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Background <p>Treatment of paediatric acute myeloid leukaemia (AML) is challenging in low- and middle-income countries (LMICs) due to resource constraints with subsequent poorer outcome. This study evaluated treatment outcomes and the determinants of survival in paediatric AML patients in Uganda.</p> Methods <p>This retrospective cohort study reviewed data from children with AML treated at three centres in Uganda between January 2016 and December 2022. Treatment comprised induction with daunorubicin and cytarabine and consolidation with high-dose cytarabine. Patients with acute promyelocytic leukaemia were treated on protocols adapted from Children's Oncology Group AAML 1331. All patients received supportive care. The data were analysed using SPSS Version 20.</p> Results <p>One-hundred and fifty-nine children with AML were included with a median age at diagnosis of 9.0&#xa0;years (IQR: 3.0–12.0). Of the 149 patients who initiated therapy, 69 (46.3%) achieved complete remission after the first induction therapy (CR1), and 81 (54.4%) achieved complete remission (CR) overall. Treatment-related mortality occurred in 50 (31.4%) of the patients, with an early death rate of 27.7% (n = 44). Among the 81 patients who achieved CR, 37 (45.7%) relapsed, of whom 27 (73.0%) subsequently died. The one-, three-, and five-year OS were 39.0%, 25.1%, and 16.7%, respectively. The corresponding EFS were 37.0%, 22.9%, and 15.2%, respectively. Median OS and EFS were 7.4&#xa0;months (95% CI: 4.3–10.6) and 6.9&#xa0;months (95% CI: 4.4–9.6), respectively. Factors significantly associated with poor OS included poor nutritional status (<i>p</i> = 0.026), delayed neutrophil recovery following induction (<i>p</i> = 0.030), failure to achieve CR1 (<i>p</i> = 0.031), and failure to complete treatment (<i>p</i> &lt; 0.001).</p> Conclusions <p>Survival rates among children with AML in this study were low. Several clinical and biological prognostic factors influenced survival outcomes in this resource-limited setting. Improving outcomes will require improving supportive care or adopting resource-adapted treatment protocols that address the supportive care challenges in such a resource-limited setting.</p>

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Treatment outcomes and prognostic factors in children diagnosed with acute myeloid leukaemia in Uganda

  • 期刊论文

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  • 发表日期：

    2025年12月18日

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  • Richard Nyeko,
  • Mariana Kruger,
  • Nixon Niyonzima,
  • Barnabas Atwiine,
  • Jennifer Zungu,
  • Joyce Balagadde Kambugu,
  • Stijn Verhulst,
  • Jaques van Heerden

摘要

Background

Treatment of paediatric acute myeloid leukaemia (AML) is challenging in low- and middle-income countries (LMICs) due to resource constraints with subsequent poorer outcome. This study evaluated treatment outcomes and the determinants of survival in paediatric AML patients in Uganda.

Methods

This retrospective cohort study reviewed data from children with AML treated at three centres in Uganda between January 2016 and December 2022. Treatment comprised induction with daunorubicin and cytarabine and consolidation with high-dose cytarabine. Patients with acute promyelocytic leukaemia were treated on protocols adapted from Children's Oncology Group AAML 1331. All patients received supportive care. The data were analysed using SPSS Version 20.

Results

One-hundred and fifty-nine children with AML were included with a median age at diagnosis of 9.0 years (IQR: 3.0–12.0). Of the 149 patients who initiated therapy, 69 (46.3%) achieved complete remission after the first induction therapy (CR1), and 81 (54.4%) achieved complete remission (CR) overall. Treatment-related mortality occurred in 50 (31.4%) of the patients, with an early death rate of 27.7% (n = 44). Among the 81 patients who achieved CR, 37 (45.7%) relapsed, of whom 27 (73.0%) subsequently died. The one-, three-, and five-year OS were 39.0%, 25.1%, and 16.7%, respectively. The corresponding EFS were 37.0%, 22.9%, and 15.2%, respectively. Median OS and EFS were 7.4 months (95% CI: 4.3–10.6) and 6.9 months (95% CI: 4.4–9.6), respectively. Factors significantly associated with poor OS included poor nutritional status (p = 0.026), delayed neutrophil recovery following induction (p = 0.030), failure to achieve CR1 (p = 0.031), and failure to complete treatment (p < 0.001).

Conclusions

Survival rates among children with AML in this study were low. Several clinical and biological prognostic factors influenced survival outcomes in this resource-limited setting. Improving outcomes will require improving supportive care or adopting resource-adapted treatment protocols that address the supportive care challenges in such a resource-limited setting.