What is effective pediatric surgical research?
Pediatric surgical research tackles with unique challenges. Pediatric surgery encompasses a few common childhood diseases and a surplus of very rare complex congenital anomalies. Practically all of them fulfil the definition of rare disease and most carry significant long-term sequela. In addition to surgery, which sometimes starts before the child is born and may continue in adulthood, successful treatment of developmental defects requires continuous input from a multitude of other specialties not only during childhood but also after transition to adult care. In most patients conclusive assessment of surgical outcomes is not possible before adulthood. (Read more)
There are efficient ways to overcome these obstacles and acquire robust evidence-based data to guide surgical care of children. Multicentre research collaboration, concentration of care and subspecialisation has benefitted patients with biliary atresia and necrotising enterocolitis by providing multicentre databases, enabling randomized control trials and advancing quality of care. Other complex neonatal surgical conditions will soon follow the suit to gain real advancement in their outcomes. Ongoing randomized controlled trials on novel medical therapies conducted by multidisciplinary group of investigators in collaborative centres have genuine possibility to augment surgical outcomes in patients with intestinal failure, biliary atresia and necrotising enterocolitis for the first time, and others will come. Importance of universal patient data bases with routine biobanking is highlighted in those congenital conditions, where surgery alone is not able to restore physiological organ function. Better understanding of the genetic disease pathophysiology and accurate genotype-phenotype correlation could allow individually tailored surgical treatment and establish basis for novel cell-replacement therapies. Research collaboration with adult physicians has started to unravel best management of long-term medical consequences of developmental defects.
At international level, recent establishment of European rare disease networks has provided a platform to pursue these targets. Hopefully, national decision makers will strengthen current development as well, when reorganising Finnish healthcare in near future. Besides economical input and pushing for multicentre collaborative research activity, European rare disease networks have brought patients amongst researchers, which will undoubtedly increase validity of clinical trials. It seems quite bizarre already that we have studied surgical outcomes for decades without asking the patients themselves what they regard essential. After all, it’s all about ourselves and our willingness to co-operate to perform effective pediatric surgical research and achieve the best possible outcomes for our patients – they deserve it.