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December 30, 2023
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ASH recap: ‘Transformative’ advance in sickle cell disease, ‘breakthrough’ in AML and ALL

Fact checked byMindy Valcarcel, MS
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This year’s ASH Annual Meeting featured several high-impact abstracts.

Highlights included a pivotal study on gene therapy for sickle cell disease, treatment advances in leukemia and lymphoma, a comparison of multiple myeloma outcomes in clinical trials vs. real-world settings, and a report on the link between a class of cancer therapies and cardiovascular adverse events.

Healio presents this recap of key ASH abstracts that may be relevant to your practice.

Graphic showing experts with whom Healio spoke at ASH 
These experts and many other key opinion leaders shared their perspective with Healio about important research presented at ASH Annual Meeting and Exposition.

1. One-time treatment with lovotibeglogene autotemcel ((Lyfgenia, Bluebird Bio) gene therapy conferred “transformative” benefit to patients with sickle cell disease, according to researcher Julie Kanter, MD. Read more.

2. The combination of ibrutinib (Imbruvica; Janssen, Pharmacyclics) and venetoclax (Venclexta; AbbVie, Genentech) used for a response-directed duration significantly improved outcomes for fit patients with untreated chronic lymphocytic leukemia. The survival curves are "very impressive" and findings show "depth of remission is critical," researcher Peter Hillmen, MB, ChB, PhD, told Healio. Read more.

3. Standard treatments for multiple myeloma conferred markedly shorter survival benefits in real-world settings than in registrational clinical trials. Cynthia E. Dunbar, MD, and James R. Berenson, MD, offer expert perspective on the findings and their implications. Read more.

4. Former ASH President Alexis Thompson, MD, MPH — who received the society’s award for leadership in promoting diversity — spoke with Healio about her efforts to help underserved populations and what more needs to be done. “There are many areas in which having diversity in person and place makes us all better,” Thompson told Healio. “The kinds of science we pursue and the ways we frame questions can be impacted by the people in the room.” Read more.

5. Emicizumab (Hemlibra, Genentech) exhibited efficacy for infants with severe hemophilia A without factor VIII inhibitors. The agent provides a “new path forward” for management of this population, researcher Steven W. Pipe, MD, told Healio. Read more.

6. Revumenib (SNDX-5613, Syndax Pharmaceuticals) appeared effective and safe for children and adults with relapsed or refractory KMT2A-rearranged acute myeloid leukemia or acute lymphoid leukemia. The results represent “a significant breakthrough” for patients with limited treatment options, researcher Ibrahim T. Aldoss, MD, told Healio. Read more.

7. Individuals with sickle cell trait are at increased risk for leukemia, specifically myeloid and monocytic leukemias. Read more.

8. Axatilimab (SNDX-6352; Incyte/Syndax Pharmaceuticals) conferred clinically meaningful and durable responses at three dose levels among individuals with chronic graft-versus-host disease. Read more.

9. Subcutaneous time-limited mosunetuzumab (Lunsumio, Genentech) exhibited “highly encouraging efficacy” among patients with newly diagnosed high-burden follicular lymphoma, according to researchers. In an accompanying perspective, Joshua Brody, MD, offers insights into the implications of what he called a “remarkable and important study.” Read more.

10. Individuals with preexisting cardiovascular disease treated with first-generation Bruton tyrosine kinase inhibitors had significantly higher risk for cardiovascular adverse events. “We knew it would be a little bit higher going in, but we did not expect to see such a significant number,” researcher Maria J. Fernandez Turizo, MD, told Healio. Read more.

11. In a special scientific session on race and science, Saad Z. Usmani, MD, MBA, FACP, FRCP, examined clinical trial inclusion from an investigator’s perspective. He highlighted challenges in trial enrollment and what can be done to achieve greater diversity in cancer trials. Read more.

12. Reduced-intensity haploidentical-bone marrow transplant showed durable donor engraftment at 2-years follow-up with low mortality among adults with severe sickle cell disease. How this approach fits in with recently approved, potentially curative gene therapies for sickle cell disease is “the $1 million question — or should I say the $2 million or $3 million question,” Mikkael A. Sekeres, MD, said when discussing the findings. Read more.

For comprehensive coverage — including researcher interviews, expert perspective and videos — visit our ASH Annual Meeting collection on Healio.