Robert Brodsky

🚨 New funding opportunity! ➡️ Research in #ThromboticMicroangiopathies and related rare disorders 🔗 Learn more and apply: grants.rarediseasesnetwork.org/grants/IMPACTp…

@psharmaMD Avoid peripheral blood. More T cells in PBSCT than marrow (~1 log) - increases gvhd which only causes harm in no malignant dz like SCD or SAA

@DocsDock200 Great! Great points. What’s your thought on use of PB as stem cell source?

Game changer for #sicklecellamemia. doi.org/10.1182/blooda… Nonmyleoablative haploBMT with PTCy using Baltimore regimen (flu/cy/tbi400) rather than thiotepa. What are the advances? 1) 95% OS and 94% DFS @ 2.4 yrs 2)ONLY curative therapy that preserves FERTILITY!

3) Engraftment > 90% even in pediatric patients possibly overcoming the reduced engraftment observed with thiotepa in haploBMT. 4) able to withdraw immunosuppression after 6mos 5) median hosp stay in adults - just 12 days! @ASH_hematology @BloodPortfolio

doi.org/10.1182/blooda… Ravulizumab appears to be safe and effective in pregnancy. We've been waiting for this data for a long time. #PNH #aHUS @ASH_hematology

Out in the Annals of the rheumatic diseases. sciencedirect.com/science/articl… Insight into what makes CAPS different from APS and finally a true target to make it treatable!

Nothing like puppy love.

Patient give to best perspective of sickle cell disease. Everyone is different but many common themes. podcasts.apple.com/us/podcast/blo…

Congrats to Valerie Bey @heartsickle1 and the Northern Va sickle cell disease association on their podcast Blood, Sweat, and Cells. Greatful to be part of but esp inspired by the patient stories Valerie brings to life. podcasts.apple.com/us/podcast/blo…

Can u stop thrombosis and treat CAPS without anticoagulation? CR1 and #complement. Great work Nikhil Ranjan. sciencedirect.com/science/articl… @eular_org @ACRheum @jhrheumatology @HTRStoday @HTRSFellows @BloodPortfolio @USTMAConsortium #MedTwitter

Five former ASH presidents in NYC @NYASciences for great symposium on sickle cell disease. One of them 🙁 still doesn't know how to wear a name tag.@ASH_hematology

notebooklm.google.com/notebook/b3751… CR1 deficiency and CAPS

Genetic and Epigenetic Dysregulation of CR1 is Associated with Catastrophic Antiphospholipid Syndrome (CAPS) | medRxiv New insight as to what makes (CAPS) catastrophic! Now we may be able to stop it from being catastrophic. nam02.safelinks.protection.outlook.com/?url=https%3A%…

Three cured #sicklecell persons in one room this am. #haplobmt. Never thought I'd see the day.

The procedure would greatly expand the pool of potential donors, in addition to costing less than one-quarter of the price of innovative gene therapies. Source: Axios search.app/qV26Ke9E6k7qVZ…

Almost every patient can have a haplo donor available, now we can say this approach is safe and effective for sickle cell disease. While gene therapies are effective too, access and cost (here and globally) will be major issues #bmtsm

Stay tuned! We already developed an even less toxic prep that allows us to stop immunosuppression in 6mos and PRESERVES FERTILITY in many. Game changer.

Out in NEJM Evidence today. KEY TAKEAWAYS •Of 42 people with severe SCD who had the procedure , 95% were alive at 2 yrs, and 88% are considered cured. •Most people with SCD are eligible, which costs a fraction of the price of gene editing/therapy #casgevy @ASH_hematology