Amongst pediatric and younger grownup sufferers with B-cell acute lymphoblastic leukemia (ALL), the removing of whole physique irradiation (TBI) from conditioning didn’t compromise efficacy, and was corresponding to printed outcomes, examine outcomes have proven.
Findings from the section 2 EndRAD examine have been offered on the 2025 American Society of Hematology Annual Assembly and Exposition.
The 2-year event-free survival (EFS) charge within the remedy arm was 76.3%, and the two-year total survival (OS) charge was 82%. These outcomes confirmed that the first finish level of the trial of EFS was met.
Glossary
Occasion-free survival (EFS): the size of time after remedy throughout which a affected person has no main setbacks, such because the most cancers coming again, getting worse, or critical unwanted side effects that cease remedy.
Total survival (OS): the whole size of time from prognosis or the beginning of remedy {that a} affected person remains to be alive, no matter whether or not the most cancers has returned or progressed.
Minimal residual illness (MRD): a really small variety of most cancers cells that stay within the physique after remedy, usually too few to be seen with normal assessments.
Allogeneic hematopoietic cell transplant: a process by which a affected person receives blood-forming stem cells from a donor — often a relative or a matched volunteer.
“Primarily based upon this information, pediatric and younger grownup sufferers who’re bone marrow next-generation sequencing (NGS)-minimal residual illness (MRD)-negative could contemplate non-TBI approaches as they select remedy,” Dr. Hisham Abdel-Azim, chief of Transplant and Mobile Remedy at Loma Linda College Well being, mentioned throughout the presentation.
A complete of 202 sufferers have been enrolled within the trial. The remedy arm (51 sufferers) was designed to estimate survival after non-TBI myeloablative conditioning previous to allogeneic hematopoietic cell transplant (HCT) for NGS-MRD-negative B-ALL. A complete of 86% of sufferers got busulfan, fludarabine and thiopeta. Different comparable therapies included fludarabine, melphalan and thiopeta (6%), and fludarabine, melphalan, clofarabine and thiopeta (6%). Within the observational arm (151 sufferers), sufferers got myeloablative HCT and weren’t eligible for the remedy arm.
Abdel-Azim said he and his colleagues hypothesized that the two-year EFS and OS charges in sufferers who’re NGS MRD destructive and receiving a non-TBI-based routine could be round 75% and 80%, respectively. The examine was performed in 45 facilities in North America between 2018 and 2025.
Infants weren’t allowed within the remedy arm, and younger youngsters who weren’t eligible and acquired non-TBI remedy have been included within the observational arm. Moreover, the observational arm included those that have been flow-negative however NGS-positive who acquired TBI.
Within the remedy arm, sufferers have been eligible for remedy in the event that they have been between 1 and 31 years previous and had high-risk full remission 1 (CR1) or CR2 standing. Receipt of prior CAR-T cell remedy and Blincyto (blinatumomab) was allowed. Sufferers have been excluded from the remedy arm however have been eligible to enroll on the observational arm in the event that they have been lower than 1 12 months previous, CR2 with a historical past of central nervous system relapse, had T-cell ALL and mixed-phenotype acute leukemia and had energetic CNS/extramedullary illness at HCT.
Within the remedy arm, the median age was 13.5 years, and 51% of sufferers have been male. Moreover, 37% had HLA-matched sibling donors, 35% had mismatched/unrelated haploidentical donors, 20% had matched unrelated donors and eight% had unrelated wire blood donors. Bone marrow graft was given in 71% of sufferers, peripheral blood stem cell graft was given to 21% of sufferers, and eight% acquired an unrelated wire blood graft. Moreover, at HCT, 49% of sufferers had CR1 and 51% had CR2.
“OS and EFS in our section 2 non-TBI remedy arm for NGS MRD-negative B-ALL matched our speculation and have been comparable with outcomes of sufferers who’re MRD-negative receiving TBI in earlier research,” Abdel-Azim concluded.
Reference
- “ Excessive event-free (EFS) and total survival (OS) after non-total physique irradiation (TBI) conditioning and allogeneic hematopoietic cell transplantation (HCT) in next-generation-sequencing minimal residual illness (NGS-MRD) destructive B-acute lymphoblastic leukemia (B-ALL): Outcomes from the EndRAD trial” by Dr. Hisham Abdel-Azim, et al., Blood.
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