Systemic anticoagulation is regarded as optimal treatment and prophylaxis of venous thromboembolism (VTE). In malignancy, bleeding risk is increased while the patients remain hypercoagulable, making anticoagulation management troublesome. Inferior vena cava (IVC) filters have emerged as an option in the management of VTE, especially when anticoagulant agents are contraindicated. There is limited data on the overall outcomes of patients with malignancy and IVC filter placement. This descriptive study identifies individuals with filters placed and reviews outcomes to guide appropriate care of patients with malignancy and VTE. We performed a retrospective chart review of 115 patients with malignancy who had a filter placed between July 2014 and December 2016. Eighty-seven patients were tracked until December 2017 for significant events (VTE and/or death). In total, 61% (n = 70) had metastatic solid tumor malignancy and 77% (n = 88) were receiving anticoagulation therapy prior to IVC filter placement. Fifty-three percent (n = 61) had bleeding events and 25% (n = 29) had thrombocytopenia. Patients with isolated solid tumors receiving frequent surgery were also common recipients of filters. Sixty-six percent (57/87) of patients had a significant event; 85% of them were anticoagulated. Eighty-two percent of events occurred within 6 months of filter placement, with death occurring on average within 5 months of placement. Overall, use of IVC filters was more common in cancer patients who developed bleeding complications on anticoagulation and with metastatic malignancy. However, in patients with metastatic or hematologic disease, filter placement did not prevent all-cause mortality. Individualized risk–benefit consideration is needed before IVC filters are placed.
Objective:
The goal of this report is to describe, through a series of 5 cases, the clinical response and safety of alpelisib (BYL719) use in children and adults with
PIK3CA
-related overgrowth spectrum (PROS) disorders at our center.
Methods:
We reviewed clinical records of 5 patients from October 2019 through September 2021 followed by the pediatric hematology and multidisciplinary vascular anomalies teams at the Monroe Carell Jr. Children’s Hospital at Vanderbilt (MCJCHV). All patients carried a clinical or genetic diagnosis of PROS and were treated with alpelisib provided by a Novartis managed access program.
Results:
We highlight improvement in reported symptoms, objective overgrowth measurements, and quality of life to varying degrees in all patients. We note dose-dependent hyperglycemia and gastrointestinal side effects in 2 of the 5 patients. No patients experienced any serious side effects.
Conclusion:
This case series reports on the real-world use of PI3K-α inhibition in the management of PROS. Ongoing clinical trials will provide efficacy and safety data as these drugs become more widely used in patients with vascular anomalies and syndromes secondary to somatic
PIK3CA
mutations.
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