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Outcomes of Acute Promyelocytic Leukaemia in Paediatric Patients: Insights from a Low-Middle-Income Country
Shaista Naz1, Tariq Ghafoor1, Rabiha Manzoor1
1Department of Paediatric Oncology, Combined Military Hospital, Rawalpindi, Pakistan.
Insights
Treatment delays significantly worsen outcomes for paediatric acute promyelocytic leukaemia (APL). Arsenic trioxide (ATO) therapy shows promise for improved survival in APL patients, especially in resource-limited settings.
Area of Science:
- Paediatric Oncology
- Hematology
- Clinical Research
Background:
- Acute promyelocytic leukaemia (APL) is a distinct subtype of acute myeloid leukaemia.
- Understanding prognostic variables is crucial for improving outcomes in paediatric APL.
- Pakistan faces unique challenges in managing paediatric cancers.
Purpose of the Study:
- To identify prognostic variables and describe the clinical course of paediatric acute promyelocytic leukaemia (APL) in Pakistan.
- To evaluate the impact of treatment delays on survival outcomes.
- To compare the efficacy of different treatment protocols.
Main Methods:
- A cohort study was conducted from January 2012 to December 2022 at the Combined Military Hospital, Rawalpindi, Pakistan.
- Included were 50 paediatric patients (1-15 years) with confirmed APL (PML-RARA positive).
- Data collected included demographics, clinical presentation, treatment received (ICC-APL vs. ATO), and outcomes; statistical analysis used SPSS 25.0.
Main Results:
- Males constituted 64% of the 50 APL cases, with a mean age of 7.02 years.
- Common symptoms included pallor (96%) and fever (88%); 54% were high-risk.
- Treatment delays over one month significantly increased induction deaths (6.7% to 35%) and reduced disease-free survival (DFS) and overall survival (OS).
- Arsenic trioxide (ATO)-based therapy in standard-risk cases showed the best OS and DFS (80%).
Conclusions:
- Neutropenic fever and bleeding are primary causes of mortality during APL induction.
- Treatment delay is a critical prognostic factor negatively impacting survival.
- ATO-based therapy is a safer and effective option, improving DFS and OS, suitable for primary healthcare settings.
Objective:
To describe the prognostic variables and course of paediatric acute promyelocytic leukaemia (APL) in Pakistan.
Study Design:
Cohort study. Place and Duration of the Study: Department of Paediatric Oncology, Combined Military Hospital, Rawalpindi, Pakistan, from January 2012 to December 2022.
Methodology:
Patients aged 1-15 years, clinically confirmed APL with promyelocytic leukaemia- retinoic acid receptor alpha (PML-RARA) were enrolled. Initial admission included a thorough examination, recording demographic and clinical data, reporting time, prior treatment, and socioeconomic status. Statistical analysis used SPSS 25.0, with significance at p <0.05.
Results:
This study included 50 cases of APL. Out of which, 32 (64%) were males and 18 (34%) were females. The mean age at diagnosis was 7.02 ± 3.86 years. Pallor (96%) and fever (88%) were common presentations. The average white blood cell count was 28.70 ± 35.39 x109/L. Treatment protocols include 48% International Consortium for Childhood (ICC)-APL, and 52% arsenic trioxide (ATO). High-risk cases were 54%. Neutropenic fever and differentiation syndrome were common induction complications. Delays over one month increased induction deaths (6.7 to 35%, p = 0.011), reducing disease-free survival (DFS), (76.7 to 35%, p = 0.001), and overall survival (OS), (80 to 45%, p = 0.007). After 40.90 ± 45.19 months' follow-up, 10-year OS and DFS were 66.0% and 60.0%, respectively. The best OS and DFS, at 80%, were observed in standard-risk cases treated with ATO.
Conclusion:
Neutropenic fever and bleeding were the primary causes of mortality in paediatric APL induction. Treatment delay was a key prognostic factor. ATO-based therapy offered safer, improved DFS, and OS suitable for primary healthcare settings.
Key Words:
Acute promyelocytic leukaemia, Chemotherapy, Neutropenic fever.
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