Cleft & Craniofacial Clinical Research
Dr. Skirko has melded his expertise in clinical research with his passion for providing care for children with cleft and craniofacial disorders. The combination of clinician and researcher allows for evidence-based care alongside cutting-edge research that is life-changing for families. As a Cleft Surgeon, he works with families to choose the best treatment plan for their child based on leading research. As a clinical researcher, he uses his observations and clinical insights to help design research that is important to improving the care of children with cleft and craniofacial disorders.
Dr. Skirko leads a team of researchers at the University of Arizona where his research is funded by the NIH, PCORI, as well as other foundations. Dr. Skirko also leads collaborative research with a consortium of cleft and craniofacial centers across the country. Creating and building a multisite consortium of cleft centers is critical to advancing our understanding of rare cleft and craniofacial disorders so that we can provide the best care to our patients.
Stakeholder Alliance for Children with Robin Sequence (StARS)
Infants with Pierre Robin Sequence (PRS) are born with a small lower jaw, causing their tongues to be pushed back. This causes them to struggle with breathing and eating because their tongue blocks their airway. They also frequently have cleft palates, causing additional feeding problems. These breathing and feeding problems have a profound impact on the child and caregiver. Despite the impact on families, PRS parents have not been engaged in patient-centered outcomes research (PCOR). Additionally, PRS researchers lack knowledge of PCOR and comparative effectiveness research (CER) methods and skills. The lack of stakeholder engagement among PRS families and the absence of PCOR and CER knowledge among researchers are barriers to improving outcomes for children with this disease.
Dr. Skirko established the Stakeholder Alliance for Children with Robin Sequence (StARS) composed of PRS parents, health systems personnel, clinicians, and researchers. The complexity of PRS children’s care requires multiple clinician types (each with unique perspectives) to be engaged and included in the StARS network. The purpose of this project is to build a PRS community able to participate in PCOR and CER. To achieve this goal, he has engaged key stakeholders in PRS treatment and research across the country.
During the project, stakeholders have been engaged through a series of educational and engaging webinars and annual meetings. These activities provide education regarding PCOR and work to identify outcomes desired by PRS families, PCOR priorities, and barriers to engaging in PCOR. In addition, stakeholders have worked collaboratively to develop a priority list for future patient-centered outcomes research.
The StARS network is positioned to undertake future PCOR projects in the field of PRS treatment. Our long-term goal is to design and implement studies and trials that answer PRS parent-/caregiver-derived questions to achieve better patient outcomes. This work has created a consortium of sites that are able and together design innovative research. This is critical to improve our understanding of how PRS impacts families as well as how to optimize their care.
Robin Sequence Quality of Life Project
Children with Pierre Robin Sequence (PRS) have breathing and feeding difficulties of varying degrees, which can profoundly affect their own and their families’ quality of life (QoL). Many infants with PRS have complicated and tenuous early lives requiring surgeries such as tracheostomy to bypass upper airway obstruction, surgical feeding tubes for nutrition, or major surgeries to improve their craniofacial anomaly. In addition to the child’s symptoms, parents often struggle to manage their own psychosocial and emotional response to their child’s disorder.
Current QoL instruments are too broad and don’t adequately measure QoL in this population. This not only limits understanding of these patients’ holistic disease burden, but it also constrains future comparative effectiveness studies to outcomes that may be less relevant to patients and families.
Our team is developing and testing a PRS-specific QoL instrument measuring both child symptoms and family QoL. We have worked closely with families to develop content based on their experiences. We have refined the instrument and are testing the instrument to understand how it functions.
Our new validated QoL instrument will be a comprehensive measure of disease burden allowing providers and clinical researchers to measure the impact of PRS on children and families. This novel measurement will advance our understanding of the impact this disorder has on families, representing a substantial shift from previous assessments in this vulnerable population. It will also facilitate critically needed future studies comparing PRS treatments.
VPI and Quality of Life – The VELO Instrument
Cleft lip and cleft palate are common birth defects. These conditions can have a substantial impact on the affected children, including altering their speech and increasing their risk of developing velopharyngeal insufficiency (VPI). VPI is characterized by hypernasal speech, nasal air emission, and nasal reflux during feeding.
The quality of life (QoL) in this population can also be greatly impacted due to intelligibility related to VPI. Measuring QoL is very important in this population. Previous patient-centered metrics are limited globally, despite their importance, and metrics that assess broader speech issues are not designed to measure VPI or hypernasality.
In order to measure the impact that VPI has on children’s lives, Dr. Skirko and his colleagues developed the VPI Effects on Life Outcomes (VELO) instrument. This instrument measures patient-reported outcomes consisting of both parent and child reports and focuses on how the symptoms of VPI affect the quality of life. Measuring quality of life is crucial to improving patients QoL. The VELO is used at cleft and craniofacial centers around the country.
The importance of this instrument has also resorted in being translated and validated in eight different languages as well as numerous other active projects around the world. Current translations include Chinese (mandarin), Dutch, French, Farsi, Italian, Napales, Norwegian, Portuguese, and Spanish (two dialects). Dr. Skirko has become an expert in translation and linguistic validation and works with providers and researchers around the globe.
The VELO instrument has become a crucial component of clinical management of children with cleft lip and palate as well as the leading measure for clinical research. The VELO instrument provides a metric that allows accurate assessment of VPI related quality of life.