Re-personalization and stratification of hemophilia care in an evolving treatment landscape.

Re-personalization and stratification of hemophilia care in an evolving treatment landscape. Hematology. 2019 Dec;24(1):737-741 Authors: Hart DP, Kessler CM, Aledort L Abstract Hemophilia therapeutics are evolving rapidly. Comprehensive care must also evolve to embrace this change. Online tools and guidelines are widely available to optimize prophylaxis with conventional clotting factor concentrates using an individual's predicted pharmacokinetic profile. Novel hemostatic agents (e.g. biphenotypic antibody) are also becoming widely available, with other agents with differing mechanisms of action in final stages of trial. Contemporary issues including challenges of prophylaxis; bleed treatment; laboratory monitoring and inhibitor risk/surveillance are summarized in this narrative review, focusing on how a re-personalization of education and treatment will be necessary to meet these challenges of the rapidly changing therapeutic landscape. PMID: 31711380 [PubMed - in process]
Source: Hematology - Category: Hematology Tags: Hematology Source Type: research

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CONCLUSION: Whereas People With Advanced Hemophilic Arthropathy Of The Ankle Are Prone To Ameliorate Pain And Range Of Motion Following Tar, There Is Deficient Knowledge To Regularly Recommend Its Use. Adverse Events And Infection Percentages Are Disturbing. Moreover, The Lack Of Survival Analysis Knowledge Makes It Difficult To Assess The Benefit To People With Hemophilia. Tar Is A Demanding Surgical Procedure And Its Survival Is Not Comparable To That After Hip Or Knee Arthroplasty. PMID: 31820705 [PubMed - as supplied by publisher]
Source: Cardiovascular and Hematological Disorders Drug Targets - Category: Drugs & Pharmacology Tags: Cardiovasc Hematol Disord Drug Targets Source Type: research
Abstract Approximately 50% of female carriers of hemophilia A have factor VIII (FVIII) levels below 0.5 IU/dL and may be categorized as having mild hemophilia. Females with hemophilia may go undiagnosed for years because the most common symptoms - menorrhagia and bleeding after childbirth - also occur in females without hemophilia. Females with hemophilia can exhibit increased bleeding tendencies despite current guidelines of expected, adequate FVIII levels. The cases described illustrate the clinical variability and presentation of hemophilia in females and highlight the importance of a timely diagnosis, effectiv...
Source: Acta Haematologica - Category: Hematology Authors: Tags: Acta Haematol Source Type: research
We report here a simple and highly specific method for detecting LA. MATERIALS AND METHODS: We examined 66 plasma samples obtained from LA-positive (LA) and 75 from LA-negative (non-LA) subjects, which included patients with acquired hemophilia and coagulation disorders, as well as from 43 healthy volunteer samples as normal controls. Activated partial thromboplastin time (APTT) was determined by adding 20 mmol of CaCl2 (Ca-APTT) or 25 mmol of a mixture of Mg and Ca (Mg-APTT). The ratio of Mg-APTT/Ca-APTT was then calculated and used as the Mg/Ca Index. RESULTS: The Mg/Ca Index value for the LA group wa...
Source: International Journal of Laboratory Hematology - Category: Hematology Authors: Tags: Int J Lab Hematol Source Type: research
CONCLUSION: Recombinant FIX Fc fusion protein activity was found to be similar to that of rFIX or pdFIX by the one-stage clotting assay. However, minimizing interlaboratory variability is vital for optimizing future patient care. PMID: 31820573 [PubMed - as supplied by publisher]
Source: International Journal of Laboratory Hematology - Category: Hematology Authors: Tags: Int J Lab Hematol Source Type: research
CONCLUSION: Oesophageal bullous detachment is rare in AIBD but, as seen here, it may be responsible for massive haemorrhage, especially in the event of associated AHA. This feature underscores the need for evaluation of haemostasis in the early stages and during relapses for all patients with AIBD. PMID: 31831217 [PubMed - as supplied by publisher]
Source: Annales de Dermatologie et de Cenereologie - Category: Dermatology Authors: Tags: Ann Dermatol Venereol Source Type: research
This study has been followed by unparalleled advances in gene therapy for hemophilia A and B, leading to clotting factor activity approaching normal or near-normal levels associated with a "zero bleed rates" in previously severely affected patients following a single administration of AAV vectors. Thus, AAV gene therapies are likely to alter the treatment paradigm for hemophilia A and B. This review explores recent progress and the remaining limitations that need to be overcome for wider availability of this novel treatment of inherited bleeding disorders. PMID: 31808868 [PubMed - in process]
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
Authors: Menegatti M, Biguzzi E, Peyvandi F Abstract Autoantibodies toward clotting factors may develop in people suffering from autoimmune or neoplastic diseases, after drug intake or even in subjects without apparent conditions. They are more commonly directed against factor VIII (FVIII) or von Willebrand factor leading to acquired hemophilia A or acquired von Willebrand syndrome, respectively. Rarely, autoantibodies develop against other clotting factors, such as fibrinogen, FII, FV, FVII, FX, FXI, and FXIII. The clinical picture of an acquired bleeding disorder includes a wide spectrum of clinical manifestation...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
PMID: 31813089 [PubMed - as supplied by publisher]
Source: International Journal of Hematology - Category: Hematology Authors: Tags: Int J Hematol Source Type: research
Condition:   Hemophilia A, Acquired Intervention:   Drug: Emicizumab Injection Sponsors:   GWT-TUD GmbH;   Hoffmann-La Roche;   Hannover Medical School Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Authors: Singh A, Mehta S, Goyal LK, Mehta S, Sharma BS Abstract Aim: To assess effect of low dose prophylaxis in hemophilics in terms of bleeding, joint function, QoL and cost-effectiveness. Methods: Analytic study done during one year among 70 adult hemophilics. In observation period (12 weeks), on-demand factor and during prophylaxis (12 weeks), low dose factor was given (Factor VIII 10 IU/KgBW biweekly for haemophilia A and Factor IX 20 IU/KgBW weekly for haemophilia B). Clinical joint assessment was done by Gilbert score and improvement by WFH definitions. Results: Bleed reduced by 68.99% in moderate h...
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research
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