C45 USE OF PROSPECTIVE REGISTERS IN DEFINING CLINICAL CHARACTERISTICS AND IMPROVING CARE PROVISION
CHIÓ A
ALS Center, University of Torino, Italy
E-mail address for correspondence: [email protected]
Keywords: Epidemiology, Italy, Registers
Amyotrophic lateral sclerosis (ALS) is a relatively rare disease with an annual incidence rate between 1.5 and 2.5/100,000 population. Over the past 10 years, the design of ALS epidemiological studies has evolved to focus on a prospective, population based methodology, employing the El Escorial criteria and multiple sources of data to ensure complete case ascertainment. Six European and North American studies based on this paradigm have been published and show remarkably consistent incidence figures among their respective Caucasian populations. Although population based studies have been useful in defining clinical characteristics and prognostic indicators in ALS, many epidemiological questions remain that cannot be resolved. The working hypotheses is that ALS, like other chronic diseases, is a complex genetic condition, and the relative contributions of individual environmental and genetic factors are likely to be relatively small. Larger studies are required to characterise risks and identify sub-populations that might be suitable for further analysis.
The Piemonte and Valle’ d'Aosta ALS register (PARALS) is a prospective register collecting all cases of ALS incident in two regions of north-western Italy (total population, 4,332,842). This was established in 1995, based on the collaboration of all the neurological departments of the area. Patients are prospectively followed-up from diagnosis. In the 10-year period of observation, 1995 to 2004, a total of 1260 new cases of ALS were detected, 687 men and 573 women. The mean age at onset was 64.9 years (SD 11.2) (men 64.6 (SD 10.1); women, 65.4 (SD 10.7); p= n.s.). The mean time delay form onset to diagnosis was 10.4 months (SD 10.1), with a significant decrease in the last 5-year period (1995–1999, 11.0 months; 2000–2004, 9.7 months; p = 0.001). The mean annual crude incidence rate in the period 1995–2004 was 2.90 (95% c.i., 2.72 to 3.09). Crude incidence rates were significantly higher in men. The incidence rates did not change when comparing the first 5 years (1995–1999, 2.87/100,000) and the second 5 years (2000–2004, 3.00/100,000) of the study. A total of 319 patients were alive at the prevalence day (December 31st, 2004), corresponding to a crude prevalence rate of 7.40 (6.6–8.3)/100,000 population. There was a slight but not significant increase of median survival time. There was significant increase of the number of patients who underwent non-invasive ventilation (NIV) (10.3% vs. 17.9%; p = 0.0001), whereas the number of patients who performed tracheotomy and PEG/PRG only slightly increased.
In conclusion, ALS incidence showed no relevant modifications during the 1995–2004 period in this population-based prospective register. The significant reduction of the mean time delay is likely to indicate an increased awareness of ALS among the neurologists and other physicians in the examined area. Despite the increased use of NIV, there was only a modest increase of median survival.
C46 DIAGNOSTIC PROCESS AND NEUROLOGICAL CARE IN PATIENTS WITH AMYOTROPHIC LATERAL SCLEROSIS IN SPAIN
MORA JS1, SALAS T1, FAJARDO M2, IVANEZ L2
1ALS Unit, Hospital Carlos III, Madrid, Spain, 2Epidemiology Unit, Consejeria de Sanidad, Huelva y Sevilla, Spain
E-mail address for correspondence: [email protected]
Keywords: Diagnosis, neurological care, Spain
Background: The adequate health care of ALS patients depends on knowledge of the disease and the adequate resources of the health care system. Identification of care deficits caused by failures of the system or the professionals is the first step that is needed to resolve them.
Objectives: To determine the health care status of Spanish patients with ALS, specifically their diagnostic process and neurological assistance in order to identify needs and to propose improvements.
Methods: We created a 156 item questionnaire that included demography, clinical status and administered health care. Diagnostic process and neurological care accounted for one third of the questions. The questionnaire was hand delivered or mailed to up to 400 patients with ALS from the Spanish ALS Foundation nationwide. Response to date is 260. A univariant analysis with SPSS 10.0 for Windows was performed.
Results: The patient's median profile is male/female (ratio 1:1); 54 years old (range 29–79); married (81%); spouse as primary caregiver (70.8%); and from Madrid (41.5%). Diagnostic and neurological care habits and deficits were identified. Among those, median diagnostic delay time was reported as 12.6 months; of those, 4.7 months in specific testing. To establish diagnosis, two or three physicians were required by 61.9% of patients; four or more by 27% of patients. The way the diagnosis was given was perceived as with scarce information or just tactless by 35.4% of the patients. In 33.8% of the cases, diagnosis was only given to the family. Seventy three percent of patients requested a second opinion; of which just over ten percent were given at our Unit. Sixty-one and a half percent of patients were being primarily attended by non-neurologists. The mean period between visits was 3.8 months. Patients reported that only 42.6% of their doctors knew and were interested in their disease. 38.2% of patients received neurological assistance in a different territorial area that of their own. Both the diagnostic and detailed therapeutic practices of neurologists as reported by the patients will be presented.
Conclusions: There is a considerable delay in establishing the diagnosis, which delays the onset of therapies and most likely increases the patient’ emotional distress. A significant percentage of patients received the diagnosis in an unsatisfactory and unhealthy way; were primarily cared for by non neurologists; sought neurological care out of their own sanitary area; or considered their doctors not knowledgeable or uninterested in the disease. Resolving the deficits identified requires the determined intervention of health care decision makers to both provide professional training and generate additional infrastructure resources.
C47 LAPAROSCOPIC PLACEMENT OF GASTROSTOMY FEEDING TUBE (LSCG-TUBE): INITIAL EXPERIENCE
NUTTLEMAN P, DENNIS C, BARKHAUS P
Medical College of Wisconsin, Milwaukee, Wisconsin, United States
E-mail address for correspondence: [email protected]
Keywords: gastostomy, nutrition, laparoscopic
Background: Gastric-feeding access has been traditionally established by endoscopic or radiologic means. Laparoscopic techniques have been described to assist endoscopic placement of the standard PEG feeding tube.
The traditional open surgical gastrostomy tube incorporates a purse-string suture sealing the stomach about the tube as well as placement of multiple sutures anchoring the stomach to the abdominal wall in a circumferential manner theoretically eliminating the devastating complication of intra-abdominal sepsis resulting from leak and/or early dislodgement real to the less invasive methods. A laparoscopic approach mimicking the original open surgical procedure preserves these technical benefits and also affords the unique advantage of correcting a hiatal hernia if present. By inference, the latter may reduce risk of aspiration of gastric contents in neurologically impaired patients.
Objectives: To explore the feasibility of LSCG-tube placement in ALS patients using general anesthetic, including repair of hiatal hernia when present.
Methods: N = 17 patients with definite or probable ALS with Laboratory Support (El Escorial criteria) from our ALS Center were referred for LPS G-tube placement. 10/17 presented with bulbar disease. These patients were evaluated by one surgical consultant (author PRN). Initially, patients were refused endoscopically or radiologically placed tubes for pulmonary or anatomic reasons. All patients showed impaired swallow. Pulmonary function varied widely (VC median: 53% predicted). All patients required general anaesthesia. Tubes were not sewn to skin nor were removable skin sutures or staples employed. Post-procedural sedatives and narcotics were judiciously minimized. A surgical follow-up was performed at one month and every three months thereafter.
Results: All 17 underwent successful LPS G-tube placement without leakage, bleeding, or intolerance to use within 24 hours. Five patients also underwent concomitant hiatal or paraesophageal hernia repair. All patients tolerated bolus gastric feedings except one patient with recurrent paraesophageal hernia salvaged by radiologic conversion to gastro-jejunal access.
Peri-operative complications include immediate reintubation (n = 1), delayed extubation (n = 1) and tube dislodgement (n = 1). Three out of seventeen patients died within 30 days of procedure. Four additional patients died within 6 months of procedure. There were no known gastric aspiration events.
Discussion and Conclusion: Initial results suggest that LSC G-tube placement is a safe procedure in ALS patients despite suboptimal pulmonary function. A general anaesthetic has the paradoxical advantage of airway control and permits liberal peri-procedural sedation and analgesia. With rehydration, management of oral-pharyngeal secretions must be anticipated. This technique has the advantage of direct visualization of a hiatal defect and repair if present.
C48 MEASURING DISEASE PROGRESSION IN ADVANCED ALS: THE ALSFRS-R EXTENSION (ALSFRS-EX)
WICKS P1, MASSAGLI M1, WOLF C2, HEYWOOD J1
1PatientsLikeMe, Cambridge, Mass, United States, 2Patient, Katonah, NY, United Kingdom
E-mail address for correspondence: [email protected]
Keywords: ALSFRS-R, advanced disease, clinical trials
Background: The Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised (ALSFRS-R) was developed for use in clinical trials where death or ventilation was considered the endpoint of study. Since the publication of the original scales, a small but increasing number of patients decide to have invasive ventilation or survive for several years on continuous non-invasive ventilation. Although dependent on others for activities of daily living, many report satisfaction with their lives and a desire to continue tracking their progress beyond the zero-point on the ALSFRS-R.
Objectives: To expand the range of the ALSFRS-R to measure disease progression in those with advanced disease.
Methods: Following interviews with patients in advanced disease states (ALSFRS-R scores <25), we developed 10 new items that could potentially be added to the scale. In August 2007 we invited 1246 patients to complete the ALSFRS plus the 10 new questions in an on-line survey. 326 patients (26%) responded between 24 August 2007 and 15 September 2007. A 1-week retest was initiated after the first response. 271 of the baseline respondents were invited between 1- and 7- September 2007 to retake the survey. 169 patients (62%) responded to the retest between 7 and 21 days after the initial survey; 80% responded in 7 to 10 days. 218 (67%) of the baseline respondents completed a 3-month follow-up survey, which was conducted to explore the ability of the extended measure to detect change in comparison with the original ALSFRS-R.
Results: Respondents were broadly representative of members of an online community for people with ALS. Adding all 10 items increased the overall Cronbach's alpha of the ALSFRS-R from 0.881 to 0.932. Factor analysis revealed 8 of the 10 new items conformed to the existing factor structure of the original scale. Test-retest reliability was r = 0.9 or higher. Following the 3-month retest we repeated our analyses and decided upon four new items: ability to get around the home, ability to move fingers, ability to show emotional expressiveness in the face, and ventilator use. Each new item loads onto the existing factors of the scale: gross motor, fine motor, bulbar, and respiratory. The new items improve discrimination at the lower end of functioning in ALS patients.
Discussion: As ventilation and assistive technologies become a more widespread option, many patients will opt to live for several years as a “zero” on the existing scale. We have concisely extended the range of the ALSFRS-R to better monitor the progression of these patients with advanced disease. The ALSFRS-EX could be particularly useful in clinical trials and studies examining end-of-life decision making.
C49 AN INVESTIGATION OF EMOTIONAL STRESSES EXPERIENCED BY MULTIDISCIPLINARY ALS CLINIC STAFF
BROMBERG M, SCHENKENBERG T
University of Utah, Salt Lake City, UT, United States
E-mail address for correspondence: [email protected]
Keywords: Stress, clinic
Background: ALS is a progressive disorder with no effective therapy that leads inexorably to death. Multidisciplinary ALS clinics may make 20–100 new diagnoses per year and experience a similar number of deaths per year. Thus, the clinical evaluation, giving the diagnosis, ongoing management, and death of the patient are emotionally taxing for clinic staff. Little is known how clinic members handle these stresses.
Objectives: We conducted a survey to better understand the types of stresses and techniques to manage them. We also queried methods of making and giving the diagnosis.
Methods: A set of questionnaires, for the neurologist (who makes the diagnosis) and the clinic manager (who continuously interacts with patients and families), was sent to 68 multidisciplinary ALS/MND clinics in the US. Included were questions on demographics; how the diagnosis is made and given; stressful factors in making and giving the diagnosis, with continued care, and at time of death; and strategies to handle stress.
Results: Demographics: the majority of neurologists were male and the majority of managers were female. The average number of new diagnoses was >50 per year. Fifty percent of neurologists and 95% of managers acknowledged being a “spiritual person”. Making the diagnosis: Two-thirds relied on the El Escorial criteria, but most acknowledged diagnostic limitations. Most ordered a battery of laboratory tests even though the diagnosis of ALS was clear. All delivered the diagnosis in person. There was a range of stress in giving the diagnosis that tended to lessen with experience. Continuing Care: There was greater stress for managers, and experience tended to lessen stress more for neurologists. Two-thirds of clinics did not write prescriptions for off-label medications, and such requests were stressful. Time of Death: There was moderate stress for both neurologists and managers that did not lessen with experience. More managers than neurologists personally contacted family and attended funerals. Overall Stress: There was a tendency for reduced stress with experience. Techniques to manage stress included exercise, spirituality, alcohol, and support from staff (discussions), but all were judged to be of low efficacy. The majority were interested in a national effort to address stress, in the form of seminars, videos or books. Overall Satisfaction: Neurologists and managers were uniformly satisfied, felt they were doing a good job, and few considered leaving the field. Challenging operational issues were finances, personnel, and time.
Discussion and Conclusions: ALS clinic providers experience considerable stresses, but they are manageable and some may lessen with experience. There are different stress management techniques, but none are clearly successful. Despite these stresses overall satisfaction is high. A formal effort to recognize and manage stress is favoured. It might be helpful to orient new personnel to these stresses and management.