Risk factors of poor early prognosis in the treatment of COVID-19 with nematevir and ritonavir tablets and the establishment of prediction model / 药学实践杂志

Objective To explore risk factors of poor early prognosis in the treatment of COVID-19 by nematevir and ritonavir tablets Paxlovid and establish the prediction model to provide reference for improving the effect of such patients. Methods 92 inpatients of COVID-19 treated with Paxlovid in three military tertiary hospital in southern Fujian from January 2023 to March 2023 were retrospectively analyzed. The clinical indicators of 92 inpatients were collected for univariate and multivariate analysis by single factor and multiple factors and the independent risk factors of poor early prognosis in Paxlovid were screened out. Logistic model equation was transformed to construct the combined predictors, and ROC curve was used to determine the area under the curve (AUC) and the optimal critical value of the combined predictors. Results Among 92 patients, 31 (33.70%) developed poor early prognosis, including 11 deaths (35.48%), 17 critical cases (54.84%) and 3 severe cases (9.68%). Multi-factor Logistic regression analysis showed that the disease days, lymphocyte count, aspartate aminotransferase(AST), C reactive protein(CRP) and ventilator-assisted ventilation were independent risk factors for poor early prognosis in Paxlovid. A formula for calculating the combined predictors (Y) was established as Ycombinedpredictors=7.875Xdisease days+126.188Xlymphocyte count+1.438XAST+XCRP+220.500Xventilator-assisted ventilation based on the above independent risk factors, and the ROC curve was drawn. With the maximum area under the ROC curve of the combined predictors being 0.939, the prediction value was best, and the optimal critical value of the ROC curve corresponding to the maximum Youden index (0.756) was 447.920.Theoretical accuracy of the model was 89.10%. Conclusion The disease days, lymphocyte count, AST, CRP and ventilator-assisted ventilation were independent risk factors for poor early prognosis in Paxlovid. Combined predictors could be calculated by the above risk factors before medication. The efficiency should be improved by taking more active treatment, including combining with other anti-COVID-19 drugs when the prediction result exceeds 447.920.

New strategies for the treatment of carcinoma of unknown primary / 中华肿瘤杂志

Carcinoma of unknown primary (CUP) is a kind of metastatic tumor whose primary origin cannot be identified after adequate examination and evaluation. The main treatment modality of CUP is empiric chemotherapy, and the median overall survival time is less than 1 year. Compared with immunohistochemistry, novel method based on gene expression profiling have improved the sensitivity and specificity of CUP detection, but its guiding value for treatment is still controversial. The approval of immune checkpoint inhibitors and pan-cancer antitumor agents has improved the prognosis of patients with CUP, and targeted therapy and immunotherapy based on specific molecular characteristics are the main directions of future research. Given the high heterogeneity and unique clinicopathological characteristics of CUP, "basket trial" is more suitable for clinical trial design in CUP.

COVID 19 INFECTION IN A PATIENT NEWLY DIAGNOSED WITH HIV/TB COINFECTION – A CASE REPORT

HIV/TB coinfection, COVID 19 with HIV/TB, immune reconstitution inflammatory syndrome (IRIS), TB lymphadenopathy

Predictive factors for poor prognosis of young patients with upper gastrointestinal bleeding in emergency department / 中华急诊医学杂志

Objective:To investigate the prognostic risk factors of young patients with upper gastrointestinal bleeding (UGIB) in emergency department (ED), so as to improve the efficiency of emergency treatment and diversion of these patients.Methods:A retrospective analysis was performed on the clinical data of young patients with UGIB in the ED of Hainan Provincial People's Hospital from January 1, 2019 to December 30, 2020. In-hospital mortality was the primary endpoint of the study, and admission to the Intensive Care Unit (ICU) and length of hospital stay were the secondary endpoints. Inclusion criteria: (1) patients met the diagnostic criteria of acute UGIB; (2) age ranged from 18 to 40 years old; and (3) complete clinical data. Exclusion criteria: (1) bleeding and hemoptysis from the mouth, nose and throat; (2) gastrointestinal bleeding occurred in hospital; (3) lower gastrointestinal bleeding; (4) incomplete clinical data.Results:Among the 383 patients, 268 (70.0%) underwent upper gastrointestinal endoscopy, and the most frequent endoscopic diagnoses were duodenal ulcer (64.6%) and esophageal-gastric varices bleeding (16.8%). Seventy-one (18.5%) patients required endoscopic treatment, 5 (1.3%) patients required surgical treatment, and 7 (1.8%) patients required intervention treatment. The mortality rate was 2.1%, the ICU admission rate was 2.3%, and the length of hospital stay was 5 (3, 6) d. The ICU admission rate and mortality rate were significantly higher in patients with liver disease and in patients with syncope/coma (all P<0.05). Patients with thrombocyte levels (<120×10 9/L) had a significantly longer length of hospital stay than that of patients with normal platelets [8 (5, 11) d vs. 4 (3, 6) d, P<0.001]. The dead patients had significantly higher white blood cell count, urea nitrogen, creatinine, aspartate aminotransferase, alanine aminotransferase and activated partial thrombin time levels (all P<0.05), and significantly lower hemoglobin, albumin, SpO 2 and Glasgow coma score (GCS) levels (all P<0.05). Low GCS was an independent risk factor of ICU admission ( OR=33.973, 95% CI: 1.582~729.417, P=0.024) and mortality ( OR=20.583, 95% CI: 1.368~309.758, P=0.029). Conclusions:The poor prognostic factors of young patients with UGIB in ED are concomitant liver disease, syncope/coma, co-infection, hyperazotemia, impaired kidney function, liver dysfunction, coagulopathy, anemia, and low SpO 2, low GCS, and low hypoproteinemia on admission.

Significance of lysophosphatidic acid receptor 6 in the large-cell transformation of mycosis fungoides and its effect on the proliferation and apoptosis of cutaneous T-cell lymphoma cells / 中华皮肤科杂志

Objective:To determine lysophosphatidic acid receptor 6 (LPAR6) expression in patients with mycosis fungoides (MF) , a variant of cutaneous T-cell lymphoma (CTCL) , and to investigate its role and mechanism of action in the development and prognosis of CTCL.Methods:A total of 110 patients with confirmed MF were collected from Department of Dermatology, Peking University First Hospital from 2011 to 2020, including 24 with large-cell transformation (LCT) and 25 with non-large cell transformation (NLCT) in the discovery cohort, and 24 with LCT and 37 with NLCT in the validation cohort. RNA sequencing and RT-PCR were conducted to determine the LPAR6 expression in patients in the discovery cohort and validation cohort respectively. LPAR6 expression was compared between patients with LCT and those with NLCT, and its effect on the prognosis of patients was evaluated. Two LPAR6-overexpressing CTCL cell lines MyLa and Sz4 were constructed to evaluate the effect of LPAR6 overexpression on proliferative activity of MyLa and Sz4 cells, with the cells normally expressing LPAR6 as the control group; after the treatment with LPAR6-related ligand lysophosphatidic acid (LPA) , 2S-OMPT, adenosine triphosphate (ATP) or adenosine (ADO) , the effects of LPAR6 activation on the proliferative activity and apoptosis of LPAR6-overexpressing MyLa and Sz4 cells were evaluated by the MTS method and flow cytometry respectively. Log-rank test was used for prognostic analysis, and t test or Mann-Whitney U test was used for comparisons between two groups. Results:As RNA sequencing showed, LPAR6 was one of the significantly underexpressed genes in the LCT group in the discovery cohort; in the validation cohort, LPAR6 expression (median[ Q1, Q3]) was significantly lower in the LCT group (204.90[81.90, 512.70]) than in the NLCT group (809.40[417.50, 1 829.20], U= 242.00, P= 0.002) ; in the two cohorts, the underexpression of LPAR6 was significantly associated with increased risk of poor prognosis (both P < 0.01) . Cell proliferation assay showed no significant difference in the proliferative activity of MyLa or Sz4 cells between the LPAR6 overexpression group and control group at 0, 24, 48 and 72 hours during the experiment (all P > 0.05) ; 48 hours after activation of LPAR6 by LPA, 2S-OMPT, ATP and ADO in MyLa cells, the LPAR6 overexpression group showed significantly decreased cellular proliferative activity (1.38 ± 0.01, 1.04 ± 0.01, 1.09 ± 0.03, 1.23 ± 0.01, respectively) compared the control group (1.73 ± 0.04, 1.23 ± 0.01, 1.24 ± 0.01, 1.42 ± 0.03, t= 30.33, 18.38, 4.78, 5.75, respectively, all P < 0.05) , but significantly increased cell apoptosis rate (17.93% ± 0.88%, 17.75% ± 0.35%, 23.97% ± 0.57%, 31.44% ± 0.34%, respectively) compared the control group (3.98% ± 0.03%, 7.81% ± 0.59%, 11.95% ± 0.85%, 12.02% ± 0.48%, t= 15.93, 14.49, 11.74, 33.01, respectively, all P < 0.05) ; 48 hours after activation of LPAR6 by 2S-OMPT and ADO in Sz4 cells, compared with the control group, the LPAR6 overexpression group also showed significantly decreased cellular proliferative activity (2S-OMPT: 1.29 ± 0.04 vs. 1.48 ± 0.01; ADO: 1.27 ± 0.01 vs. 1.51 ± 0.02; both P < 0.05) , but significantly increased cell apoptosis rate (2S-OMPT: 41.70% ± 0.70% vs. 29.35% ± 0.55%; ADO: 37.05% ± 0.15% vs. 24.60% ± 1.00%; both P < 0.05) . Conclusions:LPAR6 was underexpressed in the patients with LCT, and its underexpression was significantly associated with increased risk of poor prognosis. In vitro activation of LPAR6 could inhibit the proliferation of CTCL cells and promote their apoptosis, suggesting that the decrease of LPAR6 expression may be one of the important mechanisms underlying disease progression in patients with LCT.

Expression of CDC45 in hepatocellular carcinoma and its correlation with prognosis / 国际生物医学工程杂志

Objective:To analyze the expression of cell differentiation cyclin 45 (CDC45) in hepatocellular carcinoma (HCC) tissues, to explore the relationship between CDC45 and the prognosis of HCC, and to explore the possibility of CDC45 as a potential biomarker and therapeutic target for HCC.Methods:Bioinformatics methods were used to analyze the mRNA levels of CDC45 in HCC tissues and normal tissues, and to analyze the relationship between its expression and the survival rate of HCC patients. The clinicopathological data of 90 patients with HCC undergoing surgical treatment were retrospectively analyzed. Immunohistochemical method was used to detect the expression level of CDC45 protein in HCC tissue and normal tissues, and to analyze its relationship with clinicopathological characteristics of patients with HCC.Results:CDC45 is highly expressed in colorectal cancer, breast cancer, prostate cancer, lung cancer, liver cancer, etc. The expression of CDC45 in HCC tissues was higher than that in normal tissues adjacent to cancer ( P<0.05). The expression of CDC45 is significantly related to the clinical prognosis of HCC patients ( P<0.001), and the higher the expression of CDC45, the lower the disease-free survival rate and overall survival rate of HCC patients. CDC45 level is significantly correlated with the number of tumor nodules ( P=0.003) and tumor size ( P=0.003) in HCC patients, but the correlation between CDC45 level and age, gender and alpha-fetoprotein (AFP) levels is not statistically significant (all P>0.05). Conclusions:CDC45 plays an important role in the progression of HCC and is expected to become a therapeutic target and potential biomarker for HCC.

Analysis of related factors of poor prognosis in children with parenteral nutrition-associated cholestasis / 中华危重病急救医学

Objective:To explore the related factors affecting the prognosis of children with parenteral nutrition-associated cholestasis (PNAC).Methods:Twenty children with PNAC admitted to Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science and Technology from January 2014 to December 2020 were selected as research objects by retrospective study. According to prognosis, children were divided into good (15 cases) and poor prognosis group (5 cases). Clinical data such as general condition, intravenous nutrition duration, related biochemical examination indexes and main treatment methods of children in the two groups were collected. Spearman correlation analysis was used to quantify the correlation between alanine aminotransferase (ALT) and poor prognosis. Univariate analysis was used to analyze the risk factors affecting the prognosis of children with PNAC, and receiver operating characteristic curve (ROC curve) was drawn to evaluate the predictive value of ALT on the prognosis of children.Results:There were no significant differences in gender, body weight, gestational age, age, feeding mode, duration of intravenous nutrition, direct bilirubin (DBil), aspartate aminotransferase (AST), γ-glutamyltranspeptidase (GGT), total protein (TP), serum albumin (Alb), globulin (GLB), alkaline phosphatase (ALP), platelet count (PLT), white blood cell count (WBC), red blood cell count (RBC), hemoglobin (Hb), lymphocyte count (LYM), urine culture, AST/PLT ratio (APRI) and main treatment methods between the two groups. Total bilirubin (TBil), ALT, neutrophil count (NEU) and monocyte count (MONO) in the good prognosis group were significantly lower than those in the poor prognosis group [TBil (μmol/L): 120.00±48.63 vs. 175.26±29.14, ALT (U/L): 73.25±44.29 vs. 145.30±74.33, NEU (×10 9/L): 2.55±1.29 vs. 5.08±4.10, MONO (×10 9/L): 1.23±0.87 vs. 2.13±0.60, all P < 0.05]. Logistic regression analysis showed that ALT was the risk factor affecting the prognosis of children with PNAC, when ALT increased by 1 U/L, the probability of poor prognosis increased by 3.6% [odds ratio ( OR) = 1.04, 95% confidence interval (95% CI) was 1.00-1.07, P = 0.04]. Spearman correlation analysis showed that the incidence of poor prognosis was positively correlated with ALT ( r = 0.49, P = 0.03). ROC analysis showed that ALT had certain predictive value for the prognosis of children with PNAC [area under ROC cure (AUC) = 0.83, 95% CI was 0.00-1.00, P = 0.03]; when the cut-off value was 121.50 U/L, its sensitivity was 80% and specificity was 93%, suggesting that ALT could be used as the main indicator for clinical prediction of poor prognosis for PNAC. Conclusion:ALT is an independent risk factor of poor prognosis in children with PNAC.

Age-related gene expression alterations by SARS-CoV-2 infection contribute to poor prognosis in elderly

The ongoing pandemic caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has affected millions of people worldwide and with notable heterogeneity in its clinical presentation. Probability of contracting this highly contagious infection is similar across age groups but disease severity and fatality among aged patients with or without comorbidities are reportedly higher. Previous studies suggest that age associated transcriptional changes in lung and immune system results in a proinflammatory state and increased susceptibility to infectious lung diseases. Similarly, SARS-CoV-2 infection could augment ageing-related gene expression alterations resulting in severe outcomes in elderly patients. To identify genes that can potentially increase covid-19 disease severity in ageing people, we compared age associated gene expression changes with disease-associated expression changes in lung/BALF and whole blood obtained from publicly available data. We observed (i) a significant overlap of gene expression profiles of patients’ BALF and blood with lung and blood of the healthy group, respectively; (ii) a more pronounced overlap in blood compared to lung; and (iii) a similar overlap between host genes interacting with SARS-CoV-2 and ageing blood transcriptome. Pathway enrichment analysis of overlapping gene sets suggest that infection alters expression of genes already dysregulated in the elderly, which together may lead to poor prognosis. eQTLs in these genes may also confer poor outcome in young patients worsening with age and comorbidities. Further, the pronounced overlap observed in blood may explain clinical symptoms including blood clots, strokes, heart attack, multi-organ failure etc. in severe cases. This model based on a limited patient dataset seems robust and holds promise for testing larger tissue specific datasets from patients with varied severity and across populations

A case report of 29 year old male patient with breast carcinoma: diagnosed on fine needle aspiration cytology

Breast cancer has been considered a female dominated disease. Carcinoma of male breast is a rare disease representing 1% of all breast cancers and less than 1 % of all cancers in men. The mean age at presentation is mainly in sixties. We here present a case of male breast cancer presented at very young age of 29 years, diagnosed on fine needle aspiration which was confirmed later on histopathological examination.

Case report: A case of pericardial effusion presenting to primary care

This is a rare but interesting case of Mr. MZ who had a lifethreatening pericardial effusion presenting to the primary care clinic. Through great clinical acumen and prudent targeted investigations, diagnosis of this rare condition was reached and urgent referral made to the cardiology team which performed an emergency pericardiocentesis which proved to be life -saving. However, the hovering poor prognosis of Mr. MZ may prompt for a referral to the palliative care team to provide quality end of life care for this unfortunate patient.

Risk factors for poor prognosis of severe adenovirus pneumonia in children / 中国感染控制杂志

Objective To understand the risk factors for poor prognosis of severe adenovirus pneumonia (SAP)in children,and provide guidance for clinical diagnosis and prognosis.Methods Clinical data of 91 hospitalized children who diagnosed with SAP in Chongqing Children’s Hospital of Chongqing Medical University between January 2012 and January 2014 were analyzed retrospectively.Results Of 91 SAP children,23 (25.27%)had poor prognosis. Univariate analysis showed that risk factors for poor prognosis of SAP were age of onset,congenital heart disease and other serious underlying diseases,mechanical ventilation therapy,acute respiratory distress syndrome (ARDS), atelectasis and other serious radiological changes,and emergence of two and more extra-pulmonary complications (all P <0.05).Multivariate regression analysis showed that congenital heart disease and other serious underlying diseases,and emergence of two and more extra-pulmonary complications were independent risk factors for poor prognosis of SAP (all P <0.05).Conclusion Congenital heart disease and other serious underlying diseases,emer-gence of two and more extra-pulmonary complications are independent risk factors for poor prognosis of SAP,active intervention should be conducted during the process of clinical diagnosis and treatment,so as to improve the prognosis.

Atypical carcinoid of the esophagus.

Occurrence of carcinoid tumors in the esophagus is exceedingly rare. We present a case of an atypical carcinoid in the mid esophagus in a 56-year-old male, presenting with dysphagia. Esophagectomy was performed followed by postoperative chemotherapy. Histopathological and immunohistochemical studies were carried out. The patient succumbed to liver and lung metastasis, 6 months after the initial diagnosis, highlighting the poor prognosis of the condition.

Significance of CTEN(C-terminal tensin-like) in invasive breast carcinoma / 中国免疫学杂志

Objective:To explore C-terminal tensin-like(CTEN) gene in breast cancer tissue and its significance. Methods:Explore the prevalence and clinical significance of CTEN expression in invasive breast cancer cases ( n = 1409 ) using immunohistochemistry and tissue microarray. Results: The staining pattern for CTEN in breast tumour cells was homogenous cytoplasmic staining. Moderate to strong cytoplasmic immunoreactivity for CTEN was observed in 90% of the studied cases(1268 cases) . CTEN expression was significantly associated with poor prognostic variables including larger tumour size(χ2=4. 254,P=0. 044), higher histological grade(χ2=7. 555,P=0. 019),axillary nodal involvement(χ2=5. 842,P=0. 035),poor Nottingham Prognostic Index (χ2=7. 578,P=0. 016),Local recurrence(χ2=5. 211,P=0. 039),Regional recurrence(χ2=4. 778,P=0. 042),Distant metastases (χ2=4. 780,P=0. 041) and Histological type(χ2=7. 634,P=0. 012). Significant associations were observed between increased CTEN expression and up-regulation of phosphorylated-Akt(P-Akt)(χ2=27. 23,P<0. 001),Phosphoinositide 3 kinase(PIK3)(χ2=37. 22,P<0. 001) and N-cadherin proteins(χ2=26. 91,P<0. 001). Kaplan-Meier survival analysis demonstrated that patients with high CTEN ex-pression had significantly shorter Breast Cancer Specific Survival(P=0. 004) and Metastasis-Free Survival(P=0. 041) than those with low-CTEN expression. Multivariate analysis showed that CTEN was not an independent prognostic marker in breast cancer. Conclusion:CTEN expression increase is a cancer gene,its association with poor prognostic parameters,and the expression of CTEN associated with poor prognosis of breast cancer parameters.

Risk factors influencing short - term prognosis of acute central nervous system viral infection in children / 中华实用儿科临床杂志

Objective To study the risk factors for children with acute central nervous system(CNS)viral in-fection,so that pediatrician may identify children with poor prognosis at early stages of the disease,and provide them with a theoretical basis for clinical treatment. Methods The clinical data of a cohort patients of acute CNS viral infec-tion who were hospitalized at the First Affiliated Hospital of Fujian Medical University between January 2010 and June 2013 were retrospectively collected and analyzed. According to Glasgow outcome scale on discharge,children were di-vided into good prognosis group and poor prognosis group. Clinical data and outcomes were analyzed by using univariate analysis and binary Logistic regression multivariate analysis. Results Three hundred and one cases were enrolled,278 (92. 36% )patients were assigned to the good prognosis group,and 23(7. 64% )patients were assigned to the poor prognosis group. By univariate analysis,the patients in the poor prognosis group had longer duration of sickness before admission,longer time of fever,lower white blood cell count in cerebrospinal fluid,a relatively lower calcium level,con-scious disturbance at the early stage,multiple seizures,convulsive status epilepticus,meningeal irritation sign,muscle weakness,severe changes in electroencephalogram(EEG),and abnormal neuroimaging findings(computed tomography or magnetic resonance imaging,or both)had significant differences between the good prognosis group and the poor short - term outcome groups(all P < 0. 05). By binary Logistic regression multivariate analysis,factors indicating a poor prognosis during the early stage were conscious disturbance at the early stage(0R = 4. 885,95% CI:1. 523 - 15. 670, P = 0. 008),multiple seizures(0R = 6. 352,95% CI:1. 905 - 21. 178,P = 0. 003),severe changes in EEG( 0R =4. 269,95% CI:1. 708 - 10. 666,P = 0. 002),and abnormal neuroimaging findings( 0R = 9. 740,95% CI:2. 360 -40. 192,P = 0. 002). Conclusions Conscious disturbance at the early stage,multiple seizures,severe changes in EEG and abnormal neuroimaging findings are risk factors for acute viral infection of CNS in children.

Gastric Carcinoma with Bone Marrow Metastasis: A Case Series

Gastric cancer is a major cause of cancer-related mortality. At the time of diagnosis, majority of the patients usually have unresectable or metastatic disease. The most common sites of metastases are the liver and the peritoneum, but in the advanced stages, there may be metastases to any region of the body. Bone marrow is an important metastatic site for solid tumors, and the prognosis in such cases is poor. In gastric cancer cases, bone marrow metastasis is usually observed in younger patients and in those with poorly differentiated tumors. Prognosis is worsened owing to the poor histomorphology as well as the occurrence of pancytopenia. The effect of standard chemotherapy is unknown, as survival is limited to a few weeks. This report aimed to evaluate 5 gastric cancer patients with bone marrow metastases to emphasize the importance of this condition.

Púrpura de Henoch-Schõnlein em adulto / Henoch-Schõnlein purpura in adults

A púrpura de Henoch-Schõnlein é uma vasculite de pequenos vasos, mais comum em crianças, com acometimento cutâneo, articular, gastrointestinal e renal. É raro o acometimento em adultos, conferindo nestes pior prognóstico. Relatamos o caso de um paciente de 54 anos com púrpura de Henoch-Schõnlein com acometimento de pele, articulação, abdominal e renal, sem resposta clínica com altas doses de prednisona, mantendo quadro de claudicação abdominal e proteinúria nefrótica.

Henoch-Schõnlein purpura is a vasculitis of small vessels, more common in children with involvement cutaneous, articular, gastrointestinal and renal. It rarely affects adults, giving these worse prognosis. We report a patient aged 54 with Henoch-Schõnlein purpura with involvement of skin, joint, abdominal and renal, without clinical response with high doses of prednisone keeping a lameness abdominal and nephrotic proteinuria.

Allogeneic bone marrow transplantation for severe aplastic anemia patients with risk factors for poor prognosis: is fludarabine a requirement? / Transplante alogênico de medula óssea em portadores de anemia aplásica severa com fatores de mau prognóstico: é necessário fludarabina?

Hematopoietic progenitor cell transplantation from HLA-identical sibling donors cures 70-90 percent of Severe Aplastic Anemia (sAA) patients. Older age, heavy exposure to transfusions, immunosuppression treatment (IST) with a long interval from diagnosis to transplant and infection at procedure are associated with poor outcomes. We transplanted 18 patients with sAA and at least one risk factor (RF) for poor prognosis (age >35 years, >50 transfusions prior to transplant, unresponsiveness to previous IST and bacterial or fungal infection at transplant) from 2001 to 2005, using cyclophosphamide (CY - 5 patients) or busulfan plus CY (13 patients). Sixteen patients engrafted, two died with no engraftment, three patients had evidence of graft failure at days +67, +524 and +638 (two died and one was rescued with IST). Grade III/IV mucositis occurred in 39 percent but neither aGVHD nor cGVHD were observed. The Kaplan-Meier probability of survival was 75 percent at 2.14 years, with a trend favoring survival by number of RF (1 versus =2 RF) (P = 0.06). These results are comparable to recent data reported with fludarabine-based conditioning in patients with poor prognosis sAA. Due to the small sample size, prospective clinical trials with larger cohorts of patients are needed to confirm the real benefits of fludarabine-based conditioning, and also to define the best agent(s) to be associated with Fludarabine as preparative regimen for sAA patients with poor prognosis.

Transplante de medula óssea de doador irmão HLA-idêntico pode curar 70 por cento-90 por cento dos portadores de anemia aplásica severa (AAs). Pacientes mais idosos, muito transfundidos, longamente tratados com imunossupressão (IS) e com infecções ao tempo do transplante têm pior evolução. Nós transplantamos 18 pacientes com AAs e pelo menos um dos fatores associados a pior prognóstico (idade >35 anos, >50 transfusões antes do transplante, falta de resposta à imunossupressão prévia e infecção bacteriana ou fúngica ao transplante) de 2001 a 2005, com ciclofosfamida (Cy - 5 pacientes) ou bussulfan mais Cy (13 pacientes). Dezesseis pacientes apresentaram pega do enxerto, dois morreram sem pega, três tiveram rejeição aos dias +67, +524 e +638 (dois morreram e um foi resgatado com IS). Mucosite grau III/IV ocorreu em 39 por cento e não observamos DECH aguda ou crônica. A probabilidade de sobrevida pelo método de Kaplan-Meier foi de 75 por cento aos 2,14 anos, e uma tendência a melhor sobrevida foi encontrada entre os portadores de apenas um fator de risco ao transplante (P: .06). Estes resultados são comparáveis a recentes relatos de literatura envolvendo condicionamentos baseados em fludarabina para tratar pacientes com alto risco. Devido à pequena amostra analisada, estudos clínicos prospectivos com maior número de pacientes são necessários, visando comprovar o real benefício dos condicionamentos baseados em fludarabina, definir o melhor agente a ser a ela associado e assim obter o melhor condicionamento para portadores de AAs com fatores de mau prognóstico para o transplante.

Tetrasomy 8 in a Patient with Acute Monoblastic Leukemia / 대한진단검사의학회지

Trisomy 8 is one of the most frequent numerical chromosomal abnormalities observed in hematological malignancies, whereas tetrasomy 8 is a clonal aberration seen mainly in myeloid disorders such as acute myelod leukemia (AML) and myelodysplastic syndromes. In contrast to trisomy 8, tetrasomy 8 is a rare chromosomal aberration, in that only 17 reported AML cases with isolated tetrasomy 8 have been documented. Interestingly, the majority of reported cases were associated with monocytic-lineage leukemias. According to recent reports, tetrasomy 8 is regarded as a poor prognostic factor, and most patients having this abnormality relapsed and died within 1 yr. Here, we report a patient with acute monoblastic leukemia having tetrasomy 8 and a very aggressive disease course.

Clinical Outcome of Multicystic Dysplastic Kidney in 46 Children

PURPOSE: Conservative management of multicystic dysplastic kidney(MCDK) without nephrectomy has recently been advocated. The purpose of this study was to determine the clinical course of conservatively managed MCDK and to find out possible predictive factors for involution of MCDK by ultrasonography(US). METHODS: A retrospective analysis was made on 46 patients(26 boys and 20 girls) in whom MCDK was detected and had been traced by US between Dec. 1993 and Aug. 2005 at Severance Hospital. RESULTS: Median follow-up time was 30 months(range 2-102 months). All patients underwent radionuclide scans and voiding cystourethrograms. The serial follow-up US showed complete involution in 11(24%), partial involution in 19(41%), and no interval change or increased in cyst size in 13(28%) patients. Nephrectomy was done in 3 patients(7%) due to relapsing urinary tract infection(UTI) and severe abdominal distension. The mean age of complete involution of MCDK was 37 months(range 12-84 months). Episodes of UTI were present in 17 patients(37%) and additional genitourinary(GU) abnormalities were found in 22 patients(44%). Hypertension and renal insufficiency was complicated in one patient. No child developed malignant tumor. Univariate analysis showed that five variables were associated with complete involution of the MCDK; gender, site, UTI episode, additional GU abnormalities, and renal length on initial US. After adjusting using the Pearson model, the presence of additional GU abnormalities was exclusively associated with complete involution among the 5 variables(P=0.034). CONCLUSION: In our review of 46 cases of MCDK, non-surgical approach for patients with MCDK was advisable and we could predict poor prognosis when MCDK is associated with other GU anomalies.

Intracranial Atypical Teratoid/Rhabdoid Tumor: Retrospective Analysis in a Single Institution / 대한소아혈액종양학회지

PURPOSE: Intracranial atypical teratoid/rhabdoid tumor (ATRT) is an extremely rare and aggressive tumor of early childhood. In this study, we evaluated the clinical characteristics, therapeutic approaches and outcomes of children who were treated at Seoul National University Children's Hospital (SNUCH). METHODS: We reviewed the clinical records of seven patients who were diagnosed as ATRT at SNUCH between January 2000 and July 2005. RESULTS: Of the seven patients, three patients were male and four were female. Median age at diagnosis was 13 months ranging from 3 months to 67 months. The tumors occurred in the infratentorial area in six and at multiple sites in one patient. Metastatic disease at diagnosis was present in two patients. One showed cerebrospinal fluid (CSF) dissemination and the other showed bony metastasis. Tumor excision was performed in all patients, and with the exception of two cases that refused further treatment, five patients received postoperative chemotherpay. One patient with CSF dissemination received radiotherapy and intrathecal chemotherapy as well. Of all the patients who received chemotherapy, two patients died during treatment because of tumor progression. The chemotherapy regimen was changed in three patients during treatment because the tumor showed poor response to chemotherapy. The median length of follow-up for five patients receiving chemotherapy was 6 months and the overall survival (OS) and event free survival (EFS) were 33.3% and 0%, respectively. CONCLUSION: Though various therapeutic approaches have led to improved survival in ATRT, the prognosis of ATRT is dismal compared with other brain tumors. A precise pathologic diagnosis is crucial and intensified treatment modalities should be considered according to the extent of tumor. To establish optimal treatment guidelines, a cooperative prospective study is needed and the efficacy of individual regimens should be analyzed.