Myeloablative Hematopoietic Stem Cell Transplantation with a Non-total Body Irradiation Regimen for Treating Pediatric Acute Lymphoblastic Leukemia

임영태,이규호,김세윤,박선영,하정옥,이재민 대한소아혈액종양학회 2017 Clinical Pediatric Hematology-Oncology Vol.24 No.1

Background: Total body irradiation (TBI) has been traditionally used as a conditioning regimen prior to hematopoietic stem cell transplantation (HSCT) in patients with pediatric leukemia. However, TBI can cause late sequelae such as growth impairment, cataract, hormone abnormalities, infertility, neurocognitive effects, and secondary malignancy in pediatric patients. Methods: This single center retrospective study included 22 patients with acute lymphoblastic leukemia who were aged <18 years and underwent HSCT between May 1999 and December 2014; seven patients received a TBI-based regimen and 15 received a non-TBI regimen. Results: The overall survival and event-free survival rates in the TBI group were not significantly different from those in the non-TBI group (overall survival rate 71% vs. 73%, respectively; P=0.906; event-free survival rate 71% vs. 73%, respectively P=0.923). Conclusion: Our results indicate that non-TBI conditioning regimens can be an alternative treatment option of the treatment of pediatric acute lymphoblastic leukemia undergoing HSCT.

A Case of Hodgkin Lymphoma Developed in an Ataxia-telangiectasia Patient

임주연,전민아,김효선,한정우,유철주,한승민 대한소아혈액종양학회 2016 Clinical Pediatric Hematology-Oncology Vol.23 No.2

Ataxia-telangiectasia (AT) is characterized by cerebellar ataxia, progressive immunodeficiency, radiation sensitivity, telangiectasia, and predisposition to malignancy. AT patients have a 100-fold increased risk for the development of lymphoid malignancies. It is important to consider AT in a child with pre-existing ataxia, or lymphoid malignancy that was diagnosed at a younger age than expected. This consideration avoids the confusion between ataxia development and toxicity from chemotherapy. Hodgkin’s lymphoma (HL) is usually treated with chemotherapy and/or radiotherapy. Unfortunately, when treated with conventional doses of radiotherapy, AT patients invariably experience devastating necrosis of their normal tissues. Therefore, a new treatment protocol for patients with HL in AT must be established. In this paper, we report the case of an 8-year-old female patient with HL in AT who was treated with chemotherapy. This patient was also treated with brentuximab (which targets CD30) for salvage therapy after the disease progressed.

가능성 있는 폰빌레브란트병의 유병률에 대한 연구

이명숙,김순기,김동빈,유기영,김희진,이선호,박상규 대한소아혈액종양학회 2014 Clinical Pediatric Hematology-Oncology Vol.21 No.1

Background: Definition of possible von Willebrand disease (VWD) included levels of von Willebrand factor antigen (VWF:Ag) or von Willebrand factor ristocetin cofactor activity (VWF:RCo) below 30 U/dL. The purpose of this study was to determine the prevalence of possible VWD in Ulsan, Korea. We also analyzed the influence of demographic factors such as age, gender, and blood group on subject levels of VWF. Methods: Between March 2011 and September 2011 we prospectively investigated 1,039 subjects (271 children, 768 adults). Blood samples were collected for the determination of VWF:Ag, VWF:RCo, and factor VIII coagulation assay (FVIII:C). A standardized questionnaire was administered to evaluate hemorrhagic symptoms at the time of first examination, using a bleeding score ranging from 0 to 3. Results: Forty five subjects met the criteria for possible VWD, for a prevalence of 4.3%. Subjects of Group O had a significantly lower mean FVIII:C, VWF:Ag, and VWF:RCo value than subjects of group A,B, or AB (P<0.001). Conclusion: Our results suggest that the prevalence of VWD may be much higher than previously reported. Efforts to increase the awareness and diagnosis of VWD may help improve identification of patients with bleeding disorders and lead to early, appropriate management with safe and efficacious therapies.

폐쇄성 황달을 주증상으로 한 신생아백혈병 1예

김상범,이장훈,박준은 대한소아혈액종양학회 2014 Clinical Pediatric Hematology-Oncology Vol.21 No.1

Neonatal leukemia, which develops in first 4 weeks of life, is a rare group of hematologic neoplasm with various symptoms and prognosis. In rare cases, obstructive jaundice can be shown as initial presentation. Therefore, it is often misdiagnosed as cholestatic liver diseases. We report a case of neonatal leukemia with obstructive jaundice which was misdiagnosed as biliary atresia. This case was diagnosed with bone marrow aspirates at the age of 14th day, and the result was given that about 93% of the cells were lymphoblasts.

무쇠솥에서 유래한 철분함량 및 Heme Oxygenase-1 활성도에 관한 In Vitro 연구

김근영,안영민,김태완,전용훈,이지은,장준혁,김순기 대한소아혈액종양학회 2016 Clinical Pediatric Hematology-Oncology Vol.23 No.2

Background: Iron pots have long been used for cooking in several countries. Early studies have shown that the use of such iron pots can increase the iron content of food cooked in them and that this increased iron content has some effect on iron uptake. This study was designed to evaluate the iron content in rice cooked in a traditional iron pot and study the iron uptake by macrophages through heme oxygenase-1 (HO-1). Methods: The iron pot used in this study was round-shaped and had no legs. The iron content of rice cooked in the iron pot was measured. Thereafter, the bioavailability of iron was measured using western blot analysis. Results: A total of 35 samples were analyzed for iron concentrations, which were 10.94± 18.08 mg/L (range: 0.18-56.53 mg/L). The biochemical activity in most of materials was 1.5-9 times that of the activity observed in the control group. Conclusion: The iron concentration of rice cooked in iron pots were found to be relatively high. The introduction of iron pots in routine cooking practices may be a promising way of increasing the supply of iron, especially for people with severe iron deficiency anemia. Further, increased activity of HO-1, induced by supplementation of iron from the cast iron, may help in maintaining iron homeostasis.

T-세포형 급성림프구성백혈병에서 NOTCH1의 변형과 표적치료제 개발

노정희,전인상 대한소아혈액종양학회 2014 Clinical Pediatric Hematology-Oncology Vol.21 No.1

T-cell acute lymphoblastic leukemia (T-ALL) accounts for approximately 10-15% of entire ALL in children. The outcome of T-ALL has been improved through the intensified therapeutic strategy, however, it is still a more aggressive disease. In T-ALL a couple of transcription factor oncogenes are known to be relocated to the juxtaposition of T-cell receptor genes, potent promoter, by chromosome translocation. However the incidence of each chimeric gene formation in T-ALL is less than 5% and their clinical significance as a prognostic marker is lacking. A decade ago it was identified that activating mutations in NOTCH1 in about 60% of T-ALL. After then, activating NOTCH1 mutations present in T-ALL have been extensively investigated with regard to understanding its molecular pathogenesis, its prognostic significance, and developing molecularly tailored novel agents. Small molecule g-secretase inhibitor, blocking a proteolytic step required for creation of a fragment of NOTCH intracellular domain which actually act as a controller of its target gene expression, was tried as a target therapeutic drug for T-ALL. Although outcome of this drug was not satisfactory, challenges have been launched to develop new drugs which specifically act on the aberrant behavior of mutated NOTCH1 in T-ALL.

철 대사의 이해

서진경,전인상 대한소아혈액종양학회 2018 Clinical Pediatric Hematology-Oncology Vol.25 No.1

Iron is critical for almost all living organisms because it serves as a cofactor for many proteins and enzymes necessary for oxygen and energy metabolism. Disruption of iron homeostasis is associated with a wide range of diseases. Thus mammals have developed sophisticated mechanisms to maintain optimal range of iron concentration. Iron regulation involves processes at the systemic and cellular levels. These processes are regulated by hepcidin and iron regulatory proteins. Hepcidin modulates systemic iron homeostasis with ability to impede cellular iron export via interaction with the iron export protein, ferroportin. Whereas, iron regulatory proteins control cellular iron homeostasis by translational regulation of proteins which involve iron metabolism. Recent advances in the study of iron metabolism have shown promising results that hepcidin-targeted strategies may help to improve the diagnosis and treatment of iron related diseases. Although these strategies are now under development, ongoing studies can help to elucidate its application possibilities.

What Should We Consider Carefully When Performing Survival Analysis?

곽상규,최은진 대한소아혈액종양학회 2019 Clinical Pediatric Hematology-Oncology Vol.26 No.1

The survival data and the survival analysis are the data and analysis methods used to study the probability of survival. The survival data consist of a period from the juncture of a start event to the juncture of the end event (occurrence event). The period is called the survival period or survival time. In this way, the method of analysing the survival time of subjects and appropriately summarizing the degree of survival is called survival analysis. To understand and analyse survival analysis methods, researchers must be aware of some concepts. Concepts to be aware of in the survival analysis include events, censored data, survival period, survival function, survival curve and so on. This review focuses on the terms and concepts used in the survival analysis. It will also cover the types of survival data that should be collected and prepared when using actual survival analysis method and how to prepare them.

Successful Treatment of Severe Hemolytic Disease of the Newborn Caused by Anti-Jkb

Goo Lyeon Kim,Yoonsoo Kim,Young Pyo Chang,서주희,Mee Jeong Lee 대한소아혈액종양학회 2021 Clinical Pediatric Hematology-Oncology Vol.28 No.1

Hemolytic disease of the newborn (HDN) is a condition in which maternal antibodies cross the placenta and cause hemolytic reactions. Anti-RhD was the most common cause, but with the introduction of immunoglobulin, the frequency has decreased sig nificantly, making hemolytic d isease c aused by o ther minor b lood g roups more important. Kidd antigen is also known to cause hemolytic transfusion reactions. Only 13 cases have b een reported so far, b ecause K idd antig en dose not u sually c ause HDN. Most cases have a good outcome, and only two fatal cases have been reported. A four-day-old male patient was hospitalized for jaundice, and hemolysis was confirmed by blood test. The mother’s blood was Jkb antibody positive. The patient did not improve with phototherapy, so an exchange transfusion was performed. Additional hemolysis occurred, so we undertook transfusion of red blood cells, resulting in cessation of hemolysis. We report HDN caused by Jkb antibody that responded to exchange blood transfusion.

How I Treat Primary Immune Deficiencies with Hematopoietic Stem Cell Transplantation

Hoon Kook,Boram Kim,Hee Jo Baek 대한소아혈액종양학회 2022 Clinical Pediatric Hematology-Oncology Vol.29 No.2

Primary immune deficiencies (PID), or more recently, inborn errors of immunity (IEI), resulting from genetic defects of the immune system may present with increased sus-ceptibility to infections, persistent inflammation, and autoimmunity. With recent in-troduction of next generation sequencing, the number of IEIs increases rapidly, reaching to 484 in 2022. Hematopoietic stem cell transplantation (HSCT) has been used over decades as a mainstay of specific treatment modality, while gene therapy and pharmacologic approach have been attempted with promising results in some PID in recent years. The survival following allogeneic HSCT for PID is now generally >80%. The indication and timing of transplant must be individualized not only on the basis of the specific PID but also on the characteristics of the individual patient. For the successful transplant outcome, the choice of donor and the optimal pretrans-plant conditioning regimen is important. This article will discuss current status and recommendations from specialists in HSCT for some representative PID, including severe combined immunodeficiency (SCID), CD40 ligand or CD40 deficiency, Wiskott-Aldrich syndrome, hemophagocytic lymphohistiocytosis, and chronic gran-ulomatous disease, along with our personal experience of PID treatment in Korea.