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1.
Pediatr Blood Cancer ; 69(9): e29811, 2022 09.
Artigo em Inglês | MEDLINE | ID: mdl-35709337

RESUMO

Hemophilia is the deficiency of plasma clotting factor VIII (hemophilia A) or IX (hemophilia B) where management focuses on the prevention and treatment of acute bleeding symptoms and their sequelae. The most concerning risk is for life-threatening bleeding, including intracranial hemorrhage (ICH), which is caused by head trauma. Guidelines exist for the evaluation and management of pediatric head trauma, including the Pediatric Emergency Care Applied Research Network (PECARN) protocol, but limited evidence exists for when hemophilia patients present to the emergency department (ED), specifically with head trauma. Literature is limited regarding ICH and hemophilia, which further supports the culture of uncertainty among providers. The objective of this study is to conduct a retrospective chart review to determine the prevalence and clinical characteristics of ICH, and to describe computed tomography (CT) scan use in hemophilia patients who present to Phoenix Children's Hospital (PCH) ED with head trauma from January 1, 2007 to June 1, 2019. A total of 89 ED visits and 43 patients met inclusion criteria, and prevalence of ICH was determined to be 4% with the patients presenting with varied clinical characteristics and few commonalities. Using these data, we propose a new algorithm to aid clinicians in determining the need for CT scan in pediatric hemophilia patients who present to the ED with head trauma.


Assuntos
Traumatismos Craniocerebrais , Hemofilia A , Algoritmos , Criança , Traumatismos Craniocerebrais/complicações , Traumatismos Craniocerebrais/terapia , Serviço Hospitalar de Emergência , Hemofilia A/complicações , Hemofilia A/diagnóstico , Hemofilia A/terapia , Hemorragia/complicações , Humanos , Hemorragias Intracranianas/diagnóstico , Hemorragias Intracranianas/etiologia , Hemorragias Intracranianas/terapia , Estudos Retrospectivos
2.
Biol Blood Marrow Transplant ; 23(4): 707-712, 2017 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-28115278

RESUMO

Sinusoidal obstructive syndrome (SOS) is a potentially fatal form of hepatic injury after hematopoietic stem cell transplantation. Patients can develop liver dysfunction, portal hypertension, ascites, coagulopathies, and multisystem organ failure. The mortality rate of severe SOS has been reported as high as 98% by day 100 after transplantation. Defibrotide, which is now approved for the treatment of SOS, has significantly decreased mortality. Defibrotide is a polynucleotide with profibrinolytic, anti-ischemic, and anti-inflammatory activity. These properties can increase the risk of life-threatening bleeding in this patient population. Previous protocols have suggested maintaining international normalized ratio ≤ 1.5, platelets > 30 k/uL, and fibrinogen ≥ 150 mg/dL to minimize this risk of bleeding. However, this can be challenging in fluid-sensitive patients with SOS. Thromboelastography (TEG) is a functional assay that evaluates the balance of procoagulant and anticoagulant proteins. In this series, TEG was used to guide defibrotide therapy as well as blood product transfusions in SOS patients with abnormal coagulation studies. Each patient recovered from SOS and had no bleeding complications. A randomized clinical trial is the next step in supporting the use of TEG in SOS patients with abnormal coagulation studies receiving defibrotide therapy.


Assuntos
Hepatopatia Veno-Oclusiva/tratamento farmacológico , Polidesoxirribonucleotídeos/uso terapêutico , Tromboelastografia/métodos , Transtornos da Coagulação Sanguínea/diagnóstico , Criança , Pré-Escolar , Feminino , Fibrinolíticos/uso terapêutico , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Hepatopatia Veno-Oclusiva/diagnóstico , Hepatopatia Veno-Oclusiva/etiologia , Humanos , Lactente , Masculino , Inibidores da Agregação Plaquetária/uso terapêutico , Polidesoxirribonucleotídeos/farmacologia
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