Roshan Reshma, Shah Mubashir H, J Sherook, Parry Aidel F, Bhat Javid R
Clinical Hematology, Sher-i-Kashmir Institute of Medical Sciences (SKIMS), Srinagar, IND.
Department of Pediatrics, Government Medical College Srinagar, Srinagar, IND.
Cureus. 2025 Jul 30;17(7):e89081. doi: 10.7759/cureus.89081. eCollection 2025 Jul.
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.
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.
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.
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.
急性早幼粒细胞白血病（APL）是急性髓系白血病（AML）的一种亚型，其特征为t(15;17)易位，导致PML-RARA融合基因。虽然APL是可治疗的，但它带来了重大挑战，尤其是在资源有限的环境中，诊断延迟和获得专科护理可能会影响治疗结果。本研究旨在描述儿童APL患者的临床表现、治疗结果和生存数据。
这项观察性、回顾性、单中心研究在斯利那加的谢里夫·克什米尔医学科学研究所（SKIMS）进行，为期六年。该研究纳入了20名诊断为APL的儿童患者。分析了实验室检查结果、治疗方案和并发症。采用改良的桑斯标准进行危险分层，并根据APL0406和APML4方案对患者进行治疗。在三年的中位随访期内计算总生存期（OS）和无进展生存期（PFS）。
中位OS为88个月（95%CI = 79.612至97.188）。平均血红蛋白水平为6.51±1.82mg/dL，患者的PML-RARA转录水平较高（5175.95±3039.37）。常见症状包括皮肤黏膜出血（19例，95%）和牙龈出血（10例，50%）。全反式维甲酸（ATRA）和三氧化二砷（ATO）诱导持续时间平均为40.9天。发热性中性粒细胞减少症（16例，80%）和分化综合征（14例，70%）是常见并发症。1例患者（1例，5%）在诱导期死亡。
本研究强化了基于ATRA-ATO方案治疗儿童APL的有效性。然而，诱导相关并发症，如发热性中性粒细胞减少症、转氨酶升高和QTc延长，凸显了密切监测和有力支持治疗的必要性。及时诊断和早期开始治疗仍然是改善治疗结果的关键。
