C75 HARMONIzATION OF DATABASES
Landwehrmeyer B
University of Ulm, Ulm, Germany
No abstract available.
C76 ALS PATIENT CARE DATABASE IN JAPAN
Yamaguchi TY1, Ohbu SO2, Saito MS1, Ohashi YO1, Moriwaka FM3, Tashiro KT3, Suzukamo YS4, Fukuhara SF4, Narita YN5, Kuzuhara SK5
1University of Tokyo, Tokyo, 2Rikkyo University, Tokyo, Japan, 3Hokkaido University School of Medicine, Sapporo, Japan, 4Kyoto University School of Public Health, Kyoto, Japan, 5Mie University Graduate School of Medicine, Tsu, Japan
E‐mail address for correspondence: [email protected]
Background: The ALS Patient Care Database was created to improve the quality of care for patients with ALS in the United States, and it has provided valuable data on physicians' practices and patient‐focused outcomes (1).
Objectives: We started an ALS database in Japan to describe the status and trends in patients’ QOL, clinical conditions, therapeutic interventions, etc.; to evaluate the effects of ALS on caregivers; and to help researchers with regard to instruments and items useful for observational and interventional studies.
Methods: The database was open to all neurologists in Japan. Data were collected at intervals of six months. Patient‐reported data included demographic information, initial diagnosis, duration of symptoms, interventions, self‐reported health care, satisfaction with medical care, ALSAQ‐40, etc. Physician‐reported data included date of diagnosis, diagnostic criteria, region affected by ALS, ALSFRS‐R, WFN criteria, etc. Caregiver‐reported data included demographic information, relationship to patient, employment status, general health status, SF‐36, carer status, source of payments for caregiving, etc. Data‐collection forms were submitted to a single data center.
Results: From October 2001 to November 2004, data on 89 patients at about 40 clinical sites were collected. Patients had a mean age of 61.1 (SD 11.2) years and 63% were men. Almost all patients (96%) had sporadic ALS, and 45% had classical ALS. The mean interval from onset of the disease was 37.7 months (SD 30.1, range 1–122). About half of the patients were satisfied with the quality of their care. The mean age of caregivers was 54.0 (SD 13.9) years and 76% of them were female. The primary caregiver was the spouse (70%). About 70% of the caregivers were paid for their service by public money. One‐quarter of them had stopped other paid employment. The QOL data (SF‐36) documented that caregivers had stress and emotional burdens attributed to the disease and to caregiving. The data indicate that the Japanese version of the ALSAQ‐40 (2) had high validity and was likely to be useful in evaluating the QOL of ALS patients in Japan. Physicians (using the ALSFRS‐R) and caregivers were consistent in their evaluations of patients' abilities to carry out daily activities.
Discussion and conclusions: Longitudinal data will be needed to reveal trends in clinical conditions and interventions, and to examine the relationship between them. This database appears to provide valuable information in ALS patient care. All neurologists practising in Japan are welcome to contribute to the database and to use the data.
References
- Miller RG, Anderson FA, Bradley WG, et al. The ALS patient care database – goals, design, and early results. Neurology 2000;54:53–7.
- Yamaguchi T, Ohbu S, Saito M. et al. Validity and clinical applicability of the Japanese version of Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSAQ-40). Brain and Nerve 2004;56:483–94.
C77 TREATMENT OF PATIENTS WITH ALS IN THE COMMUNITY: NEW INSIGHTS FROM THE WEB‐BASED ALS PATIENT CARE DATABASE
Miller RG1, Scholtz D1, Moore DH5, Spitalny GM1, Fox C2, Katz J1, Kushner G1, Brooks B3, Mitsumoto H4, Anderson F6
1California Pacific Medical Center, Forbes Norris MDA/ALS Research Center, San Francisco, CA, 2University of San Francisco, Preventive Sciences Group, San Francisco, CA, 3University of Wisconsin, Department of Neurology, Madison, WI, 4Columbia University Medical Center, Neurological Institute of New York, New York, NY, , 5California Pacific Medical Center Research Institute, San Francisco, CA, 6University of Massachusetts Medical School, Center For Outcomes Research, Worcester, MA, USA
E‐mail address for correspondence: [email protected]
Background: Data on outcomes for patients cared for in arge ALS centers comes from the ALS C.A.R.E database. New data were gathered from patients who receive care in the community via a web‐based ALS registry.
Objective: To compare outcomes of patients treated in the community with those treated in large specialized ALS clinics.
Methods: Treatment and outcomes were compared from the ALS C.A.R.E. database with data entered in the web‐based, patient‐driven database, ALS Connection, supported by a grant from the MDA. The website was launched in January 2006. This analysis compares the first 111 patients enrolled in the ALS Connection (Web) with more than 6000 patients enrolled in the ALS C.A.R.E. database (CARE), 90% of whom are cared for in large ALS clinics.
Results: More Web patients obtained their information regarding ALS from the internet (CARE 3% vs. Web 28%). In general the impact of ALS was greater on Web patients: fewer Web patients could perform more than 50% of daily activities themselves (CARE 54% vs. Web 42%); more Web patients accomplished much less due to the disease (CARE 25% vs. Web 65%). However, more patients were satisfied with their care in the ALS CARE database (CARE 91% vs. Web77%).
When given the diagnosis of ALS, 71% of CARE patients received adequate information about patient advocacy organizations compared with 47% of Web. More Web patients were dissatisfied with receiving the diagnosis: too little information (CARE 2% vs. Web 17%); no hope given (CARE 3% vs. Web 13%); insensitive delivery (CARE 2% vs. Web 46%).
Access to symptomatic therapy for drooling was only 3% in the Web vs. 32% in the CARE group. Similarly, fewer Web patients used helpful therapies for pseudobulbar affect: amitriptyline (CARE 24% vs. Web 5%); SSRI (CARE 47% vs. Web 7%); other (e.g. dextromethorphan)(CARE 61% vs. Web 8%).
Web patients used less therapy: occupational therapy (CARE 59% vs. Web 42%); speech therapy (CARE 61% vs. Web 24%); nutrition (CARE 66% vs. Web 35%); social worker (CARE 65% vs. Web 27%); psychologist (CARE 65% vs. Web 14%); home nurse (CARE 63% vs. Web 27%).
Recommendation for enteral feeding was similar in both cohorts but utilization was less in CARE (CARE 30% vs. Web 42%). Utilization of non‐invasive ventilation was similar in Web and CARE patients.
Conclusions: Patients with ALS who enrolled in a web‐based registry were more impacted by ALS, were generally less satisfied with their care, had a more unsatisfactory experience in learning about the diagnosis, and less access to symptomatic therapy than those in the ALS C.A.R.E. database. ALS patient registries are needed to identify disease impact, patient needs and treatment utilization. Patients from ALS clinics and patients who access a web registry appear to have different experiences. Further research is needed to identify the reasons for these differences and how stable these differences are.
C78 CHALLENGES OF DATA MANAGEMENT IN WEB‐BASED MULTI‐SITE CLINICAL TRIALS IN AMYOTROPHIC LATERAL SCLEROSIS
Sherman AV, Cudkowicz ME, Yu H, Balakrishna S, Opoliner AF
1MA General Hospital, Boston, MA, USA, 2PharmaContent, Inc., Brookline, MA, USA
E‐mail address for correspondence: [email protected]
Background: The Neurology Clinical Trials Unit (NCTU) of the Massachusetts General Hospital serves as the coordination center for several multi‐site clinical trials in amyotrophic lateral sclerosis (ALS). Efficient data management (DM) is one of the important aspects of successful conduct of clinical trials.
Objectives: Creating an efficient and compliant environment for DM of multi‐site clinical trials in ALS is one of the major objectives for the clinical trials coordination center. Well‐organized and competent data management will reduce time in data cleaning and will allow faster locking of the database for biostatistical analyses.
Methods: NCTU has conducted multi‐site clinical trials in ALS for more than five years and has accumulated sufficient empirical data, and developed methods to maximize efficiency of DM in clinical trials. These methods may be divided into two areas, general DM and ALS‐specific DM.
The following factors may determine a successful clinical trial from the DM perspective:
Involvement of DM at the start of the trial into protocol design, Case Report Forms (CRFs) and EDC system specifications development
Constant communication with site users and feedback solicitation
Initiation of user training as combination of in‐class and web‐based sessions and periodical brief refreshment classes
Unambiguous designation of tasks for data management and study monitors in data cleaning and query closing
Clinical trials in subjects with ALS have certain ALS‐specific factors that must be discussed and resolved prior to the commencement of the trial:
Clear definition of outcome measures
Development of standard ALS‐specific common data elements (CDE) that guarantee consistency and reliability of trial data. These data elements include CRF design, associated with the form data dictionary, logic checks, data ranges, help files and reports.
Introduction of new types of visits (site, home, phone) for ‘survival’ trials
Education of sponsors, study and medical monitors on ALS‐specific adverse events reporting
Results: NCTU has developed certain approaches, techniques and recommendations on DM of clinical trials in subjects with ALS. These approaches were successfully used in several multi‐site trials. Future standardization of ALS‐specific CDE and more effective utilization of accumulated know‐how are recommended.
Conclusions: The following are recommended to the ALS research community to successfully implement this task:
Implement efficient data management
Develop standards for ALS‐specific CDE
Clearly define outcome measures
Use web portal and conferencing for communications with site users
Develop multi‐layer continuous data capture training program
Define clear line of responsibilities between DM and study monitors
Define data exchange mechanism with central laboratory
Allocate sufficient time for testing and validation of EDC system