Interesting findings with @TamerHellou and Eli Muchtar in Am J Hematology: In newly diagnosed AL patients, +1q marks a plasma cell-dominant subtype with worse long-term survival (median OS 57 vs 120 months). Other high-risk lesions didn’t! #amyloidosis
https://t.co/cXnwSuAcLU
#Myeloma Paper of the Day: Study of AL #amyloidosis pts getting ASCT finds benefit for those not in CR after induction but not if in CR, dara + ASCT linked w/ longer TTP, whereas higher BMPC% (>20%) at dx & gain/amp 1q linked with shorter TTP: https://t.co/V8BFAnDclT. #mmsm
Proud of our latest publication in American Journal of Hematology:
Determinants of Long-Term Benefit From High Dose Melphalan With Autologous Stem Cell Transplant in #AL#Amyloidosis.
https://t.co/1hEMBx7HGe
In our new paper, w/ Sunny Kim and @VPrasadMDMPH, we explain why observation should remain the standard of care in high-risk Smoldering Myeloma
What is Smoldering Myeloma?
How OpenEvidence failed?
How key data lacking from NEJM were made available by the german HTA agency?
🧵1/
🥼Big collaboration to show that the immune system may not just respond to amyloid, but actively participate in regulating its formation. Thanks to all colleagues and friends!
https://t.co/3ZOwokXf6L
🧬 Proud to share: Publication in Blood Cancer Journal:
Activity of CAR-T cells and bispecific antibodies in multiple myeloma with EMD
👉CAR T-cell therapy and effective debulking resulted in higher EMD response rates, and prolonged PFS
👉 https://t.co/3QLpa8AYFP
With the Acoramidis FDA approval, EU countries expect EMA approval in early 2025. With a hefty price tag (20k $/mo) and Tafamidis patent expiring late 2025, it will be interesting to see how prescriptions will turn out.
Head-to-head comparisons will have to be done by academia!
Fantastic news. Attruby is here. And we have had excellent experience with its long-term use.
Most unique about this announcement in my opinion - acoramidis/Attruby will be provided free for life for trial participants in the phase II and III. This is unprecedented at least in our space and many need to follow suit. Kudos to BridgeBio, Neil Kumar Jonathan Fox et al. for following on this promise. We have many patients who were elated to hear this news and felt valued in a unique way. We always prepare patients in OLE/LTE phases for unexpected news that could be disappointing. This was different.
#CardioTwitter
Happy to finally see this published! Heartfelt congratulations to Mara for the hard work and big thanks to @RascheLeo for bringing the project forward!
We are searching for a highly motivated PhD student (m/f/d) interested in developing novel CRISPR 2.0 gene editing technologies for CAR-T cell engineering and cancer immunotherapy @Uniklinikum_Wue & @Uni_WUE. Please RT 🙏.
I’m excited to share that my latest review article on AL amyloidosis is now published in the New England Journal of Medicine! @BU_Amyloidosis@BUMedicine@The_BMC
Systemic Light Chain Amyloidosis | New England Journal of Medicine https://t.co/1u1cM5HF81