Happy to share this recent publication in Cytotherapy that features a global perspective on gene therapy clinical trials for beta hemoglobinopathy. Also delighted to see content from South America 🇧🇷 Dr. Vanderson Rocha samuelcfcouto isct-cytotherapy.org/article/S1465-…

Ahead of #WorldSickleCellDay #WSCD23 we are publishing the first detailed global estimates on the burden of #sicklecell disease Read it #openaccess thelancet.com/journals/lanha… The authors show that mortality linked to #sicklecell disease is higher than previously described🧵

With early (<100d) CD4+ AND B-cell reconstitution after #BMT higher survival chances AND dramatically LOWER #GVHD Thanks team: PrinsesMáximaCentrum St. Jude and Memorial Sloan Kettering Cancer Center for fruitful collab! More to come!! sciencedirect.com/science/articl…

A randomized controlled trial with extracorporeal photopheresis as #GVHD prophylaxis found it does not prevent GVHD and prophylactic extracorporeal photopheresis does not affect survival. Read the full #openaccess article: ow.ly/R86G50OO8Sj

Today is #WorldSickleCellDay #WSCD23 #SickleCellDisease has been a priority for this Journal in the past years and the result is a Commission that will be officially launched on July 12, 9.30 – 11am East Africa Time at a hybrid event ⬇️ Register at sadacc.org/workshopsface-…

⁦seth rotz⁩ discussing fertility preservation! Great interview! podcasts.apple.com/us/podcast/thi…

In this trial, 1-year GVHD-free, relapse-free survival after stem-cell transplantation was 52.7% in the cyclophosphamide–tacrolimus–mycophenolate mofetil group and 34.9% in the tacrolimus–methotrexate group. nej.md/3pb2FN4

Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. HLA-haploidentical BMT with PTCy as upfront therapy in patients with SAA results in survival >90% and low morbidity/mortality. ashpublications.org/blood/article-…

Emapalumab can lead to good transplant outcomes for STAT1 GOF. Next step incorporating into conditioning for primary immune regulatory disorders. See our paper in JACI (sciencedirect.com/science/articl…) and details about our trial open at Memorial Sloan Kettering Cancer Center (clinicaltrials.gov/study/NCT05787……)

With CRISPR-Cas9, a repressor of fetal hemoglobin expression was disrupted in hematopoietic stem cells from three persons with sickle cell disease. Edited cells were reinfused. Hematopoiesis was restored. Read the full study results: nej.md/3L64brp

Listened 2 an inspiring talk by Prof Mamta VijayManglani @ 2nd Emirates peds bmt conference Govt hospital Mumbai, Borivalli BMT center median cost Allo 9184$, auto 2487$ #hct for the poor & the masses! 300 & counting....that's what I call impact..#HCT for all who need it

Fertility Basics for the Peds BMT patients.. chaired by seth rotz The PTCTC @BeTheMatch #HCT. Nice overview of fertility preservation options for our patients prior to #HCT Gene therapy, things we need2 know. Webinar by Dr. Allison Close, see link below bethematch.webex.com/bethematch/ldr…

duke.zoom.us/webinar/regist…. Join us tommorow at 2pm EST as 🇧🇷 Carmem Bonfim presents Duke Children's The PTCTC seminar. Dr Bonfim will speak on Brazils Haplo experience in non malignant disease.

🚨PubAlert🚨 we show how #MachineLearning can be used to leverage longitudinal data collected directly from the medical records after allo HCT to predict survival in patients. ashpublications.org/bloodadvances/…

Did you ever wonder why our marrow is located inside of our *bones*, #MedTwitter? There’s no a priori anatomical reason it should be sited there. Blood cells could form in our spleens & livers, as they do during our fetal lives; or elsewhere, as in some animals. Let’s discuss! /1

📢 Not one but TWO #GeneTherapy products approved for #SickleCell disease by the U.S. FDA 👏 Medicine will never be the same again. Now let's make these treatments AFFORDABLE and ACCESSIBLE to all who need them. fda.gov/news-events/pr…

Long time in the making: 5 year survival outcomes for pediatric stem cell transplant patients requiring intensive care. Grateful to everyone who helped with this project. CIBMTR Blood Advances The PTCTC ASTCT UC San Francisco UCSF Benioff SF ashpublications.org/bloodadvances/…

The PTCTC #Tandem24 Fertiltiy in Long term survivors of HCT Key concepts by Dr Karen Burns discussion of infertility is important.. concept of CED in calculating risk

The PTCTC #tandem24 long term followup of FA keypoints @Parinda Mehta

Huge congratulations to St. Jude Research rising star ⭐️ Shruthi suryaprakash for receiving the The PTCTC New Investigator Award