(285) Real-world Data Evaluating Safety, Effectiveness and Dosing of Dalteparin for the Treatment of Venous Thromboembolism (VTE) in Neonates Aged 0 to 28 days
Background: In May 2019, Fragmin (dalteparin sodium, administered subcutaneously) became the first FDA-approved therapy to treat Venous Thromboembolism (VTE) in pediatric patients aged ≥ 1 month. As a commitment to the FDA, a non-interventional study using real-world data was conducted to examine use of dalteparin among neonates treated with dalteparin sodium for VTE.
Objectives: To characterize safety and effectiveness and dosing of dalteparin sodium among neonates (≤ 28 days old, and ≥ 35 weeks gestation) treated with dalteparin for VTE in real-world settings.
Methods: The study utilized data from medical records (2010-2021) at pediatric hospitals in the UK. Data on dalteparin sodium use, safety (including major and minor bleeding events), effectiveness outcomes and dalteparin sodium dosing were collected.
Results: A total of 16 patients were included. Mean age of patients at first dalteparin use was 14.5 days (±5.9 SD), female 56.3%, White 81.3%. Fifteen (93.8%) patients had at least one comorbid condition or non-drug allergy; most common comorbid condition was sepsis, followed by hyperinsulinism. No patient experienced a bleeding event during treatment with dalteparin sodium and through follow up which ended 28 days after the last dose of dalteparin, death, lost to follow-up, end of study period (i.e., 31 December 2021), whichever occurred first. One patient with a history of documented hyperinsulinism, experienced a serious event of hypoglycemic brain injury. Four patients had documented resolution of VTE. The mean (±SD) daily dose of dalteparin at initiation was 364.88±196.39 IU/kg, the dose including dose at initiation, changes and restarts was 575.13±320.43 IU/kg. The mean (±SD) duration of dalteparin treatment was 62.44±30.04 days, all 16 patients experienced a dose or frequency change and then permanently discontinued treatment.
Conclusions: Overall, no safety concerns were identified in this real-world data study. The data obtained from the medical charts were insufficient to assess the effectiveness of dalteparin. Study limitations such as potential for inaccuracies and limited generalizability should be considered when interpreting the data given that this was a small non-interventional study using retrospective data.
