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. 2025 Jul 30;17(7):e89081.
doi: 10.7759/cureus.89081. eCollection 2025 Jul.

Assessing the Impact of All-Trans Retinoic Acid (ATRA)- and Arsenic Trioxide (ATO)-Based Therapy in Pediatric Acute Promyelocytic Leukemia: A Single-Center Study

Reshma Roshan  1 , Mubashir H Shah  2 , Sherook J  1 , Aidel F Parry  1 , Javid R Bhat  1
Affiliations

Assessing the Impact of All-Trans Retinoic Acid (ATRA)- and Arsenic Trioxide (ATO)-Based Therapy in Pediatric Acute Promyelocytic Leukemia: A Single-Center Study

Reshma Roshan et al. Cureus. .

Abstract

Background: Acute promyelocytic leukemia (APL) is a subtype of acute myeloid leukemia (AML) characterized by the t(15;17) translocation, leading to the PML-RARA fusion gene. While treatable, APL presents significant challenges, particularly in resource-constrained settings where delays in diagnosis and access to specialized care may impact outcomes. This study aims to describe the clinical presentation, treatment outcomes, and survival data for pediatric APL patients.

Methods: This observational, retrospective, single-center study was conducted at Sher-i-Kashmir Institute of Medical Sciences (SKIMS), Srinagar, spanning for a period of six years. The study included 20 pediatric patients diagnosed with APL. Laboratory profiles, treatment regimens, and complications were analyzed. Risk stratification was done using modified Sanz criteria, and patients were treated according to the APL0406 and APML4 protocols. Overall survival (OS) and Progression-free survival (PFS) were calculated over a median follow-up period of three years.

Results: Median OS was 88 months (95% CI= 79.612 to 97.188). The mean haemoglobin levels were 6.51 ± 1.82 mg/dL and patients had high PML-RARA transcript levels (5175.95 ± 3039.37). Common symptoms included mucocutaneous bleeding (19, 95%) and gum bleeding (10, 50%). Induction with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) lasted a mean of 40.9 days. Febrile neutropenia (16, 80%) and differentiation syndrome (14, 70%) were frequent complications. One patient (1, 5%) died during induction.

Conclusion: This study reinforces the effectiveness of ATRA-ATO-based regimens in managing paediatric APL. However, induction-related complications such as febrile neutropenia, transaminitis, and QTc prolongation highlight the need for vigilant monitoring and robust supportive care. Timely diagnosis and early initiation of therapy remain key to improving outcomes.

Keywords: acute myeloid leukemia (aml); high-risk patients; induction therapy; pediatric patients; pml-rara fusion gene; risk stratification; treatment regimen.

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Conflict of interest statement

Human subjects: Informed consent for treatment and open access publication was obtained or waived by all participants in this study. Institutional Ethics Committee of Sher-i-Kashmir Institute of Medical Sciences (SKIMS) issued approval IEC/SKIMS Protocol No. 307/2022. The study was conducted in accordance with the ethical principles outlined in the Declaration of Helsinki (2024). Animal subjects: All authors have confirmed that this study did not involve animal subjects or tissue. Conflicts of interest: In compliance with the ICMJE uniform disclosure form, all authors declare the following: Payment/services info: All authors have declared that no financial support was received from any organization for the submitted work. Financial relationships: All authors have declared that they have no financial relationships at present or within the previous three years with any organizations that might have an interest in the submitted work. Other relationships: All authors have declared that there are no other relationships or activities that could appear to have influenced the submitted work.

Figures

Figure 1
Figure 1. Consolidated Standards of Reporting Trials (CONSORT) Flow Diagram of Pediatric Patients Diagnosed With Acute Promyelocytic Leukemia (APL)
AML: acute myeloid leukemia
Figure 2
Figure 2. Overall Survival Analysis
Figure 3
Figure 3. Progression free survival (PFS) analysis
See this image and copyright information in PMC

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