Publications by authors named "Raffini L"

Article Synopsis
  • The study investigates the effectiveness of enoxaparin, a medication for preventing blood clots, in critically ill children, focusing on differences in response based on age, particularly between older children and infants.
  • It consists of two simultaneous clinical trials: one for older children aged 1-17 years, and another for infants under 1 year, with specific dosing protocols for each group.
  • The trials are set in multiple pediatric intensive care units across the U.S., using methods like randomization, ultrasonography, and blood analysis to assess outcomes and monitor safety.
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Objectives: To derive systematic review informed, modified Delphi consensus regarding monitoring and replacement of specific coagulation factors during pediatric extracorporeal membrane oxygenation (ECMO) support for the Pediatric ECMO Anticoagulation CollaborativE.

Data Sources: A structured literature search was performed using PubMed, Embase, and Cochrane Library (CENTRAL) databases from January 1988 to May 2020, with an update in May 2021.

Study Selection: Included studies assessed monitoring and replacement of antithrombin, fibrinogen, and von Willebrand factor in pediatric ECMO support.

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Objectives: To present recommendations and consensus statements with supporting literature for the clinical management of neonates and children supported with extracorporeal membrane oxygenation (ECMO) from the Pediatric ECMO Anticoagulation CollaborativE (PEACE) consensus conference.

Data Sources: Systematic review was performed using PubMed, Embase, and Cochrane Library (CENTRAL) databases from January 1988 to May 2021, followed by serial meetings of international, interprofessional experts in the management ECMO for critically ill children.

Study Selection: The management of ECMO anticoagulation for critically ill children.

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Article Synopsis
  • Pediatric pulmonary embolism (PE) is uncommon but serious, with a study analyzing treatment methods and outcomes in U.S. pediatric hospitals from 2015 to 2021.
  • Approximately 88% of pediatric patients received anticoagulation alone for PE, while treatments like systemic thrombolysis and directed therapy were less common; directed therapy showed a lower mortality risk.
  • Additional research is needed to better understand pediatric PE due to the high occurrence of comorbidities in affected patients and the limitations of current treatment practices.
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Background: Direct oral anticoagulants are commonly prescribed for adults and increasingly also for children requiring anticoagulation therapy. While household medications should not be accessible to children, accidental, and intentional overdoses occur.

Key Clinical Question: How should apixaban overdose in children be managed?.

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Background: Although children aged <1 year have a relatively high rate of venous thromboembolism (VTE) compared to older children, most have additional prothrombotic risk factors. Unprovoked VTE is rare, and little is known about this population, particularly the risk of recurrent VTE.

Objectives: We aimed to determine the rate of recurrent VTE in infants with prior unprovoked VTE and evaluate long-term, end-organ outcomes for infants with renal and intracranial vein thrombosis.

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Coronavirus disease 2019 (COVID-19) associated coagulopathy is multifactorial and involves inflammation driven hypercoagulability, endothelial dysfunction, platelet activation, and impaired fibrinolysis. Hospitalized adults with COVID-19 are at an increased risk of both venous thromboembolism and ischemic stroke, resulting in adverse outcomes, including increased mortality. Although COVID-19 in children follows a less severe course, both arterial and venous thromboses have been reported in hospitalized children with COVID-19.

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Article Synopsis
  • Pediatric hematologists in the US have been using direct oral anticoagulants (DOACs) off-label for treating venous thromboembolism (VTE) in kids and teens before official FDA approval in 2021.
  • The ATHN 15 study analyzed the use of DOACs from 2015 to 2021 across 15 specialized pediatric centers, focusing on their safety and effectiveness in patients aged 0 to 21 years.
  • Results indicated that rivaroxaban was the most commonly prescribed DOAC, with a bleeding complication rate of 13.8% and a recurrence rate of 4%, showing that DOACs have an adequate safety and effectiveness profile for young patients.
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Despite the growing number of pediatric antithrombotic clinical trials, standardized safety and efficacy outcome definitions for pediatric venous thromboembolism (VTE) clinical trials have not been updated since 2011. Many recent trials have adapted the recommended definitions, leading to heterogeneity in outcomes and limiting our ability to compare studies. The International Society on Thrombosis and Haemostasis Scientific and Standardization Subcommittee (SSC) on Pediatric and Neonatal Thrombosis and Hemostasis organized a Task Force to update the efficacy and safety outcome definitions for pediatric VTE clinical trials.

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Background: The National Hemophilia Foundation (NHF) conducted extensive, inclusive community consultations to guide prioritization of research in coming decades in alignment with its mission to find cures and address and prevent complications enabling people and families with blood disorders to thrive.

Research Design And Methods: With the American Thrombosis and Hemostasis Network, NHF recruited multidisciplinary expert working groups (WG) to distill the community-identified priorities into concrete research questions and score their feasibility, impact, and risk. WG6 was charged with identifying the infrastructure, workforce development, and funding and resources to facilitate the prioritized research.

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Article Synopsis
  • Oral anticoagulation therapy has advanced from traditional vitamin K antagonists to newer options like direct thrombin inhibitors and factor Xa inhibitors, collectively known as direct oral anticoagulants (DOACs), which are now standard for treating conditions such as atrial fibrillation and venous thromboembolism.
  • New medications targeting factors XI/XIa and XII/XIIa are being researched for various thrombotic and nonthrombotic conditions, potentially offering different benefits and risks compared to current DOACs.
  • The International Society on Thrombosis and Haemostasis has suggested a new naming system for anticoagulants based on their administration route and target, aiding clarity in their clinical use.
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Background: Unprovoked venous thromboembolism (VTE) is rare in pediatrics. Current recommendations for anticoagulation duration after unprovoked VTE differ for pediatric and adult populations.

Objectives: This single-center, retrospective cohort study aimed to determine the incidence rate of recurrent VTE in children and adolescents with unprovoked VTE, evaluate the potential risk factors for recurrence, and describe the anticoagulation regimens and bleeding in this population.

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The outcomes and characteristics of acquired thrombotic thrombocytopenic purpura (TTP) in adolescents is poorly understood due to an absence of studies focused on this population. To better understand the life-threatening disorder in this age, we performed an analysis of adolescent patients (ages 10-21) with TTP in the Pediatric Health Information Systems database from 2009 to 2020. The primary outcomes evaluated were in-hospital mortality and rate of TTP relapse.

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Caring for children and adolescents with disorders of hemostasis and thrombosis (HAT) has become more specialized and requires a unique skill set that many providers are not able to obtain in standard pediatric hematology/oncology/bone marrow transplant fellowship training programs. The influx of numerous therapeutic advances and increasing medical complexity has expanded the need for experienced HAT providers and subspecialty collaboration in the inpatient setting due to the nuances in the management of patients with HAT complications and concerns. While there are data highlighting the benefits of an inpatient hemostasis, thrombosis, and anticoagulation management service in adult hospitals, there are limited pediatric data supporting such programs.

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Purpose Of Review: To provide an update regarding what is known about thrombotic events and thromboprophylaxis in critically ill children with SARS-CoV-2 infection.

Recent Findings: Pediatric patients with SARS-CoV-2 generally have mild illness; however, intensive care is required in about 20-30% of hospitalized children with COVID-19 and an even higher proportion in those with MIS-C. Increased rates of thrombosis have been observed in adults hospitalized with COVID-19, and clinical trials have attempted to optimize thromboprophylaxis.

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Article Synopsis
  • * Recent clinical trials and studies suggest that thromboprophylaxis strategies for COVID-19 patients should be tailored to individual needs rather than applying a one-size-fits-all approach.
  • * The updated guidance from the Anticoagulation Forum covers thromboprophylaxis for patients in different care settings and addresses concerns for those with existing thrombotic conditions regarding COVID-19 vaccination.
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Article Synopsis
  • Direct oral anticoagulants (DOACs) are becoming increasingly viable options for treating and preventing blood clots in children, thanks to new trials and the recent approval of drugs like dabigatran and rivaroxaban for pediatric use.
  • Despite the growing adult data, there is still a lack of concrete real-world evidence on how well these drugs work for children who don’t fit the profiles used in clinical trials, raising concerns about their use in certain cases.
  • The review highlights the specific data relevant to pediatrics, identifies areas needing more research, and discusses important practical considerations for effectively using DOACs in pediatric patients.
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Objectives: In 2009, a large multicenter study demonstrated that the rate of pediatric venous thromboembolism (VTE) across US children's hospitals had significantly increased from 2001 to 2007. The objective of this study was to evaluate the rate of pediatric VTE from 2008 to 2019 using similar methodology.

Methods: A retrospective cohort study using the Pediatric Health Information System (PHIS) database.

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Central venous catheters (CVC) are the most significant risk factor for pediatric venous thromboembolism (VTE). After an index CVC-associated VTE (CVC-VTE), the role of secondary prophylaxis for subsequent CVC placement is uncertain. Aims of this single-center retrospective study were to evaluate the efficacy of secondary prophylaxis for patients with a prior CVC-VTE and identify risk factors associated with recurrent VTE in patients less than 19 years with an index CVC-VTE between 2003 and 2013.

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Introduction: High collision-risk physical activity can increase bleeding risk in people with hemophilia A, as can increasing the time between factor VIII (FVIII) administration and physical activity. FVIII prophylaxis may be tailored to planned activities to prevent activity-related bleeding.

Aim: To explore the relationship between physical activity levels, FVIII infusion timing, and occurrence of bleeding in patients with severe/moderately severe hemophilia A without FVIII inhibitors receiving antihemophilic factor (recombinant) (rAHF; ADVATE; Baxalta US Inc.

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Article Synopsis
  • Ichthyosis follicularis, atrichia, and photophobia syndrome (IFAP) is a rare X-linked disorder caused by changes in the MBTPS2 gene, leading to various health issues.
  • Researchers presented a case of a patient with multiple complications including skin and hair issues, heart and kidney problems, and chronic diarrhea, linked to a novel MBTPS2 variant identified through exome sequencing.
  • The study demonstrated that the identified variant disrupts normal cellular functions, contributing to the spectrum of symptoms associated with BRESHECK syndrome, including blood issues and bone marrow fibrosis.
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