Prophylaxis in hemophilia

Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B). Prophylaxis ­ the regular administration of therapeutic products to maintain hemostasis and prevent bleeding ­ is the mainstream of treatment. Addressing the development and scientific evidence for administrating prophylaxis is the goal of this review. Prophylaxis is the therapeutic modality of choice for people with severe hemophilia, being considered, in principle, a lifelong treatment. It should have an early onset, ideally as a primary, or at least secondary. Even lifelong tertiary prophylaxis seems to offer benefit, although further studies are still lacking. Individualized strategies should lead to an optimization of the dilemma between better joint outcomes versus involved costs.

Hemophilia: a biography on therapeutical approaches

The history of hemophilia is ancient, with descriptions dated to the 2nd century AD. The first modern narratives appeared in 1800s, when total blood transfusion was the only available treatment and life expectancy was remarkably low. Advances occurred with the use of plasma and cryoprecipitate, but only the discovered of factor concentrates revolutionized the treatment. The implantation of prophylaxis allowed hemophilic patients to prevent bleeding and the development of chronic arthropathy, although with a significant burdensome with the regular infusions. In the past 20 years, this field has witnessed major improvements, including the development of gene therapy and other pharmacological approaches.

Immune thrombocytopenic purpura following CoronaVac vaccination: a case report

Immune thrombocytopenic purpura (ITP), an autoimmune disorder, has been documented as a result of SARS-CoV-2 infection and a vaccination side effect. The COVID-19 pandemic has led to the creation of CoronaVac vaccine and has been widely administered in Brazil.Patient, in the case, is an 82-years-old female who received the vaccine two days before an acute episode of gingivorrhagia and diffuse cutaneous petechiae. Other exams were made to look for other causes of secondary thrombocytopenia and all the results were normal. The patient showed improvement on the platelet levels three day after the beginning of the treatment with high dosage methylprednisolone.Knowing that other kinds of vaccine can generate ITP, the SARS-CoV-2 vaccine could be related to the symptoms.

Epidemiological characteristics of hemophilia in the pre-primary prophylaxis era: a historical cohort

Introduction: Epidemiological studies on hemophilia in the Brazilian population are historically scarce. Despite the continuous effort made by the National Program of Inherited Bleeding Disorders to map this condition, little information is available, especially on the period prior to program conception. Therefore, the present study aims to assess the epidemiological, serological, and clinical characteristics of patients with hemophilia in the state of Rio Grande do Sul, Brazil. Methods: A total of 455 patients had their medical records reviewed from January 1, 2003 to December 31, 2007. Results: We observed a remarkable prevalence of hepatitis C virus (HCV) infection in patients with both hemophilia A and B, and this prevalence significantly increased along with age (p < 0.001). No positive anti-HCV results were observed among children younger than 5 years old. There was a significant correlation between the severity of hemophilia and the number of arthropathies in all age categories. Considering the presence of inhibitors, a significant difference was observed between age groups, as older patients had higher inhibitor titers. There was a significant correlation between mean coagulation factor consumption and the number of arthropathies in patients over 5 years old. Conclusions: This profile analysis of patients with hemophilia reflects a gradual improvement in treatment safety and efficiency, as well as the need for continued investment in this population. (AU)

What happens to intolerant, relapsed or refractory chronic myeloid leukemia patients without access to clinical trials?

ABSTRACT Objective: To assess clinical outcomes of intolerant, relapsed or refractory patients who could not be treated with new tyrosine kinase inhibitors or experimental therapies. Methods: A retrospective cohort of 90 chronic myeloid leukemia patients in all phases of the disease treated with imatinib mesylate as their first TKI therapy, and with dasatinib or nilotinib as the next line of therapy. We evaluated clinical outcomes of these patients, with special focus on the group that needed more than two therapy lines. Results: Thirty-nine percent of patients were refractory or intolerant to imatinib. An 8-year overall survival rate of the patients who went through three or more lines of treatment was significantly lower, compared to those who were able to maintain imatinib as their first-line therapy (83% and 22%, respectively p < 0.01). Decreased overall survival was associated with advanced-phase disease (p < 0.01), failure to achieve major molecular response in first-line treatment (p < 0.01) and interruption of first-line treatment due to any reason (p = 0.023). Failure in achieving complete cytogenetic response and major molecular response and treatment interruption were associated with the progression to the third-line treatment. Conclusion: The critical outcome observed in relapsed, intolerant or refractory chronic phase CML patients reflects the unmet need for this group of patients without an alternative therapy, such as new drugs or experimental therapies in clinical trials. Broader access to newer treatment possibilities is a crucial asset to improve survival among CML patients, especially those refractory or intolerant to first-line therapies.

Bola fúngica dos seios paranasais: Relato de dois casos e revisão de literatura / Fungus ball of the paranasal sinuses: Report of two cases and literature review

Introdução: Bola fúngica dos seios paranasais é uma infecção não invasiva que se caracteriza por sua cronicidade, sendo a maioria relacionada com tratamento endodôntico prévio. Acomete principalmente o seio maxilar, embora todos os seios possam ser envolvidos. O principal agente etiológico é o Aspergillus spp. A tomografia computadorizada, devido às apresentações radiológicas características, sugere o diagnóstico que é realizado definitivamente através de análises histopatológicas. O tratamento padrão-ouro é a cirurgia sinusal endoscópica com antrostomia meatal média. Objetivo: Relatar dois casos de bola fúngica dos seios paranasais e ressaltar aspectos importantes desta patologia. Relato dos Casos: Caso 1) Paciente do sexo feminino, 78 anos, apresentou-se com queixas de dor facial há 6 meses e história prévia de tratamento endodôntico. Ao exame físico constatou-se a presença de secreção purulenta em meato médio esquerdo. O Raio X apresentou velamento completo do seio maxilar esquerdo, enquanto a tomografia computadorizada mostrou lesão calcificada neste local. Realizou-se sinusotomia que evoluiu bem. Caso 2) Paciente do sexo feminino, 70 anos, procurou atendimento por história de sinusites de repetição. Ao exame físico não se percebeu nenhuma particularidade. A tomografia computadorizada, assim como a ressonância magnética, detectou espessamento da parede mucosa do seio maxilar esquerdo, além de uma massa calcificada. Realizou-se a mesma sequência de tratamento e a paciente também evoluiu bem. Considerações finais: A infecção fúngica deve ser considerada nos pacientes que se apresentam com sinusite crônica, que não respondem ao uso de antibióticos e que possuem história de manipulação endodôntica...

Introduction: Fungal ball of the sinuses is a not invasive infection that if characterizes for its chronicity, being the majority related with previous endodontic treatment. Affect mainly the breasts to maxillary; even so all the breasts can be involved. The main etiological agent is the Aspergillus spp. The computed tomography, had to characteristic the radiological presentations, suggests the diagnosis that is carried through definitively through histopathological analyses. The treatment standard-gold is the sinus surgery with average meatal antrostomy. Objective: Reporting two cases of fungal ball of the sinuses and to stand out important aspects of this pathology. Story of the Cases: Case 1) Patient of the feminine sex, 78 years old, presented itself with complaints of face pain has 6 months and previous history of endodontic treatment. To the physical examination it was evidenced purulent secretion presence in left average meatus. Ray X presented complete veiling of the breasts to maxillary left, while the computed tomography showed injury calcified in this place. Sinusotomy was become fulfilled that evolved well. Case 2) Patient of the feminine sex, 70 years old, looked attendance for history of sinusitis of repetition. To the physical examination no particularity was not perceived. The computed tomography, as well as the magnetic resonance, detected thickening of the mucous wall of the breasts to maxillary left, beyond a calcified mass. It was become fulfilled same sequence of treatment and the patient also evolved well. Final Considerations: The fungal infection must be considered in the patients who if present with chronic sinusitis, that they do not answer to the antibiotic use and that they possess history of endodontic manipulation...