Seven of 67 pediatric patients receiving Bluebird Bio’s elivaldogene autotemcel (eli-cel) for the treatment of cerebral adrenoleukodystrophy developed a blood cancer, according to a study published in the New England Journal of Medicine (NEJM).
Of the patients who developed a hematologic malignancy, six were diagnosed with myelodysplastic syndromes (MDS) and one with acute myeloid leukemia (AML). These hematologic malignancies occurred at 14, 26, 28, 36, 42, 57, and 92 months after receiving eli-cel treatment.
Five patients with MDS received allogeneic hematopoietic cell transplantation (alloHCT), four of whom are now free of MDS and have not had a recurrence of cerebral adrenoleukodystrophy symptoms. The fifth patient died after the transplant from graft-versus-host disease, and the final patient with MDS is awaiting alloHCT. The patient with AML received alloHCT and full donor chimerism.
“The cases are associated with clonal vector insertions within oncogenes and clonal evolution with acquisition of somatic genetic defects,” the researchers noted.
Bluebird Bio noted that blood cancers are a known risk of eli-cel, and this risk is included on the treatment’s label.
Sources: NEJM, October 9, 2024. BioSpace, October 10, 2024
