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Background <p>Pediatric-inspired regimens (PIR) yield excellent outcomes for acute lymphoblastic leukemia (ALL). Despite national guidelines recommending PIR for adolescents and young adults (AYAs; aged 15–39 years), the use of guideline-concordant PIR in AYAs is inconsistent across treatment settings.</p> Methods <p>This retrospective observational study compared overall survival (OS) and stem cell transplant (SCT) use among AYAs treated with PIR versus non-PIR. Using a deidentified, geographically representative USA health claims database, we analyzed a cohort of AYAs with newly diagnosed ALL between 1 July 2007 and 30 September 2020.</p> Results <p>Among 599 patients who met the inclusion criteria, 187 (31%) received PIR, 303 (51%) received non-PIR, and for 109 (18%), treatment was undetermined. PIR and non-PIR groups were propensity score matched (<i>n</i> = 187 each). Using Kaplan–Meier methodology, OS was significantly higher for those treated with PIR versus non-PIR (log-rank <i>P</i> = 0.0001; hazard ratio [HR], 0.31; 95% confidence interval [CI], 0.18–0.55). For PIR and non-PIR, 1-, 3-, and 5-year survival estimates (95% CI) were 98.1% (95.3–99.3%) versus 88.3% (83.5–91.7%); 88.5% (82.6–92.5%) versus 69.1% (62.1–75.1%); and 87.3% (81.0–91.6%) versus 63.3% (55.2–70.3%). Sensitivity and subgroup analyses were consistent with primary results. By Kaplan–Meier methodology, SCT use was significantly lower among AYAs treated with PIR than non-PIR (log-rank <i>P</i> = 0.0004; HR, 0.46; 95% CI 0.30–0.71); use by 2 years was 16.5% (12.2–22.2%) and 33.4% (27.6–40.0%), respectively.</p> Conclusions <p>These results support the use of guideline-concordant PIR for AYA ALL.</p>

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Survival Outcomes with Guideline-Concordant Pediatric-Inspired Regimens in Adolescent/Young Adult Acute Lymphoblastic Leukemia: Retrospective Real-World Analysis

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年1月12日

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  • David R. Freyer,
  • Jian Zhu,
  • Michael E. Roth,
  • Julie A. Wolfson,
  • Qian Meng,
  • Rachana Agarwal,
  • Rui Sammi Tang,
  • Tarun Bhagnani

摘要

Background

Pediatric-inspired regimens (PIR) yield excellent outcomes for acute lymphoblastic leukemia (ALL). Despite national guidelines recommending PIR for adolescents and young adults (AYAs; aged 15–39 years), the use of guideline-concordant PIR in AYAs is inconsistent across treatment settings.

Methods

This retrospective observational study compared overall survival (OS) and stem cell transplant (SCT) use among AYAs treated with PIR versus non-PIR. Using a deidentified, geographically representative USA health claims database, we analyzed a cohort of AYAs with newly diagnosed ALL between 1 July 2007 and 30 September 2020.

Results

Among 599 patients who met the inclusion criteria, 187 (31%) received PIR, 303 (51%) received non-PIR, and for 109 (18%), treatment was undetermined. PIR and non-PIR groups were propensity score matched (n = 187 each). Using Kaplan–Meier methodology, OS was significantly higher for those treated with PIR versus non-PIR (log-rank P = 0.0001; hazard ratio [HR], 0.31; 95% confidence interval [CI], 0.18–0.55). For PIR and non-PIR, 1-, 3-, and 5-year survival estimates (95% CI) were 98.1% (95.3–99.3%) versus 88.3% (83.5–91.7%); 88.5% (82.6–92.5%) versus 69.1% (62.1–75.1%); and 87.3% (81.0–91.6%) versus 63.3% (55.2–70.3%). Sensitivity and subgroup analyses were consistent with primary results. By Kaplan–Meier methodology, SCT use was significantly lower among AYAs treated with PIR than non-PIR (log-rank P = 0.0004; HR, 0.46; 95% CI 0.30–0.71); use by 2 years was 16.5% (12.2–22.2%) and 33.4% (27.6–40.0%), respectively.

Conclusions

These results support the use of guideline-concordant PIR for AYA ALL.