The PTCTC

@ThePTCTC #tandem24 long term followup of FA keypoints @@kidsBMTdoc

Huge congratulations to @StJudeResearch rising star ⭐️ @drshruthisurya for receiving the @ThePTCTC New Investigator Award

@ThePTCTC #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

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 @BloodAdvances @ThePTCTC @ASTCT @UCSF @UCSFChildrens ashpublications.org/bloodadvances/…

📢 Not one but TWO #GeneTherapy products approved for #SickleCell disease by the @US_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…

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

🚨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/…

duke.zoom.us/webinar/regist…. Join us tommorow at 2pm EST as 🇧🇷 @CarmemBonfim1 presents @Duke_Childrens @ThePTCTC seminar. Dr Bonfim will speak on Brazils Haplo experience in non malignant disease.

Fertility Basics for the Peds BMT patients.. chaired by @SethRotz @ThePTCTC @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…

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

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

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

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 @MSKCancerCenter (clinicaltrials.gov/study/NCT05787……)

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-…

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-…

⁦@SethRotz⁩ discussing fertility preservation! Great interview! podcasts.apple.com/us/podcast/thi…

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 🇧🇷 @dvandersonrocha @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: @prinsesmaximac @StJude and @MSKCancerCenter 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