Recombinant ADAMTS13 should be the standard of care for patients with congenital TTP. FFP is an inferior product both in terms of efficacy and safety. A paper in the journal Blood describes the experience from Japan in 14 patients. lnkd.in/eXg345SD

For anybody interested in birds, this is a great book. The author tried to learn more about birds by building a shed/hide close to his home in the south of England. Very well researched and informative. I learnt a lot about the birds we encounter in the UK.

If you are interested in birds, the Greek island of Lesbos is a great place to visit this time of the year, as it coincides with the spring migration. We saw 170 species of bird in one week thanks to the skills of "Mr Lesbos Birding" Howard Vaughan, who also took the bird pictures below.

My commentary on PITT (PF4-associated immune thrombocytopenia and thrombosis) has just been published in the British Journal of Haematology. This group of disorders is more than HIT and VITT and we should be looking for it more widely. onlinelibrary.wiley.com/doi/10.1111/...

Our editorial on the Paradox of Choice in Hemophilia has just been published in the journal Research and Practice in Thrombosis and Haemostasis. Like all papers in this journal, it is freely available. tinyurl.com/4kw9hcth @rpth.bsky.social #hemophilia #haemophilia

Thank you. In addition, there were no cases of malignancy following gene therapy, but the number of patients and years of follow-up is small.

Ben Samelson-Jones, MD, PhD@samelsonjones.bsky.social · last yr.

In addition to demonstrating long-term durability of rAAV #genetherapy, here we show: 1) Hemostatic potential of FIX Padua with multiple major trauma not needing additional treatments. 2) Importance of limiting hepatotoxic exposures with binge drinking associated with irreversible FIX loss.

A paper in @NEJM describes 14 patients with #hemophilia B who had the Pfizer gene therapy. Their achieved FIX levels remained stable over a median of 5.5 years. Confirmation that durability following hemophilia B gene therapy is very different to that in hemophilia A. tinyurl.com/2b2zzc37

An article in the current issue of @nejm.org discusses the impact of the US foreign assistance cuts on global health. Some of the impacts are not readily obvious. The authors advocate reforming the foreign assistance program rather than dismantling it.

The BIC meeting in Italy is always a great meeting, showcasing the latest science and with plenty of time for discussion. Remember the deadline for abstract submission is the 30th April 2025. #hemophilia #VWD #TMA #TTP

Today is #WorldHemophiliaDay and this year's theme is that women and girls bleed too. Despite the development of brilliant treatments to treat severe #hemophilia primarily in men, the diagnosis and treatment of women have not received as much attention.

This is the press release about the new #hemophilia B gene therapy that has been approved in China. So there are now 3 approved gene therapies for hemophilia B, 2 in US/Europe approved by the FDA/EMA and 1 in China approved by the Chinese regulator. tinyurl.com/2jc74nb9

Thank you for the copies of the paper on the UK Infected Blood Inquiry that was published in @BloodJournal. It is interesting to see the section on lessons learned. Despite the @bloodinquiry, the UK continues to use cryo and FFP without viral inactivation. #hemophilia

Article on Generative AI in medicine published today in @nejm.org. It discusses the potential uses and risks. One issue you can be sure of, is that AI will be embedded in the everyday practice of medicine in the very near future, even more than today. tinyurl.com/4sbjpjvs

Join Cedric Hermans and @profmakris.bsky.social in this E-learning Insight session as they share practical strategies and key considerations to help you navigate the world of scientific publishing. Learn how to write with purpose and make your research stand out.📺 academy.eahad.org/eahad/2023/e...

If you are a scientist who is not using AI yet, this article from today's @nature.com journal will be of great help. Whether we like it or not, AI will be everywhere very soon, just like the internet.

Everybody has an opinion about racism, but as the author of this book explains, the plural of anecdote is not data. This is a serious book looking at the scientific evidence around racism. It will not come as a surprise to read that racism is still in everything.

Pseudoscience. #MedSky

Vaccination skeptics should have a look at this figure. It shows the impact of the introduction of RSV vaccination for patients aged 75-80 on hospital admissions in the UK. tinyurl.com/5d48u7mm

Important paper: In patients with cancer and venous thrombosis, extended anticoagulation with low dose Apixaban is as good as full dose Apixaban in preventing thrombosis recurrence and is associated with less bleeding. tinyurl.com/yyfuan35

Professor Christine Lee reflects on some of the conclusions of the UK Infected Blood Inquiry. The manuscript has just been published by the journal Haemophilia. @haemophilia_jnl #hemophilia. The PDF of the manuscript is freely available at: tinyurl.com/mvh2mk22

Our paper on the severity of #hemophilia has just been published in the journal Hemasphere. Baseline clotting factors do not always accurately reflect the severity of the disease. tinyurl.com/3hn9hhrn

Our paper on inhibitor trends is now published in the journal Haemophilia. The number of inhibitors in previously untreated patients has markedly reduced, but it is possible that this is due to delayed exposure to FVIII. tinyurl.com/2mr3t5e3

The German authorities have today concluded their negotiations with CSL Behring and agreed the price they will pay for the #hemophilia B gene therapy (Hemgenix). Does anybody know how much they will pay? tinyurl.com/mpnc9kk7

CSL Behring and GKV-Spitzenverband Agree on Reimbursement Price for HEMGENIX® – Europe's First Gene Therapy for Haemophilia B

Marburg, Germany, 2 April 2025 – CSL Behring and the GKV-Spitzenverband (National Association of Statutory Health Insurance Funds) have successfully concluded negotiations on the reimbursement p...

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Comprehensive review of gene therapy in #hemophilia accepted for publication by Molecular Therapy. The pre-proof is on line at tinyurl.com/3sb4nuex

Paying reviewers to assess papers submitted to scientific journals results in more rapid reviews, but the quality does not change. News article in @nature.com. tinyurl.com/bdfxf9xy

Publishers trial paying peer reviewers — what did they find?

Two journals embarked on efforts to compensate reviewers, with different results.

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Incredible: dropbox.com/scl/fi/qy2ja.... This podcast was produced 100% by AI. It is entirely based on the editorial by myself and Brian O'Mahony published in @rpth.bsky.social. It is 21 min and remarkably accurate. Thank you, Eugene Fan, for showing me what is possible.

This is an amazingly prophetic book written by two Harvard professors who teach political science. Although published in 2018, it clearly describes what is happening in the world and the US in particular today. "History does not repeat itself; it rhymes."

Do you want the latest updates in #platelet research? 🔥 Check out these Illustrated Capsules from the Advanced Course in Platelet Research held in Spain in September 2024. Supported by @isth.org and the SETH www.rpthjournal.org/article/S247...

As expected the FDA has today announced that it has approved Fitusiran for individuals aged 12+ with #hemophilia A or B with or without inhibitors. Commercial name is Qfitlia - they must have tried hard to find a difficult to pronounce name. tinyurl.com/mr4xmawt

FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors

Today, the FDA approved a novel treatment for hemophilia A or B, with or without factor VIII or IX inhibitors (neutralizing antibodies).

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Tomorrow, March 28th, is the big day for Fitusiran as the FDA is expected to give their decision. Will they approve it? Probably. Will it have a black box warning? We do not know, but since they did not use them for Marstacimab and Concizumab, they will probably not. #hemophilia