Cancer Res Treat.  2022 Jan;54(1):269-276. 10.4143/crt.2021.313.

Clinical Characteristics and Treatment Outcomes of Childhood Acute Promyelocytic Leukemia in Korea: A Nationwide Multicenter Retrospective Study by Korean Pediatric Oncology Study Group

• Park KM
• Yoo KH
• Kim SK
• Lee JW
• Chung NG
• Ju HY
• Koo HH
• Lyu CJ
• Han SM
• Han JW
• Choi JY
• Hong KT
• Kang HJ
• Shin HY
• Im HJ
• Koh KN
• Kim H
• Kook H
• Baek HJ
• Kim BR
• Yang EJ
• Lim JY
• Park ES
• Choi EJ
• Park SK
• Lee JM
• Shim YJ
• Kim JY
• Park JK
• Kong SG
• Choi YB
• Cho B
• Lim YT

• KMID: 2524608
• DOI: http://doi.org/10.4143/crt.2021.313

Abstract

Purpose
Acute promyelocytic leukemia (APL) is a rare disease in children and there are some different characteristics between children and adult. We aimed to evaluate incidence, clinical characteristics and treatment outcomes of pediatric APL in Korea.
Materials and Methods
Seventy-nine pediatric APL patients diagnosed from January 2009 to December 2016 in 16 tertiary medical centers in Korea were reviewed retrospectively.
Results
Of 801 acute myeloid leukemia children, 79 (9.9%) were diagnosed with APL. The median age at diagnosis was 10.6 years (range, 1.3 to 18.0). Male and female ratio was 1:0.93. Thirty patients (38.0%) had white blood cell (WBC) count greater than 10×10⁹/L at diagnosis. All patients received induction therapy consisting of all-trans retinoic acid and chemotherapy. Five patients (6.6%) died during induction chemotherapy and 66 patients (86.8%) achieved complete remission (CR) after induction chemotherapy. The causes of death were three intracranial hemorrhage, one cerebral infarction, and one sepsis. Five patients (7.1%) suffered a relapse during or after maintenance chemotherapy. The estimated 4-year event-free survival and overall survival (OS) rates were 82.1%±4.4%, 89.7%±5.1%, respectively. The 4-year OS was significantly higher in patients with initial WBC < 10×10⁹/L than in those with initial WBC ≥ 10×10⁹/L (p=0.020).
Conclusion
This study showed that the CR rates and survival outcomes in Korean pediatric APL patients were relatively good. The initial WBC count was the most important prognostic factor and most causes of death were related to serious bleeding in the early stage of treatment.

Keyword

Acute promyelocytic leukemia; All-trans retinoic acid; Childhood

Figure

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  Fig. 1 Flow diagram of patients. APL, acute promyelocytic leukemia; Ara-C, anthracycline with or without cytosine arabinoside; ATRA, all-trans retinoic acid; CR, complete remission; CTx, chemotherapy; F/U, follow-up.

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  Fig. 2 Kaplan-Meier estimates of 4-year overall survival (OS) and event-free survival (EFS) for pediatric acute promyelocytic leukemia.

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  Fig. 3 Kaplan-Meier estimates of 4-year overall survival (A) (p=0.020) and event-free survival (B) (p=0.091) for pediatric acute promyelocytic leukemia according to initial white blood cell (WBC) count.

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  Fig. 4 Kaplan-Meier estimates of 4-year overall survival (A) (p=0.069) and event-free survival (B) (p=0.394) for pediatric acute promyelocytic leukemia according to all-trans retinoic acid (ATRA) dosage.

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