Spine problems Pediatric Research and Clinical Trials

For more than a century, orthopedic surgeons and investigators at Children’s Hospital Boston have played a vital role in the field of musculoskeletal research, pioneering treatment approaches and major advances in the care and treatment of ailments such as scoliosis, polio, tuberculosis, hip dysplasias and traumas to the hand and upper extremities.

Our pioneering research helps answer the most pressing questions in pediatric orthopedics today—providing children with the most innovative care available.

At Boston Children’s Orthopedic Center, we take great pride in our basic science and clinical research leaders, who are recognized throughout the world for their respective achievements. Our orthopedic research team includes:

•   eight full-time basic scientists
•   37 clinical investigators
•   a team of research coordinators and statisticians

Clinical Effectiveness Research Center

The Orthopedic Clinical Effectiveness Research Center (CERC) helps coordinate research and clinical trials to improve the quality of life for children with musculoskeletal disorders. This collaborative clinical research program is unique in the nation and plays an instrumental role in establishing—for the first time—evidence-based standards of care for pediatric orthopedic patients throughout the world.

Major areas of focus for the CERC include:

•   spinal disorders
•   hip disorders
•   upper extremity disorders
•   brachial plexus birth palsy
•   trauma/fractures

Spinal program

Physicians in the CERC Spinal Program are active in several areas of ongoing basic and clinical research based at Children’s and the Harvard Orthopaedics Biomechanics Laboratory. Research topics include:

•   congenital scoliosis and idiopathic scoliosis
•   spondylolisthesis and spondylolysis
•   bone density studies of braced patients
•   in vitromechanical testing of lumbosacral fixation devices
•   computer-assisted strength analysis of vertebral metastases

Ongoing clinical spine studies

Spine Studies Partially Sponsored by the Spinal Deformity Study Group:

The mission and purpose of the Spinal Deformity Study Group (SDSG) is to create a means and forum whereby multi-center studies can be developed and conducted both efficiently and effectively. The SDSG is comprised of fifty national and international spine surgeons from 35 participating sites worldwide. Drs. Emans and Hresko are members of the SDSG.

(SDSG) The Effect of Surgery on Sagittal Spino-pelvic Measures of Balance in Developmental Spondylolisthesis and Its Relation to Clinical Outcome

This is a prospective multi-centered study focused on the outcomes of pediatric and adolescent idiopathic scoliosis. The main purpose of this observational study is to develop a prospective comprehensive radiographic and clinical database on consecutively treated pediatric and adolescent scoliosis surgical cases to assess outcome measures in patients with operative idiopathic scoliosis being treated with current surgical techniques. A secondary objective is to obtain data on currently available surgical approaches to treat idiopathic scoliosis in the thoracic, thoracolumbar, and lumbar spine.

(SDSG) Prospective Pediatric and Adolescent Scoliosis Study

The main objective of this prospective multi-center, observational study is to assess outcome measures in pediatric and adolescent patients with kyphosis, who are being treated non-operatively or operatively with current surgical techniques. Secondarily, data on currently available surgical approaches to treat pediatric kyphosis in the thoracic and/or thoracolumbar spine will be collected.

(SDSG) Prospective Pediatric and Adolescent Kyphosis Study

The goal of this prospective multi-center study of children with “idiopathic” scoliosis is to document concomitantly: 1) control of spinal deformity, 2) growth of the thoracic spine longitudinally and transversely at a rate commensurate with the number of vertebrae involved and 3) increasing lung volume, absolute and relative to body size.

(SDSG) Prospective Study of Deformity Management and Pulmonary Function in Early-Onset Scoliosis

The short-term goal of this prospective multi-center, observational study is to confirm the predictive value of sagittal spino-pelvic measurements in the surgical treatment of L5-S1 developmental spondylolisthesis. The long-term objective is to determine the optimal surgical treatment for L5-S1 developmental spondylolisthesis based on x-ray evaluation of sagittal trunk balance and functional outcome.

Other Multicenter Studies of Importance: By combining efforts with other centers, the Division of Spine Surgery at Children’s in Boston is able to more quickly reach valid research conclusions applicable to clinical practice.

Bracing in Adolescent Idiopathic Scoliosis Trial (BrAIST)

BrAIST is a multicenter randomized trial funded by the National Institutes of Health. Children’s Hospital Boston is one of 18 pediatric centers in North America participating in this clinical trial. The overall goal of this research is to determine whether bracing can slow or halt curve progression in patients with Adolescent Idiopathic Scoliosis (AIS), alleviating the need for surgical correction.

The secondary aims of this study are to 1) investigate the effect of the diagnosis and treatment of AIS on the overall physical and mental well-being of subjects over time, 2) determine the relationship between bracing dose (wear time) and curve progression and 3) develop a predictive model for curve progression based on patient characteristics at their initial presentation and after bracing or watchful waiting.

Dual Growing Rod Instrumentation with Limited Fusion for the Treatment of Early Onset Scoliosis

This multi-center project is sponsored by the Growing Spine Study Group (GSSG). Initially a retrospective study, it has become a prospective observational study with 14 clinical centers from around the world contributing data. The main goal is to determine how successful dual growing rods and Vertical Expandable Prosthetic Titanium Rib (VEPTR™) devices are in correcting progressive scoliosis in very young children.

Secondarily, researchers want to know if children who undergo successful surgical intervention with these devices, to control their curve during growth, need to go on to receive a definitive final fusion, or if the hardware can be removed and the curve simply observed over time for possible progression.

Recently, the study was modified to include a non-operative cohort of patients who are treated for early onset scoliosis with non-surgical methods, such as bracing and casting. Outcomes associated with operative and non-operative treatment will be compared in hopes that the most effective methods of correction can be identified.

Skeletal Complications in Neurofibromatosis Type 1 (NF1)

This study is being conducted by researchers at Boston Children's Hospital and Beth Israel Deaconess Medical Center. The goal is to identify cell types associated with and responsible for skeletal defects and impaired bone healing associated with NF1.

A large proportion of patients with NF1 display skeletal abnormalities such as alterations in bone size and shape, the presence of scoliosis, and a tendency to develop pseudoarthrosis. Tissue samples from patients with and without NF1 will be analyzed by microscopic examination. We hope the results will lead to a better understanding of the cells predominantly responsible for skeletal defects in NF1 and lead to new strategies for treating this patient population.

Ongoing laboratory studies include:

•   Basic science studies
•   Biomechanical/instrumentation studies

Orthopedic basic science laboratories

Working in our labs are some of the leading musculoskeletal researchers in the nation. These labs include:

•   Orthopedic basic science research
•   Center for the study of genetic skeletal disorders
•   Sports Medicine research laboratory
•   Matthew Harris laboratory

National study on surgical outcomes

Boston Children's Hospital is one of a select group of hospitals involved in a prospective national study to determine the surgical outcomes of patients with complex spinal deformities, including idiopathic scoliosis, early onset infantile scoliosis, kyphosis and spondylolisthesis. The Division of Spinal Surgery is actively involved in the development of non-operative, minimally-invasive and non-fusion techniques for treatment of spinal deformity.