A multicentre ambispective observational study into the incidence and clinical management of aplastic anaemia in Spain (IMAS study).

Aplastic anemia (AA) is a rare, life-threatening hematological disease, with a poorly defined incidence. As the data available on AA varies substantially worldwide, a multicenter, ambispective, observational study was carried out between 2010 and 2019 to assess the incidence, clinical management and survival of AA at seven Spanish hospitals. The incidence of AA was 2.83 per million inhabitants per year, consistent with that reported previously in Europe, with a median age at diagnosis of 61 years-old (range 12-86), and a similar number of males and females. The initial diagnosis was severe or very severe AA in 55.8% of cases and 93.7% required transfusion. The most frequent first line therapy was anti-thymocyte globulin (ATG) plus cyclosporin A (CsA, 44.2%), followed by other CsA-based regimes (46.3%), with hematopoietic stem cell transplantation an infrequent 1st line therapy. The 6-month response rate was 68.2%, which then increased over a median follow-up of 3.9 years. The 5-year overall survival (5OS) was 73.6%, similar in severe (78.6%) and very severe AA patients (74.6%) but lower in moderate AA (MAA) patients (68.4%). The 5OS was 100% in 0-25 year-old patients but dropping to 58.3% in patients ≥ 60 years-old. At the last contact, 75.8% of the patients were alive. In conclusion, the incidence, characteristics and management of AA in our study are consistent with that reported previously. In terms of survival, although the global long-term OS rate was good, there is room for improvement, particularly in older patients. Finally, what appears to be a worse long-term survival of MAA patients, as reported previously, reinforces the importance of not underestimating this condition when diagnosed as MAA.

Prognostic value of molecular biomarkers in patients with metastatic colorectal cancer: a real-world study

Purpose Outcomes for patients with metastatic colorectal cancer (mCRC) have been improved by the identification of biomarkers predictive and prognostic of clinical outcome. The present retrospective analysis was undertaken to assess the utility of key biomarkers and clinical parameters in predicting outcomes in Spanish patients with mCRC. Methods We retrospectively analyzed tumor samples from a series of patients aged > 18 years with mCRC who were treated at the Hospital General Universitario Gregorio Marañón Spain. Real-time polymerase chain reaction was used to detect KRAS, NRAS, BRAF, and PIK3CA mutations. The key outcome of interest was overall survival (OS). Survival curves were estimated using the Kaplan–Meier method and stratified by the variables of greatest clinical interest. Differences were tested using the log-rank test. Results Median OS in the overall population was 24.4 months. Triple WT patients (WT KRAS, NRAS, and BRAF) and quadruple WT patients (WT KRAS, NRAS, BRAF, and PIK3CA) had significantly better OS than those who did not have triple or quadruple WT tumors. OS was significantly better in patients with left- vs. right-sided tumors, patients with resected primary tumors and metastases vs. those without resection, and patients with isolated hepatic and isolated pulmonary metastases. Conclusions This retrospective, observational study has confirmed the prognostic value of the location and resection status of the primary tumor and metastases in Spanish patients with mCRC. Triple WT status, in particular, was prognostic in this patient population, with PIK3CA adding to the prognostic value in the quadruple WT population (AU)

Prognostic value of molecular biomarkers in patients with metastatic colorectal cancer: a real-world study.

PURPOSE: Outcomes for patients with metastatic colorectal cancer (mCRC) have been improved by the identification of biomarkers predictive and prognostic of clinical outcome. The present retrospective analysis was undertaken to assess the utility of key biomarkers and clinical parameters in predicting outcomes in Spanish patients with mCRC. METHODS: We retrospectively analyzed tumor samples from a series of patients aged > 18 years with mCRC who were treated at the Hospital General Universitario Gregorio Marañón Spain. Real-time polymerase chain reaction was used to detect KRAS, NRAS, BRAF, and PIK3CA mutations. The key outcome of interest was overall survival (OS). Survival curves were estimated using the Kaplan-Meier method and stratified by the variables of greatest clinical interest. Differences were tested using the log-rank test. RESULTS: Median OS in the overall population was 24.4 months. Triple WT patients (WT KRAS, NRAS, and BRAF) and quadruple WT patients (WT KRAS, NRAS, BRAF, and PIK3CA) had significantly better OS than those who did not have triple or quadruple WT tumors. OS was significantly better in patients with left- vs. right-sided tumors, patients with resected primary tumors and metastases vs. those without resection, and patients with isolated hepatic and isolated pulmonary metastases. CONCLUSIONS: This retrospective, observational study has confirmed the prognostic value of the location and resection status of the primary tumor and metastases in Spanish patients with mCRC. Triple WT status, in particular, was prognostic in this patient population, with PIK3CA adding to the prognostic value in the quadruple WT population.

Femoral neck pseudoarthrosis in a polio patient treated with closed reduction and cell therapy.

Poliomyelitis disease affects the anterior horns cells of the spinal cord and certain motor nuclei of the brain stem. Paralysis type is flaccid and asymmetrical and result in muscular imbalance. Due to this, in case of having a hip muscles involvement, degenerative or posttraumatic, total hip arthroplasty is normally contraindicated because of the excessive risk of hip dislocation. In cases of subcapital femoral neck fractures the femoral head vascularization is a main concern, and in cases of neglected fracture with pseudoarthrosis the vascular status to the head must be investigated prior to further decisions. We report the case of a femoral neck fracture non-union after a missed femoral neck fracture in a polio affected leg treated with cannulated screws and percutaneous autologous injection of processed total nuclear cells (TNC) mixed with putty demineralized bone matrix.

Incidence of venous thromboembolism (VTE) in ambulatory pancreatic cancer patients receiving chemotherapy and analysis of Khorana's predictive model

PURPOSE: To evaluate the incidence of venous thromboembolism (VTE) in ambulatory pancreas cancer patients receiving chemotherapy and analyze Khorana's predictive model of chemotherapy-associated thrombosis. METHODS/PATIENTS: We performed a retrospective review to determine the incidence of VTE in the gastrointestinal cancer unit of our center. Between 2008 and 2011, 84 consecutives patients diagnosed with pancreas adenocarcinoma were identified and included in the analysis. Pancreatic neuroendocrine tumors were excluded. RESULTS: Thirty patients experienced VTE (35.7 %) and 66 % of the events were diagnosed during the first 6 months after diagnosis. Khorana's score: 33.3 % of the intermediate category patients developed a venous thromboembolic event and 37.5 % in the high-risk category. CONCLUSIONS: The high incidence of VTE observed in this study is consistent with prior reports. Specific predictive model for chemotherapy-associated thrombosis in pancreatic cancer must be investigated (AU)

No disponible

Incidence of venous thromboembolism (VTE) in ambulatory pancreatic cancer patients receiving chemotherapy and analysis of Khorana's predictive model.

PURPOSE: To evaluate the incidence of venous thromboembolism (VTE) in ambulatory pancreas cancer patients receiving chemotherapy and analyze Khorana's predictive model of chemotherapy-associated thrombosis. METHODS/PATIENTS: We performed a retrospective review to determine the incidence of VTE in the gastrointestinal cancer unit of our center. Between 2008 and 2011, 84 consecutives patients diagnosed with pancreas adenocarcinoma were identified and included in the analysis. Pancreatic neuroendocrine tumors were excluded. RESULTS: Thirty patients experienced VTE (35.7 %) and 66 % of the events were diagnosed during the first 6 months after diagnosis. Khorana's score: 33.3 % of the intermediate category patients developed a venous thromboembolic event and 37.5 % in the high-risk category. CONCLUSIONS: The high incidence of VTE observed in this study is consistent with prior reports. Specific predictive model for chemotherapy-associated thrombosis in pancreatic cancer must be investigated.

Association of creatine kinase and skin toxicity in phase I trials of anticancer agents.

BACKGROUND: We investigated the association between skin rash and plasma creatine kinase (CK) levels in oncology phase I trials. METHODS: We analysed data from 295 patients treated at our institution within 25 phase I trials which included CK measurements in the protocol. Trials involved drugs targeting EGFR/HER2, m-TOR, VEGFR, SRC/ABL, aurora kinase, BRAF/MEK, PARP, CDK, A5B1 integrin, as well as oncolytic viruses and vascular disrupting agents. RESULTS: Creatine kinase measurements were available for 278 patients. The highest levels of plasma CK during the trial were seen among patients with Grade (G) 2/3 rash (median 249 U l(-1)) compared with G1 (median 81 U l(-1)) and no rash (median 55 U l(-1)) (P<0.001). There was a significant reduction in CK after the rash resolved (mean 264.2 vs 100.1; P=0.012) in 25 patients, where serial CK values were available. In vitro exposure of human keratinocytes to EGFR, MEK and a PI3Kinase/m-TOR inhibitor led to the increased expression of CK-brain and not CK-muscle or mitochondrial-CK. CONCLUSION: Plasma CK elevation is associated with development of skin rash caused by novel anticancer agents. This should be studied further to characterise different isoforms as this will change the way we report adverse events in oncology phase I clinical trials.

Prognostic value of carcinoembryonic antigen level in rectal cancer treated with neoadjuvant chemoradiotherapy.

BACKGROUND: The purpose of this study was to identify clinical and pathological parameters to improve prediction of disease-free survival (DFS) and overall survival (OS) in patients treated with neoadjuvant chemoradiotherapy for rectal cancer. METHODS: Between July 1995 and May 2007, 148 patients with primary rectal adenocarcinoma received neoadjuvant chemoradiotherapy followed by mesorectal excision. Preoperative treatment included various protocols, UFT and leucovorin (28%) and oxaliplatin-based chemotherapy (72%). Clinical and pathological variables were evaluated in relation to patient outcomes. RESULTS: Thirteen percent of patients achieved a complete pathologic response. No response or minimal response as defined by Dworak (Tumor Regression Grade 0/1) was observed in 30 patients (20%). At a median follow-up of 37 months, the 3-year DFS and OS were 64% and 83%, respectively. Pre-treatment serum carcinoembryonic antigen (CEA) level

[A solitary cerebellar metastasis: a diagnostic exercise]. / Metástasis cerebelosa solitaria: un ejercicio diagnóstico.

Presentation of a solitary cerebellar mass lead us to think about the presence of a primary tumour or the possibility of a pulmonary, mammary or digestive metastasis. However, once discarded these origins, is necessary to search the primary neoplasms in less frequent organs. Approximately, 5% of all intracranial metastasis have it's origin in a renal cancer. These lesions generally appear at the supratentorial area and within a generalized disease. In the other hand, the presentation of a solitary cerebellar metastasis from a renal carcinoma without affectation of other organs is a very unusual fact. We present a case of these characteristics discussing about diagnostic, therapeutic and prognostic aspects of this unpredictable tumor.

[Vesico-ureteral reflux]. / Reflujo vesico-ureteral.

We review the current problem of vesico-ureteral reflux, commenting upon the etiological factors and the physiopathology of the same. We describe the exploratory methods (analysis, urography, cystourethrography, cystoscopy) and assess the data obtained in order to establish the indication of medical or surgical treatment.