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Strong results reported for transcatheter approach in children with certain congenital defects
Among a cohort of children, surgical and transcatheter treatment of perimembranous ventricular septal defects, one of the most common subtypes of congenital heart disease, yielded excellent midterm outcomes in terms of mortality and major complications.
However, study results also showed that several other endpoints, including minor adverse events and quantity of blood transfused, were significantly improved in the transcatheter arm, leading researchers to conclude that transcatheter device closure is the treatment of choice in these patients.
Investigators from several sites in the United States and China conducted the prospective, randomized controlled trial to determine whether the surgical or transcatheter approach was safer and more effective in correcting perimembranous ventricular septal defects in a cohort of 229 children. Although surgical closure is an established treatment, open-heart surgery with cardiopulmonary bypass is an invasive procedure, according to the researchers. The transcatheter approach, although associated with high incidence rates of complete atrioventricular block, may be attractive because of its less invasive nature.
Participants were aged 3 to 12 years and were randomly assigned to one of the two approaches between January 2009 and July 2010. The investigators compared clinical, laboratory, procedural and follow-up data during a 2-year period.
The final analysis included 101 patients (mean age, 5.5 years) in the transcatheter group and 99 patients (mean age, 5.8 years) in the surgery group.
There was no mortality or major complications in either group.
There were 32 minor adverse events (32.3%) in the surgery group compared with seven (6.9%) in the transcatheter group (P<.001).
Additional data also indicated that the transcatheter group had fewer transfusions, shorter hospital stays, reduced medical costs and faster recovery times.
The left ventricular end diastolic dimension returned to normal in both groups during the 2 years of follow-up.
Closure rates, adverse events and complications were similar between the two groups.
“Transcatheter device closure and surgical repair are effective interventions with excellent midterm results for treating [perimembranous ventricular septal defects] in children,” the researchers concluded, adding that as a result of the advantages observed in the transcatheter group, transcatheter device closure is the treatment of choice for perimembranous ventricular septal defects in children in this cohort.
Disclosure: The researchers report no relevant financial disclosures.
Perspective
Back to TopThis manuscript represents a true landmark study in the field of pediatric cardiology and the treatment of congenital heart defects. It is without doubt the first-ever truly prospective randomized controlled trial (RCT) directly comparing a surgical treatment modality to a percutaneous device treatment for a common form of congenital heart defect.
Going all the way back to the beginnings of treating congenital heart defects in 1938 when Dr. Robert E. Gross first performed a successful PDA ligation and then in 1945 when he performed the first surgical repair of an aortic coarctation, there has never been a successfully completed RCT comparing surgical vs. percutaneous device treatment of a single form of congenital heart disease.
The reasons for this lack of prospective RCTs in our field is complex, but includes factors such as a historical reluctance on the part of the congenital heart surgery community to endorse or support such direct comparison studies combined with the inherent reluctance of patients (at least in the United States) to proffer their children for RCTs in particular when they discover that there is a ‘reasonably safe’ nonsurgical alternative in the randomization mix. Example: the phase 2a IDE study of the Amplatzer Atrial Septal Occluder, which failed due to parental refusal to cooperate with the 1:1 randomization scheme which lead to the reformatting of the study as the phase 2b IDE with a change to a concurrent surgical control group.
The percutaneous device closure arm of the study shows clear superiority to the surgical arm in almost every clinically relevant endpoint. Despite what must have been a remarkable enrollment challenge to get more than 200 sets of parents to agree to enroll their children in a study where they had a 50:50 chance of either having to suffer through a median sternotomy, cardiopulmonary bypass (CPB) and the ICU recovery period, or not, the researchers were able to carry out and complete this RCT treatment comparison in an extremely well-designed trial. Simply put, the results reported reveal that the results were far from comparable for the two groups. Not only did the children randomized to the percutaneous device closure arm and treated with what turned out to be an extremely effective peri-membranous VSD occluder (pmVSDO), they were able to avoid a median sternotomy, but, most importantly, exposure to CPB and significant blood transfusions. The acute results demonstrated in remarkable fashion that the percutaneous pmVSDO closure device was able to close defects as successfully as surgery with respect to closure efficacy and mortality rates; however, there is so much more to the comparison than these relatively coarse endpoint measures. It is particularly true in the practice of taking care of children with congenital heart disease not to underemphasize the importance of reducing treatment risks other than simply mortality. Reduction of potential central nervous system injury, long-term developmental impairment, suffering and quality of life are extremely relevant to the long-term health of children. The pmVSDO group did in fact have a lower major adverse event rate than surgery, although due to the low event rates it was not statistically significant; yet it is certainly relevant when it is a children that has to go back to the operating room for more surgery, CPB and blood loss. On this point, I’m sure the child’s parents would agree.
It should therefore be strongly emphasized that the pmVSDO group had significantly lower rates for almost all other clinically relevant outcome measures such as need for blood transfusion, residual shunt at discharge, new or increased valvular regurgitation, duration of the procedure, median length of hospital stay, median ICU stay, median hospitalization cost and median blood loss. Furthermore, the study showed that at 2 years after the treatment in either arm, the curves for closure rate, adverse events or other late complications did not change (nor converge), therefore maintaining the advantages for the percutaneous pmVSDO group over time.
The time has come, after nearly 75 years of treating children with various forms of congenital heart defects, to acknowledge with deliberate enthusiasm that a first-of-its-kind, properly designed and conducted, prospective RCT comparison of two different treatment approaches with radically differing levels of physiologic stressors in children has revealed unequivocally that the percutaneous device option comes up, hands down, an overall superior treatment option going forward.
Finally, going forward, our field needs to acknowledge the dearth of RCT data particularly as it relates to other forms of congenital heart defects where there are both surgical and percutaneous treatment options, and carefully consider what treatments we recommend in our own practice.