Background and Objectives: Kaposiform hemangioendothelioma (KHE) and tufted angioma (TA) are rare vascular tumors in children historically associated with significant morbidity and mortality. This study was conducted to determine first line therapy in the absence of available prospective clinical trials. Methods: Patients from 17 institutions diagnosed with KHE/TA between 2005-2020 with > 6 months follow-up were included. Response rates to sirolimus and vincristine were compared at 3 and 6 months. Durability of response and response to other treatment modalities were also evaluated. Results: Of 159 unique KHE/TA subjects, Kasabach-Merritt phenomenon (KMP) was present in 64 (40.3%) and only 2 patients were deceased (1.3%). Over 60% (n=96) demonstrated treatment response at 3 months and >70% (n=114) by 6 months (no significant difference across groups). The vincristine group had higher radiologic response at 3 months compared to sirolimus (72.7% vs 20%, p=0.03) but there was no differences between these groups at 6 months. There were no differences in rates of recurrent or progressive disease between vincristine and sirolimus. Conclusions: In this large, multicenter cohort of 159 patients with KHE/TA, rates of KMP were consistent with historical literature but the mortality rate (1.3%) was much lower. Overall treatment response rates were high (>70%) and there were no significant difference in treatment response or durability of disease comparing sirolimus to vincristine. Our results support individualized treatment decision plans depending on clinical scenario and patient/physician preferences. Response criteria and response rates reported here will be useful for guiding future treatment protocols for vascular tumors.

Some vascular anomalies can present with challenging hematologic aberrations. Kaposiform hemangioendothelioma (KHE) may be complicated with Kasabach-Merritt phenomenon (KMP) and stagnant blood flow in slow flow malformations can promote activation and consumption of coagulation factors which results in bleeding and clotting known as localized intravascular coagulopathy (LIC). These patients can experience significant morbidity secondary to pain due to thrombosis and are at higher risk of hematologic complications during surgical procedures. No standard of care has been established to prevent or manage these complications. This review focuses on the management of coagulopathy in children and adults with vascular anomalies.

Background/Objectives: Pupil size and reactivity have been studied in adult and pediatric populations to objectively measure pain which showed that pupillometry is a promising biomarker for pain measurement. Vaso-occlusive crisis, is a common complication of sickle cell disease (SCD) and better assessment tools are needed. The objective of this study is to establish normative values for pupil size and reactivity in pediatric patients with SCD with the hope that pupillometry can be used as a tool to objectively measure pain and response to treatment. Design/Methods: Institutional review board (IRB) approved prospective study enrolled 82 patients with SCD. Readings were performed using a NeurOptics PLR-2000 pupillometer. The following measures were collected: resting pupil size, % change in pupil size, pupil constriction velocity, and pupil dilation velocity. Results: Forty-four males and 38 females, all black, were studied. Median age was 11 years (range:2-21). The mean minimum and maximum pupil sizes were 3.52mm and 5.08mm, respectively. The mean dilation velocity and maximum constriction velocity were 1.03mm/s and -4.48mm/s, respectively. When comparing our participants with white participants in a previously published pediatric study, there was a significant difference in maximum constriction velocity, maximum pupil size, and minimum pupil size.1 There was no significant difference when comparing means for these parameters. Conclusion: There was no significant difference in pupil size and reactivity between patients with SCD and black patients without the disease. Further investigation of pupillometry within the black population during vaso-occlusive crisis and in the “well state” is warranted in pediatric patients with SCD.