Extended half-life recombinant factor VIII treatment of hemophilia A in Brazil: an expert consensus statement

Introduction Treatment of hemophilia A in Brazil is offered to all patients at no cost. However, several unmet medical needs exist. Method In this study, we applied the Delphi method to discuss with seven hemophilia A specialists the challenges that patients and the health system face regarding hemophilia A treatment and opportunities for improvement. Results A consensus was obtained regarding the number of weekly infusions and patient adherence to treatment. The bleeding profile, unfavourable pharmacokinetics (PKs), low adherence and high daily activity were patient profiles that would benefit from using the extended half-life (EHL) recombinant factor VIII (rFVIII). The advantages of treatment with the EHL rFVIII were the lower number of infusions per week, which could increase patient adherence and decrease the risk of bleeds, due to a more constant plasma level, a lower value. Additionally, the EHL rFVIII could improve quality of life, especially in patients with high daily activity, such as adolescents and young adults. The panelists mentioned that EHL rFVIII, if available, could be offered first to the priority group (adolescents between 12 and 19 years old), followed by adults (20 to 64 years old) and elderly people (over 65 years old). Conclusion In summary, the EHL rFVIII offers the optimal prophylaxis by decreasing the dose frequency, increasing the treatment adherence and improving the QoL, without compromising safety and efficacy.

Consensus of the Brazilian association of hematology, hemotherapy and cellular therapy on patient blood management: Acute normovolemic hemodilution and intraoperative autotransfusion

Abstract Autologous blood transfusion can be achieved through different techniques, including by the patient donating blood before surgery (pre-deposit), collecting blood from the patient immediately before the operation and replacing the volume with colloids or plasma expanders (acute normovolemic hemodilution) or through the salvage of lost blood, during or immediately after surgery, and its retransfusion after washing (intraoperative or postoperative recovery). We will focus on the two methods used intraoperatively that are of fundamental importance in the management and conservation of the patient's own blood.

Consensus of the Brazilian association of hematology, hemotherapy and cellular therapy on patient blood management: Management of critical bleeding

Abstract The management of major bleeding is a critical aspect of modern healthcare and it is imperative to emphasize the importance of applying Patient Blood Management (PBM) principles. Although transfusion support remains a vital component of bleeding control, treating severe bleeding goes beyond simply replacing lost blood. A more comprehensive, multidisciplinary approach is essential to optimize patient outcomes and minimize the risks associated with excessive transfusions.

Consensus of the Brazilian association of hematology, hemotherapy and cellular therapy on patient blood management: Antifibrinolytics

Abstract The use of strategies to reduce blood loss and transfusions is essential in the treatment of surgical patients, including in complex cardiac surgeries and those that use cardiopulmonary bypass. Antifibrinolytics, such as epsilon-aminocaproic acid (EACA) and tranexamic acid (TXA), are widely used in these procedures, as well as in other types of surgeries. These medicines are included in the World Health Organization (WHO) list of 'essential medicines'. Scientific evidence demonstrates the effectiveness of EACA in reducing bleeding and the need for transfusions in heart surgery. EACA is highly recommended for use in heart surgery by the American Society of Anesthesiology Task Force on Perioperative Blood Management. Regarding the safety of EACA, there is no robust evidence of any significant thrombotic potential. TXA has also been shown to be effective in reducing the use of blood transfusions in cardiac and non-cardiac surgeries and is considered safer than other antifibrinolytic agents. There is no evidence of any increased risk of thromboembolic events with TXA, but doses greater than 2 g per day have been associated with an increased risk of seizures. It is also important to adjust the dose in patients with renal impairment. In conclusion, antifibrinolytics, such as EACA and TXA, are effective in reducing blood loss and transfusion use in cardiac and non-cardiac surgeries, without causing serious adverse effects.

Consensus of the Brazilian association of hematology, hemotherapy and cellular therapy on patient blood management: Inadvertent intraoperative hypothermia

Abstract Hemostasis plays a critical role in surgical procedures and is essential for a successful outcome. Advances in hemostatic agents offer new approaches to controlling bleeding thereby making surgeries safer. The appropriate choice of these agents is crucial. Volume replacement, another integral part of Patient Blood Management (PBM), maintains adequate tissue perfusion, preventing cellular damage. Individualization in fluid administration is vital with the choice between crystalloids and colloids depending on each case. Colloids, unlike crystalloids, increase oncotic pressure, contributing to fluid retention in the intravascular space. Understanding these aspects is essential to ensure safe and effective surgery, minimizing complications related to blood loss and maintaining the patient's hemodynamic status.

Consensus of the Brazilian association of hematology, hemotherapy and cellular therapy on patient blood management: Intraoperative hemostasis and volume replacement

Abstract Hemostasis plays a critical role in surgical procedures and is essential for a successful outcome. Advances in hemostatic agents offer new approaches to controlling bleeding thereby making surgeries safer. The appropriate choice of these agents is crucial. Volume replacement, another integral part of Patient Blood Management (PBM), maintains adequate tissue perfusion, preventing cellular damage. Individualization in fluid administration is vital with the choice between crystalloids and colloids depending on each case. Colloids, unlike crystalloids, increase oncotic pressure, contributing to fluid retention in the intravascular space. Understanding these aspects is essential to ensure safe and effective surgery, minimizing complications related to blood loss and maintaining the patient's hemodynamic status.

Yttrium-90 radiosynovectomy in knees and ankles (25 joints in 22 hemophilic patients). Short-term results

ABSTRACT Introduction: The radiosynovectomy (RS) is one treatment option for recurrent hemarthrosis in patients with hemophilia (PWH). A prospective cohort study was designed to evaluate the effects of the RS on the synovial membrane volume in the ankles and knees of PWH and patient characteristics related to the RS outcome. Methods: In a one-year follow-up, 25 joints of 22 PWH who presented 3 bleeds or more in the same joint over the last 6 months (target joints) were subjected to the RS. Two groups were compared: those who retained target joints following the RS and those who did not (less than 3 bleeds/6 months after the RS). The groups were analyzed according to age, hemophilia type/severity, joint, body mass index (BMI), inhibitor and Hemophilia Joint Health Score 2.1 (HJHS). The magnetic resonance images (MRI) of six ankles and six knees were acquired prior to, and 6 months after, the RS. The synovial membrane volume and arthropathy MRI scale were accessed and volumes were compared and correlated with the Yttrium-90 dose injected. Results: Patients with a mean age of 12 years and a mean HJHS of 6.7 (p < 0.05) retained target joints after the RS. The inhibitor, joint, type/severity of disease and BMI showed no significant differences between groups. The synovial membrane volume had a significant reduction after the RS (p = 0.03), but no correlation with the Yttrium-90 dose. In proportion to the synovial membrane volume, doses injected to the ankles were larger than those injected to the knees. Conclusion: The synovial membrane volume is reduced after the RS, regardless of the effective 90Y dose.

Pathologic rupture of the spleen in a patient with acute myelogenous leukemia and leukostasis

Rupture of the spleen can be classified as spontaneous, traumatic, or pathologic. Pathologic rupture has been reported in infectious diseases such as infectious mononucleosis, and hematologic malignancies such as acute and chronic leukemias. Splenomegaly is considered the most relevant factor that predisposes to splenic rupture. A 66-year-old man with acute myeloid leukemia evolved from an unclassified myeloproliferative neoplasm, complaining of fatigue and mild upper left abdominal pain. He was pale and presented fever and tachypnea. Laboratory analyses showed hemoglobin 8.3 g/dL, white blood cell count 278 × 109/L, platelet count 367 × 109/L, activated partial thromboplastin time (aPTT) ratio 2.10, and international normalized ratio (INR) 1.60. A blood smear showed 62% of myeloblasts. The immunophenotype of the blasts was positive for CD117, HLA-DR, CD13, CD56, CD64, CD11c and CD14. Lactate dehydrogenase was 2384 U/L and creatinine 2.4 mg/dL (normal range: 0.7-1.6 mg/dL). Two sessions of leukapheresis were performed. At the end of the second session, the patient presented hemodynamic instability that culminated in circulatory shock and death. The post-mortem examination revealed infiltration of the vessels of the lungs, heart, and liver, and massive infiltration of the spleen by leukemic blasts. Blood volume in the peritoneal cavity was 500 mL. Acute leukemia is a rare cause of splenic rupture. Male gender, old age and splenomegaly are factors associated with this condition. As the patient had leukostasis, we hypothesize that this, associated with other factors such as lung and heart leukemic infiltration, had a role in inducing splenic rupture. Finally, we do not believe that leukapheresis in itself contributed to splenic rupture, as it is essentially atraumatic...

Avaliação a médio prazo do controle de fatores de risco de doença cardiovascular em corte prospectiva de pacientes de alto risco tratados por intervenção coronária percutânea / Middle-term assessment of cardiovascular risk factor control in a prospective cohort of high-risk patients treated by percutaneous coronary intervention

INTRODUÇÃO: A prevenção secundária após intervenção coronária percutânea (ICP) é fundamental para melhorar a sobrevida livre de eventos e consiste principalmente no controle de fatores de risco. Analisou-se a prevenção secundária de pacientes de alto risco, incluídos prospectivamente no estudo Sequence Variation in Platelet Aggregation in Response to Clopidogrel and aspirin (SPARC). MÉTODOS: Foram arrolados 187 pacientes consecutivos entre dezembro de 2009 e fevereiro de 2011, tratados por ICP com stent e avaliados em retornos ambulatoriais de 30 dias, 3 meses, 6 meses e 12 meses quanto ao controle de hipertensão arterial, disglicemia, dislipidemia e tabagismo, e medidas terapêuticas respectivas. RESULTADOS: Houve aumento significativo de pacientes com controle pressórico (29%; P = 0,02), que cessaram tabagismo (18%; P = 0,003), e que receberam hipolipemiantes (19%; P < 0,0001) entre a internação para ICP e o primeiro retorno após o procedimento. Esse melhora do controle de fatores de risco refletiu-se em redução do escore de risco de Framingham médio observado no mesmo período (9,9%; P < 0,0001). Durante seguimento de até 12 meses o ganho atingido na internação para ICP se manteve para todos os fatores de risco. CONCLUSÕES: Observou-se efeito importante relativamente à internação índice para ICP, com aumento da prescrição de medicamentos para controle de fatores de risco e alcance de metas. Esse estudo identifica relevante janela de oportunidade para priorização do controle de fatores de risco na internação inicial, quando ganhos expressivos são observados e mantidos. Mas também explicita que esforços adicionais são necessários para expandir o benefício da prevenção secundária no seguimento a médio prazo de pacientes tratados por ICP.

BACKGROUND: Secondary prevention after percutaneous coronary intervention (PCI) is essential to increase event-free survival and consists mainly in risk factor control. We analyzed the secondary prevention of high-risk patients included prospectively in the Sequence Variation in Platelet Aggregation in Response to Clopidogrel and aspirin trial (SPARC). METHODS: From December 2009 to February 2011 we enrolled 187 consecutive patients who were submitted to PCI with stent implantation and were evaluated in outpatient visits at 1, 3, 6, and 12 months of follow-up for the control of hypertension, dysglycemia, hyperlipidemia and smoking and their respective therapeutic measures. RESULTS: There was a significant increa­se in the number of patients with controlled hypertension (29%; P = 0.02), who stopped smoking (18%; P = 0.003), and received statins (19%; P < 0.0001) between the index PCI and the first follow-up visit. The risk factor control improvement led to a decrease in the mean Framingham risk score (9.9%; P < 0.0001). During the 12 months follow-up the gains achieved at PCI admission were maintained for all risk factors. CONCLUSIONS: An important effect was observed on the index PCI admission with increased prescription of risk factor control drugs and achievement of therapeutic goals. This study identifies a relevant opportunity window for risk factor control at the index admission, when substantial gains are observed and maintained. However, it also shows further efforts are required to expand the benefit of secondary prevention in the middle-term follow-up of patients treated by PCI.

Hormônios femininos e hemostasia: [revisão] / Female hormones and hemostasis: [review]

Os hormônios femininos exógenos utilizados para contracepção ou para terapia hormonal (TH) no climatério estão associados a aumento de risco para tromboembolismo venoso (TEV), principalmente por provocarem alterações pró-coagulantes na hemostasia. Este risco não é cumulativo e parece ser maior no primeiro ano de uso. A dose de estrogênio, o tipo de estrogênio e progestagênio utilizados, a via de administração hormonal e os fatores de risco hereditários para trombose venosa de cada paciente interferem no risco final para trombose venosa. O conhecimento dos efeitos na hemostasia inerentes a cada composto hormonal é essencial para uma prescrição adequada.

Exogenous female hormones used for contraception or postmenopausal hormonal replacement therapy are associated with an increase of venous thromboembolism (VTE) risk, mainly because they cause a hypercoagulable state. The risk is highest during the first year of use and it is not cumulative. The dose of estrogen, the type of estrogen and progestogen, the route of administration of female sex steroid hormones, and the hereditary risk factors for VTE of each patient can interfere on the final risk for VTE. The knowledge of their effect on hemostasis is essential for a correct prescription.