Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder characterized by uncontrolled terminal complement activation. Eculizumab, a monoclonal antibody C5 inhibitor was introduced in Korea in 2009 and has been the standard treatment option for PNH. Methods: This study assessed the long-term efficacy/safety of eculizumab in PNH using real-world data from the Korean Health Insurance Review and Assessment Service. Eighty patients who initiated eculizumab from 2009–2020 were enrolled. Results: At eculizumab initiation, the median age was 51.5 years, lactate dehydrogenase (LDH) 6.8 × upper limit of normal, and granulocyte clone size 93.0%. All patients had at least one PNH-related complication before eculizumab initiation, including renal failure (n = 36), smooth muscle spasm (n = 24), thromboembolism (n = 20), and pulmonary hypertension (n = 15). The median (range) duration of eculizumab treatment was 52.7 (1.0, 127.3) months (338.6 total treated patient-years). Despite high disease activity in the study population before treatment initiation, overall survival was 96.2% and LDH levels were stabilized in most patients during treatment. PNH-related complications at treatment initiation were resolved in 44.4% of patients with renal failure, 95.8% with smooth muscle spasm, 70.0% with thromboembolism, and 26.7% with pulmonary hypertension. Extravascular hemolysis occurred in 28.8% of patients (n = 23; 0.09 per patient-year) and breakthrough hemolysis in 18.8% (n = 15; 0.06 per patient-year). No treatment discontinuation cases related to eculizumab were observed. Conclusion These data provided evidence for the long-term efficacy and safety of eculizumab in Korean PNH patients with high disease burdens.

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.

BACKGROUND/AIMS: Limited data are available regarding the efficacy of rivaroxaban for the treatment of cancer-associated venous thromboembolism (VTE). The aim of this study was to evaluate the effectiveness and safety of rivaroxaban for the treatment of VTE in active cancer patients. METHODS: In this prospective, multicenter, open-label trial (NCT01989845), we enrolled patients with active cancer and objectively diagnosed lower-extremity deep vein thrombosis, pulmonary embolism (PE), or both from November 2013 to June 2016. Active cancer was defined as a histologically confirmed malignancy, which was diagnosed or treated within the previous 6 months, or as a recurrent/metastatic cancer. Patients received oral rivaroxaban 15 mg twice daily for first 3 weeks, followed by 20 mg once daily for 6 months. The primary outcome was the symptomatic recurrent VTE and the secondary outcomes included any recurrent VTE, major or clinically relevant non-major (CRNM) bleeding events, and overall mortality. All study outcomes were validated by blinded central adjudication. RESULTS: Of 124 patients enrolled, 110 (88.7%) had solid cancer, 93 (75.0%) had metastatic disease, and 110 (88.7%) were receiving chemotherapy or radiotherapy. During the 6-month study period, seven patients experienced symptomatic recurrent VTE (cumulative incidence, 5.9%), and two patients experienced incidental recurrent PE (cumulative incidence of any recurrent VTE, 7.6%). Major bleeding events occurred in six patients (cumulative incidence, 5.3%) and CRNM bleeding events in 11 patients (cumulative incidence, 10.2%). Twenty-eight patients (overall mortality, 24.0%) died. CONCLUSIONS Rivaroxaban is effective and safe for the treatment of VTE in patients with active cancer.

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.
Anemia, Aplastic ; Clothing ; Consensus ; Evidence-Based Practice ; Hematology ; Humans ; Insurance ; National Health Programs ; Platelet Count ; Purpura, Thrombocytopenic, Idiopathic*

OBJECTIVE: The purpose of our study was to investigate the effect of the mode of delivery on the oxidant and antioxidant system in umbilical cord blood. METHODS: We performed gas analysis of umbilical venous blood and umbilical arterial blood immediately after delivery in 38 women; eighteen women had a vaginal delivery while 20 women delivered via cesarean section at over 37 weeks gestation. We examined lipid peroxide concentration by thiobarbituric acid reaction, protein carbonyl content by 2,4-dinitrophenylhydrazine reaction, and total antioxidant capacity by oxygen radical absorbance capacity assay. RESULTS: Lipid peroxide levels in umbilical venous blood were significantly higher in patients delivering by planned cesarean section (1.81 +/- 0.06 nmol/mg protein) than those with vaginal delivery (1.24 +/- 0.05 nmol/mg protein) (P < 0.05). Antioxidant capacity in umbilical venous blood was significantly higher in patients delivering by planned cesarean section (119.70 +/- 0.13 microM/microL) than those with a vaginal delivery (118.70 +/- 0.29 microM/microL) (P < 0.05). There was no significant difference in the carbonyl content of umbilical venous blood or in the lipid peroxide, carbonyl content, and total antioxidant capacity of umbilical arterial blood. CONCLUSION: Lipid peroxidation levels and antioxidant capacity in umbilical venous blood were higher in patients delivering by planned cesarean section than those with a vaginal delivery. Therefore, we propose that both the mother and neonate are exposed to higher oxidative stress during cesarean section delivery.
Cesarean Section ; Female ; Fetal Blood* ; Humans ; Infant, Newborn ; Lipid Peroxidation ; Mothers ; Oxidative Stress* ; Oxygen ; Pregnancy ; Umbilical Cord*

Gitelman's syndrome is a heritable renal disorder characterized by hypomagnesemia, hypokalemia and hypocalciuria. Interestingly, we have experienced one patient who had chronic hypotension, normal serum magnesium level, normal plasma ionized magnesium level, hypokalemia and hypocalciuria. Immunohistochemistry showed the absence of NCCT staining in renal tissues of the patient. We report the case of atypical Gitelman's syndrome with a brief review of related literature.
Gitelman Syndrome* ; Humans ; Hypokalemia ; Hypotension* ; Immunohistochemistry ; Magnesium ; Plasma

BACKGROUND: Rhabdomyolysis is a serious and potentially lethal condition that can develop from a variety of nontraumatic causes. Recently, there have been several reports about rhabdomyolysis which developed after alcohol abuse, drug intoxication, or vigorous exercise, but no report about all spectrums of nontraumatic rhadomyolyis in Korea. So we undertook this study to evaluate the clinical characteristics and course of nontraumatic rhabdomyolysis in a single medical center. METHODS: Several clinical and laboratory data were collected and analyzed in 68 patients with nontraumatic rhabdomyolyis from January 2002 to December 2003. RESULTS: The patients consisted of 58 males and 10 females with mean age of 48.0 +/- 16.3 years. The etiologic factors of nontraumatic rhabdomyolysis were as follows: 21 cases of alcohol abuse (30.9%), 13 cases of muscle ischemia (19%), 9 cases of shock (13%), 7 cases of infection, 6 cases of drug intoxication, 3 cases of seizure, 2 cases of malignant hyperthermia, 2 cases of diabetic ketoacidosis, 1 case of vigorous exercise, 1 case of CO poisoning, 1 case of hypophosphatemia, and 2 cases of unknown causes. Among the 68 patients, forty four patients (64%) developed acute renal failure and seventeen patients (25%) died of sepsis, acute respiratory distress syndrome, pulmonary edema or underlying diseases exacerbation. Among the 44 patients in acute renal failure, eighteen patients (41%) had oliguria during follow-up period. Thirteen patients of them needed dialysis and sixteen patients of them (36.4%) died. Another developed complications were hepatic dysfunction (34%), disseminated intravascular coagulation (28%), hypocalcemia (28%), acute respiratory distress syndrome (26%), pneumonia (22%), pulmonary edema (21%), hyperphosphatemia (7%) and hyperkalemia (4%). CONCLUSION: Nontraumatic rhabdomyolysis with acute renal failure is a serious and potentially lethal condition. Optimal treatment depends on early recognition and intervention. A high clinical suspicion for the occurrence of rhabdomyolysis in the nontraumatic conditions can lead to quicker recognition and better patient care.
Acute Kidney Injury ; Alcoholism ; Diabetic Ketoacidosis ; Dialysis ; Disseminated Intravascular Coagulation ; Female ; Follow-Up Studies ; Humans ; Hyperkalemia ; Hyperphosphatemia ; Hypocalcemia ; Hypophosphatemia ; Ischemia ; Korea ; Male ; Malignant Hyperthermia ; Oliguria ; Patient Care ; Pneumonia ; Poisoning ; Pulmonary Edema ; Respiratory Distress Syndrome, Adult ; Rhabdomyolysis ; Seizures ; Sepsis ; Shock

Neuroleptic malignant syndrome is a rare, idiosyncratic and potentially lethal side effect that occurs patients receiving neuroleptic drugs. Characteristic sings and symtoms include muscle rigidity, fever, altered consciousness, and autonomic dysfuction. The most common serious complication is rhabdomyolysis, which produces acute myoglobiuric renal failure. We present a case of 32-year-old man in whom had NMS and acute renal failure after he had received a combination of chloropromazine and haloperidol. The patients recorvered after treatment by immediate hydration, diuretics and other conservative measure.
Acute Kidney Injury* ; Adult ; Antipsychotic Agents ; Consciousness ; Diuretics ; Fever ; Haloperidol ; Humans ; Muscle Rigidity ; Neuroleptic Malignant Syndrome* ; Renal Insufficiency ; Rhabdomyolysis

Malignant fibrous histiocytoma(MFH) is a deep-seated pleomorphic sarcoma, which occurs principally as a mass of the extremities, abdominal cavity, or retroperitoneum in adults. However, it only rarely occurs in the chest wall. An 85-year-old man had undeergone excision of a small mass on the right posterior chest wall under local anesthesia 14 months age. However, the lesion did not heal and the mass recurred. He was referred to our hospital after the mass had grown to a size of 10.5x8x4cm with a 3x3cm skin defect. Intraoperative frozen biopsy revealed MFH. An en-bloc wide resection and thin-thickness skin graft from his thigh were performed. Although distant metastasis to the lund developed 14 months later and the patient died 2 months later, there was no local recurrence. Thin-thickness skin graft is a simple method for a wide range skin defect, especially in the old age. He recovered in good condition without any physical disabilities.
Abdominal Cavity ; Adult ; Aged, 80 and over ; Anesthesia, Local ; Biopsy ; Extremities ; Humans ; Neoplasm Metastasis ; Recurrence ; Sarcoma ; Skin ; Thigh ; Thoracic Wall* ; Thorax* ; Transplants

We report a case of cytomegalovirus(CMV) pneumonitis in a 21-year-old woman with systemic lupus erythematosus(SLE). She was diagnosed 3 years ago with lupus nephropathy and was on immunosuppressive therapy with prednisolone and cyclophosphamide. She developed dyspnea with fever and hypoxemia. Chest X-ray, and HRCT showed interstitial pneumonitis. We performed open lung biopsy. The diagnosis of CMV pneumonitis was made by the virus culture and immunohistochemical staining for CMV in lung tissue. Despite the admini stration of ganciclovir and high dose immunoglobulin therapy and assistance of mechanical ventilator, the patient died.
Anoxia ; Biopsy ; Cyclophosphamide ; Cytomegalovirus* ; Diagnosis ; Dyspnea ; Female ; Fever ; Ganciclovir ; Humans ; Immunization, Passive ; Lung ; Lung Diseases, Interstitial ; Lupus Erythematosus, Systemic* ; Pneumonia* ; Prednisolone ; Thorax ; Ventilators, Mechanical ; Young Adult