ProfMakris
Emeritus Professor of Haemostasis and Thrombosis in Sheffield, UK. I tweet about thrombosis, hemophilia, COVID-19, medicine, NHS & AI. Opinions are my own.
- 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.
- 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
- Reposted by ProfMakrisJoin 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.
- Reposted by ProfMakrisPseudoscience. #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
- 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
- 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."
- Reposted by ProfMakrisDo 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
- 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
- If you are still unsure whether to ask for your data to be deleted by 23andMe and any retained samples destroyed, have a read of this article in the New England Journal of Medicine @nejm.org.
- Our editorial on #hemophilia treatments and the paradox of choice is now available in the journal Research and Practice in Thrombosis and Haemostasis. www.rpthjournal.org/article/S247...
- Von Willebrand disease associated angiodysplasia can be challenging to manage. I suspect the best treatment will be targeting angiogenesis rather than VWD. Excellent review on the subject in Haematologica. tinyurl.com/vu7n9w5h
- If you have used 23andme.com for analysis of your genome like me, you may wish to read this document from the California Attorney General about requesting deletion of your data and destruction of your sample before the company is taken over. oag.ca.gov/news/press-r...
- I bought this book when META/Facebook (who claim to believe in free speech) got a Gag order to stop the author promoting the book! The author was a senior employee at Facebook, and this is her memoir. What she writes about Facebook will shock all readers because the results impact everybody.
- Reposted by ProfMakrisI am running for Clinical VP of the RCP. For those who don't know me, here is a potted bio. 🧵
- Reposted by ProfMakrisAccess to affordable rivaroxaban 2.5mg tablets could be a game changer for patients with PAD. This drug, when combined with low-dose aspirin, prevents MI, stroke, and limb amputations!
- The generic versions of the Direct Oral Anticoagulants (DOACs) are coming. First FDA approval for generic Rivaroxaban (Xarelto) 2.5mg tablets. The real need is for the 10, 15, and 20 mg tablets; let's hope the delay is not too long. Also for the other DOACs. tinyurl.com/ypmmmnmf
- The generic versions of the Direct Oral Anticoagulants (DOACs) are coming. First FDA approval for generic Rivaroxaban (Xarelto) 2.5mg tablets. The real need is for the 10, 15, and 20 mg tablets; let's hope the delay is not too long. Also for the other DOACs. tinyurl.com/ypmmmnmf
- The new dosing method for Fitusiran appears to provide equal efficacy without thrombotic events. The number of patients, however, is very small. Safety will only be demonstrated with real world data on a much larger number of patients. tinyurl.com/mupbhanr
- Reposted by ProfMakrisPublic opinion of Putin and Trump I'm Spain, France, UK and Italy. For some peculiar reason the graphs look better on their side.
- Reposted by ProfMakris"The defeat of #Ukraine would be the defeat of Europe. The Baltic States, Georgia and Moldova are already on the list. Putin’s goal is to return to Yalta, where half the continent was ceded to Stalin." - French Senator Claude Malhuret made an excellent speech.
- Reposted by ProfMakrisFrench Senator Claude Malhuret: "Washington has become Nero’s court, with an incendiary emperor, submissive courtiers and a jester high on ketamine... We were at war with a dictator, we are now at war with a dictator backed by a traitor."
- Obituary of an Australian blood donor. Despite having only one lung, he donated 1173 times between 1954 and 2018 and contributed to every batch of anti-D made in Australia. Amazingly, in Australia, the maximum age for blood donation is 81 years. tinyurl.com/yme2hck8
- Editorial in the @NEJM about the current assault on science in the US. "As editors, we should continue to do what we think makes a difference.
- Reposted by ProfMakrisI often use Google Scholar as a reliable alternative tool.
- Reposted by ProfMakris
- Reposted by ProfMakrisEuropePMC relies on a feed (feeds ?) from PubMed. As I understand, it can run on its own, but I do not know how it will get updated and synchronised with PubMed. Though it does collect some data separately. I hope EuropePMC had prepared as much as they could for this predictable event.
- Does anybody know how independent the European site (europepmc.org) is from PubMed? Can EuropePMC go it alone without any links to PubMed? 2/2 #PubMed
- With Kennedy/Trump/Musk in charge, it is possible that PubMed will be taken down permanently (unlikely), charge for use if outside the US (possible), decide what is included or excluded (probable). 1/2 #PubMed
- PubMed is down. I was hoping this was temporary, but this morning it is still down. PubMed is the site I use most for medicine. I hope this is not a Kennedy/Trump action. The European site europepmc.org works and is similar, but I do not know the relationship to PubMed.
- I really enjoyed this book about misinformation and disinformation in society and social media. Tim Caulfield is a real expert in the area and is someone I trust and follow on social media. Every single person needs to be aware of the contents of this book.
- The UK NHS will offer Efa (Altuviiio in US, Altuvoct in Europe) for the treatment of #hemophilia A. To call it a miracle drug when we have so many treatments already is a tad much. I also do not believe the NHS will pay £510,000 per year. This is 5x the cost of emicizumab. tinyurl.com/mr2xym3t
- Unidentified infectious disease affecting over 1000 persons with 60 deaths in the Democratic Republic of the Congo. News story in today's online British Medical Journal. Important surveillance work by the WHO. It is such a pity that some countries are breaking support for the WHO.
- Important lesson when regulatory approval is based on questionable secondary endpoints. Just because a drug is new, and more expensive, does not mean it is better than current treatments. #FDA #EMA #NICE #hemophilia
- The appetite for the current versions of gene therapy in #hemophilia A and B appears to be waning. Now we only have one approved product for each. For now, there is a lot of enthusiasm for the "rebalancing" therapies, but how long will it last once they are used under real world conditions?
- Pfizer has stopped the global development of Beqvez, their approved #hemophilia B gene therapy. It had a price of $3.5million per dose. tinyurl.com/ympbesmc
- This is one of the most moving, inspirational books you will ever read. If you do not have the time, have a look at the extraordinary lecture on YouTube: tinyurl.com/3kk85hwe
- Interesting case report in this week's New England Journal of Medicine describing neonatal stroke in the baby due too placental transfer of maternal anti-PF4.
- Should gene therapy be used in patients with active inhibitors and #hemophilia A? Guy Young argues that this is an area definitely worth exploring, and I agree with him. One look at this figure will convince you that a larger study is worth supporting. tinyurl.com/yfk7fh32
