Publications by authors named "Hee Jo Baek"

75 Publications

Open-Label, Multicenter Phase II Study of Combination Therapy of Imatinib Mesylate and Mycophenolate Mofetil in Pediatric Patients with Steroid-Refractory Sclerotic/Fibrotic Type Chronic Graft-versus-Host Disease.

Transplant Cell Ther 2021 Jul 24. Epub 2021 Jul 24.

Department of Pediatrics, Seoul National University College of Medicine, Seoul National University Cancer Research Institute, Seoul, Republic of Korea; Wide River Institute of Immunology, Seoul National University, Gangwon, Republic of Korea. Electronic address:

Steroid-refractory chronic graft-versus-host disease (cGVHD) is associated with high morbidity. To date, there is no standard therapy for patients who fail to respond to steroids. In this nonrandomized, open-label, single-arm, multicenter prospective phase II study, we evaluated the efficacy and safety of imatinib mesylate and mycophenolate mofetil (MMF) to treat sclerotic/fibrotic type cGVHD. The primary endpoint was the overall response rate (ORR) to imatinib mesylate plus MMF in 1 year, and the secondary endpoints included safety, quality of life, discontinuation of steroids, and overall survival (OS) rate. A total of 13 patients were enrolled, with a median age of 10.4 years (range, 5.0 to 20.1 years). All patients received a myeloablative conditioning regimen. Specifically, 6 of these patients had previously experienced acute GVHD. The most frequently affected organs were the eyes, lungs, skin, and liver. There were 2 premature deaths. One patient died of pulmonary infection and progression of cGVHD, and the other patient died from neuroblastoma progression and septic shock. The ORR was 76.9% (10 of 13 patients), and the median steroid dose was decreased from 1.0 mg/kg/day to 0.21 mg/kg/day. One-year OS was 84.6% (n = 13), and common adverse events included elevated liver enzyme and serum creatinine levels and fever. Although our sample size was limited, treatment of cGVHD with imatinib mesylate plus MMF shows promising results with acceptable toxicity.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.jtct.2021.07.019DOI Listing
July 2021

Patients' and parents' satisfaction with, and preference for, haemophilia A treatments: a cross-sectional, multicentre, observational study.

Haemophilia 2021 Jul 15;27(4):563-573. Epub 2021 Jun 15.

Korea Hemophilia Foundation Clinic (KHF Clinic, Seoul, South Korea.

Introduction: Reports on patients' satisfaction and preferred characteristics for treatments would be worthwhile when choosing an optimal treatment reflecting patients' perspectives.

Aim: To identify the characteristics and treatment patterns of patients with haemophilia A, or their caregivers, in Korea and explore patient preferences and satisfaction with their treatment.

Methods: This cross-sectional, multicentre, observational study was conducted from April 2018 to September 2019 at six nationwide hospitals and three Korea Hemophilia Foundation clinics. Patients aged ≥16 years, or legal caregivers of paediatric patients, who had used factor VIII (FVIII) concentrates for ≥1 month were enrolled. Satisfaction with treatment was measured using the Treatment Satisfaction Questionnaire for Medication (TSQM); preference was evaluated using discrete choice experiment (DCE), with 10 series of two hypothetical treatment options created from D-efficient block design, which varied across five attributes.

Results: Overall, 505 patients (mean age 31 years) were enrolled in the study. Patients had received FVIII concentrate for an average of 102.9 months (prophylaxis: 53.5%; on-demand: 22.2%). Mean TSQM scores were 64.6 (effectiveness domain), 97.9 (side effects), 57.1 (convenience) and 66.8 (global satisfaction). The number of vials per injection, and the frequency of drug administration, was significantly associated with treatment satisfaction. According to DCE, simpler treatment options were preferred by patients/caregivers.

Conclusion: The lowest satisfaction levels were shown in the treatment convenience domain. Patients/parents preferred simpler and easier treatment characteristics. In an attempt to enhance the overall satisfaction of patients and caregivers with treatment, consideration of more convenient characteristics is required in future decisions regarding treatment selection.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1111/hae.14304DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8362144PMC
July 2021

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

Cancer Res Treat 2021 Apr 20. Epub 2021 Apr 20.

Department of Pediatrics, Kosin University of Medicine, Busan, Korea.

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(AML) children, 79 (9.9%) were diagnosed with APL. The median age at diagnosis was 10.6 years (range, 1.3-18.0). Male and female ratio was 1:0.93. Thirty patients (38.0%) had WBC count greater than 10x109/L at diagnosis. All patients received induction therapy consisting of all-trans retinoic acid (ATRA) and chemotherapy. Five patients (6.6%) died during induction chemotherapy and 66 (86.8%) patients achieved complete remission (CR) after induction chemotherapy. The causes of death were 3 intracranial hemorrhage (ICH), 1 cerebral infarction, and 1 sepsis. Five patients (7.1%) suffered a relapse during or after maintenance chemotherapy. The estimated 4-year event-free survival rate (EFS) and overall survival rates (OS) were 82.1 ± 4.4%, 89.7 ± 5.1% respectively. The 4-yr OS was significantly higher in patients with initial WBC <10x109/L than in those with initial WBC ≥10x109/L (p=0.02).

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.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.4143/crt.2021.313DOI Listing
April 2021

Successful Management of Massive Congenital Hepatic Hemangioma and Systemic Hypertension With Sirolimus.

J Pediatr Hematol Oncol 2021 May 5. Epub 2021 May 5.

Department of Pediatrics, Chonnam National University Children's Hospital Department of Pediatrics, Chonnam National University Medical School Department of Radiology, Chonnam National University Hospital, Gwangju Department of Pediatrics Department of Pathology, Chonnam National University Hwasun Hospital, Hwasun, Korea.

Congenital hepatic hemangioma (CHH) is a common benign vascular tumor of the liver, seen in infancy. The clinical manifestations vary from incidental findings to life-threatening complications. The authors present here a case of an infant with massive CHH who developed systemic hypertension because of compression of the right renal artery by the CHH and did not respond to other lines of treatment. After sirolimus therapy, the CHH size decreased and antihypertensive drugs were no longer necessary. In a critical situation, if the embolization and/or steroids do not seem to control the situation, then adding sirolimus may be considered as secondary therapy with good additive effects.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1097/MPH.0000000000002146DOI Listing
May 2021

Clinical Characteristics and Treatment Outcomes in Children, Adolescents, and Young-adults with Hodgkin's Lymphoma: a KPHOG Lymphoma Working-party, Multicenter, Retrospective Study.

J Korean Med Sci 2020 Nov 30;35(46):e393. Epub 2020 Nov 30.

Department of Internal Medicine, Yeungnam University College of Medicine, Daegu, Korea.

Background: Hodgkin's lymphoma (HL) constitutes 10%-20% of all malignant lymphomas and has a high cure rate (5-year survival, around 90%). Recently, interest has increased concerning preventing secondary complications (secondary cancer, endocrine disorders) in long-term survivors. We aimed to study the epidemiologic features and therapeutic outcomes of HL in children, adolescents, and young adults in Korea.

Methods: We performed a multicenter, retrospective study of 224 patients aged < 25 years diagnosed with HL at 22 participating institutes in Korea from January 2007 to August 2016.

Results: A higher percentage of males was diagnosed at a younger age. Nodular sclerosis histopathological HL subtype was most common, followed by mixed cellularity subtype. Eighty-one (36.2%), 101 (45.1%), and 42 (18.8%) patients were classified into low, intermediate, and high-risk groups, respectively. Doxorubicin, bleomycin, vinblastine, dacarbazine was the most common protocol (n = 102, 45.5%). Event-free survival rate was 86.0% ± 2.4%, while five-year overall survival (OS) rate was 96.1% ± 1.4%: 98.7% ± 1.3%, 97.7% ± 1.6%, and 86.5% ± 5.6% in the low, intermediate, and high-risk groups, respectively ( = 0.021). Five-year OS was worse in patients with B-symptoms, stage IV disease, high-risk, splenic involvement, extra-nodal lymphoma, and elevated lactate dehydrogenase level. In multivariate analysis, B-symptoms and extra-nodal involvement were prognostic factors for poor OS. Late complications of endocrine disorders and secondary malignancy were observed in 17 and 6 patients, respectively.

Conclusion: This is the first study on the epidemiology and treatment outcomes of HL in children, adolescents, and young adults in Korea. Future prospective studies are indicated to develop therapies that minimize treatment toxicity while maximizing cure rates in children, adolescents, and young adults with HL.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.3346/jkms.2020.35.e393DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7707923PMC
November 2020

The First Korean Family with Hemoglobin-M Milwaukee-2 Leading to Hereditary Methemoglobinemia.

Yonsei Med J 2020 Dec;61(12):1064-1067

Department of Pediatrics, Chonnam National University Hwasun Hospital, Hwasun, Korea.

Hemoglobin M (HbM) is a group of abnormal hemoglobin variants that form methemoglobin, which leads to cyanosis and hemolytic anemia. HbM-Milwaukee-2 is a rare variant caused by the point mutation CAC>TAC on codon 93 of the hemoglobin subunit beta () gene, resulting in the replacement of histidine by tyrosine. We here report the first Korean family with HbM-Milwaukee-2, whose diagnosis was confirmed by gene sequencing. A high index of suspicion for this rare Hb variant is necessary in a patient presenting with cyanosis since childhood, along with methemoglobinemia and a family history of cyanosis.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.3349/ymj.2020.61.12.1064DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7700874PMC
December 2020

MEF2D-BCL9 B-Lymphoblastic Leukemia Blast Morphology Does Not Always Mimic Mature B-Cell Leukemia.

J Pediatr Hematol Oncol 2021 04;43(3):112-113

Department of Pediatrics, Chonnam National University Hwasun Hospital, Hwasun.

MEF2D (myocyte enhancer factor 2D)-rearranged acute lymphoblastic leukemia (ALL) has recently been documented by transcriptome sequencing in B-cell precursor ALL. It is associated with older age of onset (median: 14 y), and characterized by very early relapse and poorer outcomes than other B-cell precursor ALL groups. According to report by Suzuki and colleagues, all 4 cases of MEF2D-BCL9-fusion ALL among 59 children with relapsed or primary refractory ALL had leukemic blasts morphologically mimicking mature B-cell leukemia cells. However, we display morphologically different blast populations in 2 patients with MEF2D-BCL9-rearranged ALL. Mature B-cell leukemia-like morphology would aid the detection of MEF2D-BCL9 fusion, but not all cases might have typical morphology.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1097/MPH.0000000000002009DOI Listing
April 2021

Atypical Teratoid/Rhabdoid Tumor of the Central Nervous System in Children under the Age of 3 Years.

Cancer Res Treat 2021 Apr 28;53(2):378-388. Epub 2020 Oct 28.

Department of Neurosurgery, Seoul National University Bundang Hospital, Seoul National University College of Medicine, Seongnam, Korea.

Purpose: Atypical teratoid/rhabdoid tumor (ATRT) is a highly aggressive malignancy with peak incidence in children aged less than 3 years. Standard treatment for central nervous system ATRT in children under the age of 3 years have not been established yet. The objective of this study was to analyze characteristics and clinical outcomes of ATRT in children aged less than 3 years.

Materials And Methods: A search of medical records from seven centers was performed between January 2005 and December 2016.

Results: Forty-three patients were enrolled. With a median follow-up of 90 months, 27 patients (64.3%) showed at least one episode of disease progression (PD). The first date of PD was at 160 days after diagnosis. The 1- and 3-year progression-free survivals (PFS) were 51.2% and 28.5%, respectively. The 1- and 3-year overall survivals were 61.9% and 38.1%, respectively. The 3-year PFS was improved from 0% in pre-2011 to 47.4% in post-2011. Excluding one patient who did not receive any further therapy after surgery, 27 patients died due to PD (n=21), treatment-related toxicity (n=5), or unknown cause (n=1). In univariate analysis, factors associated with higher 3-year PFS were no metastases, diagnosis after 2011, early adjuvant radiotherapy, and high-dose chemotherapy (HDCT). In multivariate analysis, the use of HDCT and adjuvant radiotherapy remained significant prognostic factors for PFS (both p < 0.01).

Conclusion: Aggressive therapy including early adjuvant radiotherapy and HDCT could be considered to improve outcomes of ATRT in children under the age of 3 years.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.4143/crt.2020.756DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8053862PMC
April 2021

Case Reports of Tectal Plate Gliomas Showing Indolent Course.

Brain Tumor Res Treat 2020 Oct;8(2):109-112

Department of Neurosurgery, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea.

From 2004 to 2020, we studied three pediatric patients (age: 9-13 years, all male) and one adult patient (age: 29 years, female) with tectal plate glioma with obstructing hydrocephalus on MRI. One patient had neurofibromatosis type 1. All patients complained about headaches and vomiting, and one patient had diplopia. Endoscopic third ventriculostomy (ETV) was underwent in all patients and a biopsy was obtained from two patients. Pathologic diagnoses were a pilocytic astrocytoma and a low-grade glioma. After ETV with or without biopsy, neurological symptoms were improved in all patients. Three patients did the clinical and radiological follow-up without adjuvant treatment. One patient underwent gamma knife radiosurgery. In two pediatric patients and the adult patient, there was no clinical and radiological progression after 6.2, 6.9, and 8.0 years, respectively. One pediatric patient whose lesion had focal enhancement had radiologic progression without any neurologic symptoms after 5.1 years. Without adjuvant treatment for this lesion, there was no clinical deterioration neither further radiological progression for 6.2 years after radiological aggravation. Tectal plate gliomas showed indolent clinical courses, even after radiologic tumor progression. After the treatment of obstructing hydrocephalus, clinical and radiologic follow-up can be recommended for indolent tectal plate gliomas.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.14791/btrt.2020.8.e17DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7595851PMC
October 2020

Health-related quality of life of moderate and severe haemophilia patients: Results of the haemophilia-specific quality of life index in Korea.

PLoS One 2020 3;15(9):e0238686. Epub 2020 Sep 3.

Department of Pediatrics, Kyungpook National University Medical School, Kyungpook National University Hospital, Daegu, South Korea.

The assessment of health-related quality of life (HRQoL) as a patient-reported outcome provides information about the patients' general well-being as well as the effects of the disease and its treatment. This study aimed to investigate HRQoL using both generic and haemophilia-specific QoL instruments and to assess the clinical factors associated with HRQoL among haemophilia patients in Korea. In this cross-sectional, multicenter, observational study, moderate-to-severe haemophilia patients aged 8-64 years were recruited between November 2012 and September 2013. The EQ-5D Questionnaire, EQ Visual Analogue Scale, and Haemophilia-Specific QoL (Haemo-QoL) Questionnaire (Haemo-QoL for 8-16 years and Haemo-A-QoL for ≥17 years) were used to assess HRQoL. A total of 605 participants with a mean age of 29.32 ± 12.62 years were enrolled. The mean Haemo-QoL scores revealed significant differences by age group (children vs. adolescent vs. adult, 26.44 ± 11.3 vs. 28.88 ± 11.1 vs. 38.43 ± 17.7, respectively, p < 0.001). "Sports and leisure," "family planning," and "view" in adults and "perceived support," "friends," and "dealing" in children and adolescents were identified as the domains with the greatest HRQoL impairments. HRQoL was significantly impaired in patients with the following clinical factors: hepatitis, haemophilia-induced disability, bleeding experiences within the last 6 months, joint bleedings within the last 6 months, and haemophilic arthropathy. According to the multivariate regression analysis, HRQoL showed a negative association with the presence of haemophilia-induced disability (β = 0.222, p < 0.0001), bleeding experiences within the last 6 months (β = 0.098, p = 0.010), and haemophilic arthropathy (β = 0.212, p < 0.0001). HRQoL decreased in patients with older age and impaired clinical conditions among moderate-to-severe haemophilia patients in Korea. These study findings may provide significant insights into the adequate haemophilia management using patient-reported measurements.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0238686PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7470394PMC
October 2020

Epidemiological Study of Hereditary Hemolytic Anemia in the Korean Pediatric Population during 1997-2016: a Nationwide Retrospective Cohort Study.

J Korean Med Sci 2020 Aug 24;35(33):e279. Epub 2020 Aug 24.

Department of Pediatrics, Korea University Ansan Hospital, Korea University College of Medicine, Ansan, Korea.

Background: Hereditary hemolytic anemia (HHA) is a rare disease characterized by premature red blood cell (RBC) destruction due to intrinsic RBC defects. The RBC Disorder Working Party of the Korean Society of Hematology established and updated the standard operating procedure for making an accurate diagnosis of HHA since 2007. The aim of this study was to investigate a nationwide epidemiology of Korean HHA.

Methods: We collected the data of a newly diagnosed pediatric HHA cohort (2007-2016) and compared this cohort's characteristics with those of a previously surveyed pediatric HHA cohort (1997-2006) in Korea. Each participant's information was retrospectively collected by a questionnaire survey.

Results: A total of 369 children with HHA from 38 hospitals distributed in 16 of 17 districts of Korea were investigated. RBC membranopathies, hemoglobinopathies, RBC enzymopathies, and unknown etiologies accounted for 263 (71.3%), 59 (16.0%), 23 (6.2%), and 24 (6.5%) of the cases, respectively. Compared to the cohort from the previous decade, the proportions of hemoglobinopathies and RBC enzymopathies significantly increased ( < 0.001 and = 0.008, respectively). Twenty-three of the 59 hemoglobinopathy patients had immigrant mothers, mostly from South-East Asia.

Conclusion: In Korea, thalassemia traits have increased over the past 10 years, reflecting both increased awareness of this disease and increased international marriages. The enhanced recognition of RBC enzymopathies is due to advances in diagnostic technique; however, 6.5% of HHA patients still do not have a clear diagnosis. It is necessary to improve accessibility of diagnosing HHA.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.3346/jkms.2020.35.e279DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7445306PMC
August 2020

Dural-Based Primary Central Nervous System Lymphoma with Bone Invasion.

World Neurosurg 2020 07 30;139:415-418. Epub 2020 Apr 30.

Department of Pathology, Chonnam National University Medical School, Chonnam National University Hospital and Chonnam National University Hwasun Hospital, Gwangju, Republic of Korea.

Background: Primary central nervous system lymphoma (PCNSL) occurs frequently in a deep intraparenchymal location. It rarely occurs in the meninges, and bone invasion is uncommon.

Case Description: A 12-year-old boy was admitted to our hospital with a history of headache and seizures. Magnetic resonance imaging showed a 4-cm-sized dural-based mass with osteolytic bone change. Craniotomy and tumor removal were performed. Operative findings showed a dural-based hard tumor, including necrosis and hemorrhage, and the skull was focally destroyed by tumor cells. The tumor was completely removed. Pathologically, large atypical cells with pleomorphic nuclei and frequent mitoses were found. The tumor cells were immune-positive for CD30, epithelial cell antigen, and anaplastic lymphoma kinase. The final diagnosis was anaplastic lymphoma kinase-positive anaplastic large cell lymphoma (ALCL). There was no evidence of systemic cancer. The boy underwent chemotherapy following the Non-Hodgkin Lymphoma-Berlin-Frankfurt-Münster protocol. There was no recurrence after a 16-month chemotherapy-off period.

Conclusions: ALCL is not a common type of PCNSL, and central nervous system ALCL frequently involves the dura and meninges compared with PCNSL in general. However, osteolytic bone lesions are rarely seen in ALCL. This case thus represents a rare case of dural-based ALCL with bone invasion.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.wneu.2020.04.164DOI Listing
July 2020

Benign spindle cell tumor surrounding the bronchovascular structures in a 14-year-old male with neurofibromatosis type I.

Pediatr Pulmonol 2020 06 28;55(6):1305-1307. Epub 2020 Apr 28.

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, South Korea.

View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1002/ppul.24769DOI Listing
June 2020

Health behaviors of caregivers of childhood cancer survivors: a cross-sectional study.

BMC Cancer 2020 Apr 7;20(1):296. Epub 2020 Apr 7.

Department of Family Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, 81 Irwon-Ro, Gangnam-gu, Seoul, 06351, Republic of Korea.

Background: Caregiving for childhood cancer survivors may be burdensome for caregivers and affect their physical health and health behaviors. However, studies examining health behaviors in caregivers of childhood cancer survivors are scarce. This study aimed to examine health behaviors of caregivers of childhood cancer survivors by comparing them with those of the general population, and analyze associated factors.

Methods: This study included 326 caregivers of childhood cancer survivors recruited from 3 major hospitals in South Korea and 1304 controls from the Korean National Health and Nutritional Examination Survey matched for age, sex, and education level. We compared health behaviors between the two groups by using conditional logistic regression analyses, and investigated factors associated with unhealthy behaviors in caregivers by using multiple logistic regression analyses.

Results: Caregivers were less likely to be physically inactive (aOR: 0.69, 95% CI: 0.51, 0.92) compared to controls, and this was more evident in women (aOR: 0.65, 95% CI: 0.45, 0.94). However, caregivers were more likely to be binge drinkers (aOR: 2.26, 95% CI: 1.73, 2.97), especially if they were men (aOR: 13.59, 95% CI: 8.09, 22.82). Factors associated with unhealthy behaviors in caregivers differed by the type of behavior. Current smoking risk was lower in female caregivers and in those with more comorbidities. Increasing age, female sex, higher education level, and lower household income were associated with lower risk of binge drinking. Higher household income and anxiety were associated with lower risk of physical inactivity, while depression was associated with higher risk of physical inactivity.

Conclusions: Caregivers of childhood cancer survivors were more likely to engage in binge drinking, but less likely to be physically inactive. Strategies to promote adherence to desirable health behaviors in caregivers are needed with consideration of their socioeconomic and clinical factors, such as number of comorbidities.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s12885-020-06765-wDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7140330PMC
April 2020

Better Failure-Free Survival and Graft-versus-Host Disease-Free/Failure Free Survival with Fludarabine-Based Conditioning in Stem Cell Transplantation for Aplastic Anemia in Children.

J Korean Med Sci 2020 Feb 24;35(7):e46. Epub 2020 Feb 24.

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea.

Background: This study aimed to assess the outcome of stem cell transplantation (SCT), including overall survival (OS), failure-free survival (FFS) and graft-versus-host disease (GvHD)-free/failure-free survival (GFFS), and to analyze prognostic factors in children with aplastic anemia (AA).

Methods: From 1991 to 2018, 43 allogeneic SCT recipients were enrolled in the study to investigate the demographic characteristics, survival outcomes and prognostic factors.

Results: With the median follow-up of 7.1 years, the estimated 10-year OS, FFS, GFFS were 86.0%, 60.5%, and 51.2%, respectively. Matched related donors (MRD, n = 28) showed better 10-year OS than unrelated donors (n = 15) (96.4% vs. 66.7%; = 0.006). Engraftment failure was seen in 13 patients (30.2%). Donor-type aplasia was seen in 13.8% (4/29) after fludarabine (Flu)-based conditioning (Flu-group), while in 42.6% (6/14) after cyclophosphamide (Cy)-based regimen (Cy-group) ( = 0.035). Six patients died. The 10-year OS in Cy-group was 92.9% (n = 14, all MRD), while that of Flu-group was 82.1% (n = 29; = 0.367). But Flu-group tended to have better FFS and GFFS than Cy-group, although Flu-group had less MRDs (41.4% vs. 100%; = 0.019), and higher proportion of previous immunosuppressive treatment (IST; 62% vs. 21.4%, = 0.012). In MRD transplants, OS was similar between Flu-group (100%, n = 14) and Cy-group (92.9%, n = 14), while FFS (100.0% vs. 42.9%; = 0.001) and GFFS (85.7% vs. 35.7%; = 0.006) were significantly better in Flu-group. Stem cell sources, irradiation in the conditioning, and method of GvHD prophylaxis did not significantly influence the outcome.

Conclusion: This study reviewed SCT outcomes for pediatric AA with changes of transplant strategies over the last 25 years. The FFS and GFFS were higher in Flu-group than in Cy-group, especially in matched related transplantation. Graft failure including donor-type aplasia remains troublesome even with Flu-based conditioning. Further refinement of transplant strategies to ensure better quality-of-life should be pursued.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.3346/jkms.2020.35.e46DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7036341PMC
February 2020

Correction to: influenza vaccination in caregivers of childhood cancer survivors.

J Cancer Surviv 2020 Feb;14(1):100

Department of Family Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.

The original version of this article unfortunately contained a mistake in affiliation of the 1st, 4th, and corresponding author.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11764-019-00848-9DOI Listing
February 2020

Subgroup analysis of a phase 2/3 study of rurioctocog alfa pegol in patients with severe hemophilia A: efficacy and safety in previously treated Korean patients.

Blood Res 2019 Sep 25;54(3):198-203. Epub 2019 Sep 25.

Baxalta US Inc., a Takeda Company, Cambridge, MA, USA.

Background: The efficacy and safety of extended half-life, full-length, pegylated recombinant factor VIII rurioctocog alfa pegol [BAX 855, ADYNOVATE (USA)/ADYNOVI (Europe); Baxalta US Inc., a Takeda company, Lexington, MA, USA] was investigated in previously treated Korean patients with severe hemophilia A (HA).

Methods: A post hoc data analysis from the international, multicenter, phase 2/3 PROLONG-ATE study of rurioctocog alfa pegol in patients with severe HA (NCT01736475) determined annualized bleeding rates (ABRs) and rates of adverse events (AEs) in Korean patients treated in this study.

Results: All 10 enrolled Korean patients receiving rurioctocog alfa pegol (9 prophylaxis, 1 on-demand) completed the study [median (range) age, 28.0 (12-50) yr; weight, 64.8 (45-90) kg; 8 patients had ≥1 target joint at screening]. Median (range) ABR was 1.9 (0.0-14.5) for patients on prophylaxis and 62.2 for the patient receiving on-demand treatment. The hemostatic efficacy of rurioctocog alfa pegol was rated "excellent" or "good" and only single infusions were required per bleeding episode. ABRs improved in most patients compared with prestudy values. No dose adjustments were required for prophylaxis, and the dosing frequency was reduced in 8 patients, compared with their previous prophylaxis regimen. No serious AEs were reported; all 9 nonserious AEs (in 3 patients) were mild in severity and unrelated to the study treatment.

Conclusion: This post hoc analysis of a small group of Korean patients with severe HA indicated that rurioctocog alfa pegol was effective, and no serious AEs were observed. For most patients, the dosing frequency was also reduced compared with their previous regimen.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.5045/br.2019.54.3.198DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6779944PMC
September 2019

Influenza vaccination in caregivers of childhood cancer survivors.

J Cancer Surviv 2019 Dec 4;13(6):993-1001. Epub 2019 Nov 4.

Department of Family Medicine, Healthcare System Gangnam Center, Seoul National University Hospital, Seoul, Republic of Korea.

Purpose: An increased risk of influenza-related morbidity and mortality in childhood cancer survivors could be significantly reduced through vaccinations. This study aimed to examine influenza vaccination practices by caregivers of childhood cancer survivors.

Methods: Study subjects were 364 caregivers, recruited at three major hospitals in South Korea. We collected demographic and medical information through self-administered questionnaires, along with clinical information of childhood cancer survivors by reviewing medical records. Multiple logistic regression analysis was performed to analyze factors associated with influenza vaccination of caregivers.

Results: A total of 55.2% of caregivers had received influenza vaccination at least once and 24.2% had received yearly influenza vaccinations during the last 3 years. Caregivers with highest education level were more likely to receive influenza vaccination than caregivers with lowest education level: aOR (95% CI) was 3.42 (1.17, 9.99) for ever-receipt of vaccination and 3.33 (1.15, 9.58) for yearly vaccination. One-year increase in caregiver age was inversely associated with ever-receipt of vaccination (aOR (95% CI), 0.94 (0.90, 0.98)) as well as yearly vaccination (aOR (95% CI), 0.92 (0.88, 0.97)). Increasing time lapse since the end of treatment was also associated with lower vaccinations.

Conclusion: Influenza vaccination rates of childhood cancer survivor caregivers were suboptimal. Time lapse after completing cancer treatment, caregiver age, and educational level were independently associated with receipt of influenza vaccination by caregivers.

Implications For Cancer Survivors: To promote influenza vaccination in caregivers of childhood cancer survivors to prevent influenza-related morbidity and mortality in childhood cancer survivors is necessary.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s11764-019-00825-2DOI Listing
December 2019

Molecular diagnosis of hereditary spherocytosis by multi-gene target sequencing in Korea: matching with osmotic fragility test and presence of spherocyte.

Orphanet J Rare Dis 2019 05 23;14(1):114. Epub 2019 May 23.

Department of Pediatrics, Kyungpook National University School of Medicine, Daegu, Republic of Korea.

Background: Current diagnostic tests for hereditary spherocytosis (HS) focus on the detection of hemolysis or indirectly assessing defects of membrane protein, whereas direct methods to detect protein defects are complicated and difficult to implement. In the present study, we investigated the patterns of genetic variation associated with HS among patients clinically diagnosed with HS.

Methods: Multi-gene targeted sequencing of 43 genes (17 RBC membrane protein-encoding genes, 20 RBC enzyme-encoding genes, and six additional genes for the differential diagnosis) was performed using the Illumina HiSeq platform.

Results: Among 59 patients with HS, 50 (84.7%) had one or more significant variants in a RBC membrane protein-encoding genes. A total of 54 significant variants including 46 novel mutations were detected in six RBC membrane protein-encoding genes, with the highest number of variants found in SPTB (n = 28), and followed by ANK1 (n = 19), SLC4A1 (n = 3), SPTA1 (n = 2), EPB41 (n = 1), and EPB42 (n = 1). Concurrent mutations of genes encoding RBC enzymes (ALDOB, GAPDH, and GSR) were detected in three patients. UGT1A1 mutations were present in 24 patients (40.7%). Positive rate of osmotic fragility test was 86.8% among patients harboring HS-related gene mutations.

Conclusions: This constitutes the first large-scaled genetic study of Korean patients with HS. We demonstrated that multi-gene target sequencing is sensitive and feasible that can be used as a powerful tool for diagnosing HS. Considering the discrepancies of clinical and molecular diagnoses of HS, our findings suggest that molecular genetic analysis is required for accurate diagnosis of HS.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s13023-019-1070-0DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6533652PMC
May 2019

Biphenotypic acute leukemia or acute leukemia of ambiguous lineage in childhood: clinical characteristics and outcome.

Blood Res 2019 Mar 21;54(1):63-73. Epub 2019 Mar 21.

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea.

Background: Acute leukemia (AL), not clearly assigned to myeloid, B-lymphoid, or T-lymphoid lineage, is classified as either biphenotypic acute leukemia (BAL) based on the European Group for Immunological Classification of Leukemias (EGIL) or acute leukemia of ambiguous lineage (ALAL) encompassing acute undifferentiated leukemia (AUL) and mixed-phenotype acute leukemia (MPAL) based on the World Health Organization (WHO) criteria.

Methods: Medical records of children newly diagnosed with BAL or ALAL, based on the EGIL or the 2008/2016 WHO criteria, respectively, admitted at Chonnam National University Hospital in 2001-2017 were retrospectively reviewed.

Results: Twelve (3.2%) of 377 AL patients satisfied the BAL or ALAL definitions based on the EGIL or the WHO criteria, respectively. Among 12 patients including 11 with BAL and another with undefined case based on the EGIL criteria, 7 (1.9%) had ALAL based on more stringent 2016 WHO criteria (AUL, 2; MPAL, 5). One patient had MPAL with t(9;22)(q34;q11.2), +, and two had gene abnormality. ALL-directed regimen was associated with better complete remission rate compared with AML-directed regimen (100.0% vs. 16.7%; =0.015). The 5-year overall survival (OS) and event-free survival (EFS) were 51.1±15.8% and 51.9±15.7%, respectively. AUL was associated with poor OS and EFS compared with MPAL (0.0% vs. 75.0±21.7%; =0.008).

Conclusion: Due to the rarity of the cases, future multicenter, prospective studies incorporating large number of cases are urgently warranted to identify the clinical, biologic, and molecular markers for the prediction of prognosis and determine the best tailored therapy for each patient.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.5045/br.2019.54.1.63DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6439300PMC
March 2019

External Jugular Vein Aneurysm in a Child With Hemophilia A.

J Craniofac Surg 2019 Jun;30(4):e377-e378

Department of Otolaryngology-Head and Neck Surgery.

An external jugular vein (EJV) aneurysm is an extremely rare disease. Ultrasonography with color doppler methods is considered the criterion standard for diagnosis of a venous aneurysm. The treatment of choice for an EJV aneurysm is surgical excision. Herein, we report an unusual case of an EJV aneurysm in a child with hemophilia A. Because the incidence of an EJV aneurysm is low, it is important to distinguish it from other neck lesions, including congenital disease. In addition, it should be noted that in hemophilia patients, an EJV aneurysm may occur in a spontaneous or small trauma.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1097/SCS.0000000000005434DOI Listing
June 2019

Risk Factor Analysis for Secondary Malignancy in Dexrazoxane-Treated Pediatric Cancer Patients.

Cancer Res Treat 2019 Jan 14;51(1):357-367. Epub 2018 May 14.

Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.

Purpose: Dexrazoxane has been used as an effective cardioprotector against anthracycline cardiotoxicity. This study intended to analyze cardioprotective efficacy and secondary malignancy development, and elucidate risk factors for secondary malignancies in dexrazoxane-treated pediatric patients.

Materials And Methods: Data was collected from 15 hospitals in Korea. Patients who received any anthracyclines, and completed treatment without stem cell transplantation were included. For efficacy evaluation, the incidence of cardiac events and cardiac event-free survival rates were compared. Data about risk factors of secondary malignancies were collected.

Results: Data of total 1,453 cases were analyzed; dexrazoxane with every anthracyclines group (D group, 1,035 patients) and no dexrazoxane group (non-D group, 418 patients). Incidence of the reported cardiac events was not statistically different between two groups; however, the cardiac event-free survival rate of patients with more than 400 mg/m2 of anthracyclines was significantly higher in D group (91.2% vs. 80.1%, p=0.04). The 6-year cumulative incidence of secondary malignancy was not different between both groups after considering follow-up duration difference (non-D, 0.52%±0.37%; D, 0.60%±0.28%; p=0.55). The most influential risk factor for secondary malignancy was the duration of anthracycline administration according to multivariate analysis.

Conclusion: Dexrazoxane had an efficacy in lowering cardiac event-free survival rates in patients with higher cumulative anthracyclines. As a result of multivariate analysis for assessing risk factors of secondary malignancy, the occurrence of secondary malignancy was not related to dexrazoxane administration.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.4143/crt.2017.457DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6333985PMC
January 2019

Treatment Decisions of World Health Organization Grade II and III Ependymomas in Molecular Era.

J Korean Neurosurg Soc 2018 May 1;61(3):312-318. Epub 2018 May 1.

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea.

Surgery and radiotherapy are mainstays of treatment for ependymomas (EPNs). Recent molecular subgrouping could be superior to histopathological grading for predicting the prognosis of patients with EPNs. Gross total resection is an effective treatment approach regardless of its locations or pathologic grades. Adjuvant therapeutic strategies could be decided based on molecular subgrouping with risk-stratification. Information of histologic-molecular biology is now providing clues to therapeutic insights.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.3340/jkns.2018.0003DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5957310PMC
May 2018

Improvement of treatment outcome over 2 decades in children with acute myeloid leukemia.

Blood Res 2018 Mar 27;53(1):25-34. Epub 2018 Mar 27.

Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea.

Background: The prognosis of pediatric acute myeloid leukemia (AML) has recently improved. This study aimed to describe the epidemiology, changes in treatment strategies, and improvement of outcomes in Gwangju-Chonnam children with AML over 2 decades.

Methods: Medical records of 116 children with newly diagnosed AML were retrospectively reviewed for demographic characteristics, prognostic groups including cytogenetic risks, treatment protocols, and survival rates over the periods between 1996 and 2005 (Period I, N=53), and 2006 and 2015 (Period II, N=38).

Results: The annual incidence of AML has decreased with reduced pediatric population. The 5-year Kaplan-Meier (K-M) estimated overall survival (OS) and event-free survival (EFS) rates in 110 AML patients were 53.2±5.1% and 43.8±5.1%, respectively. The 5-year OS rate significantly improved during period II (70.3±7.0%) as compared to that during period I (40.0±6.8%) ( =0.001). The 5-year OS was not significantly different among cytogenetic risk groups ( =0.11). Fifty-eight patients underwent hematopoietic stem cell transplantation (HSCT). The K-M 5-year estimated survival for transplanted patients was 53.7±7.0%, while that for chemotherapy-only patients was 30.1±9.1% ( =0.014). Among the prognostic factors, treatment modality was the only independent factor. The chemotherapy-only group had a relative risk of 2.06 for death compared with the transplantation group (=0.015).

Conclusion: The survival of Korean children with AML has improved to a level comparable with that of developed countries over 2 decades, owing to a change in induction strategy, better supportive care with economic growth, refinement of HSCT techniques including a better selection of patients based on prognostic groups, and stem cell donor selection.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.5045/br.2018.53.1.25DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5898990PMC
March 2018

Endoscopic transpterygoid approach to a mass in a child.

Int J Pediatr Otorhinolaryngol 2018 Feb 18;105:115-117. Epub 2017 Dec 18.

Department of Otolaryngology-Head and Neck Surgery, Chonnam National University Medical School & Hwasun Hospital, Hwasun, South Korea. Electronic address:

The endoscopic transterygoid approach to the petrous apex is a feasible/alternative approach in carefully selected patients with specific favorable anatomy, even children. This approach, unlike traditional approaches, spares cochlear and vestibular function. We report a case of a six-year-old boy with embryonal rhabdomyosarcoma of the petrous apex that was diagnosed via the endoscopic transpterygoid approach.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ijporl.2017.12.020DOI Listing
February 2018

Disseminated juvenile xanthogranuloma occurring after treatment of Langerhans cell histiocytosis: a case report.

Childs Nerv Syst 2018 04 5;34(4):765-770. Epub 2017 Dec 5.

Department of Dermatology, Chonnam National University Medical School, Chonnam National University Hwasun Hospital, Jeollanam-do, Republic of Korea.

Case Presentation: An 11-year-old boy presented with a complaint of a painful temporal mass. Brain magnetic resonance imaging (MRI) showed a 3-cm-sized, homogeneously enhancing mass in the greater wing of the left sphenoid bone, which was diagnosed as Langerhans cell histiocytosis (LCH). Chemotherapy with vincristine and prednisolone was performed for 1 year. After 1 year and 11 months off treatment, he developed symptoms such as polydipsia and polyuria. Brain MRI showed thickening of the pituitary stalk with enhancement, suggestive of LCH involvement, and no recurrence in the sphenoid bone. After 4 years and 4 months off treatment, he developed multiple, subcutaneous, asymptomatic, and yellowish variable-sized papules on his face, posterior neck, and back, which were pathologically diagnosed as juvenile xanthogranuloma (JXG). Brain MRI revealed multifocal enhancing skull lesions in the left parietal, right frontal, and left occipital bones, which were also diagnosed as JXG. After 5 years and 8 months off treatment, the number of variable-sized skin lesions was increased without changes in the lesions in the skull and pituitary stalk.

Conclusion: We report a case of disseminated JXG occurring after treatment of LCH. These clinical co-presentations suggested a close relationship between their pathogenesis.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s00381-017-3675-8DOI Listing
April 2018

Awareness about past diagnosis and treatment history: nationwide survey of childhood cancer survivors and their parents.

Jpn J Clin Oncol 2017 Oct;47(10):962-968

Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea.

Objective: To assess the awareness of past medical history and long-term care issues of childhood cancer survivors (CCS) in Korea.

Methods: A nationwide survey was conducted on CCS and their parents in 10 regional cancer centers in Korea. Answers regarding cancer diagnosis and treatment history were compared with the treatment summary and categorized into three ('specific,' 'general,' and 'no') or two ('yes' and 'no') groups.

Results: Out of 343 contacts, 293 dyads completed the survey, and 281 dyads were analyzed. Awareness of cancer diagnosis was mostly specific for parents (76.5%) and CCS (35.2%). Awareness of anti-cancer treatment exposure was mostly general (84.6% for surgery, 67.9% for chemotherapy, and 53.9% for hematopoietic stem cell transplantation) rather than specific. In particular, more than half of the parents were not aware of the exposure to cardiotoxic agents (72.9%) or radiation therapy (56.3%). Providing information about long-term side effects and prevention of secondary cancer was significantly correlated only with more concern and more follow-up visits (P ≤ 0.001, respectively), without correlation with more specific awareness of exposure to cardiotoxic agents or radiation.

Conclusion(s): Most of the parents of CCS were not aware of treatment-related risk factors necessary for long-term care. Providing information was significantly correlated with more concern and more follow-up visits, without improving corresponding knowledge about their past medical history. Effort aimed towards improving awareness about risk factors, the manner of providing information, and the patient referral system within which we use this information is warranted.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1093/jjco/hyx102DOI Listing
October 2017

Characteristics and Outcomes of Second Malignant Neoplasms after Childhood Cancer Treatment: Multi-Center Retrospective Survey.

J Korean Med Sci 2016 Aug 18;31(8):1254-61. Epub 2016 May 18.

Division of Pediatric Hematology/Oncology, Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea .

This retrospective study investigated the clinical characteristics and outcomes of second malignant neoplasms (SMNs) in survivors of childhood cancer from multiple institutions in Korea. A total of 102 patients from 11 institutions who developed SMN after childhood cancer treatment between 1998 and 2011 were retrospectively enrolled. The most common primary malignant neoplasms (PMNs) were central nervous system (CNS) tumors (n = 17), followed by acute lymphoblastic leukemia (n = 16), non-Hodgkin lymphoma (n = 13), and osteosarcoma (n = 12). The most common SMNs were therapy-related myeloid neoplasms (t-MNs; acute myeloid leukemia [AML], 29 cases; myelodysplastic syndrome [MDS], 12 cases), followed by thyroid carcinomas (n = 15) and CNS tumors (n = 10). The median latency period was 4.9 years (range, 0.5-18.5 years). Among 45 patients with solid tumors defined as an SMN, 15 (33%) developed the lesion in a field previously subjected to radiation. The 5-year overall survival (OS) rate of patients with an SMN was 45% with a median follow-up time of 8.6 years. Patients with AML, MDS, and CNS tumors exhibited the poorest outcomes with 5-year OS rates of 18%, 33%, and 32%, respectively, whereas those with second osteosarcoma showed comparable outcomes (64%) to patients with primary counterpart and those with second thyroid carcinoma had a 100% OS rate. Further therapeutic efforts are recommended to improve the survival outcomes in patients with SMNs, especially in cases with t-MNs and CNS tumors.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.3346/jkms.2016.31.8.1254DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4951555PMC
August 2016
-->