Pectus Study Update
A Multicenter Study of the Clinical Outcomes of the Nuss and Ravitch Procedures for Repair of Pediatric Pectus Excavatum
Robert E. Kelly, MD, Principal Investigator
This is a prospective observational study with detailed data collection pre, peri and post- operatively on patients who have already decided to have surgery for pectus excavatum with the participating surgeon. Randomization is not appropriate since patients usually come with preconceived ideas of which surgery is preferable and surgeons should be free to do the procedure they believe they do best. A major focus of the study is to document outcomes of repair regardless of procedure.
Each patient clinically judged to have a severe pectus will undergo a CT scan of the chest according to a standard protocol. If the clinical impression is confirmed radiographically, further evaluation will proceed. Each patient will be assessed by a pediatric pulmonologist according to a standard protocol. If repair is deemed clinically indicated after all evaluations, the patient will be scheduled for operative repair by the method of choice of that patient and surgeon. Demographic and medical information will be recorded in a database by a research coordinator specifically designated at each center to be responsible for data collection and entry (PRC). The written consenting process for participation in the rest of the study will take place at the first visit with the surgeon.
Before surgery and one year post-op, each patient and their parent will be contacted by phone and asked to complete a brief questionnaire designed to assess body image. After surgery, data regarding pain management will be collected and submitted to the database during the immediate post-operative period. An additional pain assessment will be mailed to patients and their parents 30 days after their surgery. Patients will be followed via phone, mail, and/or clinical visit at yearly intervals on or near the anniversary of their surgery for a period of up to 5 years. The surgical, research, and consulting physician teams will assess and administer clinical exam, body image questionnaires and pulmonary function studies.
One year after treatment is completed, the patient will undergo CT scan of the chest and pulmonary function tests to assess results. CT scans will be scored according to percent change from the pre-operative scan. This will constitute the main outcome of the study in terms of clinical effectiveness of the two procedures.
As discussed at the May 19, 2001 APSA Meeting regarding the Pectus Study, surgical centers that participate in the study must do a minimum of 5 pectus excavatum repairs/year and surgeons must have performed a minimum of 20 surgeries in order to eliminate the learning curve. For this reason, as well as because of the extensive data collection involved, this trial cannot have open enrollment. In addition, the extensive data collection requires intense training for the Research Coordinators at each site.
Currently there are ten sites in addition to Children’s Hospital of The King’s Daughters enrolling subjects in the study and one site whose contract is in process. Current enrollment is 307 with a goal of 360. An addendum to the study, a multicenter comparison of cardio-pulmonary function before and after surgical repair of pectus excavatum, was initiated December 2003 at sites capable of performing progressive exercise testing. A subset of 64 subjects participating in the main study will be enrolled in the exercise portion of the study. A total of nine subjects are participating in the exercise substudy as of September 2004. Dr. Robert Mellins of Columbia University serves as the study pulmonologist.
Enrollment as of September 2004 is summarized below:
|All Children's Hospital, St. Petersburg, FL (André Hebra, MD)
|Children's Hospital Boston (Robert Shamberger, MD)
|Children's Hospital for The King's Daughters (Robert Kelly, MD)
|Children's Mercy Hospital, Kansas City, MO (Ronald Sharp, MD)
|Children’s Hospital of Wisconsin (Keith Oldham, MD)
|Cincinnati Children's Hospital Medical Center (Richard Azizkhan, MD)
|Kapio’lani Medical Center for Women and Children (Walton Shim, MD)
|The Hospital for Sick Children (Annie Fecteau, MDCM)
|Children’s Medical Center of Dallas (Steve Megison, MD)
|Yale New Haven Children’s Hospital (Lawrence Moss, MD)
|Johns Hopkins (Paul Colombani, MD)
|Children’s Hospital of New York-Presbyterian (Charles Stolar, MD)
For more information, please contact Dr. Robert Kelly at (757) 668-7703 or [email protected] or the study coordinator:
Karen Mitchell, BSN, CCRC
Clinical Research Coordinator, Pectus Multicenter Trial
Children's Hospital of The King's Daughters
Toll free # (866) 279-6430
Email: [email protected]