HJAR Nov/Dec 2021

46 NOV / DEC 2021 I  HEALTHCARE JOURNAL OF ARKANSAS CHILDREN’S HEALTH gical and medical treatment and has the availability to participate in clinical trials. Patient advocate groups are also essential in the multidisciplinary approach as they can provide resources, outreach and guid- ance to patients looking for a program. To treat a patient with a vascular anomaly, having a teamwho can diagnose, treat and contribute to research is crucial in provid- ing care and producing great outcomes. n Joana M. Mack, MD, is an assistant professor at the University ofArkansas for Medical Sciences (UAMS) practicing at Arkansas Children’s Hospital (ACH). She is a pediatric hematologist-oncologist and joined the group in 2018 after completing a residency and fellowship training at Arkansas Children’s Hospital. She is also the Pediatric Hematology-Oncology Fel- lowship program director and is actively involved in educating trainees.She is amember of theVascular Anomalies Center of Excellence and provides care for these complex patients.Mack leads several research opportunities in the treatment of patients with Vas- cular Anomalies. Gresham T. Richter, MD, FACS, is a head and neck surgeonwho spendsmuch of his clinical time treating children with vascular anomalies and head and neck tumors. He is the chief of Pediatric Otolaryngology, Vascular Anomalies Clinic and research director at UAMS and ACH. He is a professor and vice chair in the UAMSCollege of Medicine and holds the Hamlen and Siebert Endowed Chair in Pediatric Otolaryngol- ogy at ACH. Richter is the founder and director of the Arkansas Vascular Biology Program, a research center dedicated to investigating normal and abnor- mal vascular development at Arkansas Children’s Research Institute. observation, or extremely complex, need- ing the tools and expertise of several spe- cialists. Most require staged multimodal and multidisciplinary care. Skin malformations and hemangiomas can be lasered. Vascular anomalies and their complications may require surgical intervention by otolaryngologists, ortho- pedic surgeons and/or general surgeons, based upon the anatomic location. Deep vascular malformations of the veins, ar- teries or lymphatic vessels benefit from interventional radiologists for sclero- therapy, a procedure that causes endo- thelial damage and fibrosis of the vessel. Other patients may need a hematologist due to bleeding and thrombotic issues or oncologic support for medical therapy. Involvement of the genitourinary region may need a gynecologist, especially for family planning. Overgrowth syndromes benefit from genetics, oncology and or- thopedics. Arkansas Children’s team has discov- ered that patients benefit most from being seen and followed by a vascular anoma- lies multidisciplinary group who is knowl- edgeable about the most up-to-date sur- TARGETED THERAPY Rapamycin (Sirolimus), anmTOR inhib- itor, was the first targeted therapy used in vascular malformations with overgrowth syndrome. Several studies have shown that it can prevent further growth and, in some instances, decrease the size of the vascular malformation. However, its in- hibitive properties are further down the pathway, allowing several elusive feed- back pathways to continue activation and possible growth. Alpelisib, a PIK3CA inhibitor, shows promise in patients with PIK3CA-related overgrowth syndromes (PROS). A study that included 19 patients with PROS showed a decrease in tumor size and improvement in symptoms in all patients. Several therapies are making their way down the pipeline, and this ge- netic era and targeted therapy is exploding for vascular anomalies. There are several clinical trials exploring new pathways to treat vascular malformations with medi- cines at Arkansas Children’s Hospital. MULTIDISCIPLINARY APPROACH Every vascular anomaly is unique. The anomaly can be simple, requiring active “Every vascular anomaly is unique. The anomaly can be simple, requiring active observation, or extremely complex, needing the tools and expertise of several specialists. Most require staged multimodal and multidisciplinary care.”