Background: Rixubis (recombinant factor IX, nonacog gamma) is indicated for the control and prevention of bleeding episodes, perioperative management, and routine prophylaxis in hemophilia B patients. This real-world, postmarketing surveillance study aimed to evaluate the safety and effectiveness of Rixubis in adult and pediatric hemophilia B patients in South Korea. Methods: This prospective, observational, multicenter study (clinicaltrials.gov identifier: NCT029 22231) was conducted in hemophilia B patients between April 2015 and April 2019, who were observed for up to 6 months after the initiation of Rixubis treatment. Safety was evaluated based on the number and severity of adverse events (AEs) and serious AEs (SAEs). Hemostatic effectiveness was assessed by physicians and patients by using a four-point scale and rated as excellent, good, fair, or no response based on treatment type. Results: In all, 58 patients were enrolled from four centers by seven physicians during the study period. The safety and effectiveness analysis sets included 57 and 54 patients, respectively. Overall, 11 AEs were reported in eight patients (14.0%), of which three were SAEs and occurred in three patients (5.3%). All 11 AEs were reported as unexpected and mild in severity, with no anaphylactic reaction, and 10 AEs (90.9%) resolved. The majority of AEs (10) were unrelated to Rixubis. Of the 142 hemostatic effectiveness assessments, 123 (86.6%) were reported as good or excellent. Conclusion Rixubis demonstrated an acceptable safety and effectiveness profile in the treatment of bleeding, perioperative management, and prophylaxis in hemophilia B patients in a real-world setting in South Korea.

It is important to identify therapeutic compounds with no adverse effects for use in the chemotherapy of patients with bone-related diseases. The aim of this study was to identify a new compound that inhibits osteoclast differentiation and bone resorption. Herein, we examined the effects of 1',2'-dihydrorotenone on osteoclast differentiation and bone resorption in vitro and in vivo. 1',2'-dihydrorotenone inhibited receptor activator of NF-kappaB ligand (RANKL)-induced osteoclast differentiation of cultured bone marrow macrophages (BMMs) in a dose-dependent manner. However, 1',2'-dihydrorotenone did not exert cytotoxic effect on BMMs. 1',2'-dihydrorotenone suppressed the expression of c-fos and NFATc1 as well as osteoclast-specific genes in BMMs treated with RANKL. Treatment with RANKL inhibited the expression of inhibitors of differentiation/DNA binding (Id)1, 2, and 3; however, in the presence of 1',2'-dihydrorotenone, RANKL did not suppress the expression of Id1, 2, and 3. Furthermore, 1',2'-dihydrorotenone inhibited bone resorption and considerably attenuated the erosion of trabecular bone induced by lipopolysaccharide treatment. Taken together, these results suggest that 1',2'-dihydrorotenone has the potential to be applied in therapies for bone-related diseases.
Bone Marrow ; Bone Resorption ; Humans ; Macrophages ; Osteoclasts ; Receptor Activator of Nuclear Factor-kappa B ; Rotenone

OBJECTIVE: The aim of this study was to illustrate the circumferential comfortable retainer (CCR) as a removable maxillary retainer with good potential patient compliance and to evaluate the discomfort of the retainers including distorted speech, gagging sensation and appliance discomfort. METHODS: Sixty-six orthodontic patients (male, 23; female, 43; mean age, 23.42 +/- 10.19 years) who received orthodontic treatment with fixed orthodontic appliances were randomly assigned to two groups after debonding, a conventional wraparound retainer (CWR) group that fully covers the palate with an acrylic plate and a highly polished surface, and a circumferential comfortable retainer (CCR) group which has a horseshoe shaped base plate with three folds on the anterior region. A questionnaire that had a visual analog scale (VAS) which consists of a 100-mm horizontal line with 2 end-points labeled "no discomfort" on the left and "worst discomfort" on the right, with regard to distorted speech, gagging sensation and discomfort, was administered to patients after 4 weeks of retainer wear. The Mann-Whitney test was used to test the hypothesis that there was no difference between the two retainers. RESULTS: Comparing distorted speech and discomfort, the CCR group significantly had lower values than the CWR group (p < 0.05). Comparing gagging sensation, the CCR group had lower values than the CWR group but there were no statistically significant differences between groups (p = 0.146). CONCLUSIONS: In conclusion, the results suggest that the circumferential comfortable retainer (CCR) might facilitate patient compliance and thereby improve the maintenance of the fixed orthodontic treatment outcome.
Female ; Gagging ; Humans ; Orthodontic Appliances ; Palate ; Patient Compliance ; Surveys and Questionnaires ; Sensation ; Treatment Outcome

Adult ; Humans ; Hypogonadism ; Mitochondrial Diseases ; Nose ; Ophthalmoplegia ; Tokyo

BACKGROUND: On performing umbilical cord blood (UCB) transplantation, faster engraftment may lead better clinical outcome. Because transplanted viable cell count in UCB is related to the engraftment, accurate evaluation of viability of CD34+cells in cryopreserved UCB has clinical implication. We examined the difference in viability of cells in cryopreserved UCB according to the duration of cryopreservation and different methods. METHODS: A total of 60 UCB samples which were cryopreserved for 1 to 4 years were used in this study. Viability of cryopreserved cells were examined with trypan blue exclusion assay, DNA contents analysis, caspase-3 activation test, intracellular esterase activity and Annexin-V/PI staining. RESULTS: After thawing the cryopreserved UCB, 89% of the total MNCs and 84% of CD34+cells were viable as identified by trypan blue exclusion assay. In the CD34+cell population, the cell death rate was found to be 47% by Annexin-V/PI staining and less than 5% by DNA contents analysis. However, cspase-3 activity failed to document apoptosis. The intracellular esterase activity test also showed a cell death rate of about 10~20% at 2, 4, and 6 hours after thawing. CONCLUSION: Viable cells in UCB should be measured by several compensatory techniques rather than a single method. Discordance among Annexin-V/PI staining versus trypan blue exclusion, DNA contents analysis, and the caspase-3 activation test or intracellular esterase activity should be clarified in order to apply these techniques for actual cord blood transplantation.
Apoptosis ; Caspase 3 ; Cell Count ; Cell Death ; Cryopreservation ; Diminazene ; DNA ; Fetal Blood ; Transplants ; Trypan Blue

BACKGROUND: Acute leukemias co-expressing myeloid and lymphoid antigens but does not meet the criteria for biphenotypic acute leukemia (BAL) is common, however its clinical significance is not fully defined. METHODS: In this study, clinical features of 68 co-expressing (myeloid and lymphoid) acute leukemias diagnosed between January 2000 and December 2006 were studied and compared with those of a control group of patients (pure AML or ALL). RESULTS: Age, gender, initial Lactate dehydrogenase (LDH) level and cytogenetics were not different between the co-expressing group and the control group. But, the initial bone marrow blast percent was significantly higher in the co-expressing group (70% vs. 54.5%, P=0.003). Fifty five percent (16/29) of ALL and 30% (52/172) of AML patients showed myeloid and lymphoid markers concomitantly. The lymphoid antigen positive AML (Ly+AML) patients showed significantly shorter survival rates than pure AML patients (4 year survival rate, 17.6% vs. 45.6%, P=0.002). However hematopoietic stem cell transplantation (HST) abrogated the difference (4 year survival rate, 54.7% vs. 50.6%, P=0.894). In ALL patients, survival rate was not affected by myeloid antigen co-expression (4 year survival rate 26.1% vs. 20%, P=0.954). CONCLUSION: Co-expression of lymphoid markers in AML should be regarded as a poor prognostic factor and more aggressive treatment such as HST should be considered.
Bone Marrow ; Cytogenetics ; Hematopoietic Stem Cell Transplantation ; Humans ; Immunophenotyping ; L-Lactate Dehydrogenase ; Leukemia ; Leukemia, Biphenotypic, Acute ; Prognosis ; Survival Rate

Successful treatment of the non-growing patient with an open bite of either dental skeletal pattern often presents a difficult challenge. The morphologic pattern in anterior open bite is characterized by longer vertical dimensions, an increase in development of the maxillary posterior dento-alveolar structure and a steep mandibular plane. In such cases, molar intrusion would be a good remedy for treatment. This article reports the successful treatment and retention of two anterior open-bite cases. We used orthodontic mini-implants for treatment and a circumferential retainer with posterior bite block or skeletal fixed retainer for retention. The diagnostic criteria and mechanics for appropriate treatment are discussed. Our results suggest that open bite can be reduced successfully with intrusion of molars using orthodontic mini-implants (OMI) without orthognathic surgery, and that circumferential retainer with posterior bite block and skeletal fixed retainer are effective for retention.
Adult ; Bites and Stings ; Humans ; Mechanics ; Molar ; Open Bite ; Orthognathic Surgery ; Retention (Psychology) ; Vertical Dimension

OBJECTIVE: The purpose of this study was to evaluate the efficacy of a slim bristled toothbrush compared with a V-shaped orthodontic toothbrush in patients with fixed orthodontic appliances. METHODS: Thirty four orthodontic patients receiving edgewise treatment were randomly assigned to two groups, a slim bristled toothbrush (Nano silver slim care) and a V-shaped toothbrush (Oral-B). Plaque index, gingival index and bleeding index were recorded at the beginning of the study (baseline), 2 weeks, 4 weeks and 6 weeks after new toothbrushes were supplied and the results were analyzed statistically. RESULTS: No statistically significant differences were found for plaque index, gingival index and bleeding index between toothbrush groups during the 6 weeks. Plaque and gingival indices were decreased at 2 weeks and 4 weeks but increased at 6 weeks in both toothbrush groups. Bleeding index in the V-shaped toothbrush group showed the lowest value at 2 weeks then increased at 4 weeks and 6 weeks but in the slim bristled toothbrush group decreased from 2 weeks through 6 weeks to under baseline levels. Patients in their twenties had significantly lower values than teenagers in the slim bristled toothbrush group (p < 0.013). CONCLUSIONS: The results would suggest that both of the toothbrushes are equally effective but the use of a slim bristled toothbrush may be of benefit in promoting gingival health for fixed orthodontic appliance patients in their twenties and over.
Adolescent ; Hemorrhage ; Humans ; Oral Hygiene ; Orthodontic Appliances ; Periodontal Index ; Silver

BACKGROUND: It is still obscure how dendritic cells (DCs) can orchestrate whole immune reactions according to the host age. We studied changes of murine splenic DCs after total body irradiation (TBI), with regards to age. METHODS: Young (8~14 wk) and old (12~16 mo) C57Bl/6 mice were irradiated with a dose of 1,100 cGy and were assessed 6 h later for phenotypic and functional changes of the DCs. The mean fluorescence intensities and cytokine producing cell proportions were analyzed with the student's t-test. RESULTS: Interleukin-12 (IL-12), interferon (IFN gamma) and tumor necrosis factor (TNF alpha) producing classical DCs (cDCs) were more numerous in the young untreated mice than in the old mice. However, the number of these cells decreased in the young mice and increased in the old mice after TBI. IL-12, IFN gamma and TNF alpha producing plasmacytoid DCs (pDCs) were more frequent in the old mice than in the young mice before TBI both mice showed an increased frequency of cells producing these cytokines after TBI. Overall, the highest numbers of cDCs and pDCs producing IL-12, IFN gamma and TNF alpha were present in the old mice after TBI. In both the cDC and pDC populations, the old mice had a higher frequency of IL-10+ cells prior to TBI. After irradiation, the young mice had a higher frequency of IL-10+ cells. CONCLUSION: With TBI, the DCs showed dramatic differences between young and old mice. Young mice turned to an immuno-suppressive response whereas the old mice changed to an immuno-stimulation of DCs after TBI. From these dramatic aging effects, we hope to explain the different frequencies and severities of acute GvHD after allogeneic hematopoietic stem cell transplantation according to host age.
Aging* ; Animals ; Centers for Disease Control and Prevention (U.S.) ; Cytokines ; Dendritic Cells* ; Fluorescence ; Hematopoietic Stem Cell Transplantation ; Hope ; Interferons ; Interleukin-12 ; Mice* ; Tumor Necrosis Factor-alpha ; Whole-Body Irradiation*

Idiopathic retroperitoneal fibrosis is a relatively rare disease that is characterized by the proliferation of fibrous tissue with an inflammatory process in the retroperitoneal cavity. It can cause an obstruction and compression of the ureter, abdominal aorta, and finally progress to renal failure. During the initial stages, the histology shows active inflammation. However, in the late stages, fibrous scarring occurs and the tissue becomes relatively avascular and acellular. Increased 18F-FDG accumulation was observed in our patient at the time of disease onset, which was attributed to the presence of inflammatory cells and actively metabolizing fibroblasts. We describe two patients with idiopathic retroperitoneal fibrosis, who were examined by 18F-FDG-PET, and discuss the efficiency of positron emission tomography in the diagnosis and management of idiopathic retroperitoneal fibrosis patients.
Aorta, Abdominal ; Cicatrix ; Diagnosis ; Fibroblasts ; Fluorodeoxyglucose F18 ; Humans ; Inflammation ; Positron-Emission Tomography* ; Rare Diseases ; Renal Insufficiency ; Retroperitoneal Fibrosis* ; Ureter