of Orthopedics at The Children’s Hospital of Philadelphia has been consistently top ranked in U.S. News & World Report’s annual survey of the Best Children’s Hospitals in the United States. The Hospital shared the No. 1 spot on U.S. News'  2014-15 Honor Roll. CHOP's orthopedic team has vast experience treating all types of orthopedic disorders.

The Division of Orthopedics team, led by John P. Dormans, MD, performs more than 300 surgeries each year on children with spinal deformities, and also has extensive expertise in limb-sparing surgery for bone tumors.

Scoliosis and thoracic insufficiency syndrome

When Salma arrived at CHOP in the fall of 2011, she was evaluated by a team of specialists, including Robert M. Campbell Jr., MD, director of the Hospital’s Center for Thoracic Insufficiency Syndrome.

The severe curve in Salma’s spine had affected the development of her ribs and lungs and caused her to develop thoracic insufficiency syndrome (TIS), a condition characterized by the inability of the thorax to support normal respiration or lung growth.

The team also found that Salma suffered from several other serious medical problems, including an obstructed trachea and basilar invagination, a rare condition in which the cervical spine migrates into the skull.

Multidisciplinary care

Patients like Salma benefit from the Center’s multidisciplinary medical and surgical evaluation and care, which involves orthopedics, pulmonology and general surgery, along with consultations by neurosurgery, gastroenterology, nutrition and other services.

“No other center has the multispecialty approach that we have,” says Dr. Campbell. “It’s very comprehensive.”

The Center also employs the most advanced diagnostic imaging, including dynamic lung MRI, a technique pioneered at CHOP that allows clinicians to see the precise movements of a patient’s lungs and breathing muscles.

Like the more than 120 other families from the Middle East who have come to CHOP for care over the past year, Salma’s family worked closely with the Hospital’s International Patient Services staff, who assist with visas, travel logistics and accommodations, arrange for medical interpreters, and help families feel right at home at CHOP — and in Philadelphia.

A pioneering treatment

To stabilize the curve in Salma’s spine and to expand her chest to help her lungs grow, Dr. Campbell performed a VEPTR implantation in October 2011. He implanted two vertical expandable prosthetic titanium ribs (VEPTR) from Salma’s ribs to her pelvis, which allow her chest to expand so she can breathe more easily. He also added a bone graft to stabilize her spine long term.

Dr. Campbell invented the VEPTR device, one of several “growth-sparing” options CHOP offers for children with thoracic insufficiency syndrome and early-onset scoliosis.

The device, which is often the best option for TIS patients who have chest wall deformities, allows the ribs, spine and lungs to expand and grow and is expanded over time.

Patients whose chest walls are intact typically receive growing rods, which are placed alongside the spine and are lengthened as the child grows.

Safety first

Children undergoing complex spinal surgeries face a substantial risk of spinal cord injury — a risk CHOP is working to reduce with its comprehensive neural monitoring program.

During spine surgeries at CHOP, sensors are applied to the patient’s body to monitor signals between the brain and the upper and lower extremities. Working with anesthesiologists, neurophysiologists in the OR continuously assess the integrity of sensory and motor pathways and nerve roots, and are able to alert the team when there is an indication the patient may be at risk for an adverse event.

Safety is key,” says Dr. Dormans. “The risks are high for these children. And so having a team that really dedicates itself to the intricacies of care for these children with very, very difficult and complex problems is important to us.”

Hope for the future

Less than a week after her surgery, Salma went home to the Philadelphia apartment that her family had rented for the duration of her treatment. In March 2012, CHOP neurosurgeon Phillip B. Storm Jr., MD, performed an operation to stabilize her cervical spine, and she had her first VEPTR-expanding surgery a month later.

Salma’s father, Khamis, is thrilled with the progress his daughter has made over the past several months — and he’s grateful to CHOP for providing such exceptional care.

“Every day,” he says, “we gain something.”


Originally posted: July 2012