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Purpose@#Despite the recent success of Bruton’s tyrosine kinase (BTK) inhibitors for the treatment of Waldenstrom macroglobulinemia (WM), their indefinite treatment duration ultimately tantamount to substantial financial and emotional burden. On the other hand, fixed duration of proteasome inhibitors (PI) have shown rapid and reasonable response in WM treatment. Despite the well-known synergism between PI and immunomodulatory drugs (IMiD), there is no trials evaluating such combination in WM. @*Materials and Methods@#Based on above, we designed this phase II study to investigate the efficacy and safety of 6 cycles of 28-day bortezomib-thalidomide-dexamethasone (VTD) regimen for treatment-naïve WM. @*Results@#A total of 15 patients were enrolled: major response rate was 64.3%, and overall response rate was 78.6%. During the median follow-up of 41 months, median progression-free survival (PFS) was 13 months and overall survival 40 months. For responders, median duration of response was 13 months and median PFS 19 months. The most common adverse event (AE) of any grade was constipation (57.1%). The most common grade ≥ 3 AE was anemia (21.4%). @*Conclusion@#All in all, we hereby provide proof-of-concept that PI + IMiD may be an attractive backbone for fixed duration treatment. It should be noted that granting the same level of access to newer drugs globally is virtually impossible. Thus efforts to develop regimens using readily available drugs to yield similar or adequate treatment outcomes should not be disregarded. In this sense, we believe our study holds its place for its novelty and eloquently addresses achieving the daunting societal quest of health equity.

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Background/Aims@#Little attention is paid to chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) in Korea due to the rarity of the disease. With its rising incidence, we aimed to evaluate recent changes in treatment patterns and survival outcomes of patients with CLL/SLL. @*Methods@#A total of 141 patients diagnosed with CLL/SLL between January 2010 and March 2020 who received systemic therapy were analyzed in this multicenter retrospective study. @*Results@#The median patient age was 66 years at diagnosis, and 68.1% were male. The median interval from diagnosis to initial treatment was 0.9 months (range: 0–77.6 months), and the most common treatment indication was progressive marrow failure (50.4%). Regarding first-line therapy, 46.8% received fludarabine, cyclophosphamide, plus rituximab (FCR), followed by chlorambucil (19.9%), and obinutuzumab plus chlorambucil (GC) (12.1%). The median progression-free survival (PFS) was 49.3 months (95% confidence interval [CI], 32.7–61.4), and median overall survival was not reached (95% CI, 98.4 mo– not reached). Multivariable analysis revealed younger age (≤ 65 yr) (hazard ratio [HR], 0.46; p < 0.001) and first-line therapy with FCR (HR, 0.64; p = 0.019) were independently associated with improved PFS. TP53 aberrations were observed in 7.0% (4/57) of evaluable patients. Following reimbursement, GC became the most common therapy among patients over 65 years and second in the overall population after 2017. @*Conclusions@#Age and reimbursement mainly influenced treatment strategies. Greater effort to apply risk stratifications into practice and clinical trials for novel agents could help improve treatment outcomes in Korean patients.

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Palbociclib, in conjunction with endocrine therapy, has been approved for the treatment of patients with advanced breast cancer. The common hematological toxicities associated with palbociclib are leukopenia and neutropenia. However, hematological malignancies have not been reported for palbociclib treatment. Here, for the first time, we present a case of acute lymphoblastic leukemia that was diagnosed in a patient undergoing treatment with letrozole and palbociclib for metastatic breast cancer. This case emphasizes the need for long term follow up of patients treated with palbociclib.

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BACKGROUND: Mast cell leukemia (MCL) is the most aggressive form of systemic mastocytosis disorders. Owing to its rarity, neither pathogenesis nor standard treatment is established for this orphan disease. Hence, we tried to treat a patient with MCL based on the exome and transcriptome sequencing results of the patient's own DNA and RNA. METHODS: First, tumor DNA and RNA were extracted from bone marrow at the time of diagnosis. Germline DNA was extracted from the patient's saliva 45 days after induction chemotherapy and used as a control. Then, we performed whole-exome sequencing (WES) using the DNA and whole transcriptome sequencing (WTS) using the RNA. Single nucleotide variants (SNVs) were called using MuTect and GATK. Samtools, FusionMap, and Gene Set Enrichment Analysis were utilized to analyze WTS results. RESULTS: WES and WTS results revealed mutation in KIT S476I. Fusion analysis was performed using WTS data, which suggested a possible RARα-B2M fusion. When RNA expression analysis was performed using WTS data, upregulation of PIK3/AKT pathway, downstream of KIT and mTOR, was observed. Based on our WES and WTS results, we first administered all-trans retinoic acid, then dasatinib, and finally, an mTOR inhibitor. CONCLUSION: We present a case of orphan disease where we used a targeted approach using WES and WTS data of the patient. Even though our treatment was not successful, use of our approach warrants further validation.

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BACKGROUND: Intention to leave was an important managerial issue among physicians working in public health centers. This study was conducted to explore the relationship between job participations and intention to leave among physicians working in health centers. METHODS: A cross-sectional questionnaire survey was conducted to gather information about job participation, intention to leave and demographics among physicians (n=243) in public health centers in Korea. Job participation was measured by 15 items categorized 3 dimensions. Multiple regression analysis was performed to determine the effect of job participation on intent to leave among physicians working in public health center. RESULTS: Participation of medical treatment and administrative job were significantly associated with intention to leave adjusted for sex, age, income, working area, working duration, tenure, and overall job satisfaction. Therefore, physicians who actively participated in administrative job showed a lower turnover intention. Physicians who actively participated in medical treatment job had a higher quit intention. CONCLUSION: To retain qualified physicians in public health center, education should be reinforced to physician for administrative capacity building.

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Cholangiocarcinoma with paraneoplastic dermatomyositis (DM) is extremely rare, and the whole body positron emission tomography-computed tomography (PET-CT) finding of paraneoplastic DM is rarely reported. We report a 66-year-old woman with metastatic cholangiocarcinoma, initially presented with bilateral proximal muscle uptake on PET-CT without clinical muscle symptoms. The initial interpretation of the high muscle uptake was metastasis to the muscles. However, while awaiting for chemotherapy, muscle weakness evolved and rapidly progressed. The level of creatine phosphokinase was significantly elevated. Electromyography revealed moderate myopathy, and a muscle biopsy showed degenerating myofibers with variable sizes. The diagnosis of paraneoplastic dermatomyositis was made. This case highlights that, although rare, paraneoplastic dermatomyositis can be present with cholangiocarcinoma. Also, muscle inflammation can precede the clinical muscle symptoms, and paraneoplastic DM should be considered as a possible differential diagnosis in the assessment of cancer patients who present with abnormal muscle tracer uptake in PET-CT scans.

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Cholangiocarcinoma with paraneoplastic dermatomyositis (DM) is extremely rare, and the whole body positron emission tomography-computed tomography (PET-CT) finding of paraneoplastic DM is rarely reported. We report a 66-year-old woman with metastatic cholangiocarcinoma, initially presented with bilateral proximal muscle uptake on PET-CT without clinical muscle symptoms. The initial interpretation of the high muscle uptake was metastasis to the muscles. However, while awaiting for chemotherapy, muscle weakness evolved and rapidly progressed. The level of creatine phosphokinase was significantly elevated. Electromyography revealed moderate myopathy, and a muscle biopsy showed degenerating myofibers with variable sizes. The diagnosis of paraneoplastic dermatomyositis was made. This case highlights that, although rare, paraneoplastic dermatomyositis can be present with cholangiocarcinoma. Also, muscle inflammation can precede the clinical muscle symptoms, and paraneoplastic DM should be considered as a possible differential diagnosis in the assessment of cancer patients who present with abnormal muscle tracer uptake in PET-CT scans.

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Catastrophic antiphospholipid syndrome (APS) is defined as a rare, life-threatening autoimmune disorder leading to multiorgan failure. Probable APS, with clinical manifestations similar to APS without antiphospholipid antibodies, was suggested to be seronegative catastrophic APS. The triggering factors of catastrophic APS are various, including infection, trauma, malignancy, and surgery. In approximately 40% of patients, catastrophic APS develops from an unknown cause. We report a case of seronegative catastrophic APS due to an unknown origin. A 20-year-old man presented with cough, abdominal pain, skin lesions, tunnel vision, and watery diarrhea without fever. His symptoms and laboratory test suggested disseminated intravascular coagulation. Considering seronegative catastrophic APS, we treated with intravenous steroid and intravenous immunoglobulin, but the effects were limited. After weekly treatment with rituximab, an immune-modulating agent, his laboratory findings including thrombocytopenia and coagulation tests, returned to normal. We conclude that rituximab can be an effective treatment for seronegative catastrophic APS.

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BACKGROUND/AIMS: Colonic diverticular bleeding cases account for 30-40% of the lower gastrointestinal bleeding, among which, 3-5% appear to be massive bleeding. The purpose of this study was to evaluate the risk factors for colonic diverticular bleeding diagnosed by colonoscopic examination. METHODS: Among the 1,003 patients, who were identified to have colonic diverticulosis including sleeding by diverticulitis and diverticular bleeding coding search, 216 patients had diverculosis, and they were divided into two groups: one with diverticular bleeding, and the other without bleeding. We evaluated the potential risk factors for diverticular bleeding, based on age, gender, location of diverticulum, comorbidities related to atherosclerosis, smoking, alcohol and medications, and compared them between both groups. RESULTS: Among the 216 patients, we observed colonic diverticular bleeding in 35 patients (16.2%). The mean age of the bleeding group was significantly older than that of non-bleeding group. No difference was observed regarding gender ratio. Right colonic diverticula were common in both groups, but there were higher proportion of patients with bleeding in bilateral diverticuosis. Old age, bilateral diverticulosis, presence of atherosclerosis related diseases (hypertension, diabetes mellitus, ischemic heart disease, obesity), use of aspirin, NSAIDs and calcium channel blocker, increased the risk of bleeding. In a multivariate analysis, use of aspirin and bilateral diverticulosis were identified as independent risk factors for colonic diverticular bleeding. CONCLUSIONS: Since the patients who took aspirin and/or had bilateral colonic diverticulosis increased the risk of bleeding from divertuculi. As such, caution and education of patients are required.

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BACKGROUND/AIMS: Gastroesophageal reflux disease is one of the most common and frequent chronic disease requiring considerable cost. We investigated the medical care costs in the erosive reflux disease (ERD) and non-erosive reflux disease (NERD). METHODS: The risk factors and the direct medical care costs were analyzed retrospectively in the ERD (178 patients) and NERD (183 patients) groups for a follow up period of 2 years. RESULTS: Logistic regression analysis showed that the ERD was more frequent in the groups of male gender, alcohol consumption, higher body mass index (> or =25 kg/m2), hiatal hernia, and higher triglyceride levels (> or =150 mg/dL). The direct medical care costs per person for 2 years were found to be $384.8 (ERD) and $412.9 (NERD) without statistically significant differences (p = 0.364). However, 9.3% (17/183) of the NERD patients had visited the emergency room compared to 3.4% (6/178) of the ERD patients (p = 0.029). In addition, more NERD patients were hospitalized than ERD patients (p = 0.006), and because of the longer hospitalization period, the medical costs in NERD patients were higher than ERD patients (p = 0.038). CONCLUSIONS: In spite of the different risk factors for ERD and NERD, total direct medical care costs were similar between the ERD and NERD group. However, more visits to emergency room and longer hospitalization period with more hospitalization costs in NERD patients account for the differences in medical service and usage distribution between the 2 groups.

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Persistent pulmonary hypertension of the newborn (PPHN) is defined as a failure of normal pulmonary vascular relaxation at or shortly after birth, resulting in impedance to pulmonary blood flow which exceeds systemic vascular resistance, such that unoxygenated blood is shunted to the systemic circulation. Perinatal stressors including hypoxia, hypoglycemia, cold stress, sepsis, and direct lung injury alter the course of transition. The initial clinical picture of PPHN is one of dynamic pulmonary vasospasm, with labile flow through the pulmonary circuit and right-to-left shunting of blood across the ductus arteriosus and foramen ovale. The normal postnatal decline in pulmonary vascular tone is absent following exposure to chronic hypoxia. The pathophysiology of neonatal pulmonary hypertension can involve multiple pathways of injury, from altered circulating agonist balance, to endothelial dysfunction, to smooth muscle dysfunction and phenotypic change. The treatment for PPHN has evolved over the past 10 to 15 years but reported mortality remains at 10% to 20% in newborns with PPHN. Extracorporeal membrane oxygenation (ECMO) has been proven of value for this condition, and several "alternative" therapies such as high-frequency ventilation (HFV), surfactant, and inhaled NO (iNO) have been used in a rescue mode.

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Persistent pulmonary hypertension of the newborn (PPHN) is defined as a failure of normal pulmonary vascular relaxation at or shortly after birth, resulting in impedance to pulmonary blood flow which exceeds systemic vascular resistance, such that unoxygenated blood is shunted to the systemic circulation. Perinatal stressors including hypoxia, hypoglycemia, cold stress, sepsis, and direct lung injury alter the course of transition. The initial clinical picture of PPHN is one of dynamic pulmonary vasospasm, with labile flow through the pulmonary circuit and right-to-left shunting of blood across the ductus arteriosus and foramen ovale. The normal postnatal decline in pulmonary vascular tone is absent following exposure to chronic hypoxia. The pathophysiology of neonatal pulmonary hypertension can involve multiple pathways of injury, from altered circulating agonist balance, to endothelial dysfunction, to smooth muscle dysfunction and phenotypic change. The treatment for PPHN has evolved over the past 10 to 15 years but reported mortality remains at 10% to 20% in newborns with PPHN. Extracorporeal membrane oxygenation (ECMO) has been proven of value for this condition, and several "alternative" therapies such as high-frequency ventilation (HFV), surfactant, and inhaled NO (iNO) have been used in a rescue mode.

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