RESUMO
INTRODUCTION AND AIM: Open questions in haemophilia, such as effectiveness of innovative therapies, clinical and patient-reported outcomes (PROs), epidemiology and cost, await answers. The aim was to identify data attributes required and investigate the availability, appropriateness and accessibility of real-world data (RWD) from German registries and secondary databases to answer the aforementioned questions. METHODS: Systematic searches were conducted in BIOSIS, EMBASE and MEDLINE to identify non-commercial secondary healthcare databases and registries of patients with haemophilia (PWH). Inclusion of German patients, type of patients, data elements-stratified by use in epidemiology, safety, outcomes and health economics research-and accessibility were investigated by desk research. RESULTS: Screening of 676 hits, identification of four registries [national PWH (DHR), national/international paediatric (GEPARD, PEDNET), international safety monitoring (EUHASS)] and seven national secondary databases. Access was limited to participants in three registries and to employees in one secondary database. One registry asks for PROs. Limitations of secondary databases originate from the ICD-coding system (missing: severity of haemophilia, presence of inhibitory antibodies), data protection laws and need to monitor reliability. CONCLUSION: Rigorous observational analysis of German haemophilia RWD shows that there is potential to supplement current knowledge and begin to address selected policy goals. To improve the value of existing RWD, the following efforts are proposed: ethical, legal and methodological discussions on data linkage across different sources, formulation of transparent governance rules for data access, redefinition of the ICD-coding, standardized collection of outcome data and implementation of incentives for treatment centres to improve data collection.
Assuntos
Pesquisa Biomédica , Bases de Dados Factuais , Hemofilia A/terapia , Sistema de Registros , Adulto , Criança , Alemanha , Humanos , Resultado do TratamentoRESUMO
INTRODUCTION: This report summarizes recommendations relating to haemophilia therapy arising from discussions among experts from 36 European countries during the 'Kreuth IV' meeting in May 2016. AIM: The objective of the meeting was for experts in the field of haemophilia from across Europe to draft resolutions regarding current issues relating to the treatment of haemophilia. RESULTS: Hospitals providing clinical care for people with haemophilia and related disorders are strongly recommended to seek formal designation as either European Haemophilia Treatment Centres (EHTC) or European Haemophilia Comprehensive Care Centres (EHCCC). There should be agreed national protocols or guidelines on management of the ageing patient with haemophilia. The minimum consumption of factor VIII and IX concentrate in any country should be 4 IU and 0.5 IU per capita of general population respectively. Treatment for hepatitis C with direct-acting antiviral agents should be provided to all people with haemophilia on a priority basis. Genotype analysis should be offered to all patients with severe haemophilia. Genetic counselling, when given, should encompass the recommendation that genetic relatives of the affected person be advised to seek genetic counselling. People with inhibitors should have access to bypassing agents, immune tolerance and elective surgery. National or regional tenders for factor concentrates are encouraged. Outcome data including health related quality of life should be collected. Treatment with extended half-life factors should be individualized and protection against bleeding should be improved by increasing trough levels. Steps should be taken to understand and minimize the risk of inhibitor development. CONCLUSION: It is hoped that these recommendations will help to foster equity of haemophilia care throughout Europe.
Assuntos
Fatores de Coagulação Sanguínea/uso terapêutico , Consenso , Hemofilia A/tratamento farmacológico , Europa (Continente) , HumanosRESUMO
Payers in European countries request studies with high levels of evidence for decision making also for rare diseases like haemophilia B (HB). The objective of the study was to determine the status quo of current studies in HB regarding the overall level of evidence generated. The methods used for performing the study were systematic literature research in EMBASE and MEDLINE, search terms 'HB' and 'factor IX' (FIX). The inclusion criteria were journal articles (JA), conference abstracts (CA), English language, published between January 2009 and March 2013, studies only; screening of titles, abstracts, full texts subsequently. ClinicalTrials.gov search: unpublished registered trials (RT) concerning HB or FIX. The analysis was performed on research topic, sponsor, recruitment status and study design. Screening of 1639 hits yielded 31 JA describing 35 studies, and 62 CA. FIX was subject of 21 studies (60.0%) and 29 CA (46.8%). Seven studies focused on various aspects of HB, six on haemophilia studies with separate HB data. Screening of 173 hits from ClinicalTrials.gov yielded 42 RT. Overall, 32 RT (76.2%) related to FIX. Measurement of health-related quality of life (HRQoL) was identified in none of these studies, four CA (6.5%), four RT (9.5%). Randomized study design was found in one study (2.9%), four RT (9.5%). Three studies (8.6%) and seven RT (16.7%) were prospective, observational and comparative. The majority of published clinical studies do not meet payers' expectations for evidence. Therefore, clinical investigation concepts addressing randomization, outcomes research including HRQoL and comparison of therapy options should be discussed. Refined statistical methods and exploitation of complementary real-life data sources may fill current evidence gaps concerning rare diseases.
Assuntos
Hemofilia B , Humanos , Qualidade de Vida , Doenças RarasRESUMO
UNLABELLED: Haemophilic arthropathy is a defining feature and a debilitating condition of persons with haemophilia (PwH) in low resource countries. Orthopaedic surgery is unavoidable for patients with high occurrence of joint damage. AIMS: We aimed to evaluate the spectrum and outcome of invasive orthopaedic therapies in PwH and von Willebrand diseases (VWD). PATIENTS AND METHODS: Our descriptive observational retrospective study included 131 invasive surgical procedures, performed on 76 consecutive patients, most of them (93.4%) with severe disease, treated in Timisoara's Haemophilia Center over a period of 12 years; 17.1% had pre-operation anti-FVIII inhibitors. Invasive elective procedures were predominant (90.8%) as compared to emergency measures (9.2%); according to their invasiveness, 20.6% of interventions were major, 44.3% intermediate and 35.1% minor. Results were good in the majority of cases; significantly reduced joint bleed rate and pain score were the most consistent achievements. The greatest proportion of complications occurred after major (66.7%), compared to moderate (25.6%) and minor (7.7%) interventions. The main threatening complication was the development (3.8%) or increase (4.6%) of inhibitor titer. Local bacterial infections and wound dehiscence complicated the evolution in 4.6% and 0.8 % of cases, respectively; we noticed no blood-borne infections or thrombotic accidents. Low dosage (10.7%) and short duration of substitution (21.4%) led to increased post-surgical bleeding and post-haemorrhagic anaemia. CONCLUSIONS: Surgery is a highly demanding intervention in haemophilia, which cannot be ignored in a low resource country. It represents a life or limb-saving and quality of life-improving measure.
Assuntos
Artrodese/estatística & dados numéricos , Países em Desenvolvimento/estatística & dados numéricos , Hemartrose/epidemiologia , Hemartrose/terapia , Prótese Articular/estatística & dados numéricos , Complicações Pós-Operatórias/epidemiologia , Pré-Medicação/estatística & dados numéricos , Adolescente , Adulto , Criança , Fator VIII/uso terapêutico , Feminino , Humanos , Estudos Longitudinais , Masculino , Complicações Pós-Operatórias/diagnóstico , Prevalência , Estudos Retrospectivos , Fatores de Risco , Romênia/epidemiologia , Distribuição por Sexo , Adulto JovemRESUMO
UNLABELLED: Prophylaxis in adults can be necessary and reasonable for clinical reasons. The aim was to evaluate from an economic viewpoint prophylactic factor VIII substitution in adult patients with haemophilia in Germany. PATIENTS, METHODS: A decision model (time frame: one year; perspective: statutory health insurance; reference patient (RP) and 2 patient profiles) was developed. Calculations are based on data from a structured literature search and a pharmacovigilance study: therapy switch on-demand/prophylaxis (OD/Proph). RESULTS: RP: 45 years, 20 bleeds p.a. OD, 16 bleeds avoided with 8.5 I.U./kg/d Proph, additional cost Euro 141,113 p.a.; profile 1: 50 years, 55 bleeds p.a. OD, factor consumption per bleed 20 I.U./kg higher than RP, 39 bleeds avoided with 8.5 I.U./kg/d Proph, additional cost Euro 19,134 p.a.; profile 2: 60 years, 35 bleeds p.a. OD, factor consumption per bleed 40-80 I.U./kg higher than RP, 34 bleeds avoided with 11 I.U./kg/d Proph, cost reduction Euro 660 p.a. CONCLUSIONS: Prophylactic factor VIII substitution in adult haemophilia patients is depending on the individual clinical situation not only clinically but also economically reasonable. To evaluate this effects in the future comprehensively, longitudinal real-life data from patient-centered care are needed including clinical outcomes, quality of life and adherence.
Assuntos
Fator VIII/economia , Fator VIII/uso terapêutico , Custos de Cuidados de Saúde/estatística & dados numéricos , Hemofilia A/economia , Hemofilia A/prevenção & controle , Modelos Econômicos , Adulto , Idoso , Idoso de 80 Anos ou mais , Coagulantes/economia , Coagulantes/uso terapêutico , União Europeia , Feminino , Alemanha/epidemiologia , Hemofilia A/epidemiologia , Humanos , Masculino , Pessoa de Meia-IdadeRESUMO
OBJECTIVE: Fertiloscopy is a simple minimal invasive method which allows salpingoscopy and microsalpingoscopy in order to examine the mucosa of the fallopian tubes of patients with unexplained infertility. Infectious tubal damage is a common cause of tubal infertility. In 1998 it was demonstrated that nuclear staining of cellular nuclei during microsalpingoscopy with methylene blue provides a simple in vivo method to evaluate cellular damage of the tubal epithelium. The purpose of this study was to introduce and statistically test a new computerized method to objectively evaluate the extent of tubal damage. DESIGN OF RETROSPECTIVE STUDY: Cooperation of two Departments of Gynecology and Obstetrics (Krankenanstalt Rudolfstiftung, Vienna, Austria and CRES Center, Hôpital Natecia, Lyon, France) with the University of Art and Design, Linz, Austria and University Hospital, Vienna, Austria. MATERIALS AND METHODS: Microsalpingoscopic images from ten female patients, aged between 18 and 45 years with primary infertility, showing stained nuclei in damaged intrafallopian tubal epithelium were provided by Antoine Watrelot, CRES Center, Hôpital Natecia, Lyon, France. These images were evaluated by an experienced medical expert staff examiner and a computerized standard method called cross-correlation and template matching. The obtained numbers of nuclear stainings were statistically evaluated. RESULTS: Computerized evaluation of nuclear staining of damaged intrafallopian epithelial cells in female patients with infertility obtains similar but more reproducible results compared to manual evaluation (p = 0.007). CONCLUSION: Normalized cross-correlation can be used to measure tubal damage diagnosed by in vivo methylene blue dyeing during microsalpingoscopy and might facilitate the decision for in vitro fertilisation in patients with unclear unexplained infertility in further studies.
Assuntos
Endoscopia/métodos , Tubas Uterinas/patologia , Processamento de Imagem Assistida por Computador/métodos , Epitélio/patologia , Feminino , Humanos , Infertilidade Feminina/diagnóstico , Azul de Metileno , Mucosa/patologiaRESUMO
This report summarizes recommendations relating to haemophilia therapy arising from discussions among experts from 36 European countries during the Kreuth III meeting in April 2013. To optimize the organization of haemophilia care nationally, it is recommended that a formal body be established in each country to include the relevant clinicians, national haemophilia patient organization, health ministry, paying authority and (if appropriate) regulatory authorities. The minimum factor VIII consumption level in a country should be 3 I.U. per capita. Decisions on whether to adopt a new product should not be based solely on cost. Prophylaxis for children with severe haemophilia is already recognized as the optimum therapy. Ongoing prophylaxis for individual adults should also be provided when required based on clinical decision making by the clinician in consultation with the patient. Children with inhibitors who have failed, or who are not suitable for, immune tolerance therapy should be offered prophylaxis with bypassing agents. Single factor concentrates should be used as therapy wherever possible in patients with rare bleeding disorders. Orphan drug designation for a factor concentrate should not be used to hinder the development, licencing and marketing of other products for the same condition which have demonstrably different protein modification or enhancement.
Assuntos
Fatores de Coagulação Sanguínea/uso terapêutico , Hemofilia A/tratamento farmacológico , Criança , Consenso , Europa (Continente) , Humanos , Guias de Prática Clínica como AssuntoRESUMO
The development of neutralizing antibodies to factor VIII (FVIII) is the most serious complication of therapy for haemophilia A. There is now excellent documentation that a large number of both genetic and environmental factors contribute to the risk of FVIII inhibitor incidence. One of the environmental factors that has been proposed as an influence on this complication is the occurrence of FVIII product switching. There are only a small number of clinical studies that have addressed this question, and thus, the amount of objective information available to assess this association is limited. In this review, in addition to summarizing past evidence pertinent to this subject, we present the results of a complementary strategy, a Delphi analysis, to add to the considerations of product switching and FVIII immunogenicity. With the imminent arrival in the clinic of several new FVIII products, the haemophilia community must be prepared to collect prospectively controlled data to better address this important management issue.
Assuntos
Anticorpos Neutralizantes/imunologia , Substituição de Medicamentos , Fator VIII/imunologia , Fator VIII/uso terapêutico , Hemofilia A/tratamento farmacológico , Hemofilia A/imunologia , Hemofilia A/epidemiologia , Humanos , IncidênciaRESUMO
UNLABELLED: The German Haemophilia Registry records online data from patients with haemophilia A, haemophilia B, von Willebrand`s disease and other coagulation factor deficiency disorders since 2009. Patient's pseudonymised data will only be enrolled in the German Haemophilia Registry if the patient signs an informed consent. Without the informed consent, only aggregated data according to §21 German Transfusion Law are reported. These data include the number of persons with congenital haemostasis disorders classified to type of disease and severity as well as patients' age, and the consumption of clotting factor according to each group. RESULTS: The highest number of patients with haemophilia was reported in 2010: 3375 patients with haemophilia A and 614 with haemophilia B respectively; the highest number of patients with von Willebrand's disease was 1473, reported in 2011. CONCLUSION: In comparison to data from registries in Austria and Switzerland it can be assumed that most of the patients with severe haemophilia are registered in the German Haemophilia Registry whereas patients with moderate and mild forms are still missing.
Assuntos
Fatores de Coagulação Sanguínea/uso terapêutico , Pesquisas sobre Atenção à Saúde , Hemofilia A/mortalidade , Hemofilia A/terapia , Sistema de Registros/estatística & dados numéricos , Doenças de von Willebrand/mortalidade , Doenças de von Willebrand/terapia , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Áustria/epidemiologia , Criança , Pré-Escolar , Feminino , Alemanha/epidemiologia , Humanos , Incidência , Lactente , Recém-Nascido , Masculino , Pessoa de Meia-Idade , Fatores de Risco , Taxa de Sobrevida , Suíça/epidemiologia , Adulto JovemRESUMO
Since 1982, the multicenter study of the epidemiology of patients with haemophilia is carried out annually with the support of all sizes of haemophilia centers and treatment facilities. The long period of investigation has not only enabled the reporting of up-to-date mortality and morbidity status, but also of retrospective observations. The participating centers have made anonymized data available for patients with Haemophilia A, B and von Willebrand's disease (total counts for treated patients, type and severity of the disease, HIV status and causes of death). For 2011-2012 3331 patients were reported with haemophilia A or B from 32 centers. Although the mortality from HIV/AIDS in haemophilia patients continues to decline, HIV infection remains clinically relevant since an HIV/HCV coinfection can raise the risk of severe liver disease. The data from the multicenter survey were, together with the anonymized data from the Foundation for Humanitarian Aid for Persons Infected with HIV though Blood Products, comparatively descriptively compared and the temporal progression illustrated by a so-called moving average. Expectedly, haemophilia patients were shown to be under-represented by the data from the multicenter survey. Otherwise, the data are consistent.
Assuntos
Infecções por HIV/mortalidade , Hemofilia A/mortalidade , Hepatite C/mortalidade , Doenças de von Willebrand/mortalidade , Idoso , Idoso de 80 Anos ou mais , Comorbidade , Feminino , Alemanha/epidemiologia , Humanos , Incidência , Masculino , Pessoa de Meia-Idade , Fatores de Risco , Taxa de SobrevidaRESUMO
Patients with haemophilia A and inhibitors are at high risk for severe bleeding, progression of joint disease and deterioration of health-related quality of life (HRQoL). To determine the impact of prophylaxis with an activated prothrombin complex concentrate (aPCC) on HRQoL, HRQoL was assessed using the Short-Form (SF)-36 Health Survey and the EQ-5D questionnaire in subjects ≥ 14 years participating in a prospective, randomized, crossover study comparing 6 months of aPCC prophylaxis with 6 months of on-demand therapy. Eighteen of 19 patients completed the survey or questionnaire before and after the on-demand therapy and prophylaxis periods. A general trend towards improved HRQoL after prophylaxis was observed for the 18 evaluable patients in all SF-36 dimensions except for vitality/energy and physical functioning. After prophylaxis, 'good responders,' defined as patients experiencing ≥ 50% reduction in bleeding, exhibited statistically and clinically significant differences in the physical component score (P = 0.021), role - physical (P = 0.042), bodily pain (P = 0.015), and social functioning (P = 0.036). Similarly, the EQ-5D health profile showed a trend towards improvement after prophylaxis in all evaluable patients. Among the good responders, improvements did not differ from those observed after on-demand treatment. EQ visual analogue scale values were slightly improved following prophylaxis for all evaluable patients and the EQ-5D utility index improved in the good responders only. During prophylaxis, patients missed significantly fewer days from school or work because of bleeding than during on-demand treatment (P = 0.01). In conclusion, by significantly reducing bleeding frequency in good responders, aPCC prophylaxis improved HRQoL compared with on-demand treatment.
Assuntos
Fator VIII/imunologia , Hemofilia A/tratamento farmacológico , Hemofilia A/psicologia , Isoanticorpos/imunologia , Protrombina/administração & dosagem , Adolescente , Adulto , Idoso , Criança , Pré-Escolar , Estudos Cross-Over , Feminino , Hemofilia A/imunologia , Humanos , Isoanticorpos/biossíntese , Masculino , Pessoa de Meia-Idade , Estudos Prospectivos , Qualidade de Vida , Inquéritos e Questionários , Adulto JovemRESUMO
BACKGROUND: Decision makers request increasingly for high levels of evidence when allocating resources in medical care. This is hardly feasible for rare diseases. The objective was to analyze clinical and economic aspects of different immune tolerance induction (ITI) strategies for children with severe haemophilia A and inhibitors. METHODS: A decision model, time frame 18 years (base case: 2 year old boy), was constructed from a German statutory health insurance (SHI) perspective. Compared were high-dose (HD) ITI, low-dose (LD) ITI, 'ITI with risk assessment', on-demand (OD) treatment with bypassing agents. Clinical data were derived from structured literature research and expert opinion. Sensitivity analyses were conducted for parameters with wide statistical ranges. RESULTS: Base case analysis: total costs for HD ITI amounted to 3.4 million with 40.9% ITI costs, 51 joint bleeds, 36 hospital days; LD ITI, 2.4 million with 21.4% ITI costs, 74 joint bleeds, 52 hospital days; 'ITI with risk assessment', 2.7 million with 27.6% ITI costs, 53 joint bleeds, 37 hospital days; OD treatment, 1.7 million, 146 joint bleeds, 104 hospital days. Incremental costs per bleed avoided with HD ITI decreased from 1 million to 0.15 million with increase of joint bleeds from 3 to 20 per year, when compared to 'ITI with risk assessment' in sensitivity analysis. CONCLUSION: 'ITI with risk assessment' is cost-saving with comparable outcomes to HD ITI. However, patient-related factors like bleeding frequency have to determine treatment decisions in individual patients. More clinical data is needed to increase the significance of model -calculations.
Assuntos
Fator VIII/imunologia , Fator VIII/uso terapêutico , Hemofilia A/economia , Hemofilia A/terapia , Tolerância Imunológica , Programas Nacionais de Saúde/economia , Doenças Raras/economia , Doenças Raras/terapia , Pré-Escolar , Análise Custo-Benefício , Técnicas de Apoio para a Decisão , Relação Dose-Resposta a Droga , Esquema de Medicação , Medicina Baseada em Evidências/economia , Fator VIII/antagonistas & inibidores , Alemanha , Alocação de Recursos para a Atenção à Saúde/economia , Hemartrose/economia , Hemartrose/imunologia , Hemartrose/terapia , Hemofilia A/complicações , Hemofilia A/imunologia , Humanos , Tempo de Internação/economia , Masculino , Cadeias de Markov , Computação Matemática , Modelos EconométricosRESUMO
BACKGROUND AND AIM: Diabetes mellitus is a widespread chronic disease. Diabetes prevalence was already analyzed in multiple complex studies. The goal of this analysis was the determination of the regional drug treatment prevalence and medication of diabetes mellitus in Germany. Simultaneously, we examined and validated the applicability of prescription data of the German statutory health insurance (SHI) as the basis for reliable and comparable prevalence estimations. PATIENTS AND METHODS: For the analysis, we used anonymized data from a prescription database as well as the member statistics of the German federal Ministry of Health. By using defined prescription profiles with anti-diabetic medication, we determined the diabetic patients and treatment schemes per Association of SHI Physicians. Subsequently, we calculated and described the drug treatment prevalence and medication. A differentiation between type 1 and type 2 diabetes was not possible. RESULTS: The total prevalence of drug-treated patients in Germany was 7.77%. The regional values ranged from 6.40% in Schleswig-Holstein until 11.37% in Saxony-Anhalt. The highest numbers of drug treatment prevalence were found in the East of Germany. Insulin treatment was frequent in Hamburg, but rare in Bavaria. Insulin combined with oral anti-diabetic medication was mainly dispensed in Saxony-Anhalt and Brandenburg, least in Hamburg. A treatment with one or more oral anti-diabetics was most common in Bavaria and Bremen, lowest in Rhineland-Palatinate and Mecklenburg-West Pomerania. CONCLUSION: We developed an easy to use evaluation algorithm for prescription data to estimate the drug treatment prevalence of a chronic disease. The estimated prevalence confirms the increasing trend reported from other studies and appears to be plausible in comparison. More research is required to validate and enhance the method.
Assuntos
Diabetes Mellitus/tratamento farmacológico , Diabetes Mellitus/epidemiologia , Hipoglicemiantes/uso terapêutico , Padrões de Prática Médica/estatística & dados numéricos , Prescrições/estatística & dados numéricos , Programas Médicos Regionais/estatística & dados numéricos , Geografia Médica , Alemanha/epidemiologia , Humanos , PrevalênciaRESUMO
Since 1982 an annual multicentric survey regarding the epidemiology of patients suffering of haemophilia is performed with support of haemophilia treating centres of any size. Again, the actual compilation is resting upon a broad database returning to over 30 years of inquiry well representing both the actual and retrospective status of mortality. Prompted was exclusively information about patients with haemophilia A, B and von Willebrand disease. In particular anonymous data concerning the preceeding 12 months about number of treated patients, type and severity of illness, HIV-status and detailed information about causes of death was inquired. This data was merged with existing data and analysed statistically. In the 2009/2010 survey, a total number of 9448 patients with bleeding disorders have been reported from 47 participating centres. Despite mortality from HIV in patients with haemophilia is keeping on decreasing, HIV still remains an important factor as an HIV/HCV coinfection seems to increase risk of progression of severe liver disease. In the actual investigation the findings of the foundation "Humanitäre Hilfe für durch Blutprodukte HIV-infizierte Personen" were compared for the first time to our data. Time trends were visualised with a moving average. Our investigation has a smaller number of deceased patients, but contains consistent data.
Assuntos
Infecções por HIV/mortalidade , Hemofilia A/mortalidade , Doenças de von Willebrand/mortalidade , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Criança , Pré-Escolar , Comorbidade , Feminino , Alemanha/epidemiologia , Humanos , Lactente , Recém-Nascido , Masculino , Pessoa de Meia-Idade , Prevalência , Medição de Risco , Análise de Sobrevida , Taxa de Sobrevida , Adulto JovemRESUMO
Haemophilia, a lifelong congenital bleeding disease, is a highly demanding disorder, due to the costs of its replacement therapy. In the absence of this pivotal treatment, life expectancy and quality of life are deleteriously affected. As illustration, we present a 14 years long follow-up of a patient with severe haemophilia A, treated sporadically with fresh plasma, cryoprecipitate and factor concentrates, who developed a giant iliopsoas pseudotumor. Since he was an infant, under on demand therapy with fresh frozen plasma, cryoprecipitate and low doses of factor concentrates he presented many spontaneous bleedings, developing multiple disabling arthropathies. At the age of 14 years, an iliopsoas hematoma occurred, which relapsed several times, developing an iliopsoas pseudotumour. After 5 years, sepsis with Klebsiella was diagnosed. A CT scan revealed fistula between the pseudotumor and the gut. Under antibiotics, the evolution of sepsis improved, but over a period of 10 months 5 episodes of haematemesis and melena, followed by one episode of macroscopic haematuria occurred; two months later he developed an inguino-crural mass, which fistulized through the abdominal wall. A mixt german-romanian team solved the clinical concern. After 108 hospitalization days and consumption of 104840 IU factor VIII he left the clinic in good condition. One year later, the temporary colostomy with anus praeter was closed. The follow-up reveals now, after almost 10 years with favourable outcome, that the patient is well, active within his family and profession.
Assuntos
Granuloma de Células Plasmáticas/complicações , Granuloma de Células Plasmáticas/terapia , Hemofilia A/complicações , Hemofilia A/terapia , Miosite/complicações , Miosite/terapia , Adolescente , Adulto , Granuloma de Células Plasmáticas/diagnóstico , Hemofilia A/diagnóstico , Humanos , Estudos Longitudinais , Masculino , Miosite/diagnóstico , Adulto JovemRESUMO
The aim of this study was to determine the clinical conditions of patients with haemophilia within Europe as recommended by the European Commission. In this multicentre, cross-sectional, ambispective study, conducted within 21 European countries patients' clinical data were collected, amongst others haemophilia type, severity, treatment pattern, use of factor products, bleeding, orthopaedic joint scores and infections. A total of 1400 patients, 84.3% with haemophilia A and 15.7% with haemophilia B were enrolled by 42 centres between 2004 and 2006. Thereof, 417 were children (30.0%) and 983 were adults (70.0%). About 70% of patients had severe factor deficiency (<1%). More than half of the adults were carriers of chronic infections (12.6% HIV, 55.8% HCV), compared to only 3.8% children (no HIV, 2.9% HCV). Patients were grouped according to per capita amount of clotting factor used in patients' region of residence in 2005: region 1: >5 IU; region 2: 2-5 IU; region 3: <2 IU. Paediatric and adult patients in region 3 had median numbers of three and eight joint bleeds, respectively, with worse joint scores compared to region 1 with zero and one bleed. Prophylactic therapy was used in only 31.3% children and 8.9% adults with severe haemophilia in region 3 compared to 93.7% and 54.1%, respectively, in region 1. Statistical analysis revealed that residence in areas with low factor consumption/availability is the most prominent risk factor for joint disease. Access of European patients with haemophilia to optimal care with safe factor VIII concentrates is limited and depends on the region of residence.
Assuntos
Hemofilia A/terapia , Hemofilia B/terapia , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Fatores de Coagulação Sanguínea/administração & dosagem , Fatores de Coagulação Sanguínea/economia , Criança , Pré-Escolar , Estudos Transversais , Europa (Continente)/epidemiologia , Acessibilidade aos Serviços de Saúde , Hemartrose/etiologia , Hemofilia A/complicações , Hemofilia A/economia , Hemofilia A/epidemiologia , Hemofilia B/complicações , Hemofilia B/economia , Hemofilia B/epidemiologia , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Prospectivos , Qualidade de Vida , Estudos Retrospectivos , Fatores de Risco , Adulto JovemRESUMO
We are entering a new phase in the management of patients with bleeding disorders such as haemophilia. This is the result of the positive effects that disease management strategies have had on patient longevity over the last 10-15 years. A greater number of individuals are entering middle- to old-age and, as a result, we face a new era of having to manage haemophiliac patients at risk of, or suffering from, age-related diseases. We can clearly learn from the experiences of geriatricians who have made many advances in the management of chronic disorders such as cardiovascular diseases and osteoporosis. However, the hypocoagulable state brings challenges of its own and it is important that we communicate our experiences so that the shared information can help drive improved levels of care and better clinical outcomes. In this article we look at factors that have impacted the life expectancy of patients with haemophilia over the last few decades, and we also review some of the early literature relating to cardiovascular risk management and the treatment of osteoporosis.
Assuntos
Doenças Cardiovasculares/complicações , Hemofilia A/complicações , Osteoporose/complicações , Idoso , Fatores de Coagulação Sanguínea/uso terapêutico , Densidade Óssea/fisiologia , Comorbidade , Hemofilia A/tratamento farmacológico , Hemofilia A/fisiopatologia , Humanos , Expectativa de Vida , Pessoa de Meia-Idade , Osteoporose/fisiopatologia , Fatores de RiscoRESUMO
Severe haemophilia results in increased mortality and poorer quality of life. Factor prophylaxis leads to a more normal life, but is very costly; most of the cost is due to the high cost of replacement factor. Despite its high cost, factor prophylaxis has been adopted throughout the developed world--even in different health care systems. We argue that there are at least five possible reasons why societies may value factor prophylaxis despite its cost: (i) it is directed towards an inherited disease, (ii) the treatment is largely directed towards children, (iii) the disease is rare and the overall cost to society is small, (iv) the treatment is preventative, and v) the high cost is largely the result of providing safe products. In an era of rising health care costs, there is a strong research agenda to establish the factors that determine the value of expensive therapies for rare diseases like haemophilia.
Assuntos
Fatores de Coagulação Sanguínea/economia , Fatores de Coagulação Sanguínea/uso terapêutico , Custos de Medicamentos , Hemofilia A/economia , Hemofilia A/terapia , Hemorragia/economia , Hemorragia/prevenção & controle , Humanos , Masculino , Medicina Preventiva/economiaRESUMO
A number of 513 consecutive patients (494-haemophilia A and 19-haemophilia B) from eight haemophilia treatment centers have been investigated with Bethesda assay for the presence of factor VIII or IX inhibitors. The overall prevalence of inhibitors was 15.20%, 18.48% in severe, 5.60% in moderate and 12.24% in mild forms. The prevalence was higher than reported in most of the western countries. The age at start of substitution (p = 0.9775), the frequent switching of factor concentrates (p = 0.8931) were not relevant factors for the development of inhibitors. It is worth to be mentioned the unexpectedly occurrence of inhibitors in prior inhibitor negative (6/72) patients (during surgical interventions) probably due to their previous scarce substitution, occurrence which seems not being connected with the continuous infusion modality of factor VIII administration (p = 0.8341). In controversial situations, in the field of low titer (≤ 1 BU/ml) inhibitors for a reliable interpretation of the results the performance of recovery index and half-life time assessment of FVIII/IX was undertaken.
Assuntos
Inibidores dos Fatores de Coagulação Sanguínea/sangue , Fator IX/antagonistas & inibidores , Fator IX/análise , Fator VIII/antagonistas & inibidores , Fator VIII/análise , Hemofilia A/sangue , Hemofilia A/epidemiologia , Adulto , Feminino , Alemanha/epidemiologia , Humanos , Masculino , PrevalênciaRESUMO
UNLABELLED: At present (2010), we investigate in a long term study on psychosocial aspects of haemophilia at the Munich Haemophilia Centre factors which could be held responsible for the large number of still living HIV+ patients while many patients died from AIDS or from diseases caused by HCV during the 1990ies. PATIENTS, METHODS: We retrospectively compare medical and psychosocial data of 15 HIV+ long term survivors (L-S), 15 HIV+ later on deceased patients and 15 HIV-, all of them deriving from the original investigation group from 1985 including 52 patients (30 HIV+, 22 HIV-). We prove the validity of our psychosocial factors by the SF 36 which currently serves as gold standard. The actual psychosocial factors and medical parameters of the 15 L-S are checked against the data of the 15 HIV- patients. RESULTS, CONCLUSION: In 1985 already, we retrospectively found significantly higher values regarding psychosocial factors within the group of the L-S as opposed to the later on deceased ones. Highly significant are the facts that more than 90 % of HIV+ L-S have had a good relationship to their fathers and are still employed today in contrast to the deceased HIV+ patients. At present, the differences regarding psychosocial factors between HIV+ L-S and HIV-negative patients are vanishing.