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

Park, Kyung Mi; Yoo, Keon Hee; Kim, Seongkoo; Lee, Jae Woo; Chung, Nack‐Gyun; Ju, Hee Young; Koo, Hong Hoe; Lyu, Chuhl Joo; Han, Seung Min; Han, Jung Woo; Choi, Jung Yoon; Hong, Kyung Taek; Kang, Hyoung Jin; Shin, Hee Young; Im, Ho Joon; Koh, Kyung‐Nam; Kim, Hyery; Kook, Hoon; Baek, Hee Jo; Kim, Bo Ram; Yang, Eu Jeen; Lim, Jae Young; Park, Eun Sil; Choi, Eun Jin; Park, Soo Young; Lee, Jae Min; Shim, Ye Jee; Kim, Ji Yoon; Park, Ji Kyoung; Kong, Seom Gim; Choi, Young Bae; Cho, Bin; Lim, Young Tak

doi:10.4143/crt.2021.313

Cited by 5 publications

(4 citation statements)

References 21 publications

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“…There was no difference in median age between the standard-and high-risk groups [43]. These patterns have been maintained across multiple clinical trials and are comparable to risk breakdown in adult studies [44,45]. Thus, children do not appear to be more likely to have a high or standard risk of disease compared to adults.…”

Section: Risk Categorymentioning

confidence: 64%

“…The severity can range from isolated lab abnormalities to severe bleeding, including intracranial or pulmonary hemorrhage, which can be lethal. Severe bleeding events have been reported in up to 15% of children with APL, leading to early death in up to 10% [43][44][45]. In particular, central nervous system (CNS) haemorrhage can be challenging to diagnose in young children who are unable to communicate symptoms early in the disease process.…”

Section: Severe Coagulopathy At Diagnosismentioning

confidence: 99%

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Management of Acute Promyelocytic Leukemia at Extremes of Age

Käyser

Conneely

2023

Cancers

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Tailored treatment with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) has revolutionized the outcome of acute promyelocytic leukemia (APL) from a uniformly fatal disease to one of the most curable malignant diseases in humans. Due to its high efficacy, ATO/ATRA is the standard first-line therapy in younger adult, non-high-risk APL patients. However, early death is still a major issue in APL, particularly in older patients. Thus, rapid diagnostics, immediate access to ATRA-based therapy, and supportive care are of utmost importance. Nevertheless, challenging situations occur, particularly in patients excluded from controlled studies with clinical knowledge mainly based on case reports and registries. Besides the treatment of newly diagnosed patients, managing toxicities and complications remains challenging. This review discusses the approach to the treatment of APL in elderly and pediatric patients.

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Section: Risk Categorymentioning

confidence: 64%

Section: Severe Coagulopathy At Diagnosismentioning

confidence: 99%

Management of Acute Promyelocytic Leukemia at Extremes of Age

Käyser

Conneely

2023

Cancers

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“…Most KPHOG studies have been retrospective because of limited resources, and six articles have been published in peer-reviewed journals in the recent 2 years. 18 - 21 Currently, four multicenter prospective clinical trials are ongoing for childhood cancers including three ALL studies participated by three institutions and one AML study by four institutions. Although the numbers of participating centers are small, these include about 40% and 50% of patients in Korea, respectively.…”

Section: Methodsmentioning

confidence: 99%

Current Challenges of Asian National Children's Cancer Study Groups on Behalf of Asian Pediatric Hematology and Oncology Group

Li,

Kurkure,

Arora

et al. 2023

JCO Global Oncology

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In Asia, a few countries have a long and established history of collaborative clinical trials successfully formed national children's cancer study groups, but many still do not have such groups. The process of forming national children's cancer groups is fraught with many hurdles, which varies among the countries. One of the basic requirements for running clinical trials is an affordable health care system in which most of the children with cancer can receive the proposed treatment. The health insurance coverage for children with cancer varies from <20% to as high as 100% among Asian countries, and the operation of clinical trials must also be adjusted accordingly. Shortage of research personnel is common, including medical, nursing, research coordinators, and data managers. The establishment of the Asian Pediatric Hematology and Oncology Group aims to provide a good platform for promotion of international clinical trials in the Asian countries.

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“…Furthermore, APL is a rare type of leukemia, accounting for only 5-10% of pediatric AML cases [1]. A recent study conducted in patients aged ＜18 y from Korea who were diagnosed with AML from 2009 to 2016 reported that 10% of them were diagnosed with APL [2]. APL is usually associated with coagulopathy and thrombotic complications [3], which increase the risk of fatal hemorrhage.…”

Section: Introductionmentioning

confidence: 99%

Successful Treatment of Isolated Central Nervous System Relapse with Intrathecal Chemotherapy in an Adolescent with Acute Promyelocytic Leukemia

Song

2022

Clin Pediatr Hematol Oncol

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Acute promyelocytic leukemia (APL) is a subtype of acute myeloid leukemia (AML) characterized by translocation of chromosomes 15 and 17 (t(15;17)), which rarely occurs in the pediatric population. Isolated central nervous system (CNS) relapse is particularly rare in children with APL, and a standard treatment has not yet been established. In this case, a 15-year-old male adolescent with APL had no CNS involvement at the initial diagnosis. After remission induction therapy with idarubicin, all-trans retinoic acid, and intrathecal (IT) cytarabine, a bone marrow (BM) study revealed morphological and cytogenetic complete remission. However, immature myeloid cells and blasts were observed in the cerebrospinal fluid (CSF). IT triple chemotherapy (ITT) consisting of cytarabine, methotrexate, and hydrocortisone was administered in addition to the planned systemic chemotherapy. ITT was performed weekly for 6 weeks with consolidation therapy, and then every 3 months for 2 years during the period of maintenance therapy. Abnormal immature cells were no longer detected after the second ITT administration. The patient survived without evidence of BM or CNS relapse until 1 year after treatment completion. The combination of IT chemotherapy might be a possible treatment option for CNS relapse in patients with APL, and further studies are needed to establish an optimal treatment strategy for them.

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

Abstract: contributed equally to this work. Running title: Characteristics and outcomes of childhood APL.

Cited by 5 publications

References 21 publications

Management of Acute Promyelocytic Leukemia at Extremes of Age

Management of Acute Promyelocytic Leukemia at Extremes of Age

Current Challenges of Asian National Children's Cancer Study Groups on Behalf of Asian Pediatric Hematology and Oncology Group

Successful Treatment of Isolated Central Nervous System Relapse with Intrathecal Chemotherapy in an Adolescent with Acute Promyelocytic Leukemia

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