Knee replacement surgery in a patient with acquired von Willebrand disease: a case study with recommendations for patient management.

Introduction And Importance: Acquired von Willebrand disease (AvWD) is a rare underdiagnosed bleeding disorder caused by alterations in the levels of the major blood-clotting protein von Willebrand factor (vWF). The clinical and laboratory parameters of AvWD are similar to congenital vWD, but it is found in individuals with no positive family history with no underlying genetic basis. The disease remains multifactorial and incompletely understood.

A post hoc comparative real-world analysis of HEAD-US score for joint health assessment of patients with severe haemophilia A and B in Spain.

Aim: Joint damage due to haemarthrosis can be effectively monitored with point-of care ultrasound using the Haemophilia Early Arthropathy Detection with US (HEAD-US) scoring system. A post hoc comparative analysis of the joint status of patients with severe haemophilia A (HA) or B (HB) was performed.

Methods: The databases of two observational, cross-sectional studies that recruited patients with HA or HB from 12 Spanish centres were analysed to compare the status of the elbows, knees and ankles in patients with severe disease according to treatment modality.

Study of platelet kinetics in immune thrombocytopenia to predict splenectomy response.

Despite the efficacy of splenectomy for chronic immune thrombocytopenia (ITP), its considerable failure rate and its possible related complications prove the need for further research into potential predictors of response. The platelet sequestration site determined by In-labelled autologous platelet scintigraphy has been proposed to predict splenectomy outcome, but without standardisation in clinical practice. Here, we conducted a single-centre study by analysing a cohort of splenectomised patients with ITP in whom In-scintigraphy was performed at La Paz University Hospital in Madrid to evaluate the predictive value of the platelet kinetic studies.

Development of a telematic pharmaceutical care app (Haemoassist) for multidisciplinary follow-up of patients with congenital coagulopathies.

Background: Guidelines for congenital coagulopathies recommend that patients record treatment administrations and bleeding episodes to help healthcare professionals monitor the disease.

Research Design And Methods: We studied over two years which patient profiles (age, treatment regimen, treatment compliance) were most likely to accept the use of an app to collect this information. We validated the quality of patient-reported data by comparing it with data obtained from hospital electronic records, pharmacy dispensing records and patient interview, collected in an access database used as a reference.

Acquired Haemophilia A: A 15-Year Single-Centre Experience of Demography, Clinical Features and Outcome.

Acquired haemophilia A (AHA) is a rare severe bleeding disorder resulting from the production of autoantibodies directed against coagulation factor VIII. At presentation, bleeding events can be severe, and an early diagnosis and treatment are of major importance. The current study aims to analyse the treated patients who have been diagnosed with AHA for a better understanding of our population and treatment outcome.

Arthropathy in people with mild haemophilia: Exploring risk factors.

Background: It is important to investigate which factors are associated with the development of arthropathy in people with mild haemophilia (PWMH), in order to prevent it and to limit its effect on function and quality of life.

Purpose: To examine the risk factors associated with arthropathy and to predict its presence and degree of involvement in our PWMH population.

Methods: This was an observational, cross-sectional cohort study of 85 PWMH under follow-up in our centre.

Clinical assessment and point of care ultrasonography: How to diagnose haemophilic synovitis.

Background: The ability of clinical tools to identify early joint changes is limited. Synovitis is a fundamental finding in understanding haemophilia activity and the response to its therapies; thus, there is a need for sensitive methods to better diagnose subclinical synovitis early.

Purpose: Our aim was to compare the frequency with which clinical assessment and ultrasound detected synovial hypertrophy in the most frequently affected joints in patients with haemophilia (elbows, knees and ankles).

The Importance of Platelet Glycoside Residues in the Haemostasis of Patients with Immune Thrombocytopaenia.

Loss of sialic acid from the carbohydrate side chains of platelet glycoproteins can affect platelet clearance, a proposed mechanism involved in the etiopathogenesis of immune thrombocytopaenia (ITP). We aimed to assess whether changes in platelet glycosylation in patients with ITP affected platelet counts, function, and apoptosis. This observational, prospective, and transversal study included 82 patients with chronic primary ITP and 115 healthy controls.

Impact of COVID-19 Pandemic on Patients with Immune Thrombocytopaenia.

: The aim of this study was to determine the impact of the COVID-19 pandemic on the lives of patients with immune thrombocytopaenia (ITP) treated at our hospital. : The study was conducted in the Community of Madrid, which has the highest number of COVID-19 cases in Spain. We included 143 adult patients with ITP (130 with chronic ITP, 8 with persistent ITP, and 5 with newly diagnosed ITP).

Ultrasound evaluation of joint damage and disease activity in adult patients with severe haemophilia A using the HEAD-US system.

Introduction: The Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) system and scoring scale has proven to be an accurate and time-efficient imaging method for identifying joint damage in patients with haemophilia.

Aim: Observational, multicentre, cross-sectional study conducted in 8 centres in Spain that assessed the joint status of adult patients with severe haemophilia A (SHA) using HEAD-US.

Methods: Joint status of the elbow, knee and ankle was evaluated in adults with SHA receiving on-demand (OD) treatment, or primary (PP), secondary (SP), tertiary (TP) or intermittent (IP) prophylaxis.

Paradoxical effect of SARS-CoV-2 infection in patients with immune thrombocytopenia.

Thrombocytopenia has been identified as a common complication of severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection in the general population. In an attempt to determine the impact of coronavirus disease 2019 (COVID-19) in patients with immune thrombocytopenia (ITP), a retrospective single-centre study was performed. Thrombocytosis was observed in patients with chronic ITP after SARS-CoV-2 infection, frequently needing treatment adjustment or even discontinuation of therapy.

HJHS 2.1 and HEAD-US assessment in the hemophilic joints: How do their findings compare?

: In hemophilic patients methods are needed to better diagnose joint damage early, so that treatments can be adjusted to slow the progression of hemophilic arthropathy. The aim of this study is to investigate the relationship between the Hemophilia Joint Health Score version 2.1 (HJHS 2.

Thromboprophylaxis in a patient with COVID-19 and severe hemophilia A on emicizumab prophylaxis.

COVID-19 can be associated with coagulopathy (CAC, COVID-19-associated coagulopathy) with a high prothrombotic risk based on an intense inflammatory response to viral infection leading to immunothrombosis through different procoagulant pathways. Emerging evidence suggests that the use of heparin in these patients could be associated with lower mortality. Emicizumab is a bispecific humanized monoclonal antibody that bridges activated factor IX and factor X, thereby restoring the function of missing factor VIIIa in hemophilia A.

Registry of patients with congenital bleeding disorders and COVID-19 in Madrid.

Introduction: We present the first registry of patients with congenital bleeding disorders and COVID-19. The study has been carried out in the Community of Madrid, which has the highest number of cases in Spain. The objective is to understand the incidence of COVID-19, the course of the disease if it occurs and the psychosocial and occupational impact on this population.

'Do not Do' Recommendations in Hemophilia.

Background: It is important to discard those practices that do not add value. As a result, several initiatives have emerged. All of them try to improve patient safety and the use of health resources.

Platelet and immune characteristics of immune thrombocytopaenia patients non-responsive to therapy reveal severe immune dysregulation.

Multifactorial mechanisms leading to diminished platelet counts in immune thrombocytopaenia (ITP) might condition the ability of patients with ITP to respond to treatments. Examining their platelet and immune features, we aimed to detect singular characteristics of patients with ITP who do not respond to any treatment. We studied patients with chronic primary ITP who had been without treatment, or untreated (UT-ITP), for at least six months; included were responders to agonists of thrombopoietin receptors (TPO-RA), patients who showed no response to first- and second-line treatments (NR-ITP), and healthy controls.

Health education and empowerment in adult patients with haemophilia.

: The purpose of this study is to evaluate the degree of education and empowerment of the patients of the Haemophilia Unit at 'La Paz' University Hospital (Madrid, Spain).: Haemophilic patients attending routine haemophilia consultations were asked to complete a questionnaire with a view to determining the patient´s perceived knowledge about the disease; their ability to make individual and group decisions; and whether the frequency of their hospital visits could be reduced.: Patients were shown to have ample knowledge about the disease.

Accelerating recovery from acute hemarthrosis in patients with hemophilia: the role of joint aspiration.

: Arthrocentesis of an acute hemarthrosis in hemophilia remains a controversial issue. The purpose of this study is to define the role that joint aspiration can play in the recovery from acute hemarthrosis in patients with hemophilia. The study sample included 33 hemophilic patients (55 joints) with acute elbow, knee, and ankle hemarthrosis as confirmed by ultrasonography.

Platelet Apoptosis and PAI-1 are Involved in the Pro-Coagulant State of Immune Thrombocytopaenia Patients Treated with Thrombopoietin Receptor Agonists.

The treatment goal for patients with immune thrombocytopaenia (ITP) is to raise platelet counts to levels that minimize or stop bleeding. Thrombopoietin receptor agonists (TPO-RAs) have been successfully and extensively employed as second-line therapy for ITP. However, TPO-RAs have a small but significant increase in the risk of thrombosis.

Hindfoot malalignment in adults with haemophilic ankle arthropathy: The importance of early detection and orthotic treatment.

Introduction: Haemophilic arthropathy (osteoarthritis secondary to haemophilia) of the ankle may result in painful hindfoot malalignment.

Purpose: To analyse hindfoot alignment in subjects with haemophilic arthropathy of the ankle and evaluate the response (improvement of pain, function and alignment) to the orthotic treatment prescribed in patients with malalignment.

Methods: The study included 163 patients with haemophilia, all of them over 16 years of age.

Evaluation of EC50 of factor VIII as predictor of prophylaxis efficacy in patients with severe haemophilia A.

Trough factor (F) VIII level is a not reliable bleeding risk indicator to predict prophylaxis efficacy in severe haemophilia A (SHA), therefore, accurate biomarkers are much needed. Thrombelastography (TEG) monitors both thrombin and clot formation addressing the global haemostatic status but its usefulness to tailor prophylaxis in haemophilia has been poorly evaluated. In this study, correspondence between individual pharmacodynamic/pharmacokinetic profile of FVIII and joint condition, physical activity and bleeding phenotype of SHA patients under prophylactic treatment was assessed.