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<p>With increasing survival in acute lymphoblastic leukemia (ALL), long-term toxicities have become a critical aspect. A novel measure, designated Severe Toxicity-free survival (STFS), was developed through international consensus to integrate the most severe, symptomatic organ toxicities in outcome evaluation. This measure has not been applied to real-world data before. We assessed the incidence of 21 predefined Severe Toxicities in a nationwide cohort of 506 ALL patients aged 1–45 years treated according to the NOPHO ALL2008 protocol. At five years, event-free survival was 84.4% (95% CI: 81.3–87.7%) and Severe Toxicity-event-free survival was 78.4% (95% CI: 74.9–82.1%), with significantly lower values in adults (aged 18–45 years) than children (61.6% [52.6–72.2%] vs 82.4% [78.8–86.2%]; log-rank <i>p</i> &lt; 0.001). The most common Severe Toxicities were severe osteonecrosis limiting activities of daily function (<i>N</i> = 20) and disabling paralytic and neuropathic conditions (<i>N</i> = 16). Exploratory analyses showed that 10–17-year-olds had the highest risk of Severe Toxicities similar to that of adults. These findings highlight a burden of severe, long-term toxicities in ALL survivors overlooked by traditional outcome measures, also following frontline therapy only. STFS should be incorporated in future trials for meaningful outcome evaluation and international comparisons across treatment strategies.</p>

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Severe toxicity-free survival following acute lymphoblastic leukemia in patients aged 1–45 years: a Danish cohort study

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年2月10日

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  • Camilla Grud Nielsen,
  • Bodil Als-Nielsen,
  • Birgitte Klug Albertsen,
  • Ólafur Birgir Davídsson,
  • Andreja Dimitrijevic,
  • Henrik Hjalgrim,
  • Marianne Ifversen,
  • Louise Lundgren,
  • Line Stensig Lynggaard,
  • Marianne Olsen,
  • Ulrik Malthe Overgaard,
  • Cecilie Utke Rank,
  • Mathias Rathe,
  • Klaus Rostgaard,
  • Kjeld Schmiegelow,
  • Liv Andrés-Jensen

摘要

With increasing survival in acute lymphoblastic leukemia (ALL), long-term toxicities have become a critical aspect. A novel measure, designated Severe Toxicity-free survival (STFS), was developed through international consensus to integrate the most severe, symptomatic organ toxicities in outcome evaluation. This measure has not been applied to real-world data before. We assessed the incidence of 21 predefined Severe Toxicities in a nationwide cohort of 506 ALL patients aged 1–45 years treated according to the NOPHO ALL2008 protocol. At five years, event-free survival was 84.4% (95% CI: 81.3–87.7%) and Severe Toxicity-event-free survival was 78.4% (95% CI: 74.9–82.1%), with significantly lower values in adults (aged 18–45 years) than children (61.6% [52.6–72.2%] vs 82.4% [78.8–86.2%]; log-rank p < 0.001). The most common Severe Toxicities were severe osteonecrosis limiting activities of daily function (N = 20) and disabling paralytic and neuropathic conditions (N = 16). Exploratory analyses showed that 10–17-year-olds had the highest risk of Severe Toxicities similar to that of adults. These findings highlight a burden of severe, long-term toxicities in ALL survivors overlooked by traditional outcome measures, also following frontline therapy only. STFS should be incorporated in future trials for meaningful outcome evaluation and international comparisons across treatment strategies.