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Background <p>Down syndrome (DS) is a common genetic disorder resulting from an extra copy of genetic material from all or part of chromosome 21. Individuals with DS have a higher burden of co-occurring structural birth defects, neurocognitive delay, and chronic health conditions when compared to those without DS, as well as a 10 to 20-fold excess risk for acute leukemia (AL). Few studies have reported the late effects of cancer treatment in DS-AL survivors, and even fewer have compared outcomes to children with DS and no cancer history. The Children’s Oncology Group study ALTE22C1 was developed to address this knowledge gap.</p> Methods <p>This study leverages both registry and site-based DS-AL survivor recruitment and a prospective/retrospective cohort design to compare chronic health conditions and neurocognitive outcomes experienced by DS-AL survivors to age and sex-matched individuals enrolled to a DS cohort for which cancer is exclusionary. Survivors 6–39&#xa0;years old, ≥ 3&#xa0;years from end of AL treatment, and in remission are eligible. Participants complete a medical conditions survey and neuropsychological battery by parent proxy and may also participate in an in-person physical and neurocognitive assessment. Biological samples are collected to evaluate molecular features associated with outcomes.</p> Discussion <p>This cooperative group study will identify the prevalence and severity of medical and neurocognitive outcomes in DS-AL survivors compared with non-DS AL and DS controls without cancer history. Results are anticipated to inform clinical practice guidelines for DS-AL survivors and improve survivor outcomes through mitigation of outcome disparities in this vulnerable population.</p> Trial registration <p>Children’s Oncology Group study ALTE22C1 is registered under the ClinicalTrials.gov identifier NCT05702645.</p>

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A multicenter observational cohort study in survivors of Down Syndrome-associated acute leukemia (ALTE22C1): a report from the Children’s Oncology Group

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年10月20日

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  • M. Monica Gramatges,
  • Lauren N. Sanclemente,
  • Lacey Hall,
  • Olga A. Taylor,
  • Michelle M. Nuño,
  • Smita Bhatia,
  • Eric J. Chow,
  • Kelly D. Getz,
  • Johann K. Hitzler,
  • Amanda M. Li,
  • Kaitlin McCloskey,
  • Paul C. Nathan,
  • Maureen M. O’Brien,
  • Serina Patel,
  • Anupam Verma,
  • Angela R. Yarbrough,
  • Melissa A. Richard,
  • Tracie C. Rosser,
  • Lisa M. Jacola,
  • Philip J. Lupo,
  • Karen R. Rabin

摘要

Background

Down syndrome (DS) is a common genetic disorder resulting from an extra copy of genetic material from all or part of chromosome 21. Individuals with DS have a higher burden of co-occurring structural birth defects, neurocognitive delay, and chronic health conditions when compared to those without DS, as well as a 10 to 20-fold excess risk for acute leukemia (AL). Few studies have reported the late effects of cancer treatment in DS-AL survivors, and even fewer have compared outcomes to children with DS and no cancer history. The Children’s Oncology Group study ALTE22C1 was developed to address this knowledge gap.

Methods

This study leverages both registry and site-based DS-AL survivor recruitment and a prospective/retrospective cohort design to compare chronic health conditions and neurocognitive outcomes experienced by DS-AL survivors to age and sex-matched individuals enrolled to a DS cohort for which cancer is exclusionary. Survivors 6–39 years old, ≥ 3 years from end of AL treatment, and in remission are eligible. Participants complete a medical conditions survey and neuropsychological battery by parent proxy and may also participate in an in-person physical and neurocognitive assessment. Biological samples are collected to evaluate molecular features associated with outcomes.

Discussion

This cooperative group study will identify the prevalence and severity of medical and neurocognitive outcomes in DS-AL survivors compared with non-DS AL and DS controls without cancer history. Results are anticipated to inform clinical practice guidelines for DS-AL survivors and improve survivor outcomes through mitigation of outcome disparities in this vulnerable population.

Trial registration

Children’s Oncology Group study ALTE22C1 is registered under the ClinicalTrials.gov identifier NCT05702645.