Purpose: Chronic myeloid leukemia (CML) in children, adolescents, and young adults is rare and differs from older adults. This study evaluated the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) in young Korean CML patients during the tyrosine kinase inhibitor (TKI) era. Materials and Methods: A retrospective analysis of 35 CML patients aged < 40 years who underwent allogeneic HSCT from 2009 to 2019 was conducted using Korean Blood and Marrow Transplantation Registry data. Patients were grouped by age < 20 years at HSCT (group 1, n=15) and 20-40 years at HSCT (group 2, n=20). Survival outcomes including overall survival (OS), relapse-free survival (RFS), and event-free survival (EFS) were analyzed using the Kaplan-Meier method. Results: The median time between diagnosis and HSCT was 8.9 months. All the patients achieved engraftment but platelet recovery was significantly slower in group 1 (p=0.034). Acute and chronic graft-versus-host disease occurred in 54.3% and 34.3%, respectively. Five-year OS, RFS, and EFS rates of total patients were 66.8%, 50.8%, and 47.6%, with better OS was observed in group 1 by multivariable analysis (p=0.048). Disease status at HSCT was a significant predictor of OS (p=0.028), RFS (p=0.003), and EFS (p=0.004). Disease progression occurred in 13 out of 35 patients (37.1%); treatment-related mortality accounted for 63.6% of deaths (7 out of 11). Conclusion When performed at a younger age, allogeneic HSCT result in superior outcome in CML. Achieving remission before HSCT is critical for improved outcomes, highlighting the importance of pretransplant remission via optimal TKI strategies and minimal residual disease monitoring.

BACKGROUND: The number of patients diagnosed with hereditary hemolytic anemia (HHA) has increased since the advent of novel diagnostic techniques that accurately identify this disorder. Here, we report data from a survey on the prevalence and characteristics of patients diagnosed with HHA in Korea from 2007 to 2011. METHODS: Information on patients diagnosed with HHA in Korea and their clinical and laboratory results were collected using a survey questionnaire. Globin gene and red blood cell (RBC) enzyme analyses were performed. In addition, we analyzed data collected by pediatricians. RESULTS: In total, 195 cases of HHA were identified. Etiologies identified for HHA were RBC membranopathies, hemoglobinopathies, and RBC enzymopathies, which accounted for 127 (64%), 39 (19.9%), and 26 (13.3%) cases, respectively. Of the 39 patients with hemoglobinopathies, 26 were confirmed by globin gene analysis, including 20 patients with beta-thalassemia minor, 5 patients with alpha-thalassemia minor, and 1 patient with unstable hemoglobin disease. CONCLUSION: The number of patients diagnosed with hemoglobinopathies and RBC enzymopathies has increased considerably since the previous survey on HHA in Korea, dated from 1997 to 2006. This is likely the result of improved diagnostic techniques. Nevertheless, there is still a need for more sensitive diagnostic tests utilizing flow cytometry and for better standardization of test results to improve the accuracy of diagnosis of RBC membranopathies in Korea. Additionally, more accurate assays for the identification of RBC enzymopathies are warranted.
alpha-Thalassemia ; Anemia, Hemolytic, Congenital ; beta-Thalassemia ; Diagnostic Tests, Routine ; Erythrocytes ; Flow Cytometry ; Globins ; Hematology ; Hemoglobinopathies ; Hemoglobins ; Humans ; Korea ; Prevalence ; Spherocytosis, Hereditary ; Thalassemia ; Surveys and Questionnaires

PURPOSE: Hemophagocytic lymphohistiocytosis (HLH) is a rare but fatal disorder characterized by fever, splenomegaly, pancytopenia, and hemophagocytosis in the bone marrow and other tissues. In this study, we investigated the clinical manifestations and prognostic factors in patients with HLH. METHODS: We retrospectively analyzed the data from 29 patients who were diagnosed whit HLH in the Severance Children's Hospital from Jan. 1996 to Feb. 2007. RESULTS: The median age at diagnosis was 3.8 years (range 0.1-12.2). The ratio of male to female patients was 1.1:1. The 5-year overall survival rate was 55.2% with a median follow-up duration of 32 months. In a multivariate analysis, the duration of fever before admission (survival vs. non-survival, 6.5 days vs. 14 days, P=0.010), the interval from the day of fever onset to the day of initiation of etoposide (survival vs. non-survival, 10 days vs. 35 days, P=0.002) and the presence of neurologic symptoms (survival vs. non-survival, 1 case vs. 7 cases, P=0.010) were independent, poor prognostic factors of HLH. EBV infection, gender, and the level of serum ferritin had no relations to the poor prognosis of the disease. CONCLUSION: This study showed that the presence of neurologic symptoms and a longer duration of fever were related to a poor prognosis. Therefore, if a patient develops neurologic symptoms and the duration of fever is prolonged, a prompt diagnostic approach and aggressive treatment for HLH are necessary.
Bone Marrow ; Epstein-Barr Virus Infections ; Etoposide ; Female ; Ferritins ; Fever ; Follow-Up Studies ; Humans ; Lymphohistiocytosis, Hemophagocytic ; Male ; Multivariate Analysis ; Neurologic Manifestations ; Pancytopenia ; Prognosis ; Retrospective Studies ; Splenomegaly ; Survival Rate