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Michael Makris Profile picture
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9 Jan, 4 tweets, 3 min read
Biomarin has just announced their year 2 phase 3 (134 patients) ValRox gene therapy in #hemophilia results. Yes on a Sunday evening! Stable and durable bleed control but the FVIII levels are falling significantly. tinyurl.com/2p92ksj8 1/4
Key questions: How many patients needed steroids, what was the variability in levels (and how many had levels <10% and <5%), how long is the response predicted to last? #hemophilia #genetherapy #ValRox 2/4
Considering the mean FVIII levels, it is not surprising that stable and durable annual bleed control was observed. I would expect not many patients in the <10% group. Loss of ABR control would only be expected when the levels fall below 10%. #hemophilia #genetherapy 3/4
I understand there is a presentation to investors at 5:00pm Eastern Time today. I am not an investor but if anybody knows if I can join please email me at m.makris@sheffield.ac.uk 4/4

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Michael Makris Profile picture
9 Jan
I joined the Biomarin conference this evening. Not much more info than the press release but here are a few snippets that I learned. 1/7
Biomarin phase 3 steroid use. Number of patients who needed steroids not mentioned. No mention of steroid adverse events. They said they just completed recruitment of a study that uses prophylactic steroids. 2/7
I believe that in #hemophilia gene therapy studies the adverse events due to steroids should be mentioned. These are high dose steroids for several weeks and adverse events are common. 3/7
Read 7 tweets
Michael Makris Profile picture
3 Jun 21
What is the incidence of VITT after 2nd AZ vaccination? The MHRA are saying 1.3 per 1 million. I say it may be a lot less and could be zero. #VITT #COVIT19 1/7
We do not know much about the 2nd dose VITT cases that the MHRA claims to have had reported to them. They are all >50 but we do not know anything else. Why do they not publish type of thrombosis, age, sex, co-morbidities, anti-PF4? 2/7
It is accepted that the MHRA have had 18 reports of thrombosis with thrombocytopenia after 2nd dose AZ vaccine. But are they VITT? Do they definitely not have cancer or sepsis? Do they fulfil the attached typical VITT definition? 3/7 Image
Read 7 tweets
Michael Makris Profile picture
29 May 21
Health care workers and carers >18 years in the UK could get any vaccine relatively early on. Because of the Pfizer storage requirements, it was used largely in hospital vaccination sites. So in UK, hospital staff largely got Pfizer in contrast to Norway/Germany who got AZ. 1/4
In the UK this was a logistics issue since prior to March we knew nothing about VITT. Many European countries felt the efficacy data for the AZ vaccine were not strong for >65, so gave Pfizer to >65 and AZ to <65. 2/4
In Norway and Germany where the decision was to use AZ for <65 years, they also decided to give it to health care staff first. Since health care staff are more likely to be female, there was a large female excess in the persons who developed VITT. 3/4
Read 4 tweets
Michael Makris Profile picture
22 May 21
What is the risk of vaccine-induced immune thrombocytopenia and thrombosis (VITT) after a 2nd AZ vaccine dose? This is the commonest question I get at present on twitter, email and in clinic. 1/6
The information released by the MHRA (the UK regulator) two days ago on their website stated that they have had 15 cases of 2nd dose VITT after 9 million 2nd doses of the AZ vacc administered (ie 1 VITT case every 600,000 2nd doses of the AZ vaccine). tinyurl.com/s53nf2w 2/6
If the rare of VITT in the UK after 2nd doses of the vaccine was the same as after the the 1st dose, we should have had (309-15)x9/23.9= 110.7 cases. According to the MHRA we have had 15 but expected 110. 3/6
Read 6 tweets
Michael Makris Profile picture
20 May 21
I am pro-vaccine and especially so when it comes to #COVID19. I have participated in the vaccine clinical trial and will do so again. I have had AZ x2. I believe people should be provided with all the available information to allow them to make a decision before vaccination. 1/4
Q1 to @MHRAgovuk: It is great that you provide the cases of VITT post AZ vaccine by decade of age. Please also provide the number of persons vaccinated with AZ by decade of age so the rates of VITT can be calculated. 2/4
Q2 to @MHRAgovuk: Have there been any VITT cases after Pfizer vaccination reported? If yes, how many? 3/4
Read 4 tweets
Michael Makris Profile picture
22 May 20
Today has been the most important day in terms of high quality publications on COVID-19. Up to now amazingly 15425 manuscripts have been published in scientific journals. However 4 of the top 10 papers ever published on COVID-19 were published today. #COVIDー19 #Coronavirus 1/5
PAPER 1: The trial which was presented by Fauci on Remdesivir in COVID-19 has just been published by the New England Journal of Medicine. The benefit is modest but it is one of the very few RCTs with a positive result. tinyurl.com/y8s46vc7 2/5
PAPER 2: Huge observational study shows that the use of hydroxychloroquine in COVID-19 is associated with reduced survival. OK not an RCT but sufficiently powerful to limit use of hydroxychloroquine to clinical trials only. tiny.cc/gywjpz 3/5
Read 5 tweets

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