Penn Medicine at the 2023 ASH Meeting

SAN DIEGO – Researchers from the Abramson Cancer Center and Perelman School of Medicine at the University of Pennsylvania will present data on the latest advances in blood cancer and benign hematology research at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition from Dec. 9-12 in San Diego, California. Follow us @PennMDForum and @PennCancer for updates.

Expert Interviews

Experts from Penn Medicine are available to comment on a wide range of hematology-oncology research and care topics during the meeting by video call, telephone, or email. To arrange interviews, please contact Meagan Raeke at Meagan.Raeke@pennmedicine.upenn.edu or 267-693-6224.

News Releases

• First-in-human clinical trial of CAR T cell therapy with new binding mechanism shows promising early responses

• New Drug Helps Narrow Racial Survival Disparity in Patients with Acute Myeloid Leukemia

Key Presentations

Penn Medicine experts will present new data across a variety of topics, including health services and outcomes research, long-term follow-up of two Phase II drug studies for follicular lymphoma patients, and translational research in blood cancers and sickle cell disease.

Health Services and Outcomes Research

• Frailty influences survival for older adults with a new diagnosis of acute myeloid leukemia (AML) (Abstract 376): While research has shown that physical activity has proven benefits on cancer outcomes, this prospective observational study looked at the inverse effect, by measuring frailty. Patients age 60 and older with AML who were considered “frail” had three times the risk of early mortality compared to those considered "fit." Similarly, patients who were “pre-frail” had nearly double the risk of early mortality compared to “fit” patients. Frailty was measured by Freid’s Frailty Phenotype (FFP), which uses subjective measures (reported weight loss, exhaustion, and activity level) and objective measures (four-meter walk speed and grip strength) to determine fitness to endure stressors. Two-year overall survival estimates were 57%, 42%, and 21% in fit, pre-frail, and frail patients, respectively. Ashley D. Hadjis, MD, an Internal Medicine resident who is working with Shannon R. McCurdy, MD, an assistant professor of Hematology-Oncology, will present the findings in an oral session on Saturday, Dec. 9 at 4:45 p.m. PT in the Marriott Marquis San Diego Marina Pacific Ballroom Salons 15-17.
• Financial navigation program for patients with multiple myeloma (Abstract 909): Despite being insured, 55 percent of patients in this study reported a significant financial burden from their treatment for multiple myeloma, a particularly high-cost cancer where improved survival often comes with lifelong therapy, and consequently, financial burdens. In this trial, patients who were identified as at risk of financial toxicity – determined using the COmprehensive Score for financial Toxicity (COST) questionnaire – were randomized to a proactive and coordinated financial navigation program. On average, all at-risk participants reported improvement in their financial toxicity scores, while participants who were not at risk of financial toxicity reported worsening COST scores at study completion, indicating a potential benefit for proactive financial toxicity screening even among those who do not appear at risk at the time of diagnosis. Mia Djulbegovic, MD, MHS, an assistant professor of Hematology-Oncology, will present the findings in an oral session on Monday, Dec. 11 at 3:15 p.m. PT in the Marriott Marquis San Diego Marina Marriott Grand Ballroom 2-4.
• Non-transfusion-dependent thalassemia may evolve to transfusion-dependent thalassemia in adulthood (Abstract 2362): This retrospective study evaluated 94 patients with thalassemia referred to the Penn Comprehensive Adult Thalassemia Program between 2013 and 2023. Of the 34 patients who didn’t have a history of regular blood transfusions to manage their disease (non-transfusion-dependent thalassemia), 14 (41 percent) eventually met criteria to begin a chronic transfusion regimen to maintain their hemoglobin levels and/or treat clinical complications of anemia. These findings show how transfusion needs can evolve in adulthood much more commonly than was previously known. Aaron Cheng, MD, a fellow in Hematology-Oncology, will present the findings in a poster session on Saturday, Dec. 9 at 5:30 p.m. PT in the San Diego Convention Center Halls G-H.

Long-term follow-up from Phase II treatment studies for follicular lymphoma

• Durable responses continue for CAR T cell therapy and bispecific antibody treatment (Abstract 601 and Abstract 603): Last year, patients with relapsed/refractory follicular lymphoma gained access to several newly approved therapies, including the CAR T cell therapy tisagenlecleucel, originally developed at Penn, and the bispecific antibody mosunetuzumab. Clinical trials that paved the way to approval are continuing to mature. At the three-year follow-up point, the Phase II ELARA clinical trial (NCT03568461) continues to produce durable responses with tisagenlecleucel: 82 percent overall survival; 68 percent complete response (CR) rate; and 86 percent overall response rate (CR plus partial responses). After a median follow-up of more than three years, the pivotal Phase II study of mosunetuzumab (NCT02500407), continued to see durable responses: progression-free survival (PFS) rate of 57.1 percent and median PFS of 24 months; CR of 60 percent; and 77.8 percent overall response rate. Stephen J. Schuster, MD, director of the Lymphoma Program at Penn’s Abramson Cancer Center, will present both abstracts in an oral session on Sunday, Dec. 10, at 4:30 p.m. PT in the Manchester Grand Hyatt San Diego Grand Hall C.

Translational Research

• Potential therapeutic target in sickle cell disease (Abstract 13): New data from this study indicate that the protein phosphatase 6 (PP6) complex is a novel regulator of fetal hemoglobin (HbF), a protective form of hemoglobin that reduces mortality in patients with sickle cell disease. Using a CRISPR-Cas9-based genetic screen to identify druggable molecules—those able to be therapeutically modulated by medicine—involved in HbF control, this study uncovered that depletion of either the PP6 catalytic subunit (PPP6C) or its red-cell selective subunit (PPP6R1) led to significantly increased HbF levels, providing potential therapeutic targets in the treatment of sickle cell disease. Scott A. Peslak, MD, PhD, an assistant professor of Hematology, will present the findings in an oral session on Saturday, Dec. 9 at 9:30 a.m. PT in San Diego Convention Center Room 29.
• New Target for TP53 mutant AML (Abstract 408): With few effective treatment options, patients with TP53-mutated AML have very poor outcomes. This research team found that chemotherapy resistance in TP53 mutant AML depends on the mevalonate pathway, making the pathway an ideal target to improve response to chemo.  Sarah J. Skuli, MD, PhD, an instructor of Hematology-Oncology, will present the findings in an oral session on Sunday, Dec. 10 at 10:45 a.m. PT in the Marriott Marquis San Diego Marina Marriott Grand Ballroom 11-13.
• CD5 knockout is a universal strategy to improve CAR T cell therapy (Abstract 102): This research team previously showed that a CD5 knockout strategy could improve CAR T cell function against B and T cell lymphomas. In this update, the team has extended the strategy to pancreatic cancer models and melanoma cell lines, indicating that it could work across multiple cancer types. Ruchi Patel, a PhD candidate in the laboratory of Marco Ruella, MD, an assistant professor of Hematology-Oncology, will present the findings in an oral session on Saturday, Dec. 9 at 10:45 a.m. PT in the San Diego Convention Center Room 6DE.