RESUMO
BACKGROUND: Extended half-life (EHL) factor VIII (FVIII) and IX (FIX) products are intended to decrease the burden of prophylaxis for patients with haemophilia A or B. Whether these newer concentrates have led to meaningful clinical practice change remains vague. AIM: To characterize the longitudinal use of standard (SHL) and EHL factor concentrates at haemophilia treatment centres (HTCs), using the ATHNdataset, a US database of 138 ATHN-affiliated HTCs. METHODS: Factor concentrate use among moderate and severe haemophilia A and B patients without inhibitors was analysed at three time points over 18 months. RESULTS: Use of EHL concentrates rose from 10% of patients to 22% during this study. EHL FVIII prophylaxis is prescribed to the minority of patients, 28%; EHL FIX now predominates for prophylaxis, 52%. Rates of prescribed EHL products varied significantly by age group and HTC region. Median prescribed prophylaxis for SHL compared to EHL products was FVIII 6240 and 5200 and FIX 6968 and FIX 3900 IU/kg/y, respectively. On-demand EHL use has grown but has minimal contribution to overall usage (2%). CONCLUSION: Haemophilia treatment centre region and patient age impact the rate of adoption of EHL products; however, EHL prescribing continues to rise nationally, particularly for EHL FIX. Careful attention to annual cost of prophylaxis is imperative as the decrease in median EHL prophylaxis consumption is not offset by the higher unit cost of these products. It is unclear how further growth in use of EHLs will be impacted by emerging non-factor replacement and gene therapies.
Assuntos
Custos e Análise de Custo , Fator IX/economia , Fator IX/uso terapêutico , Fator VIII/economia , Fator VIII/uso terapêutico , Hemofilia A/tratamento farmacológico , Hemofilia B/tratamento farmacológico , Adolescente , Adulto , Fatores Etários , Criança , Prescrições de Medicamentos/economia , Prescrições de Medicamentos/estatística & dados numéricos , Fator IX/farmacocinética , Fator VIII/farmacocinética , Feminino , Geografia , Meia-Vida , Hemofilia A/metabolismo , Hemofilia B/metabolismo , Humanos , Estudos Longitudinais , Masculino , Estados Unidos , Adulto JovemAssuntos
Anemia Falciforme/complicações , Sobrecarga de Ferro/diagnóstico , Sobrecarga de Ferro/etiologia , Adolescente , Adulto , Anemia Falciforme/sangue , Anemia Falciforme/terapia , Criança , Pré-Escolar , Estudos Transversais , Gerenciamento Clínico , Transfusão de Eritrócitos/efeitos adversos , Feminino , Ferritinas/sangue , Humanos , Lactente , Sobrecarga de Ferro/sangue , Sobrecarga de Ferro/terapia , Masculino , Pessoa de Meia-Idade , Adulto JovemRESUMO
Case of LMC in a BRCA2-mutated breast cancer patient shows clinical improvement with Olaparib therapy.
Assuntos
Proteína BRCA2 , Neoplasias da Mama , Mutação em Linhagem Germinativa , Carcinomatose Meníngea , Ftalazinas , Piperazinas , Humanos , Ftalazinas/uso terapêutico , Feminino , Neoplasias da Mama/tratamento farmacológico , Neoplasias da Mama/genética , Neoplasias da Mama/patologia , Piperazinas/uso terapêutico , Proteína BRCA2/genética , Carcinomatose Meníngea/tratamento farmacológico , Carcinomatose Meníngea/secundário , Carcinomatose Meníngea/genética , Pessoa de Meia-Idade , Inibidores de Poli(ADP-Ribose) Polimerases/uso terapêuticoRESUMO
Background: During pregnancy and in the postpartum period women are at increased risk of venous thromboembolism (VTE) owing to hypercoagulability and mechanical issues, as well as nonpregnancy conditions including inherited and acquired thrombophilia. Although guidelines exist for the use of thromboprophylaxis in this setting, there are differences in the specifics of the recommendations among expert societies. We assessed the current practice patterns of North American providers in the prevention of pregnancy-associated VTE in women with thrombophilia. Methods: A survey was created and distributed with case studies and questions addressing VTE prevention during the antepartum and postpartum periods. Results: Surveys were completed by 28% of adult providers queried, with broad geographic representation. There was consistent use of a prophylactic dose of low-molecular weight heparin (LMWH) ante- and postpartum for individuals with low-risk thrombophilia and past estrogen-provoked VTE but a lack of a consensus of anticoagulant (AC) use and dose in individuals with higher risk thrombophilia. There was variability in the dose selection and monitoring of AC when using induction versus spontaneous labor, with 47% of providers switching from LMWH to unfractionated heparin for those not having a scheduled delivery, and there were differences in the duration of postpartum prophylaxis based upon delivery mode. Conclusion: In this survey of North American experienced specialists' responses to a variety of commonly encountered scenarios of thrombophilia and pregnancy and the management of AC were not always consistent with published guidelines.
Assuntos
Trombofilia , Tromboembolia Venosa , Adulto , Anticoagulantes/efeitos adversos , Feminino , Heparina , Heparina de Baixo Peso Molecular/uso terapêutico , Humanos , América do Norte , Padrões de Prática Médica , Gravidez , Fatores de Risco , Trombofilia/tratamento farmacológico , Tromboembolia Venosa/tratamento farmacológico , Tromboembolia Venosa/prevenção & controleRESUMO
With advances in care, increasing numbers of people with hemophilia (PWH) achieve near-normal life expectancies and present with typical age-related cardiovascular conditions. Evidence-based guidelines for medical or surgical management of cardiovascular conditions in individuals with hemophilia are limited. Published recommendations exist for the management of some common cardiovascular conditions (eg, ischemic heart disease, atrial fibrillation), but identifying optimal strategies for anticoagulant or antithrombotic therapy constitutes the primary challenge of managing nonoperative cardiovascular disease (CVD) in PWH. In general, as long as factor concentrates or other hemostatic therapies maintain adequate hemostasis, the recommended medical and surgical management of CVD in PWH parallels that in individuals without hemophilia. The presence of factor inhibitors complicates hemophilia management. Published outcomes of CVD treatment in PWH are similar to those in the general population. Specific knowledge about factor replacement, factor inhibitors, and disease-specific treatment distinguishes the cardiovascular care of PWH from similar care of individuals without this rare bleeding disorder. Furthermore, a multidisciplinary approach incorporating a hematologist with an onsite coagulation laboratory, ideally associated with a hemophilia treatment center, is integral to the management of CVD in PWH.
Assuntos
Coagulação Sanguínea/efeitos dos fármacos , Fármacos Cardiovasculares/farmacologia , Doenças Cardiovasculares , Hemofilia A , Hemofilia B , Doenças Cardiovasculares/complicações , Doenças Cardiovasculares/tratamento farmacológico , Consenso , Gerenciamento Clínico , Hemofilia A/sangue , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemofilia B/sangue , Hemofilia B/complicações , Hemofilia B/tratamento farmacológico , Humanos , Conduta do Tratamento MedicamentosoRESUMO
Little information is available regarding late relapse in patients with T-lymphoblastic leukemia/lymphoma (T-LBL). Because of the aggressive nature of this disease, relapse is common and often happens early. Late relapses are rare and generally occur within a few years after initial remission. The relapse rate after 3 years has been reported to steadily decrease over time yet does not parallel with cure. We report a case of a 26-year-old female with T-LBL and relapse 16 years after her first remission with successful treatment with HyperCVAD and L-asparaginase.
RESUMO
Hemophilia type A is a rare inherited bleeding disorder with a diversity of clinical manifestations ranging from persistent bleeding after minor trauma, spontaneous deep muscle or joint hemorrhage, to intracranial hemorrhage. As an X-linked disorder, hemophilia is rare in females and therefore there is little experience with pregnancy and no standardized guidelines to prevent bleeding antepartum, at delivery, and postpartum. We report the clinical course and management of a woman with severe hemophilia A who on two occasions had uncomplicated pregnancies and vaginal deliveries at term utilizing bolus recombinant factor VIII concentrate.
RESUMO
BACKGROUND: Atrial thrombus formation in patients with atrial flutter raises concerns of stroke risk. We investigated patients with isthmus-dependent atrial flutter for coagulation abnormalities before and after cardioversion to sinus rhythm by catheter ablation, and evaluated the relationship of the abnormalities to the echocardiographic risk markers of stroke. METHODS AND RESULTS: Plasma samples were drawn prior to insertion of catheters, immediately after the procedure, and 24 hours afterwards. At baseline, coagulation abnormalities were found in 22 out of 25 patients (88%). von Willebrand factor antigen (vWF-Ag) and factor VIII:C were elevated in 17 patients (68%) and 15 patients (60%), respectively. At baseline, mean plasma levels of vWF-Ag (250.1 +/- 144.4%) and factor VIII:C (215.0 +/- 77.1%) were increased. Key markers of thrombin generation, thrombin-antithrombin III complex (TAT; 47.8 +/- 30.9 microg/L vs 14.5 +/- 13.8 microg/L; p < 0.05) and prothrombin fragments 1.2 (F1.2; 2.5 +/- 0.5 nmoL/L vs 1.2 +/- 1.0 nmoL/L) were significantly elevated in the presence of spontaneous echo contrast. Further, both markers of thrombin generation inversely correlated with left atrial appendage emptying velocity (r = -0.42 and -0.63, p < 0.05). Levels of TAT and F1.2 increased after conversion and ablation. CONCLUSIONS: Endothelial-dependent coagulation factors were enhanced in most patients with atrial flutter. Spontaneous echo contrast and decreased atrial contractility were associated with increased thrombin generation. After conversion and ablation, an increase in thrombin generation and fibrinolysis suggest a transient pro-thrombotic state.