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Mashup Score: 0Breaking ground in haemophilia B gene therapy: insights from the HOPE-B trial and beyond - 1 month(s) ago
Since the first gene therapy clinical trial for haemophilia, which used adeno-associated viral (AAV) vectors for liver-directed delivery of wild-type factor IX, remarkable advancements have ensued.1 In the dose-escalation phase 1/2 study, seven individuals with severe haemophilia B (factor IX activity<1%) were enrolled.2 These participants received hepatic-directed administration of an AAV2-factor IX construct, regulated by a liver-specific promoter (rAAV2-hAAT-F9-WT), delivered via the hepatic artery.
Source: www.thelancet.comCategories: General Medicine News, Onc News and JournalsTweet
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Mashup Score: 9
Primary prophylaxis to prevent spontaneous bleeds and development of haemophilia arthropathy was first recommended by the World Federation of Hemophilia (WFH) in 1994 for people with severe haemophilia A (ie, factor VIII <1%).1 Haemophilia severity has been defined by factor VIII concentrations, based on a 1958 paper describing the relationship between bleeding and factor concentrations in 135 patients,2 although the fact that there is heterogeneity within these severity thresholds is recognised.
Source: www.thelancet.comCategories: General Medicine News, Onc News and JournalsTweet-
"Re-evaluation of previous assumptions of factor VIII break points for severity distinctions is needed to close equity gaps. One consideration would be to redefine mild haemophilia..." Comment by @acweyand, Lynn Malec & Steven W Pipe #bleedingdisorders https://t.co/KfDtaCxoFD https://t.co/d5opzQCk78
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Mashup Score: 1Emicizumab prophylaxis in patients with acquired haemophilia A (GTH-AHA-EMI): an open-label, single-arm, multicentre, phase 2 study - 6 month(s) ago
This study suggests that emicizumab prophylaxis prevents bleeding in patients with acquired haemophilia A and that immunosuppressive therapy can be deferred while patients are receiving this treatment. The low number of thromboembolic events, severe infections, and fatalities observed in this study are promising.
Source: www.thelancet.comCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
NEW: results from the GTH-AHA-EMI trial show that emicizumab prophylaxis protects patients with acquired #haemophilia A from bleeding and allows deferral of immunosuppression during the first 12 weeks after diagnosis #hemophilia #bleedingdisorders https://t.co/Cc07bH1KJ6 https://t.co/RqMjNMquzi
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Mashup Score: 0
Acquired haemophilia A is a rare disorder that affects older adults with no previous bleeding history who develop life-threatening bleeding, accompanied by a prolonged activated partial thromboplastin time that fails to correct in a 1:1 mix and caused by a specific autoantibody to factor VIII.1,2 The aim of acquired haemophilia treatment is two-fold: to stop the bleeding, and to eradicate the inhibitor. To date, standard treatment includes haemostatic bypassing agents to prevent bleeding and immunosuppressives to eradicate the inhibitor, which is 90% effective.
Source: www.thelancet.comCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
"The key message to take from this study is that early rapid emicizumab loading is safe and effective alone, without immunosuppression, in preventing bleeding in acquired #haemophilia A" #hemophilia #bleedingdisorders Linked comment by @ragnmv https://t.co/M0DfipV6bo
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Mashup Score: 17
Explore the current issue of The Lancet Haematology, a monthly journal dedicated to publishing content that advocates change in, or illuminates, haematological clinical practice
Source: www.thelancet.comCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
Our August issue is out! Packed with #sicklecell disease content: our Commission, a #GBD study on #sicklecell, and several associated pieces Also trials on AML, #HIV-related HL, and #menstrualbleeding in VWD #leusm #lymsm #bleedingdisorders ➡️https://t.co/EyrqUdHpyd https://t.co/Uva1tqkJHW
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Mashup Score: 0AAV-based gene therapy in hemophilia A and B: valoctocogene roxaparvovec & etranacogene dezaparvovec - 10 month(s) ago
Johannes Oldenburg, MD, University Clinic Bonn, Bonn, Germany, discusses the promise of AAV-based gene therapies in hemophilia A and B…
Source: VJHemOncCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
Johannes Oldenburg (@UniBonn) spoke with us at #ISTH2023 on the promise of AAV-based gene therapies in hemophilia A and B: 🎥: https://t.co/DvElCZwdEr @isth #Hemophilia #RareDiseases #BleedingDisorders
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Mashup Score: 13Information for Adolescents - 11 month(s) ago
Bleeding disorders in tweens and teens may go undiagnosed
Categories: General Medicine News, Latest HeadlinesTweet-
Test your knowledge of bleeding disorders. Share free social media resources from CDC’s new adolescent toolkit to help raise awareness of bleeding disorders among tweens and teens. https://t.co/mn92IeEkzd #HeavyPeriods #BleedingDisorders #HeavyBleeders https://t.co/X1zLO7da6e
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Mashup Score: 31I Want to Know - 11 month(s) ago
Congratulations! You are taking the first step in better understanding your bleeding symptoms. Simply respond to the questions* below and click Submit at the end. Then we will tell you if your symptoms show if you are maybe at risk for a bleeding disorder . Either way, we have lots of information and resources once you find out, so get started now, in your journey to feeling better.
Source: Better You KnowCategories: General Medicine News, Latest HeadlinesTweet-
If you have heavy periods, talk to your doctor to find out what is causing them. Think you might have a bleeding disorder? Take a quiz and find out your risk today: https://t.co/jPjHI50Ral #HeavyPeriods #BleedingDisorders #HeavyBleeders https://t.co/yMojPxZgPp
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Mashup Score: 0Information for Adolescents - 11 month(s) ago
Bleeding disorders in tweens and teens may go undiagnosed
Categories: Latest Headlines, PediatricsTweet-
Help raise awareness of bleeding disorders among tweens and teens! Use CDC’s new adolescent toolkit to share information and graphics on social media: https://t.co/yLJo5HaVqk #HeavyPeriods #BleedingDisorders #HeavyBleeders https://t.co/AI5VQeQnDU
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Mashup Score: 1
In participants with haemophilia A or B without inhibitors, fitusiran prophylaxis resulted in significant reductions in annualised bleeding rate compared with on-demand clotting factor concentrates and no bleeding events in approximately half of participants. Fitusiran prophylaxis shows haemostatic efficacy in both haemophilia A and haemophilia B, and therefore has the potential to be…
Source: The Lancet HaematologyCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
ICYMI: Fitusiran, a subQ investigational siRNA therapeutic, shows haemostatic efficacy as prophylaxis in both #haemophilia A and B Check the results of the ATLAS A/B study https://t.co/egppH04LT7 And @TheLancet ATLAS-INH #hemophilia #bleedingdisorders https://t.co/98c94qIKKE
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"Contrary to the observed trends in haemophilia A... individuals with haemophilia B undergoing AAV-based gene therapy appear to exhibit a sustained and consistent expression of factor IX" Linked comment by Margareth Ozelo #bleedingdisorders https://t.co/Y2v9MEQSzg