A pediatric perspective on Infantile Hemangioma

Abstract

Infantile hemangiomas (IH) are the most common benign vascular tumors in infancy. Most often IH follow an uncomplicated course without the need for treatment. However a minority of patients experience complications that require a more active approach. A proper diagnosis made by an experienced vascular team of physicians is warranted to predict which infants need treatment. Nowadays beta-blockers are the first-line treatment. The aim of this thesis was to further optimize care and treatment of IH in children. Part I gives an overview of the treatment of IH in historical perspective. The beta-blocker propranolol is an effective and tolerated treatment of hemangiomas of which the safety is still to be elucidated. The studies described in the first part of this thesis showed that propranolol is an effective treatment in patients with IH but that many patients encounter side effects of this therapy. Atenolol became of interest at our institution based on its alternative working mechanism. It subsequently showed similar efficacy and probably less (severe) side effects in comparison to propranolol. In part II the pediatric perspective on IH treatment guidelines is elaborated. We confirmed that an electrocardiogram is probably of no additional value as baseline assessment in otherwise healthy patients without a cardiac (family) history and a normal heart rate and blood pressure (BP) at the start of beta-blocker treatment. We revealed that dose response effects of beta-blocker therapy on BP were seen during start and follow up. Furthermore we found that side effects and adverse events during propranolol treatment are commonly seen. An overview of therapeutic options for IH in children was presented to guide physicians in finding the most effective and safe treatment and how to monitor each individual patient with IH. Other medications used in the treatment of IH are probably less effective, while information about side effects and long-term outcome is also lacking and precise information from adult pharmaceutical databases is incomplete. Part III shows that vascular lesions have an extensive differential diagnosis and can be associated with syndromes. We sought for PHACES (posterior fossa malformations, hemangiomas, arterial anomalies, cardiac defects, eye anomalies, supraumbilical raphe and/or sternal pit) in children diagnosed with obstructive aortic arch pathology though unfortunately we were not able to recognize a significant association between the cardiovascular defects and PHACES. For a patient with a congenital hemangioma close antenatal evaluation by ultrasound and involvement of a multidisciplinary team warranted a proper diagnosis and treatment plan. Finally we showed that adherence to the current therapeutic guideline for kaposiform hemangioendothelioma has not been studied so far. While most Dutch KHE patients are treated according to the therapeutic guideline, many cases from literature did not reveal outcomes according to the proposed treatment, because different therapies were used.