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The PTCTC

@ThePTCTC

Supports research and education to improve the availability, safety, and efficacy of HCT and other cellular therapeutics for children and adolescents.

theptctc.org/
Joined January 2021
  • Tweets592
  • Following346
  • Followers423
27Media
Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)Photo by The PTCTC (@ThePTCTC)
Replying to @ThePTCTC
@ThePTCTC #tandem24 long term followup of FA keypoints @@kidsBMTdoc
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
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Huge congratulations to @StJudeResearch rising star ⭐️ @drshruthisurya for receiving the @ThePTCTC New Investigator Award
Image from Akshay Sharma MD (@AkshaySharmaMD)
Image from Akshay Sharma MD (@AkshaySharmaMD)
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Replying to @ThePTCTC
@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
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
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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/bloodadv…
Intensive care risk and long-term outcomes in pediatric allogeneic hematopoietic cell transplant...

Intensive care risk and long-term outcomes in pediatric allogeneic hematopoietic cell transplant...

Among 1067 critically ill pediatric patients with HCT, survival to ICU discharge was 85%, but half of patients required ICU readmission.For pediatric patie

ashpublications.org
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📢 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/press-an…
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

The FDA approved the first cell-based gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease in patients 12 years and older.

fda.gov
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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
Image from David Steensma, MD (@DavidSteensma)
Image from David Steensma, MD (@DavidSteensma)
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🚨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/bloodadv…
Longitudinal clinical data improve survival prediction after hematopoietic cell transplantation...

Longitudinal clinical data improve survival prediction after hematopoietic cell transplantation...

We developed a risk-prediction model using machine learning, incorporating clinical measurements performed before and after allo-HCT.By using longitudinal

ashpublications.org
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duke.zoom.us/webinar/registe…. 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.
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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/bethema…
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
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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
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
Image from Hemalatha Rangarajan MD https (@hmgcoa2)
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The PTCTC retweeted
NEJM (@NEJM) profile picture
NEJM
@NEJM30 Aug 2023
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

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The PTCTC retweeted
NEJM (@NEJM) profile picture
NEJM
@NEJM21 Jun 2023
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

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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/ar…) and details about our trial open at @MSKCancerCenter (clinicaltrials.gov/study/NCT……)
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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/ar…
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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-fac…
Image from The Lancet Haematology (@TheLancetHaem)
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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…
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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🧵
Image from The Lancet Haematology (@TheLancetHaem)
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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/ar…
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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
Image from Transplantation and Cellular Therapy Journal (@ASTCT_Journal)
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