Electronic Case Report Form Research Articles

P.399The international spinal muscular atrophy (SMA) registry: longitudinal collection and refinement of outcome measures for spinal muscular atrophy

The international Spinal muscular atrophy consortium (iSMAc) is an academic collaboration linking 3 national networks (Italy, United States, United Kingdom), focused on natural history in infants, children and adults affected by SMA. The three networks, originally funded by their national advocacy groups, have since 2015 contributed to the development of novel and SMA specific outcome measures used in natural history studies, allowing the definition of trajectories of SMA patients. Since 2017, the network has been collaborating with Biogen to capture, using a comprehensive electronic case report form (eCRF), the evolving history of SMA patients. The iSMAc registry is a prospective multicenter natural history study, linking previous retrospective data of the iSMAc network to the prospective data collection from SMA patients, both naïve or treated with nusinersen. Initial efforts focused on identifying data elements and creating a data dictionary. Detailed information on clinical features, supportive care, and functional measures, of hospitalization and other health related issues are captured in standardized eCRFs that allow the assessment of the impact of therapeutic intervention(s) on burden of disease and survival. Data are collected at regular clinical intervals, curated at the network level and entered into a registry database. Regular physiotherapy training sessions ensure quality control of the outcome measures studied. To date, baseline data on 735 SMA patients have been recorded in the registry, including SMA type 1 (182), type 2 (285), type 3 (262) and other forms (6), mostly in the pediatric age range (80%). The majority of patients (64%) are treated with nusinersen. Relevant data is shared on a quarterly basis with Biogen for surveillance of patients receiving nusinersen. Data from the iSMAc registry are contributing to the "real-world" assessment of the contemporary natural history and response to emerging therapies in a broadly based population of patients with SMA.

Methodological description of clinical research data collection through electronic medical records in a center participating in an international multicenter study.

ABSTRACTData collection for clinical research can be difficult, and electronic health record systems can facilitate this process. The aim of this study was to describe and evaluate the secondary use of electronic health records in data collection for an observational clinical study. We used Cerner Millennium®, an electronic health record software, following these steps: (1) data crossing between the study’s case report forms and the electronic health record; (2) development of a manual collection method for data not recorded in Cerner Millennium®; (3) development of a study interface for automatic data collection in the electronic health records; (4) employee training; (5) data quality assessment; and (6) filling out the electronic case report form at the end of the study. Three case report forms were consolidated into the electronic case report form at the end of the study. Researchers performed daily qualitative and quantitative analyses of the data. Data were collected from 94 patients. In the first case report form, 76.5% of variables were obtained electronically, in the second, 95.5%, and in the third, 100%. The daily quality assessment of the whole process showed complete and correct data, widespread employee compliance and minimal interference in their practice. The secondary use of electronic health records is safe and effective, reduces manual labor, and provides data reliability. Anesthetic care and data collection may be done by the same professional.

Linguistic and Cultural Acceptability of a Spanish Translation of the Ohio State University Traumatic Brain Injury Identification Method Among Community-Dwelling Spanish-Dominant Older Adults

ObjectiveOur objective was to (1) evaluate the linguistic and cultural acceptability of a Spanish translation of the Ohio State University traumatic brain injury identification method (OSU TBI-ID) and (2) to assess the usability and acceptability of a tablet-based version of this instrument in a cohort of Spanish-dominant older adults. SettingUniversity clinical research center and local community center. ParticipantsCommunity-dwelling Spanish-dominant adults age 50 years or older without dementia residing in the Bay Area of California (N=22). DesignCross-sectional cohort study. Main Outcome MeasuresQualitative assessment of linguistic or cultural acceptability of a Spanish translation of the OSU TBI-ID as well as usability or acceptability of a tablet-based self-administered version of this instrument. ResultsThe Spanish translation had high linguistic and cultural acceptability and was further optimized based on participant feedback. Cognitive interviews to review survey wording revealed high levels of homogeneity in the clinical definitions and synonyms given by participants—for example, results for the clinical term “Quedó Inconsciente/Pérdida (temporal) de la conciencia” (To be unconscious/[Temporary] loss of consciousness) used in the survey included “perder el conocimiento” (loss of consciousness), “knockeado” (knocked out), “No es que esté dormida, porque está inconsciente, pero su corazón está todavía palpitando” (it’s not that they’re sleeping, because they’re unconscious, but their heart is still palpitating). The tablet interface had low observer-based usability, revealing that participants with <13 years of education (n=6) had more difficulty using the tablet which could be improved with minor changes to the coding of the application and minimal in-person technology support. Acceptability of the tool was low among all but 1 participant. ConclusionThis linguistically optimized Spanish translation of the OSU TBI-ID is recommended for use as a semistructured interview among Spanish-dominant older adults. Although the tablet-based instrument may be used by interviewers as an efficient electronic case report form among older adults, further research is needed, particularly among older adults with varying levels of education, to validate this instrument as a self-administered survey.

Assessment of dialysis initiation by a fuzzy mathematics equation (ADIFE): a study protocol for a randomised controlled trial

IntroductionStarting dialysis early or late both result in a low quality of life and a poor prognosis in patients undergoing haemodialysis. However, there remains no consensus on the optimal timing...

Chronic obstructive pulmonary disease exacerbation episodes derived from electronic health record data validated using clinical trial data

PurposeTo validate an algorithm for acute exacerbations of chronic obstructive pulmonary disease (AECOPD) episodes derived in an electronic health record (EHR) database, against AECOPD episodes collected in a randomized clinical trial using an electronic case report form (eCRF).MethodsWe analyzed two data sources from the Salford Lung Study in COPD: trial eCRF and the Salford Integrated Record, a linked primary‐secondary routine care EHR database of all patients in Salford. For trial participants, AECOPD episodes reported in eCRF were compared with algorithmically derived moderate/severe AECOPD episodes identified in EHR. Episode characteristics (frequency, duration), sensitivity, and positive predictive value (PPV) were calculated. A match between eCRF and EHR episodes was defined as at least 1‐day overlap.ResultsIn the primary effectiveness analysis population (n = 2269), 3791 EHR episodes (mean [SD] length: 15.1 [3.59] days; range: 14‐54) and 4403 moderate/severe AECOPD eCRF episodes (mean length: 13.8 [16.20] days; range: 1‐372) were identified. eCRF episodes exceeding 28 days were usually broken up into shorter episodes in the EHR. Sensitivity was 63.6% and PPV 71.1%, where concordance was defined as at least 1‐day overlap.ConclusionsThe EHR algorithm performance was acceptable, indicating that EHR‐derived AECOPD episodes may provide an efficient, valid method of data collection. Comparing EHR‐derived AECOPD episodes with those collected by eCRF resulted in slightly fewer episodes, and eCRF episodes of extreme lengths were poorly captured in EHR. Analysis of routinely collected EHR data may be reasonable when relative, rather than absolute, rates of AECOPD are relevant for stakeholders' decision making.

Passive enhanced safety surveillance of GSK’s quadrivalent seasonal influenza vaccine in Belgium, Germany and Spain, an observational study: protocol for the 2018/2019 influenza season

IntroductionThe European Medicines Agency requires Marketing Authorisation Holders providing seasonal influenza vaccines in Europe to conduct enhanced safety surveillance accounting for the different age groups based on the vaccine indication,...

Visual and Quantitative Evaluation of Emphysema: A Case-Control Study of 1111 Participants in the Pilot Swedish CArdioPulmonary BioImage Study (SCAPIS)

Emphysema is a hallmark of chronic obstructive pulmonary disease. The primary aim of this study was to investigate inter- and intraobserver agreement of visual assessment of mild emphysema in low-dose multidetector computed tomography of subjects in the pilot SCAPIS in order to certify consistent detection of mild emphysema. The secondary aim was to investigate the performance of quantitative densitometric measurements in the cohort. Participants with emphysema (n = 100, 56 males and 44 females) reported in the electronic case report form of pilot SCAPIS and 100 matched controls (gender, age, height, and weight) without emphysema were included. To assess interobserver variability the randomized examinations were evaluated by two thoracic radiologists. For intraobserver variability three radiologists re-evaluated randomized examinations which they originally evaluated. The results were evaluated statistically by Krippendorff's α. The dataset was also assessed quantitively for % lung attenuation value -950 HU (LAV950), mean lung density and total lung volume by commercially available software. Emphysema was visually scored as mild and Krippendorff's α was ≥0.8 for both the inter- and intraobserver agreement regarding presence of emphysema and approaching 0.8 regarding presence and extent of emphysema by location in the upper lobes. Mean LAV950 was not different between the emphysematous and the nonemphysematous participants; 8.3% and 8.4%, respectively. The inter- and intraobserver agreement for visual detection of mild emphysema in low-dose multidetector computed tomography was good. Surprisingly, quantitative analysis could not reliably identify participants with mild emphysema, which hampers the use of automatic evaluation.

Protocol of the European Registry on the management of Helicobacter pylori infection (Hp‐EuReg)

Helicobacter pylori selectively infects the human stomach, being the most prevalent chronic infection in the world. Hpylori presence causes chronic gastritis in 100% of infected patients and is the major cause of relevant diseases such as atrophic gastritis, peptic ulcer disease, and gastric cancer; it is for this reason that from a public health standpoint, it is considered a high-impact pathogen, responsible of a significant morbidity and mortality. Nowadays, there are consensus and clinical guidelines regarding the infection management at a European level and in most of European countries, but no data have shown the level of implementation of these recommendations. The high costs that this infection carries both socially and to the health system require the continuous and systematic assessment of the diagnostic and treatment strategies, as well as the accessibility to diagnostic methods and most efficient drugs. To register the diagnosis, management strategies, and treatment of Hpylori-infected adult patients in the Digestive Services outpatient clinics throughout Europe. Noninterventionist prospective multicentre international Registry promoted by the European Helicobacter and Microbiota Study Group. National Coordinators will select recruiting gastroenterologists in their country that will register the Hpylori-related routine clinical practice consultations they receive in an electronic case report form (e-CRF) provided by AEG-REDCap. Variables retrieved will include clinical, diagnostic, treatment, eradication confirmation, and outcome data. The database will allow researchers to perform specific subanalyses after approval by the Scientific Committee of the study.

Development of a Duchenne Muscular Dystrophy registry for children in South Africa to optimize care

Background&#x0D; Duchenne Muscular Dystrophy (DMD) is the most prevalent and lethal of the inherited dystrophies. Globally, the incidence is reported at 1 in 3500 live male births. There is currently no cure for the disease. With the possibility of gene therapy becoming available, patients who would qualify for such treatment need to be identified. Further, understanding disease expression in a population is essential to focus targeted interventions, such as corticosteroids, to ensure they are safe and effective in the local setting. By maintaining a DMD specific disease registry this information can be attained.&#x0D; Objective: This report describes the concept and design of the first South African DMD disease registry using Research Electronic Data Capture (REDCap) &#x0D; Methods: The registry was developed using REDCap’s web-based online design, accessed through the Clinical Research Centre (CRC) in the Faculty of Health Sciences at the University of Cape Town. Electronic case report forms were created from these clinical data using REDCap and for specific variables serial entries were possible relating to disease progression. International data standards were adopted as proposed by TREAT-NMD, a global network of registries on DMD to ensure our data is compatible with this and other international registries.&#x0D; Results: Retrospective data entry combined with dynamic prospective recording of data was utilized in this project. Building on an existing database, 100 confirmed DMD boys are currently eligible for inclusion into the registry.&#x0D; As our registry is an on-going study, sequential analysis of accumulated data will be done going forward to review trends on our patients with DMD.&#x0D; &#x0D; Conclusions: This report describes the concept and design of a DMD registry and the steps followed to its establishment with REDCap. The focus is to consolidate clinical and genetic information on South African patients with DMD, commencing with the local centre’s patient cohort but rolling out access to other South African centres to create a national resource, which is internationally relevant. Ideally this template could be duplicated in the conceptualisation of disease registries for other key conditions.&#x0D; &#x0D; &#x0D; &#x0D;

The French glioblastoma biobank (FGB): a national clinicobiological database

BackgroundGlioblastomas (GB) are the most common and lethal primary brain tumors. Significant progress has been made toward identifying potential risk factors for GB and diagnostic and prognostic biomarkers. However, the current standard of care for newly diagnosed GB, the Stupp protocol, has remained unchanged for over a decade. Large-scale translational programs based on a large clinicobiological database are required to improve our understanding of GB biology, potentially facilitating the development of personalized and specifically targeted therapies. With this goal in mind, a well-annotated clinicobiological database housing data and samples from GB patients has been set up in France: the French GB biobank (FGB).MethodsThe biobank contains data and samples from adult GB patients from 24 centers in France providing written informed consent. Clinical and biomaterial data are stored in anonymized certified electronic case report forms. Biological samples (including frozen and formalin-fixed paraffin-embedded tumor tissues, blood samples, and hair) are conserved in certified biological resource centers or tumor tissue banks at each participating center.ResultsClinical data and biological materials have been collected for 1087 GB patients. A complete set of samples (tumor, blood and hair) is available for 66%, and at least one frozen tumor sample is available for 88% of the GB patients.ConclusionsThis large biobank is unique in Europe and can support the large-scale translational projects required to improve GB care. Additional biological materials, such as peritumoral brain zone and fecal samples, will be collected in the future, to respond to research needs.

Nurses' decision-making, practices and perceptions of patient involvement in medication administration in an acute hospital setting.

To describe nurses' decision-making, practices and perceptions of patient involvement in medication administration in acute hospital settings. Medication errors cause unintended harm to patients. Nurses have a major role in ensuring patient safety in medication administration practices in hospital settings. Investigating nurses' medication administration decision-making and practices and their perceptions of patient involvement, may assist in developing interventions by revealing how and when to involve patients during medication administration in hospital. A descriptive exploratory study design. Twenty nurses were recruited from two surgical and two medical wards of a major metropolitan hospital in Australia. Each nurse was observed for 4hr, then interviewed after the observation. Data were collected over six months in 2015. Observations were captured on an electronic case report form; interviews were audio-recorded and transcribed verbatim. Data were analysed using descriptive statistics and content and thematic analysis. Ninety-five medication administration episodes, of between two and eight episodes per nurse, were observed. A total of 56 interruptions occurred with 26 of the interruptions being medication related. Four major themes emerged from the interviews: dealing with uncertainty; facilitating, framing and filtering information; managing interruptions and knowing and involving patients. Nurses work in complex adaptive systems that change moment by moment. Acknowledging and understanding the cognitive workload and complex interactions are necessary to improve patient safety and reduce errors during medication administration. Knowing and involving the patient is an important part of a nurses' medication administration safety strategies.

Abstract P6-17-28: Outcomes of real-world use of eribulin plus trastuzumab for HER2-positive metastatic breast cancer

Abstract Background Eribulin mesylate is approved for the treatment of metastatic breast cancer (mBC) after two prior chemotherapy regimens including an anthracycline or a taxane in either the metastatic or adjuvant setting. Eribulin in combination with trastuzumab (E+T) has demonstrated tolerability and anti-tumor activity in phase I and II trials but is not FDA-approved for the treatment of HER2-positive mBC. Case series and retrospective research have noted the use of E+T in clinical practice. We sought to describe patient characteristics and long-term outcomes of treatment with E+T for HER2-positive mBC patients treated outside of clinical trials in the US. Methods US-based community oncologists from an open network of over 7,000 oncologists, hematologists, and urologists were invited to participate in identifying HER2-positive mBC patients treated with E+T between 01/01/11 and 12/31/13 outside of clinical trials. Data were collected from 03/18/2016 until 09/01/2016. Providers completed an electronic case report form (CRF) by abstracting data on disease characteristics, treatment patterns, disease response (per provider assessment), adverse events (Aes), and date of death. Duration of treatment and overall survival (OS) were calculated from the initiation of the E+T regimen. The target sample size was 60 patients and patients were selected according to resource available for chart data abstraction. Results Twenty-three providers submitted CRFs for 62 total patients. After data collection, 59 of 62 submitted records were validated for analysis. At mBC diagnosis, 69.4% of patients were ER/PR negative and 42.4% of patient had de novo stage IV disease. At initiation of E+T, the median age was 57 years and 81.4% were ECOG-OS 0/1. Mean length of follow-up from the initiation of any therapy was 33.6 months. Twenty-two (37.3%) patients initiated E+T as their first- or second-line of treatment; those remaining were in third-line or greater. At initiation of E+T, 72.8% of patients had prior treatment with trastuzumab in combination with chemotherapy, 25.4% had prior trastuzumab in combination with pertuzumab and chemotherapy, and 16.9% had received TDM-1. Mean duration of E+T treatment was 5.2 months (SD=2.4). A response (complete [CR] or partial [PR]) was recorded by the providers for 64.4% of patients (not independently verified). The most common Aes reported were fatigue (67.8%), weakness (50.8%), decreased appetite (28.8%), decreased hemoglobin (27.1%), peripheral neuropathy (25.4%), and neutropenia (18.6%). At the end of the study period, 34 patients (57.6%) were deceased; the median OS from the initiation of E+T was 23.9 months (95% CI: 17.8-30.4). Conclusions In a small cohort of patients treated with E+T in the community setting, the observed response rate of 64.4% (CR+PR) was comparable to that of a prior phase II trial of E+T which reported an ORR with first-line E+T of 71.2% overall, 77.4% among T-naïve and 61.9% in T-pretreated patients. Further research is warranted to examine the tolerability and efficacy of E+T for metastatic HER2-positive breast cancer patients in different treatment settings. Citation Format: Mougalian SS, Copher R, McAllister L, Radtchenko J, Wang EC, Broscious M, Yu H-T, Kish J. Outcomes of real-world use of eribulin plus trastuzumab for HER2-positive metastatic breast cancer [abstract]. In: Proceedings of the 2018 San Antonio Breast Cancer Symposium; 2018 Dec 4-8; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2019;79(4 Suppl):Abstract nr P6-17-28.

A National Iranian Cochlear Implant Registry (ICIR): cochlear implanted recipient observational study.

Patients who receive cochlear implants (CIs) constitutes a significant population in Iran. This population needs regular monitor on long-term outcomes, educational placement and quality of life. Currently, there is no national or regional registry on the long term outcomes of CI users in Iran. The present study aims to introduce the design and implementation of a national patient-outcomes registry on CI recipients for Iran. This Iranian CI registry (ICIR) provides an integrated framework for data collection and sharing, scientific communication and collaboration inCI research. The national ICIR is a prospective patient-outcomes registry for patients who are implanted in one of Iranian centers. The registry is based on an integrated database that utilizes a secure web-based platform to collect response data from clinicians and patient's proxy via electronic case report forms (e-CRFs) at predefined intervals. The CI candidates are evaluated with a set of standardized and non-standardized questionnaires prior to initial device activation(as baseline variables) and at three-monthly interval follow-up intervals up to 24 months and annually thereafter. The software application of the ICIR registry is designed in a user-friendly graphical interface with different entry fields. The collected data are categorized into four subsets including personal information, clinical data, surgery data and commission results. The main parameters include audiometric performance of patient, device use, patient comorbidities, device use, quality of life and health-related utilities, across different types of CI devices from different manufacturers. The ICIR database could be used by the increasingly growing network of CI centers in Iran. Clinicians, academic and industrial researchers as well as healthcare policy makers could use this database to develop more effective CI devices and better management of the recipients as well as to develop national guidelines.

Spanish national registry of patients undergoing pancreaticoduodenectomy for periampullary tumors: effects of hospital volume on outcomes

Background: Several studies have reported that hospitals with a high volume (HV) of pancreaticoduodenectomies (PD) have better outcomes and lower mortality rates. Currently, no data describe the situation in Spain. The aim of this study is to assess the effect of hospital volume on outcome after PD in a real situation of clinical practice. Methods: The Spanish National Registry of PD is a multicenter, web-based registry, which is open to any institution performing PD during 2015. Demographic, perioperative data, and final follow-up were recorded in an electronic Case Report Form. Patients were stratified by annual pancreatic resection volume (low volumen (LV)< 9, medium volumen 10–25, high > 25). Results: 1.016 patients undergoing PD from 74 hospitals in Spain were registered. Thirty-day mortality was 6.5%. 55% experienced complications. Patients > 70 years were more common at LV hospitals compared with HV hospitals (P = 0.02). HV centers had more vascular resections (P < 0.001), lower mortality (P = 0.007) and a shorter median length of stay (P = 0.01). Total number of examined LN and tumor size were also significantly higher (p < 0.001). There was no significant difference for radicality of the resection, comorbidities, and complications after surgery. Conclusion: The present study is the first nationwide population-based study to assess the effect of hospital volume on outcome after PD in Spain. Our data suggest that increasing operative volume in DP is associated with significantly better outcomes.

Epidemiology and Microbiology of Skin and Soft Tissue Infections: Preliminary Results of a National Registry

Skin and soft tissue infections (SSTIs) represent a wide range of clinical conditions characterized by a considerable variety of clinical presentations and severity. Their aetiology can also vary, with numerous possible causative pathogens. While other authors previously published analyses on several types of SSTI and on restricted types of patients, we conducted a large nationwide surveillance programme on behalf of the Italian Society of Infectious and Tropical Diseases to assess the clinical and microbiological characteristics of the whole SSTI spectrum, from mild to severe life-threatening infections, in both inpatients and outpatients. Twenty-five Infectious Diseases (ID) Centres throughout Italy collected prospectively data concerning both the clinical and microbiological diagnosis of patients affected by SSTIs via an electronic case report form. All the cases included in our database, independently from their severity, have been managed by ID specialists joining the study while SSTIs from other wards/clinics have been excluded from this analysis. Here, we report the preliminary results of our study, referring to a 12-month period (October 2016–September 2017). During this period, the study population included 254 adult patients and a total of 291 SSTI diagnoses were posed, with 36 patients presenting more than one SSTIs. The type of infection diagnosed, the aetiological micro-organisms involved and some notes on their antimicrobial susceptibilities were collected and are reported herein. The enrichment of our registry is ongoing, but these preliminary results suggest that further analysis could soon provide useful information to better understand the national epidemiologic data and the current clinical management of SSTIs in Italy.

Real-World Risk Assessment and Treatment of Patients with Myelofibrosis at Community Oncology Practices in the United States

Real-World Risk Assessment and Treatment of Patients with Myelofibrosis at Community Oncology Practices in the United States

Non-Interventional Retrospective Multicenter Study Evaluating Real Word Idelalisib Use in Chronic Lymphocytic Leukemia and Indolent Non-Hodgkin Lymphoma Patients Enrolled in the French Early Access Program

INTRODUCTIONIdelalisib (IDELA), the first-in-class PI3K-delta inhibitor, is indicated in Europe in combination with rituximab (R) or ofatumumab for the treatment (tt) of adult patients (pts) with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy, or as first-line tt of pts with 17p deletion (del(17p)) or TP53 mutation (TP53m), not eligibles for any other therapies and as monotherapy for pts with follicular lymphoma (FL) refractory to two prior lines of tt. IDELA has significant clinical activity with a manageable safety profile. However, there is a paucity of real word data regarding its effectiveness and safety. The REALIST study was initiated to better describe pts who started IDELA treatment during the French Early Access Program (EAP).AIMSThe aim of this retrospective multicenter study was to describe efficacy, adverse events (AEs), serious AEs (SAEs), AEs of special interest (AESIs, defined as diarrhea/colitis, pneumonitis, liver enzyme elevation, neutropenia, infection, and rash) and IDELA use in adult pts previously enrolled in the French EAP for CLL or iNHL.METHODSAll Investigators who enrolled at least one pt in the EAP were contacted by email inviting them to participate in this study. Only pts who initiated IDELA tt between June and October 2014 have been included in this study. For each pt, 12 months (m) follow up data were collected via an electronic Case Report Form and monitored by the Lymphoma Academic Research Organization (LYSARC). The Primary endpoint was the overall response rate (ORR) at 6 m and its 95% confidence interval (95% CI). Statistical analysis was descriptive.RESULTSSeventy-five pts were included, 41 in CLL group (39 in third-line or late and 2 in first-line with del(17p)/TP53m unsuitable for chemo-immunotherapy (CIT)) and 34 in iNHL group, all refractory after two prior lines of tt. Table 1 lists key pt characteristics at the time of initiation of IDELA. In CLL/iNHL groups respectively, median IDELA tt duration was 25.1 weeks/18.9 weeks, ORR, was 82.8%/56.5% at 6 m (data reported in 29 pts/23 pts) and 100%/57.1% at 12 m (data reported in 12 pts/14 pts), median PFS was not reached (NR)/ 6.7 m, median time to next tt (TTNT) defined as the time between the date of the last IDELA intake and the start of next tt after progression of disease (PD) was NR/11.6 m, median OS was NR in both groups and OS estimate at M12 was 69.9%/67.6%. Additional efficacy data are presented in Table 2, Fig 1 and Fig 2. In CLL/iNHL groups respectively, at least one AE was reported during the study in 35 pts (85.4%)/26 pts (76.5%), most frequently reported AEs by SOC/PT were: infections and infestations 61.0%/50.0% (pneumocystis jirovecii pneumonia (PJP) 2.4%/5.9%), gastrointestinal disorders 56.1%/35.3% (diarrhea 41.5%/29.4%), blood and lymphatic system disorders 41.5%/41.2% (neutropenia 17.1%/23.5%) and investigation 43.9%/47.1% (hepatic enzyme increase 19.5%/14.7%). 24 pts (58.5%)/15 pts (44.1%) discontinued temporary IDELA, 6 pts (14.6%)/8 pts (23.5%) discontinued permanently for AE, 38 pts (92.7%)/25 pts (73.5%) had at least one AESI, 10 pts (24.4%)/11 pts (32.4%) had at least one serious AESI, 3 pts (7.3%)/6 pts (17.6%) permanently discontinued IDELA for AESI and deaths were reported during the study in 13 pts (31.7%)/11 pts (32.4%), mainly due to PD (46.2%/72.7%).CONCLUSIONThe results of this non-Interventional study of heavily pretreated CLL and iNHL population treated by IDELA monotherapy or in combination with rituximab indicate that IDELA is an effective treatment in routine clinical practice with an acceptable safety profile. ORR at 6 months was 83% in CLL full analysis set and 79% in del(17p)/TP53m sub-group and 55% in FL sub group. These results mirror those of clinical trials. No unexpected IDELA safety information was identified in this study and the pattern of AEs corresponds to that reported in previous clinical studies and might be improved by specific management of AESI including PJP prophylaxis to be administered to all patients throughout IDELA treatment. DisclosuresYsebaert:Roche: Consultancy, Research Funding; Gilead Sciences, Inc.: Consultancy, Research Funding; Janssen: Consultancy, Research Funding. Feugier:Gilead: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Roche: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Abbvie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding. Salles:Novartis: Consultancy, Honoraria; Roche: Honoraria, Research Funding; Celgene: Honoraria, Research Funding; Abbvie: Honoraria; Acerta: Honoraria; Amgen: Honoraria; Epizyme: Honoraria; Gilead Sciences: Honoraria; Janssen: Honoraria; Merck: Honoraria; Morphosys: Honoraria; Pfizer: Honoraria; Servier: Honoraria; Takeda: Honoraria. Sylvain:Gilead: Other: scientific advisor board. Glorian Kergaravat:Gilead Sciences: Employment. Simpson:Gilead Sciences: Employment. Ramroth:Gilead Sciences: Employment. Abdelhadi:Gilead Sciences: Employment. Haioun:Takeda: Consultancy, Honoraria; Amgen: Consultancy, Honoraria; Gilead Sciences: Consultancy, Honoraria; Janssen: Consultancy, Honoraria; Sciences: Consultancy, Honoraria; Celgene: Consultancy, Honoraria; Roche: Consultancy, Honoraria. Troussard:Gilead: Other: scientific advisory board.

Survival in Patients with Relapsed/Refractory Multiple Myeloma: Outcomes after 4 Years of the Ongoing Multinational Observational Preamble Study

Survival in Patients with Relapsed/Refractory Multiple Myeloma: Outcomes after 4 Years of the Ongoing Multinational Observational Preamble Study

Real-World Data on the Use of rFIXFc in Subjects With Hemophilia B for Up to 3.7 Years Demonstrates Improved Bleed Control and Adherence With Reduced Treatment Burden

Introduction: Recombinant factor IX Fc fusion protein (rFIXFc) was the first extended half-life FIX product approved in the United States to treat children and adults with hemophilia B. Long-term data from clinical trials have demonstrated the safety and efficacy of rFIXFc as well as an extended dosing interval (once weekly or every 10‒14 days based on individual needs); however, real-world data are limited (Wang et al. Haemophilia, 2018; Buckley et al. AMCP NEXUS, 2015). We therefore performed a retrospective chart review to further understand the clinical experience and outcomes associated with real-world treatment of hemophilia B with rFIXFc. Methods: This retrospective chart review is being conducted at 6 sites across different regions of the United States and aims to include 70 patient charts. Data entry for 43 patient charts has been completed to date (cutoff: June 29, 2018). Data collection is ongoing. Inclusion criteria were diagnosis of hemophilia B and receipt of rFIXFc for ≥6 months. Subjects with other coagulation disorders or any record of positive FIX inhibitor titers were excluded. De-identified subject-level data were transcribed onto anonymous electronic case report forms. Endpoints included changes in FIX therapy dosing interval, factor consumption, bleed control, and patient adherence before and after rFIXFc initiation. Descriptive statistics were used to summarize the results. Results: For the 43 charts available for analysis, the duration of follow-up receiving rFIXFc ranged from 0.5 to 3.7 years. Of these, 58% of subjects (25/43) were >18 years of age, 77% (33/43) were of white race, and 51% (22/43) had severe hemophilia B (Table 1). The most common genotype was missense, occurring in 63% of subjects (27/43). Among subjects with comorbidity, 37% reported hemophilic arthropathy (16/43) and 28% had hepatitis C (12/43). All 22 subjects with severe hemophilia B were treated with rFIXFc prophylactically compared with 9/15 moderate and 3/6 mild cases. From the data collected thus far, 94% of prophylaxis subjects were on a dosing interval of weekly or longer (every 7 days, n=20; every 10 days, n=3; and every 14 days, n=9). The total weekly dose before and after switching to rFIXFc prophylaxis were available for 20 subjects. Of the 12 adults (9 severe, 2 moderate, and 1 mild), the median weekly factor consumption decreased from 111 IU/kg to 52.5 IU/kg. A similar pattern was observed for subjects who were 12-18 years of age (n=4). For subjects The annualized bleeding rates (ABRs), annualized spontaneous bleeding rates (AsBRs), and annualized joint bleeding rates (AjBRs) were available for 17 subjects treated with prophylaxis regimens pre- and post-rFIXFc (Table 2). Of these subjects, 11 had severe, 5 had moderate, and 1 had mild hemophilia B. Among 11 subjects with severe hemophilia B, median (interquartile range) ABRs decreased from 8.2 (4.4‒11.5) to 2.3 (0.6‒10.2), AsBR from 1.2 (0‒9.7) to 0.3 (0‒8.7), and AjBR from 2.3 (1.4‒8.2) to 0.7 (0‒6.9) before and after rFIXFc treatment. Subjects with moderate disease had a similar pattern (Table 2). The most common reason for switching to rFIXFc was to reduce the burden associated with therapy (21/43, 49%). No rationale for switching was documented in 40% (17/43) of subjects, and 7% (3/43) switched due to lack of efficacy with previous treatment. The other reasons, including difficult venous access, lack of adherence, and failure to reach desired trough were mentioned by Conclusions: This real-world study of rFIXFc demonstrates improved bleed control, reduced overall consumption, and reduced frequency of injection for subjects with moderate and severe hemophilia B. The data also show that rFIXFc provides an opportunity to tailor dosing and improve adherence. These results echo the findings of the pivotal trials and add to the pool of evidence supporting rFIXFc in the treatment of hemophilia B. These data also reflect the use of rFIXFc for mild hemophilia patients in the real-world setting. Disclosures Shapiro:Kedrion Biopharma: Consultancy, Research Funding; Novo Nordisk: Membership on an entity9s Board of Directors or advisory committees, Research Funding; Bioverativ, a Sanofi Company: Consultancy, Membership on an entity9s Board of Directors or advisory committees, Research Funding; Prometic Life Sciences: Consultancy, Research Funding; Shire: Membership on an entity9s Board of Directors or advisory committees, Research Funding, Speakers Bureau; Genetech: Membership on an entity9s Board of Directors or advisory committees, Research Funding, Speakers Bureau; Bayer Healthcare: Other: International Network of Pediatric Hemophilia; BioMarin: Research Funding; Bio Products Laboratory: Consultancy; Octapharma: Research Funding; OPKO: Research Funding; Sangamo Biosciences: Consultancy; Daiichi Sankyo: Research Funding. Chaudhury:Bioverativ, a Sanofi Company: Consultancy, Research Funding; Bayer: Membership on an entity9s Board of Directors or advisory committees. Jain:Bioverativ: Employment. Tsao:Bioverativ: Employment. Barnowski:Bioverativ, a Sanofi Company: Employment. Feng:Bioverativ: Employment. Quon:Shire: Speakers Bureau; Genetech: Consultancy, Speakers Bureau; NovoNordisk: Consultancy, Speakers Bureau; Bayer: Consultancy; Octapharma: Consultancy; Bioverativ, a Sanofi Company: Speakers Bureau.

Clinical and Hematological Risk Factors for Postpartum Hemorrhage: A 2 Years Population Based Cohort Study in France

Objective: To determine clinical and hematological risk factors for immediate postpartum hemorrhage (PPH).Design: A cohort study.Setting: Obstetric unit of Brest University Hospital (France).Population: All women who underwent a vaginal delivery between April 1, 2013 and May 29, 2015.Methods: Clinical data were collected by obstetricians or midwives during antenatal care visits, labor and delivery, and recorded by trained research assistants in a standardized electronic case report form. Hematological variables, including immature platelet fraction, were measured from a blood sample systematically performed at the entrance in the delivery room.Main Outcome Measures: Postpartum hemorrhage (PPH), measured with a graduated collector bag, was defined as blood loss of at least 500ml.Results: 2742 women were studied. PPH occurred in 141 (5%) women. Seven clinical factors were independently associated with PPH: pre-eclampsia (OR 5.85, 95%CI 2.02-16.90), multiple pregnancy (OR 3.28 (1.21-8.91), assisted reproduction (OR 2.75, 95%CI 1.45-5.20), ante partum bleeding (OR 2.15, 95%CI 1.24-3.73), post-term delivery (OR 1.93, 95%CI 1.17-3.17), obesity (OR 2.95, 95%CI 1.76-4.93) and episiotomy (OR 2.51, 95%CI 1.63-3.74). Three hematological factors were additionally identified as potential predictive factors for PPH: platelets < 150 000 Giga/L (OR 2.98, 95%CI 1.63-5.46), fibrinogen < 4.5 g/dL (OR 1.86, 95%CI 1.21-2.87) and APTT ratio ≥ 1.1 (OR 2.16, 95%CI 1.31-3.57).Conclusion: Besides classical risk factors, this study highlighted the role of assisted pregnancy and obesity in the PPH occurrence, and identified simple hematological predictive risk factors for PPH. How these parameters could be integrated in a helpful prediction score of PPH remains to be determined. DisclosuresPan-Petesch:LFB: Other: Investigator.