Congenital thrombotic thrombocytopenic purpura results from hereditary deficiency of ADAMTS13. Prophylactic administration of recombinant ADAMTS13 achieved normal blood levels with limited toxicity and no neutralizing antibodies. Full cTTP trial results: nej.md/3UEqGc2

Good to see koalas got a mention #gattii

Dr David Griffin 🦠🧫💉 Steven Tong

Acknowledging Infection Risk in Bispecific Antibody Trials in the Treatment of Multiple Myeloma [Jan 30, 2023] Eddie Cliff et al. Journal of Clinical Oncology ascopubs.org/doi/full/10.12… #mmsm #ImmunoOnc #IDonc

🔥 Tune in to our new episode on maintenance therapy in #MultipleMyeloma with Hira Mian & Manni Mohyuddin ! We discuss a wide range of RCTs from CALGB to FORTE, ideal endpoints for maintenance trials, and role of MRD!
tinyurl.com/4mtjyz6m
Here is clip on Dara maint. in CASSIOPEIA!

Congrats Jeff on an outstanding career!
And looking forward to many more years of playing music together cc Linda Mileshkin

Highly recommended to apply!

A great chance to meet FDA reviewers

ASH | FDA Collaboration: A Workshop on Regulatory Science in Hematology

ASH FDA Oncology Jennifer Gao Kelly Norsworthy Kelly Norsworthy, MD Greg
Talal Hilal 🩺

hematology.org/advocacy/get-i…

5-year follow-up from the LEGEND-2 study of cilta-cel
⏳ 5-year PFS 21% and OS 49% (despite variable LD). 3 progressions after 5 years. No plateau!
🧠No delayed neurotox
🩸No 2nd heme malignancies
🏋️T cells persisted >1 year for VGPR+ (1 at 5.6 years!)
jhoonline.biomedcentral.com/articles/10.11…

Heck of a deal!

Bispecifics for autoimmune disease: watch this space

🧬🩸 Happy to share a review I wrote w/ Alex Herrera on ctDNA in DLBCL with a focus on applications (now and in the new future) for trials and in the clinic. We must aim high to incorporate ctDNA analysis to the routine care of pts #lymsm #ctDNA #MRD

rdcu.be/dFGlj

Great to see this exciting data on sickle cell disease gene therapy out in NEJM

Majority of patients appear functionally cured at this stage

New frontier

Access & long term toxicity remain unanswered

What a wild ride

Bispecifics and autoimmune disease seem like a logical next step, given the degree of B-cell/plasma cell aplasia they can achieve 👀

Benjamin Sutu Mike Putman Chris McMaster Eric Morand 🇺🇦

Helpful study from Joshua D. Wallach Joseph Ross & co - highlighting the major challenges with surrogate endpoints outside of oncology

If we want drugs that work, we need endpoints that provide more certainty of benefit

#MedicalEthics “They stated that optimal postprogression therapy is not only critical ‘to support the ethical justification for enrolling patients in the RCT’ but also ‘essential to the scientific integrity and clinical value of the trial results.’
Gary Lyman 🇺🇸🇪🇺🇺🇦💛💙 Pavlos Msaouel

Nice summary by Eli Darnell, MD & Dr Marcela Maus doi.org/10.1016/j.xcrm… on a recent analysis comparing #TME between SOC and #CART ( #ZUMA7 ) nature.com/articles/s4159…. Main points==>

got this one on repeat

Risk of therapy related myeloid neoplasms (tMN) after ASCT for Hodgkin
• 321 pts
• clonal haematopoiesis in PBSC prod: 14%
• TP53 and / or PPM1D mutation ⬆️ risk tMN (HR 7.3)
• DNMT3a alone did NOT ⬆️ risk

Worth avoiding in high risk pts? #lymsm

ascopubs.org/doi/10.1200/JC…

Superb discussion on bispecific antibodies in FL by David Russler-Germain, MD/PhD
Agents leading to durable remissions for our patients with manageable toxicities and ? more exciting approvals to come. #IUCLS2024 WashUOncology Soni Smith MD Andrew M. Evens, DO, MBA, MSc Tim Fenske

What clinicians should know about surrogate endpoints

Nice work Côme Bommier Matthew J. Maurer, DMSc

Many great examples woven into a thoughtful piece

ashpublications.org/blood/article/… Blood Journal