By Beth Gilbert HealthDay Reporter
The annual meeting of the American Society of Hematology was held from Dec. 9 to 12 in San Diego and attracted participants from around the world, including hematology specialists as well as clinical practitioners and other health care professionals. The conference featured presentations focusing on the diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems.
In one study, Julie Kanter, M.D., of the University of Alabama in Birmingham, and colleagues found that treatment of patients with sickle cell disease (SCD) with lovotibeglogene autotemcel (lovo-cel) results in sustained antisickling hemoglobin production and nearly complete resolution of vaso-occlusive events and severe vaso-occlusive events up to 18 months following treatment.
The authors reported on efficacy and safety from the phase 1/2 HGB-206 and phase 3 HGB-210 studies of lovo-cel. Data were included from adult and pediatric (age 12 to <18 years) patients in the HGB-210 study and health-related quality-of-life data from the HGB-206 study. The researchers found that one-time treatment with lovo-cel resulted in near-complete resolution of acute pain events (vaso-occlusive crises) for all patients, with complete resolution of moderate and severe crises in 88 and 94 percent of patients, respectively. In addition, 100 percent of adolescent patients demonstrated complete resolution of all vaso-occlusive crises. A majority of patients were free of acute pain events through last follow-up (median, 35.5 months). The safety profile of the lovo-cel treatment regimen was consistent with underlying SCD and known effects of myeloablative conditioning.
“Lovo-cel uses a gene that makes hemoglobin A (similar to the adult HbA that is naturally occurring) and is highly effective and durable at improving hemoglobin, almost completely resolving hemolysis and reducing patient reported pain intensity and interference,” Kanter said. “We will certainly be discussing this therapy with people with frequent acute pain events who are interested in pursuing a transformative treatment and also pursuing payment options.”
Several authors disclosed financial ties to pharmaceutical and biotechnology companies, including Bluebird Bio, which manufactures lovo-cel.
Abstract No. 1051
In another study, Jatinder Lamba, Ph.D., of the University of Florida in Gainesville, and colleagues found that low ACS10 (10-SNP Ara-C_SNP) scores are more prevalent in Black patients with acute myeloid leukemia (AML) and are associated with inferior outcomes among patients treated with standard (low-dose cytarabine) induction therapy.
The authors compared results from an investigation of ACS10 differences between Black and White patients with AML across three treatment arms. The researchers observed a greater abundance of low ACS10 scores in Black patients (70 percent) compared with White patients (30 percent). They also found a low ACS10 score was associated with poor outcomes in patients treated on a standard dose of cytarabine, with the use of an intensified regimen improving outcomes in the groups with low ACS10 scores.
“The negative impact of low ACS10 score was mitigated by intensification of cytarabine during induction therapy, suggesting that Black and White patients with low ACS10 scores may benefit from this intervention,” Lamba said. “Using genotype-guided intensification regimens can help overcome the negative impact of low ACS10 score by intensification of cytarabine during induction therapy. This can further mitigate racial differences in outcomes given that Black patients have high abundance of low ACS10 scores.”
Several authors disclosed financial ties to the pharmaceutical and biotechnology industries.
Abstract No. 386
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