Pediatric and AYA B-ALL Treatment shows Promise Without Harsh Total Body Irradiation
PHILADELPHIA – A new study offers hope for young patients with B-acute lymphoblastic leukemia (B-ALL), demonstrating that allogeneic hematopoietic cell transplantation (HCT) can be highly effective even without the use of total body irradiation (TBI), a historically standard but often debilitating component of the treatment regimen. Results from the phase 2 EndRAD trial, presented by Dr. Hisham Abdel-Azim and colleagues, reveal comparable event-free survival (EFS) and overall survival (OS) rates to those achieved with TBI-based conditioning in patients with minimal residual disease (MRD)-negative B-ALL.
This finding is particularly significant for pediatric and adolescent/young adult (AYA) patients, as TBI is associated with long-term side effects impacting growth, fertility, and neurocognitive growth. The EndRAD trial suggests a path toward reducing these toxicities without compromising treatment success, potentially improving the quality of life for survivors. B-ALL is the most common childhood cancer, and while survival rates have improved dramatically, the intensive nature of treatment continues to pose challenges. These results offer a crucial step toward more refined, less harmful therapies.
The study focused on patients with B-ALL who achieved MRD negativity using next-generation sequencing (NGS) prior to HCT. The treatment arm included patients with a median age of 13.5 years,with 51% being male. Donor sources varied, with 37% receiving grafts from HLA-matched siblings, 35% from mismatched/unrelated haploidentical donors, 20% from matched unrelated donors, and 8% from unrelated cord blood donors. The majority (71%) received bone marrow grafts, while 21% received peripheral blood stem cell grafts and 8% received cord blood grafts. At the time of HCT, 49% of patients were in first complete remission (CR1) and 51% were in second complete remission (CR2).
“OS and EFS in our phase 2 non-TBI treatment arm for NGS MRD-negative B-ALL matched our hypothesis and were comparable with outcomes of patients who are MRD-negative receiving TBI in previous studies,” Dr. Abdel-Azim concluded.
The research,published in Blood,provides compelling evidence for a potential shift in standard practice,offering a less toxic yet equally effective treatment option for a vulnerable patient population.
Reference:
“High event-free (EFS) and overall survival (OS) after non-total body irradiation (TBI) conditioning and allogeneic hematopoietic cell transplantation (HCT) in next-generation-sequencing minimal residual disease (NGS-MRD) negative B-acute lymphoblastic leukemia (B-ALL): Results from the EndRAD trial” by Dr. Hisham Abdel-Azim, et al., Blood.
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