THE MEDICAL COLLEGE OF WISCONSIN, INC.

The FDA approved remestemcel-L (Ryoncil) for pediatric steroid-refractory acute graft-vs-host disease (SR-aGVHD), supported by phase 3 GVHD001/002 trial data. Remestemcel-L showed higher day 28 overall response (70.4%) and day 100 survival (87%) compared to best available therapy. A 4-year survival study indicated durable benefits, with 49% survival at 4 years. The approval followed a resubmission addressing FDA concerns.

Real-world data on CD19-directed CAR T-cell therapy lisocabtagene maraleucel (liso-cel) in relapsed/refractory large B-cell lymphoma (LBCL) show similar safety and efficacy to pivotal trials. Median progression-free survival (PFS) and overall survival (OS) were not reached (NR) at 6.4 months follow-up. Objective response rate (ORR) was 84%, with a complete response (CR) rate of 70%. Liso-cel demonstrated deep responses in a broad patient cohort, supporting its use as a second-line treatment regardless of age.

Real-world data on liso-cel (Breyanzi) in relapsed/refractory large B-cell lymphoma (LBCL) showed safety and efficacy comparable to pivotal trials, with median progression-free survival (PFS) and overall survival (OS) not reached (NR) at 6.4 months follow-up. Objective response rate (ORR) was 84% with a complete response (CR) rate of 70%, and median duration of response (DOR) was NR. These results support liso-cel as a second-line standard-of-care treatment for LBCL.

Liso-cel therapy in relapsed/refractory large B-cell lymphoma showed consistent PFS and OS with prior trials, with median PFS and OS not reached at 6.4 months follow-up. ORR was 84% with a CR rate of 70%, and median DOR was NR. Safety profile consistent with previous reports, with most patients experiencing low-grade CRS and ICANS.

Kite, a Gilead Company, announced findings from three new Yescarta analyses at the 66th ASH Annual Meeting, demonstrating improved outcomes for R/R LBCL patients. The largest RWE analysis showed a 71% OS rate in second-line treatment, consistent with ZUMA-7 outcomes. RWE also indicated a decreasing trend in CRS and ICANS incidence and severity over time in the third-line-plus setting. The ALYCANTE study revealed stable long-term HRQoL for R/R LBCL patients treated with Yescarta.

The FDA approved StemCyte’s Regenecyte, an allogeneic hematopoietic stem cell therapy from umbilical cord blood, for use in unrelated donor hematopoietic progenitor cell transplantation. Regenecyte aids in hematopoietic and immunologic reconstitution in patients with inherited or acquired disorders affecting the hematopoietic system. Clinical trial data showed significant recovery rates in neutrophils, platelets, and erythrocytes, with varying median recovery times. Common adverse reactions include hypertension, vomiting, nausea, bradycardia, and fever, with additional warnings and precautions for hypersensitivity reactions, graft versus host disease, and transmission of serious infections. Regenecyte is contraindicated for patients with known sensitivity to DMSO, Dextran 40, or plasma proteins.

Pediatric SCT disparities are influenced by SDOH like race, SES, and neighborhood poverty, affecting access, outcomes, and resource utilization. High-poverty neighborhoods correlate with higher TRM, and Medicaid insurance increases TRM risk. Black and Hispanic children face higher mortality and fewer suitable donors. Newborn screening reduces survival disparities, emphasizing early detection's importance. Addressing these disparities requires multifaceted strategies, including policy interventions and community engagement.

Research highlights measurable residual disease in acute myeloid leukemia (AML) before allogeneic transplant, emphasizing the importance of DNA sequencing and precision medicine. Studies also address racial and ethnic disparities in access to medical advancements, clinical trials, and allogeneic hematopoietic cell transplantation, with initiatives aiming to reduce barriers and improve outcomes for diverse populations.