Pediatric-Inspired Regimen Leads to Higher OS in Teens, Young Adults with ALL

By Cecilia Brown - Last Updated: December 20, 2022

Adolescents and young adults with acute lymphoblastic leukemia (ALL) who received an asparaginase-containing, pediatric-inspired regimen had significantly longer overall survival (OS) compared with patients who received other regimens, according to research presented at the 2022 American Society of Hematology Annual Meeting.

“Pediatric oncologists have long understood the effectiveness of pediatric-inspired regimens for treating children and teenagers with most types of [ALL], but this analysis indicates those benefits extend to a broader population of older adolescents and young adults,” David R. Freyer, DO, MS, study co-author and Co-Director of the Adolescent and Young Adult Cancer Program at the University of Southern California Norris Comprehensive Care Center, said in a statement. “Although the use of pediatric-inspired regimens in young adults is inconsistent across adult centers, we believe this analysis supports their use in this patient population and hope it promotes further investigation to better understand their impact on overall survival and long-term outcomes.”

The researchers conducted a retrospective study using the Optum de-identified Clinformatics Data Mart, which comes from a database of administrative health claims from members of large United States commercial health plans and Medicare Advantage health plans. They examined patient records to ensure there was no evidence of ALL remission or relapse prior to the index date, no evidence of stem cell transplant prior to the index date, and at least six months of continuous health plan enrollment before the index date

The researchers found 599 young adult and adolescent patients with ALL who met the selection criteria, with 30% of those patients receiving a pediatric-inspired regimen and 51% receiving a non-pediatric-inspired regimen. The remaining 18% of patients were not included in the primary analyses due to insufficient information about their treatment. The researchers performed propensity score matching, resulting in 187 patients in each treatment group with balanced baseline characteristics.

Patients who received pediatric-inspired regimens had significantly higher OS (P<.01), with a 69% lower likelihood of death compared to those who received non-pediatric-inspired regimens. The estimated one-year OS was 98.1% in patients who received pediatric-inspired regimens, while it was 88.3% in those who received non-pediatric-inspired regimens. In patients who received pediatric-inspired regimens, the estimated five-year OS rate was 87.3%, while it was 63.3% in those who received non-pediatric-inspired regimens.

Infection was the most commonly reported adverse event within the one-year follow-up period, occurring in 61% of patients who received pediatric-inspired regimens and in 76% of those who received non-pediatric-inspired regimens (See TABLE 1 ).

TABLE 1: The Prevalence of Other Common Adverse Events within the First Year of Follow-Up

Adverse event type Pediatric-inspired regimen Non-pediatric-inspired regimen
Hyperglycemia 31% 22%
Hypokalemia 30% 40%
Hyperuricemia 26% 25%
Mucositis 25% 16%
Thrombosis 9% 20%


The researchers obtained results “consistent with the primary analysis” in multiple sensitivity analyses using conservative regimen definitions and varied time periods, according to the study’s authors.

“[Adolescent and young adult] patients with ALL treated with a [pediatric-inspired regimen] had significantly better OS as compared to those treated with a non-[pediatric-inspired regimen], in aggregate and within pre-defined subgroups,” the study’s authors concluded. “These results suggest that [pediatric-inspired regimens] may represent a superior treatment approach for most [adolescents and young adults] with newly-diagnosed ALL.”


Bhagnani T, Zhu JJ, Freyer DRR, et al. Comparison of overall survival among adolescents and young adults (AYA) with newly-diagnosed acute lymphoblastic leukemia (ALL) treated with pediatric-inspired or non-pediatric inspired regimens: a real-world analysis using claims data. Abstract #906. Presented at the 64th ASH Annual Meeting and Exposition; December 10-13, 2022; New Orleans, Louisiana.

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