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Background@#There has been remarkable progress in hemophilia A (HA) treatment in Korea.Viral inactivation products were developed in 1989, use of recombinant factor VIII (FVIII) concentrates started in 2002, and prophylaxis expanded thereafter. This study was conducted to identify the changes in complications in HA before and after 1989 or 2002. @*Methods@#The study was performed using the 2007–2019 Healthcare Big Data Hub of the Health Insurance Review and Assessment Service. @*Results@#Among 2,557 patients, 1,084 had ≥ 1 complication; 829 had joint problems, 328 had viral infections, 146 had neurologic sequelae, and 87 underwent 113 surgeries or procedures due to complications. Patients born after 1989 had a significantly lower risk of viral infections than those born before 1989; 27.1% vs. 1.4% (P 10 years to expand prophylaxis widely. Neurologic sequelae have not decreased; thus, additional efforts to decrease intracranial hemorrhage are needed. We suggest personalized dosing of FVIII and more meticulous care during childbirth to further reduce the complications.
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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 children, 79 (9.9%) were diagnosed with APL. The median age at diagnosis was 10.6 years (range, 1.3 to 18.0). Male and female ratio was 1:0.93. Thirty patients (38.0%) had white blood cell (WBC) count greater than 10×109/L at diagnosis. All patients received induction therapy consisting of all-trans retinoic acid and chemotherapy. Five patients (6.6%) died during induction chemotherapy and 66 patients (86.8%) achieved complete remission (CR) after induction chemotherapy. The causes of death were three intracranial hemorrhage, one cerebral infarction, and one sepsis. Five patients (7.1%) suffered a relapse during or after maintenance chemotherapy. The estimated 4-year event-free survival and overall survival (OS) rates were 82.1%±4.4%, 89.7%±5.1%, respectively. The 4-year OS was significantly higher in patients with initial WBC < 10×109/L than in those with initial WBC ≥ 10×109/L (p=0.020). @*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.
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Purpose@#Children with Down syndrome (DS) show a higher risk of acute leukemia than those without DS. In this study, we investigated the nationwide incidence of acute leukemia among children with DS and compared their epidemiologic characteristics with those of children with acute leukemia but without DS. @*Materials and Methods@#Using the National Health Insurance Service database, we selected patients with acute leukemia aged 0–19 years at diagnosis between 2007 and 2016. @*Results@#Among the 4,697 children with acute leukemia, 54 (1.1%) had DS. The median incidence rate of leukemia with DS by year was 1.3% (range, 0.2%–2.0%). Sixteen patients with acute lymphoblastic leukemia (ALL; 29.6%) and 36 with acute myeloid leukemia (AML; 66.7%) had DS. The DS group showed younger age at diagnosis than the non-DS group, and diagnosis of AML was more frequent in the DS group than in the non-DS group (3 years vs. 9 years, p<0.001; 66.7% vs. 32.4%, P<0.001, respectively). The 5-year overall survival was comparable between the DS and non-DS groups (88.0% vs. 81.9%, p=0.375). Among all the Koreans born between 2007 and 2008, the incidences of acute leukemia, ALL, and AML were 49.25, 20.75, and 163.38 times higher, respectively, in the DS group than in the non-DS group. @*Conclusion@#Our findings support the fact that the incidence of acute leukemia is higher among patients with DS than among those without DS in Korea. However, the DS and non-DS groups in this study had a comparable overall survival rate.
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Despite the availability of therapies to treat patients with immune thrombocytopenia (ITP), there is currently little data from randomized trials to assist clinicians in managing patients. The evidence-based guidelines of the Korean Society of Hematology Aplastic Anemia Working Party (KSHAAWP) are intended to support patients and physicians in the management of ITP. Experts from the KSHAAWP discussed and described this guideline according to the current treatment situation for ITP in Korea and finalized the guidelines. The expert panel recommended the management of ITP in adult and pediatric patients with newly diagnosed, persistent, and chronic disease refractory to first-line therapy with minor bleeding. Management approaches include observation and administration of corticosteroids, intravenous immunoglobulin, anti-D immunoglobulin, and thrombopoietin receptor agonists. Currently, evidence supporting strong recommendations for various management approaches is lacking. Therefore, a large focus was placed on shared decision-making, especially regarding second-line treatment.
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When a patient with malignant lymphoma develops new lymph node enlargement, a recurrence of lymphoma is usually suspected first. However, painless and rapid lymph node enlargement, a manifestation of non-Hodgkin’s lymphoma, could also be due to other causes. A 3-year-old boy who was previously diagnosed with Burkitt lymphoma was admitted for routine tumor evaluation one year following completion of treatment. Abdominal computed tomography showed several enlarged lymph nodes in the right lower quadrant, and 18 F-fluoro-2-deoxy-D-glucose positron emission tomography revealed hypermetabolic enlarged lymph nodes in the corresponding lesion. The patient underwent ileocecal lymph node biopsy for pathologic confirmation, which revealed reactive hyperplasia without lymphoma recurrence. Serologic test results for cytomegalovirus immunoglobulin G and immunoglobulin M were positive. Additionally, the polymerase chain reaction test performed using a urine sample was positive for cytomegalovirus. After several outpatient follow-ups, we concluded cytomegalovirus infection that mimicked a recurrence of lymphoma on imaging as the cause for lymph node enlargements. This case highlights the importance of using prompt and multiple approaches after detecting a possible tumor recurrence through imaging studies.
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When a patient with malignant lymphoma develops new lymph node enlargement, a recurrence of lymphoma is usually suspected first. However, painless and rapid lymph node enlargement, a manifestation of non-Hodgkin’s lymphoma, could also be due to other causes. A 3-year-old boy who was previously diagnosed with Burkitt lymphoma was admitted for routine tumor evaluation one year following completion of treatment. Abdominal computed tomography showed several enlarged lymph nodes in the right lower quadrant, and 18 F-fluoro-2-deoxy-D-glucose positron emission tomography revealed hypermetabolic enlarged lymph nodes in the corresponding lesion. The patient underwent ileocecal lymph node biopsy for pathologic confirmation, which revealed reactive hyperplasia without lymphoma recurrence. Serologic test results for cytomegalovirus immunoglobulin G and immunoglobulin M were positive. Additionally, the polymerase chain reaction test performed using a urine sample was positive for cytomegalovirus. After several outpatient follow-ups, we concluded cytomegalovirus infection that mimicked a recurrence of lymphoma on imaging as the cause for lymph node enlargements. This case highlights the importance of using prompt and multiple approaches after detecting a possible tumor recurrence through imaging studies.
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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 (P = 0.021). Five-year OS was worse in patients with B-symptoms, stage IV disease, highrisk, 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.
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In the initial published version of this article, there was a mistake in the title. The correct title should be “Impact of Day 14 Peripheral Blood Chimerism after Allogeneic Hematopoietic Stem Cell Transplantation on the Treatment Outcome of Non-Malignant Disease”.
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BACKGROUND: The impact of early peripheral blood chimerism on the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) is unclear. We aimed to determine whether day 14 peripheral blood chimerism after allo-HSCT predicts outcomes in patients with non-malignant diseases. METHODS: Data from 56 patients who received allo-HSCT between April 2007 and March 2016 were retrospectively analyzed. Chimerism was evaluated using short-tandem repeat polymerase chain reaction, with mixed chimerism (MC) defined as greater than 1% recipient cells which was further categorized into low-level MC (> 1% and < 15% of recipient-derived cells) and high-level MC (≥ 15% of the recipient-derived cells). RESULTS: Thirty-six patients showed complete donor chimerism (CC), 14 low-level MC, and 6 high-level MC at day 14 post-transplant. The estimated 5-year event-free survival (EFS) was higher in the CC or low-level MC groups than in the high-level MC group (86.1% vs. 71.4% vs. 33.3%; P = 0.001). In BM or peripheral blood stem cell (BM/PBSC) transplants, the 5-year EFS was higher in the CC or low-level MC group than in the high-level MC group (93.1% vs. 66.7% vs. 0%; P < 0.001). However, in cord blood transplants, the 5-year OS and EFS according to the day 14 peripheral blood chimerism did not reach statistical significance. CONCLUSION: Although CC is not always necessary after allo-HSCT for non-malignant diseases, our data suggest that day 14 peripheral blood chimerism may predict outcomes in patients with non-malignant diseases who underwent BM/PBSC transplants.
Subject(s)
Humans , Bone Transplantation , Chimerism , Disease-Free Survival , Fetal Blood , Hematopoietic Stem Cell Transplantation , Hematopoietic Stem Cells , Polymerase Chain Reaction , Retrospective Studies , Stem Cells , Tissue Donors , Treatment OutcomeABSTRACT
PURPOSE: We aimed to identify the impact of NUDT15 variants on thiopurine intolerance and 6-thioguanine nucleotide (6-TGN) levels in Korean children with acute lymphoblastic leukemia (ALL). MATERIALS AND METHODS: Genotyping of NUDT15 was tested in 258 patients with ALL registered at Samsung Medical Center. Patients were classified into normal-activity (wild-type), intermediate-activity (heterozygous variant), and low-activity groups (homozygous or compound heterozygous variant). Clinical and laboratory features during the first year of maintenance therapy were investigated. RESULTS: A total of 182 patients were included in the final analysis. There were five (2.7%), 46 (25.3%), and 131 (72.0%) patients in low-, intermediate-, and normal-activity groups, respectively. The lowest 6-mercaptopurine (6-MP) dose (mg/m2/day) was administered to the low-activity group (low-activity group 7.5 vs. intermediate-activity group 24.4 vs. normalactivity group 31.1, p < 0.01) from three months to a year after beginning maintenance therapy. The low-activity group experienced the longest duration of therapy interruption during the first year (low-activity group 169 days vs. intermediate-activity group 30 days vs. normal-activity group 16 days, p < 0.01). They also showed the lowest blood cell counts and had a longer duration of leukopenia (low-activity group 131 days vs. intermediate-activity group 92 days vs. normal-activity group 59 days, p < 0.01). 6-TGN level and its ratio to 6-MP dose were lowest in the low-activity group. CONCLUSION: NUDT15 variants cause hematopoietic toxicity with low 6-TGN levels. NUDT15 genotyping should be conducted before administering thiopurine, and dose adjustments require caution regardless of 6-TGN levels.
Subject(s)
Child , Humans , Mercaptopurine , Blood Cell Count , Leukemia , Leukopenia , Precursor Cell Lymphoblastic Leukemia-Lymphoma , ThioguanineABSTRACT
PURPOSE: Although the prognosis is generally good in patients with intermediate-risk neuroblastoma, no consensus has been reached on the ideal treatment regimen. This study analyzed treatment outcomes and toxicities in patients younger than 18 months with stage 4 MYCN nonamplified neuroblastoma. METHODS: We retrospectively analyzed 20 patients younger than 18 months newly diagnosed with stage 4 MYCN nonamplified neuroblastoma between January 2009 and December 2015. Patients received 9 cycles of chemotherapy and surgery, with or without local radiotherapy, followed by 12 cycles of differentiation therapy with 13-cis-retinoic acid. Chemotherapy consisted of alternating cycles of cisplatin, etoposide, doxorubicin, and cyclophosphamide (CEDC) and ifosfamide, carboplatin, and etoposide (ICE) regimens. RESULTS: The most common primary tumor site was the abdomen (85%), and the most common metastatic sites were the lymph nodes (65%), followed by the bones (60%), liver (55%), skin (45%), and bone marrow (25%). At the end of induction therapy, 14 patients (70%) achieved complete response, with 1 achieving very good partial response, 4 achieving partial response, and 1 showing mixed response. Nine patients (45%) received local radiotherapy. At a median follow-up of 47 months (range, 17–91 months), none of these patients experienced relapse, progression, or secondary malignancy, or died. Three years after chemotherapy completion, none of the patients had experienced grade ≥3 late adverse effects. CONCLUSION: Patients younger than 18 months with stage 4 MYCN nonamplified neuroblastoma showed excellent outcomes, without significant late adverse effects, when treated with alternating cycles of CEDC and ICE, followed by surgery and differentiation therapy.
Subject(s)
Child , Humans , Infant , Abdomen , Bone Marrow , Carboplatin , Cisplatin , Consensus , Cyclophosphamide , Doxorubicin , Drug Therapy , Etoposide , Follow-Up Studies , Ice , Ifosfamide , Isotretinoin , Liver , Lymph Nodes , Neoplasm Metastasis , Neuroblastoma , Prognosis , Radiotherapy , Recurrence , Retrospective Studies , SkinABSTRACT
OBJECTIVE: This study was conducted to investigate the efficacy of laser-assisted hatching (LAH) and various vitrification times for embryonic development and blastocyst cell numbers. METHODS: First, 2-cell and 8-cell embryos were collected by flushing out the oviducts. In the control groups, they were vitrified for 8 or 10 minutes without LAH. The LAH groups underwent quarter laser zona thinning-assisted hatching before vitrification (4, 6, and 8 minutes or 4, 7, and 10 minutes, respectively). After incubation, double-immunofluorescence staining was performed. RESULTS: The hatched blastocyst rate 72 hours after the 2-cell embryos were thawed was significantly higher in the 2LAH-ES8 group (33.3%) than in the other groups (p < 0.05). In the control-8 group (22.1±4.6), the cell number of the inner cell mass was higher than in the LAH groups (p < 0.05). The number of trophectoderm cells was higher in the 2LAH-ES6 group (92.8±8.9) than in the others (p < 0.05). The hatched blastocyst rate 48 hours after the 8-cell embryos were thawed was higher in the 8LAH-ES4 group (45.5%) than in the other groups, but not significantly. The inner cell mass cell number was highest in the 8LAH-ES7 group (19.5±5.1, p < 0.05). The number of trophectoderm cells was higher in the 8LAH-ES10 group (73.2±12.1) than in the other groups, but without statistical significance. CONCLUSION: When LAH was performed, 2-cell embryos with large blastomeres had a lower hatched blastocyst rate when the exposure to vitrification solution was shorter. Conversely, 8-cell embryos with small blastomere had a higher hatched blastocyst rate when the exposure to vitrification solution was shorter.
Subject(s)
Animals , Female , Mice , Pregnancy , Blastocyst , Blastomeres , Cell Count , Embryonic Development , Embryonic Structures , Flushing , Herpes Zoster , Oviducts , VitrificationABSTRACT
Management options for patients with immune thrombocytopenia (ITP) have evolved substantially over the past decades. The American Society of Hematology published a treatment guideline for clinicians referring to the management of ITP in 2011. This evidence-based practice guideline for ITP enables the appropriate treatment of a larger proportion of patients and the maintenance of normal platelet counts. Korean authority operates a unified mandatory national health insurance system. Even though we have a uniform standard guideline enforced by insurance reimbursement, there are several unsolved issues in real practice in ITP treatment. To optimize the management of Korean ITP patients, the Korean Society of Hematology Aplastic Anemia Working Party (KSHAAWP) reviewed the consensus and the Korean data on the clinical practices of ITP therapy. Here, we report a Korean expert recommendation guide for the management of ITP.
Subject(s)
Humans , Anemia, Aplastic , Clothing , Consensus , Evidence-Based Practice , Hematology , Insurance , National Health Programs , Platelet Count , Purpura, Thrombocytopenic, IdiopathicABSTRACT
Adequate organ growth is an important aspect of growth evaluation in children. Renal size is an important indicator of adequate renal growth; computed tomography (CT) can closely estimate actual kidney size. However, insufficient data are available on normal renal size as measured by CT. This study aimed to evaluate the relationships of anthropometric indices with renal length and volume measured by CT in Korean pediatric patients. Renal length and volume were measured using CT images in 272 pediatric patients (age < 18 years) without renal disease. Data for anthropometric indices—including height, weight, and body surface area (BSA)—were obtained using medical records. Using the equation for an ellipsoid, renal volume was calculated in cubic centimeters. Height showed greatest correlation with renal length on stepwise multiple linear regression analysis; BSA showed the strongest significant correlation with renal volume. The mean renal size for each age group and height group was determined; it showed a tendency to increase with age and height. This is the first Korean study to report the relationship between body indices and renal size measured by CT. These results can serve as normative standards for assessing adequate renal growth.
Subject(s)
Child , Humans , Body Surface Area , Kidney , Linear Models , Medical Records , Regression AnalysisABSTRACT
PURPOSE: The purpose of this study is to investigate the clinical significance of tyrosine hydroxylase (TH) expression in peripheral blood (PB) at diagnosis in patients with neuroblastoma. MATERIALS AND METHODS: TH mRNA expression in PB was measured by reverse transcription quantitative real-time polymerase chain reaction in 210 patients who were newly diagnosed with neuroblastoma from July 2005 to June 2015 and the clinical significance of TH expression in PB at diagnosis was evaluated. RESULTS: TH expression was positive in 60 patients (28.6%). Fifty of 60 TH-positive patients had metastatic tumors and the remaining 10 had localized tumors. TH expression was associated with high-risk features (i.e., advanced stage, older age, unfavorable pathology, and MYCN amplification) at diagnosis. Among TH-positive patients, higher TH expression level was observed in high-risk patients than in low- or intermediate-risk patients (p=0.035). The probability of 5-year progression-free survival (PFS) was lower in TH-positive patients than in TH-negative patients (63.8±6.9% vs. 94.7±2.1%, p < 0.001). In analysis confined to high-risk patients, the 5-year probability of PFS remained lower in TH-positive patients (55.7±8.2% vs. 89.6±5.8%, p < 0.001). Among TH-positive patients, a higher expression level of TH was associated with a worse outcome. In multivariate analyses, positive TH expression in PB at diagnosis was an independent poor prognostic factor for PFS. CONCLUSION: The treatment intensity should be tailored according to TH expression in PB at diagnosis.
Subject(s)
Humans , Diagnosis , Disease-Free Survival , Multivariate Analysis , Neuroblastoma , Pathology , Polymerase Chain Reaction , Real-Time Polymerase Chain Reaction , Reverse Transcription , RNA, Messenger , Tyrosine 3-Monooxygenase , TyrosineABSTRACT
PURPOSE: We prospectively evaluated the effectiveness of tandem high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) in improving the survival of patients with atypical teratoid/rhabdoid tumors while reducing the risks of late adverse effects from radiotherapy (RT). MATERIALS AND METHODS: For young children ( 3 years old), RT including reduced-dose craniospinal RT (23.4 or 30.6 Gy) was administered either after two cycles of induction chemotherapy or after surgery, and tandem HDCT/auto-SCT was administered after six cycles of induction chemotherapy. RESULTS: A total of 13 patients (five young and eight older) were enrolled from November 2004 to June 2012. Eight patients, including all five young patients, had metastatic disease at diagnosis. Six patients (four young and two older) experienced progression before initiation of RT, and seven were able to proceed to HDCT/auto-SCT without progression during induction treatment. Three of six patients who experienced progression during induction treatment underwent HDCT/auto-SCT as salvage treatment. All five young patients died from disease progression. However, four of the eight older patients remain progression-freewith a median follow-up period of 64 months (range, 39 to 108 months). Treatment-related late toxicities were acceptable. CONCLUSION: The required dose of craniospinal RT might be reduced in older patients if the intensity of chemotherapy is increased. However, early administration of RT should be considered to prevent early progression in young patients.
Subject(s)
Child , Humans , Brain Neoplasms , Diagnosis , Disease Progression , Drug Therapy , Follow-Up Studies , Hematopoietic Stem Cell Transplantation , Induction Chemotherapy , Prospective Studies , Radiotherapy , Recurrence , Salvage Therapy , Stem Cell Transplantation , Stem CellsABSTRACT
Despite increasing evidence that high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) might improve the survival of patients with high-risk or recurrent solid tumors, therapy effectiveness for bone and soft tissue sarcoma treatment remains unclear. This study retrospectively investigated the feasibility and effectiveness of HDCT/auto-SCT for high-risk or recurrent bone and soft tissue sarcoma. A total of 28 patients (18 high-risk and 10 recurrent) underwent single or tandem HDCT/auto-SCT between October 2004 and September 2014. During follow-up of a median 15.3 months, 18 patients exhibited disease progression and 2 died of treatment-related toxicities (1 veno-occlusive disease and 1 sepsis). Overall, 8 patients remained alive and progression-free. The 3-year overall survival (OS) and event-free survival (EFS) rates for all 28 patients were 28.7% and 26.3%, respectively. In the subgroup analysis, OS and EFS rates were higher in patients with complete or partial remission prior to HDCT/auto-SCT than in those with worse responses (OS, 39.1% vs. 0.0%, P = 0.002; EFS, 36.8% vs. 0.0%, P < 0.001). Therefore, careful selection of patients who can benefit from HDCT/auto-SCT and maximal effort to reduce tumor burden prior to treatment will be important to achieve favorable outcomes in patients with high-risk or recurrent bone and soft tissue sarcomas.
Subject(s)
Adolescent , Child , Child, Preschool , Female , Humans , Infant , Male , Young Adult , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Bone Neoplasms/mortality , Disease-Free Survival , Follow-Up Studies , Retrospective Studies , Sarcoma/mortality , Soft Tissue Neoplasms/mortality , Stem Cell Transplantation , Survival Rate , Transplantation, Autologous , Treatment OutcomeABSTRACT
BACKGROUND: This retrospective study aimed to characterize and analyze the outcome of therapy-related myeloid neoplasms (t-MNs) in children and adolescents. METHODS: The medical records of 16 patients under 21 years of age at the time of t-MN diagnosis were reviewed. RESULTS: The median patient age was 11.5 years (range, 1.6–20.4 yr). Twelve patients had therapy-related acute myeloid leukemia, 3 patients had myelodysplastic syndrome, and 1 patient had chronic myelomonocytic leukemia. The median latency period was 29 months (range, 11–68 mo). Fourteen patients had cytogenetic aberrations, 8 of whom had an 11q23 abnormality. Of the 13 patients treated with curative intent, 12 patients received myeloid-type induction therapy that led to complete remission (CR) in 8 patients. Nine patients underwent allogeneic transplantation; 4 patients did not undergo transplantation due to chemotherapy-related toxic death (N=3) or parental refusal (N=1). The 5-year overall survival and event-free survival of the 13 patients treated with a curative intent were 46.2% and 30.8%, respectively. For the 9 patients who underwent allogeneic transplantation, the 5-year event-free survival was 66.7%. CONCLUSION: A significant proportion of young patients with t-MNs can experience long-term survival, and allogeneic transplantation plays a key role for attaining cure in these patients.
Subject(s)
Adolescent , Child , Humans , Chromosome Aberrations , Diagnosis , Disease-Free Survival , Latency Period, Psychological , Leukemia, Myeloid, Acute , Leukemia, Myelomonocytic, Chronic , Medical Records , Myelodysplastic Syndromes , Parents , Retrospective Studies , Transplantation, HomologousABSTRACT
BACKGROUND: Kasabach-Merritt syndrome (KMS) is a rare but life-threatening illness. The purpose of this study is to report our single-center experience with KMS. METHODS: We reviewed the medical records of 13 patients who were diagnosed with KMS between 1997 and 2012 at Samsung Medical Center. Treatment response was defined as follows: 1) hematologic complete response (HCR) – platelet count >130×10⁹/L without transfusion; 2) clinical complete response (CCR) – complete tumor disappearance or small residual vascular tumor displaying lack of proliferation for at least 6 months after treatment discontinuation. RESULTS: Participants included 7 male and 6 female patients. The median initial hemoglobin levels and platelet counts were 9.7 g/dL (range, 6.6–11.6 g/dL) and 11×10⁹/L (range, 3–38×10⁹/L), respectively. Twelve patients received corticosteroid and interferon-alpha as initial treatment, and the remaining patient received propranolol instead of corticosteroid. Two patients with unsatisfactory response to the initial treatment received weekly vincristine. Successful discontinuation of medication was possible at a median of 301 days (range, 137–579) in all patients except one who was lost to follow-up. The median times to achieve HCR and CCR were 157 days and 332 days, respectively. The probabilities of achieving HCR and CCR were 77% and 54% at 1 year, and 88% and 86% at 2.5 years, respectively. CONCLUSION: The prognosis of KMS in our cohort was excellent. Our data suggest that individualized treatment adaptation according to response may be very important for the successful treatment of patients with KMS.
Subject(s)
Female , Humans , Male , Cohort Studies , Interferon-alpha , Kasabach-Merritt Syndrome , Lost to Follow-Up , Medical Records , Platelet Count , Prognosis , Propranolol , VincristineABSTRACT
Neuroblastoma (NB) is the most common extra-cranial solid tumor of childhood and is characterized by a wide range of clinical behaviors. Amplification of MYCN is a well-known poor prognostic factor in NB patients. As the MYCN amplification status is usually tested using tumor specimens, lengthy and invasive procedures are unavoidable. To evaluate the possibility of detecting MYCN amplification without invasive procedure, we performed conventional polymerase chain reaction (PCR) analysis to identify MYCN amplification using the preserved serum DNA. PCR of serum DNA was done in 105 NB patients whose MYCN status had been confirmed by fluorescence in situ hybridization. MYCN amplification was evaluated as the ratio of signal intensities between MYCN and NAGK (M/N ratio). When regarding the tissue FISH results as a reference, 10 patients had MYCN-amplified (MNA) NB, and 95 had non-MNA NB. The M/N ratio of the MNA group (median 2.56, range 1.01-3.58) was significantly higher than that of the non-MNA group (median 0.97, range 0.67-5.18) (P < 0.001). In the receiver operating characteristic curve analysis, the area under the curve was 0.957 (95% confidence interval 0.898-1.000; P < 0.001), and it showed 90.9% sensitivity and 97.9% specificity with the selected cut-off value set as 1.6. The detection of MYCN amplification using conventional PCR analysis of serum samples seems to be a simple and promising method to evaluate the MYCN status of NB patients. Further study with a larger set of patients is needed to confirm the accuracy of this result.