Clin Pediatr Hematol Oncol.  2016 Oct;23(2):105-115. 10.15264/cpho.2016.23.2.105.

Characteristics and Therapeutic Outcomes of Acute Promyelocytic Leukemia in Children and Adolescents

• Kim G
• Lee HO
• Choi JS
• Baek HJ
• Kook H

• KMID: 2358366
• DOI: http://doi.org/10.15264/cpho.2016.23.2.105

Abstract

BACKGROUND
Acute promyelocytic leukemia (APL) is rare in children, accounting for 5-7% of all acute myeloid leukemia (AML) cases. This study aimed to review clinical and laboratory characteristics of pediatric APL patients, and to analyze the therapeutic outcomes.
METHODS
Twenty pediatric patients with APL, diagnosed from January 1998 to April 2016, were retrospectively reviewed. Clinical and laboratory findings were collected, and morphologic, immunophenotypic, cytogenetic and molecular characteristics were evaluated. Therapeutic outcomes and prognostic factors of patients were analyzed.
RESULTS
The number of pediatric APL patients was 20, which was 18.5% of all the cases of AML. The 5-year overall survival (OS) and event-free survival (EFS) were 88.5±7.6% and 83.0±9.0%, respectively. As compared to typical APL patients, patients with microgranular variants (M3v) (n=4, 20%) showed a tendency for a younger age, low white blood cell counts, and a shorter prothrombin time (P=0.045), but their 5-year OS and EFS were not significantly different. Better survival was observed in ATRA plus chemotherapy group both for 5-year OS (100% vs. 60.0±21.9%, P=0.018), and for 5-year EFS (60.0±21.9% vs. 91.7±8.0%, P=0.080) than ATRA only group. Stem cell transplantations were given to 3 patients and they are alive without disease for 8.3-16.5 years of follow-up. One death after relapse, another death in remission and development of secondary leukemia were encountered during the study period.
CONCLUSION
This study analyzed clinical characteristics of pediatric APL and demonstrated very good outcome with the combination of ATRA and chemotherapy.

Keyword

Acute promyelocytic leukemia; Children; All-trans retinoic acid

MeSH Terms

Adolescent*
Child*
Cytogenetics
Disease-Free Survival
Drug Therapy
Follow-Up Studies
Humans
Leukemia
Leukemia, Myeloid, Acute
Leukemia, Promyelocytic, Acute*
Leukocyte Count
Prothrombin Time
Recurrence
Retrospective Studies
Stem Cell Transplantation