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hemehub.bsky.social @hemehub.bsky.social · 19h

SCD and Gene Therapy:

Additional facts about the LentiGlobin Study

@asgct.bsky.social @bsochaem.bsky.social @ash.edu.hematology.org @ash.hematology.org @cuhemeonc.bsky.social @ucsdmedschool.bsky.social @bcmhouston.bsky.social @dukemedschool.bsky.social @dgsomucla.bsky.social #Hematology

hemehub.bsky.social @hemehub.bsky.social · 19h

🧬 Today’s question explores how the lasting success of gene therapy is assessed beyond symptom resolution.

See the answer and explanation on the next slide!

@asgct.bsky.social @ash.edu.hematology.org @bsochaem.bsky.social @ash.hematology.org @vjhemonc.bsky.social @sicklecelluk.bsky.social

hemehub.bsky.social @hemehub.bsky.social · 1d

Case submissions are a vital part of our educational seminars.
You can submit cases directly on our website!

🩸Highlight real-world diagnostic challenges and share knowledge that supports education across all levels.

Visit hemehub.org to learn more!

#Hematology #MedicalEducation

hemehub.bsky.social @hemehub.bsky.social · 3d

SCD and Gene Therapy:

Additional facts about the LentiGlobin Study

@ash.hematology.org @asgct.bsky.social @ash.edu.hematology.org
#Lyfgenia #LentiGlobin #GeneTherapy #SickleCellDisease #Hematology #CellTherapy #ClinicalTrials #RareDiseases #BloodDisorders #StemCellTherapy

hemehub.bsky.social @hemehub.bsky.social · 3d

Facts about Sickle Cell Disease and Gene Therapy

Study Focus: LentiGlobin (Lovotibeglogene Autotemcel, Lyfgenia)

@ash.hematology.org @asgct.bsky.social @ash.edu.hematology.org
#Lyfgenia #GeneTherapy #SickleCellDisease #Hematology #CellTherapy #RareDiseases #BloodDisorders #MedicalEducation

hemehub.bsky.social @hemehub.bsky.social · 3d

Today’s question!

Check out the answer on the next slide along with its explanation.

#Lyfgenia #GeneTherapy #SickleCellDisease #Hematology #CellTherapy #RareDiseases #BloodDisorders @ash.hematology.org @rarediseasectn.bsky.social @cuhemeonc.bsky.social @ash.edu.hematology.org @asgct.bsky.social

hemehub.bsky.social @hemehub.bsky.social · 6d

Why might anemia persist in a patient treated with Lyfgenia despite elimination of VOCs?

See the answer and explanation on the next slide!

#Lyfgenia #LentiGlobin #GeneTherapy #SickleCellDisease #Hematology #CellTherapy #ClinicalTrials #RareDiseases #BloodDisorders #StemCellTherapy

hemehub.bsky.social @hemehub.bsky.social · 8d

Today’s question explores how vector copy number relates to treatment outcomes in patients receiving Lyfgenia.

Check out the answer on the next slide along with its explanation!

#Lyfgenia #GeneTherapy #SickleCellDisease #Hematology #RareDiseases #BloodDisorders #MedicalEducation

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hemehub.bsky.social @hemehub.bsky.social · 8d

Facts about #SickleCellDisease and #GeneTherapy

Study Focus: #LentiGlobin

The HGB-206 clinical trial refined the treatment process, including transfusion regimens, busulfan conditioning, and manufacturing methods, leading to improved and consistent outcomes for patients with sickle cell disease.

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hemehub.bsky.social @hemehub.bsky.social · 9d

Today’s question highlights the clinical effects of Lyfgenia™ in a patient with SCD.

Check out the answer on the next slide along with its explanation!

@ascaa.bsky.social @sicklecell101.bsky.social @sicklecelluk.bsky.social @asgct.bsky.social #SickleCellDisease #Hematology #BloodDisorders

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hemehub.bsky.social @hemehub.bsky.social · 9d

An overview of Lyfgenia and the antisickling hemoglobin HbAT87Q, highlighting the mechanism by which autologous stem cells are engineered to restore functional red blood cells.

@ascaa.bsky.social @sicklecell101.bsky.social @sicklecelluk.bsky.social #SickleCellDisease #Hematology #CellTherapy

hemehub.bsky.social @hemehub.bsky.social · 10d

This Month on HemeHub: SCD and Gene Therapy

Join us as we explore advances in the treatment of #sicklecelldisease through #genetherapy, featuring pivotal trials of Lyfgenia and Casgevy.

Stay tuned for our upcoming session featuring Dr. John Tisdale, Senior Investigator at the NHLBI.

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hemehub.bsky.social @hemehub.bsky.social · 14d

Today's second question is about the diagnostic approach to JAK2-unmutated #erythrocytosis and the appropriate next step in evaluation.

The third question focuses on the mechanism by which HFE mutations contribute to #erythrocytosis.

Check out the answers to both questions on the next slides!

hemehub.bsky.social @hemehub.bsky.social · 14d

Today’s first question highlights a key distinguishing feature of TEMPI syndrome in clinical practice.

#JAK2NegativeErythrocytosis #NonPVErythrocytosis #Erythrocytosis #Hematology #MyeloproliferativeDisorders #SecondaryErythrocytosis #EPO #Hypoxia #GermlineMutations #ClinicalHematology

hemehub.bsky.social @hemehub.bsky.social · 14d

Facts about JAK2 unmutated erythrocytosis:

Genetic variants in Epo signaling, oxygen affinity, and iron metabolism can all contribute to inherited erythrocytosis.

@bsochaem.bsky.social @ash.edu.hematology.org @vjhemonc.bsky.social @rarediseasectn.bsky.social #Erythrocytosis #Hematology

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hemehub.bsky.social @hemehub.bsky.social · 15d

Today’s question is about the complications of erythrocytosis in congenital heart disease and their underlying associations.

#JAK2NegativeErythrocytosis #Erythrocytosis #Hematology @bsochaem.bsky.social @vjhemonc.bsky.social @ash.hematology.org @ash.edu.hematology.org @cuhemeonc.bsky.social

hemehub.bsky.social @hemehub.bsky.social · 15d

Facts about JAK2 unmutated erythrocytosis:

The HIF-PHD-VHL pathway, driven by HIF2A, links oxygen and iron sensing to Epo production and hypoxia-induced erythrocytosis.

@vjhemonc.bsky.social @ash.hematology.org @ash.edu.hematology.org @bsochaem.bsky.social #Erythrocytosis #Hematology

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hemehub.bsky.social @hemehub.bsky.social · 18d

Missed our session with Dr. Naseema Gangat on JAK2 Unmutated Erythrocytosis? The full recording is now available on our YouTube channel!

➡️Watch the full session: www.youtube.com/watch?si=Qeq...

@bsochaem.bsky.social @vjhemonc.bsky.social @cuhemeonc.bsky.social @bsochaem.bsky.social #Hematology

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hemehub.bsky.social @hemehub.bsky.social · 18d

Today’s question is about evaluating causes of erythrocytosis in a JAK2-negative patient with menorrhagia.

Check out the answer on the next slide along with its explanation!

@ash.edu.hematology.org @stanfordmedicine.bsky.social @bcmhouston.bsky.social @medicine.stitches.today @bsochaem.bsky.social

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hemehub.bsky.social @hemehub.bsky.social · 18d

Facts about JAK2 unmutated erythrocytosis:

Post-renal transplant erythrocytosis declines with ACEi/ARBs; management is monitoring and CV risk control, not routine phlebotomy or JAK2 inhibitors.

#JAK2NegativeErythrocytosis #Erythrocytosis #Hematology #MyeloproliferativeDisorders

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hemehub.bsky.social @hemehub.bsky.social · 20d

Today’s question is about predisposing factors for erythrocytosis.

Check out the answer on the next slide along with its explanation!

@ash.edu.hematology.org @stanfordmedicine.bsky.social @bcmhouston.bsky.social @yaleschoolofmed.bsky.social @medicine.stitches.today @dukemedschool.bsky.social

hemehub.bsky.social @hemehub.bsky.social · 20d

Facts about JAK2-unmutated erythrocytosis:

How SGLT-2 inhibitors and testosterone therapy can raise hemoglobin/hematocrit and what current guidelines recommend for management.

#Hematology @ash.edu.hematology.org @rarediseasectn.bsky.social @bsochaem.bsky.social
@vjhemonc.bsky.social

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hemehub.bsky.social @hemehub.bsky.social · 21d

Today’s question!

Check out the answer on the next slide along with its explanation.

@vjhemonc.bsky.social @bsochaem.bsky.social @ash.edu.hematology.org @rarediseasesint.bsky.social @ucsdmedschool.bsky.social @stanfordmedicine.bsky.social @bcmhouston.bsky.social @cuhemeonc.bsky.social

hemehub.bsky.social @hemehub.bsky.social · 21d

Facts about JAK2 unmutated erythrocytosis:

In Chuvash polycythemia and high oxygen affinity hemoglobin, thrombosis risk is linked to age and cardiovascular factors—not hematocrit—and phlebotomy or hydroxyurea are generally not beneficial.

#Erythrocytosis #Hematology #MyeloproliferativeDisorders

hemehub.bsky.social @hemehub.bsky.social · 22d

Facts about JAK2-unmutated erythrocytosis:

Phlebotomy isn’t recommended for asymptomatic patients, may be harmful in physiologic cases, and can cause its own side effects.

@ash.edu.hematology.org @vjhemonc.bsky.social @rarediseasectn.bsky.social @bsochaem.bsky.social @ash.hematology.org