Recent evidence suggests that cell-free plasma DNA has potential use as a prognostic marker in many clinical settings. The aim of the present study was to evaluate the prognostic role of cell-free plasma DNA in the prediction of clinical outcome in intensive treatment unit (ITU) patients. Cell-free plasma DNA was measured by real-time polymerase chain reaction assay for the beta-globin gene and SOFA score, APACHE II score, CRP concentrations, and clinical outcome (duration of stay, ventilation time, and mortality) were noted in 94 patients on admission to the ITU. The median plasma DNA concentration in ITU patients was 5493 GE/mL and this was significantly (P <0.001) higher than the DNA concentration in healthy subjects (1970 GE/mL). DNA concentration demonstrated a significant correlation with serum C-reactive protein (CRP) (r = 0.363) concentration and Sepsis-related Organ Failure Assessment (SOFA) (r = 0.360) score (P <0.001 for both by Pearson correlation) but not with Acute Physiology And Chronic Health Evaluation (APACHE II) score. Patients on ventilation had significantly higher DNA concentrations compared to nonventilated patients (7362 GE/mL versus 4479 GE/mL; P = 0.004). The median DNA concentration in nonsurvivors was 9148 GE/mL, and this was 2.3-fold greater than that in survivors (3921 GE/ml, P <0.001). ROC analysis of the data indicated a sensitivity of 85% and a specificity of 80% when DNA concentration of 6109 GE/mL was taken as a predictor of death. The data suggest that cell-free plasma DNA concentration is potentially useful as a prognostic marker in ITU patients.

Background Efficiently sharing health data produced during standard care could dramatically accelerate progress in cancer treatments, but various barriers make this difficult. Not sharing these data to ensure patient privacy is at the cost of little to no learning from real-world data produced during cancer care. Furthermore, recent research has demonstrated a willingness of patients with cancer to share their treatment experiences to fuel research, despite potential risks to privacy. Objective The objective of this study was to design, pilot, and release a decentralized, scalable, efficient, economical, and secure strategy for the dissemination of deidentified clinical and genomic data with a focus on late-stage cancer. Methods We created and piloted a blockchain-authenticated system to enable secure sharing of deidentified patient data derived from standard of care imaging, genomic testing, and electronic health records (EHRs), called the Cancer Gene Trust (CGT). We prospectively consented and collected data for a pilot cohort (N=18), which we uploaded to the CGT. EHR data were extracted from both a hospital cancer registry and a common data model (CDM) format to identify optimal data extraction and dissemination practices. Specifically, we scored and compared the level of completeness between two EHR data extraction formats against the gold standard source documentation for patients with available data (n=17). Results Although the total completeness scores were greater for the registry reports than those for the CDM, this difference was not statistically significant. We did find that some specific data fields, such as histology site, were better captured using the registry reports, which can be used to improve the continually adapting CDM. In terms of the overall pilot study, we found that CGT enables rapid integration of real-world data of patients with cancer in a more clinically useful time frame. We also developed an open-source Web application to allow users to seamlessly search, browse, explore, and download CGT data. Conclusions Our pilot demonstrates the willingness of patients with cancer to participate in data sharing and how blockchain-enabled structures can maintain relationships between individual data elements while preserving patient privacy, empowering findings by third-party researchers and clinicians. We demonstrate the feasibility of CGT as a framework to share health data trapped in silos to further cancer research. Further studies to optimize data representation, stream, and integrity are required.

We present here the case of a 39-year-old man with metastatic pancreatic carcinoma receiving chemotherapy with the combination of gemcitabine and nabpaclitaxel as part of a clinical trial. Despite an impressive response to therapy, he ultimately developed profound anasarca, renal insufficiency, progressive cytopenias, and malignant hypertension 6 months into his treatment course. The diagnosis of gemcitabine-associated thrombotic microangiopathy (G-TMA) was made based on renal biopsy, and receipt of the anti-C5 monoclonal antibody eculizumab proved successful at reversing his deteriorating clinical course and improving his laboratory parameters. This case illustrates the importance of recognizing this rare but serious complication, and highlights one potential therapeutic option that can be used in the appropriate clinical context.The nucleoside analogue gemcitabine, originally approved by the Food and Drug Administration in 1996 for the treatment of advanced pancreatic cancer, is now used extensively as part of combination chemotherapy regimens for a variety of solid tumor indications, including not only carcinomas of the pancreas but also those of the breast, ovary, lung, and biliary tract (1-5). While stereotypical side effects of gemcitabine (most commonly cytopenias, nausea, flu-like symptoms, myalgias, and rash) are well-recognized and generally manageable with appropriate supportive care measures, other less common toxicities associated with this cytotoxic agent can also contribute to significant morbidity and even mortality (6). However, these rare events may often go unrecognized by treating medical providers and have less well-defined algorithms for appropriate management and treatment. One of the more serious complications, thrombotic microangiopathy (TMA), can be associated with severe kidney injury, dialysis dependence, and death. We present here the case of a patient with metastatic pancreatic cancer receiving gemcitabine-based chemotherapy as part of a clinical trial who developed florid TMA 6 months into treatment. After progressively worsening symptoms, his disease course was successfully reversed with administration of a single dose of the terminal complement inhibitor eculizumab. Case PresentationThe patient was a previously healthy 39 year-old man who presented with progressive left calf pain and swelling which extended proximally up to the knee over the next several months. A Doppler ultrasound showed an extensive deep venous thrombosis involving the proximal to distal left lower extremity including the femoral vein, popliteal vein and peroneal vein, extending into the inferior vena cava and right common femoral vein, for which he was started on low molecular weight heparin (enoxaparin, 1 mg/kg BID dosing).Further diagnostic evaluation included computed tomographic (CT) scans of the chest, abdomen, and pelvis which revealed a 6.9 cm pancreatic tail hypoattenuating mass, innumerable hepatic lesions measuring up to 4.7 cm, and multiple retroperitoneal and peripancreatic lymph nodes up to 1.5 cm...

Authors NA and JC contributed equally to this study and their names have been listed in alphabetical order of their last name.

e19358 Background: Efficiently sharing health data produced during standard care could dramatically accelerate progress in cancer treatments but various barriers make this difficult. Not sharing these data to ensure patient privacy is at the cost of little to no learning from real-world data produced during cancer care. Furthermore, recent research has demonstrated a willingness of cancer patients to share their treatment experiences to fuel research, despite potential risks to privacy. The objective of this study was to design, pilot, and release a decentralized, scalable, efficient, economical, and secure strategy for dissemination of de-identified clinical and genomic data with a focus on late stage cancer. Methods: We created and piloted a blockchain-authenticated system to enable securely sharing de-identified patient data derived from standard of care imaging, genomic testing, and electronic health records (EHR), called the Cancer Gene Trust (CGT). We prospectively consented and collected data for a pilot cohort (n = 18), which we uploaded to CGT. EHR data were extracted from both a hospital cancer registry and a common data model format to identify optimal data extraction and dissemination practices. Specifically, we scored and compared the level of completeness between two EHR data extraction formats against the gold-standard source documentation for patients with available data (n = 17). Results: While the total completeness scores were greater for the registry reports than the common data model, this difference was not statistically significant. We did find that some specific data fields, such as histology site, were better captured using the registry reports, which can be used to improve the continually adapting common data model. In terms of the overall pilot study, we found that CGT enables rapid integration of real-world cancer patient data in a more clinically useful timeframe. We also developed an open-source web application to allow users to seamlessly search, browse, explore, and download CGT data. Conclusions: Our pilot demonstrates the willingness of cancer patients to participate in data sharing and how blockchain-enabled structures can maintain relationships between individual data elements while preserving patient privacy, empowering findings by third party researchers and clinicians. We demonstrate the feasibility of CGT as a framework to share health data trapped in silos to further cancer research. Further studies to optimize data representation, stream, and integrity are required.