Abstract
Background: The American Academy of Neurology (AAN) ALS Practice Parameter was published in April 1999. The ALS CARE Database has been collecting data on the management of patients with ALS in North America since 1996.
Objective: To compare the management of patients with ALS in North America as recorded in the ALS CARE Database with the recommendations of the AAN ALS Practice Parameter. Methods: Data were analyzed from 2018 patients at enrollment and from 373 of these patients who died between enrollment and May 1999.
Results: Eighty-two percent of the enrolled patients reported that they had been given enough information about ALS. Only 54% of patients with drooling were receiving medication for this problem. Only 41% of those who reported being depressed most of the time were receiving antidepressant medications. Only 28% of those with dyspnea and only 9.2% of those with a forced vital capacity <40% predicted were receiving noninvasive positive pressure ventilator support. Only 30% of those with moderate to severe dysphagia had a gastrostomy tube. Half of the patients who died did so at home, but only 47% of them received residential hospice services. Although 89% of patients who died were recorded as having done so peacefully, 17% were reported to have had breathing difficulties (i.e., respiratory distress), 8% anxiety, 3.3% pain, and 2.5% choking. Advance directives were in place for 90% of the patients who died, and in 97% of cases these directives were followed.
Conclusions: These findings indicate that in the 3-year period prior to the publication of the AAN Practice Parameter, many but not all patients received the care that is recommended in that parameter; there were deficiencies, particularly in the key areas of gastrostomy and noninvasive positive pressure ventilation.
The ALS CARE Database has been collecting information on patients with ALS, their caregivers, and their management since July 1996.1-4⇓⇓⇓ Currently there are 91 clinical sites that have enrolled more than 2500 patients. The American Academy of Neurology (AAN) ALS Practice Parameter was published in the April 1999 issue of Neurology.5 One of the goals of this practice parameter is to influence the clinical management of patients with ALS. To observe whether this goal has been achieved, it will be necessary to monitor the management of patients with ALS over time after the publication of the practice parameter. We report here a comparison of the recommendations of the AAN ALS Practice Parameter with the baseline features of the management of patients with ALS extracted from the ALS CARE Database for patients enrolled in the 3-year period before the publication of the practice parameter. Our long-term goal is to repeat this study after an interval of a few years to examine the effect of the publication and dissemination of the AAN ALS Practice Parameter.
We also intend to determine whether the current ALS CARE Database questionnaire provides sufficient data for the prospective analysis of the effect of publication of the AAN Practice Parameters, and to search for temporal trends in management that may have occurred during the baseline period of 1996 through May 1999.
Methods.
The ALS CARE Database was created to measure and to improve the quality of care for patients with ALS.1,4⇓ Key features of this program include 1) providing neurologists with data to evaluate and to improve their practices; 2) publishing data on temporal trends in the care of patients with ALS; and 3) developing hypotheses to be tested in clinical trials. This observational database is open to all neurologists practicing in North America. Longitudinal data are collected at intervals of 3 to 6 months using standard data collection instruments. Forms are submitted to a central data-coordinating center, which mails quarterly reports to participating neurologists. Participating clinical sites are encouraged to enroll all their patients with ALS, including both incident and prevalent cases. Data are collected in the following domains: diagnosis, prognostic factors, natural history, and therapeutic interventions. In addition to data reported by neurologists, self-reports are collected from patients and their caregivers. A university-based Study Coordinating Center manages the data, performs analyses, and protects the confidentiality of participants, including patients, neurologists, and clinical sites.
Items relevant to the AAN ALS Practice Parameter5 were extracted from the enrollment data of 2018 patients who were entered into the ALS CARE Database before May 1999. The demographics of these 2018 patients at enrollment were (mean ± SD) age 58.9 ± 12.8 years, males 61.0%, symptom duration 2.5 ± 2.9 years, and ALS functional rating scale score 27.3 ± 8.0. By nature of the enrollment process, cases were entered by the contributing centers at all stages of the disease. We also analyzed the completion data on 373 of these patients who had died at some time after enrollment but before May 1999. There was a significant trend for the cases enrolled in the first year of the study to have had the disease for a longer time (i.e., that were prevalence cases derived from the population of ALS patients in the clinic) than those enrolled later (i.e., that were patients newly presenting with ALS to the clinic). In 1996–1997, only 19% of the cases were enrolled within 3 months of diagnosis, whereas in 1998–1999 this figure was 37%. Allowance for this enrollment bias was made in a number of subsequent analyses.
Results.
In the paragraphs below we compare the data from the ALS CARE Database concerning management of patients enrolled before May 1999 with the recommendations of the AAN ALS Practice Parameter.5 Additional data are provided in a table on the Neurology Web site (go to www.neurology.org) that lists the main recommendations of the AAN ALS Practice Parameter5 and the findings from the ALS CARE Database.
Was sufficient information about ALS provided to patients?
Overall, 82% of patients felt that they had received enough information about ALS. The perception of having received sufficient information increased with a longer interval from diagnosis to enrollment, being 74% for those enrolled less than 3 months after diagnosis, 82% for those enrolled between 3 and 6 months after diagnosis, and 87% for those enrolled longer than 6 months after diagnosis. The ALS Association was recorded as being the most valuable source of ALS information by 37% of respondents, and the Muscular Dystrophy Association by 16%. The category “Other” (e.g., the neurologist, the ALS Center, the Internet, etc.) was recorded as the most valuable source of information by 29%.
How often were patients with sialorrhea treated?
Patients with increasing degrees of sialorrhea were more likely to receive treatment with atropine, glycopyrrolate, or amitriptyline (p < 0.001) (figure 1). Of the patients taking these drugs, 73% reported that they were helpful.
Figure 1. Proportion of patients having problems with sialorrhea as indicated by the ALS functional rating scale salivation subscores (open boxes), and proportion of patients at each subscore receiving medication for sialorrhea (filled boxes).
Were emotional lability and depression adequately treated?
The ALS CARE Database does not specifically record features of emotional lability. However, of the patients recorded as having upper motor neuron bulbar signs at enrollment, 29% had taken or were taking medications such as tricyclic or selective serotonin reuptake inhibitors that suppress emotional lability, compared with 21% of those without upper motor neuron bulbar signs (p < 0.001). Interestingly, the figures were the same for patients with lower motor neuron bulbar signs.
One question on the Sickness Impact Profile asked the patient to grade the amount of time spent “downhearted and blue.” Sixteen percent of patients overall admitted to being depressed “a good bit,” “most,” or “all of the time”; the proportion in each group who were taking or had taken antidepressants was 39, 41, and 50%, respectively. There was a significant (p < 0.001) relationship between symptoms of depression and the use of antidepressant medications (figure 2).
Figure 2. Proportion of patients having problems with depression as indicated by the Sickness Impact Profile depression subscore (open boxes), and proportion of patients at each subscore receiving medication for depression (filled boxes).
Was hypoventilation treated with noninvasive positive pressure ventilator (NIPPV) support?
Of the total 2018 patients, 13% had a severe degree of respiratory impairment (ALSFRS breathing subscores of 2 to 0, i.e., from shortness of breath at rest to ventilator-dependent). Of those with severe dyspnea who were not ventilator-dependent (i.e., those with ALSFRS breathing subscores of 1 or 2), 28% were using NIPPV.
Of the total 2018 patients, 38% had a forced vital capacity (FVC) <40% of predicted at the time of enrollment. Four percent of patients with FVC 40% and greater were receiving NIPPV support, compared with 9.2% of those with FVC below 40% (p < 0.001). Of the patients with FVC <40%, 3.2% were using a ventilator with a tracheostomy, and 0.3% had a tracheostomy alone.
Was severe dysphagia treated with a gastrostomy tube?
Of the total 2018 patients, 20% had significant dysphagia (ALSFRS swallowing subscores of 2 to 0, i.e., from needing a change of dietary consistency to being unable to eat by mouth). Only 30% of those with moderate to severe dysphagia had a gastrostomy tube. Of those patients with FVC of less than 50%, 12% had a gastrostomy tube compared with 3.3% of patients with FVC >50% (p < 0.001). There was a significant (p < 0.001) relationship between the FVC and the percent of patients having a gastrostomy tube (figure 3).
Figure 3. Distribution of forced vital capacities in the total 2018 patients (open boxes), and proportion of patients within each range of forced vital capacity that had a percutaneous gastrostomy tube (filled boxes).
What proportion of patients used hospice services?
Of the 373 patients who had died, 56% did so at home, 19% in a hospital, and 7% in a residential hospice facility. Forty-seven percent of those who died at home had received hospice services.
Was palliative care used effectively in the terminal stages of ALS?
Of the 373 patients who had died, 89% were recorded as having died peacefully. Symptoms during the dying process included breathing difficulty (i.e., respiratory distress) in 17%, pain in 3.3%, anxiety or fear in 8%, and choking in 2.5%. Medications such as benzodiazepines, narcotics, other sedatives, and analgesics were used to control pain and distress in the preterminal stage in 66% of the 373 patients who died. The route of administration was parenteral in 38%, enteral in 53%, and both in 9%. There was a trend for an increase in the use of such medications over the 3-year period of the study. Thirty-seven percent of patients received oxygen in the preterminal phase, and a further 17% received oxygen for a more prolonged period.
Were advance directives used in patients with ALS who died?
Advance directives were in place for 90% of patients who died, and in 97% of cases the advance directives were followed.
Discussion.
This study was undertaken to define the pattern of care of patients with ALS in the 3-year period before the publication of the AAN Practice Parameter for the management of ALS.5 It was also undertaken to determine to what extent the ALS CARE Database4 could be used to follow future changes in the pattern of ALS care. There is a considerable amount of information in the ALS CARE Database concerning the management of more than 2500 patients in the period July 1996 to the present. We have restricted our analysis to the data recorded for the 3-year period up to May 1999, thereby covering the management of ALS before the publication of the AAN ALS Practice Parameter.5
It is important to recognize the limitations of the data available in the ALS CARE Database. Most of the current patients were enrolled by specialized ALS centers, rather than by neurologists in practice. Also, it is evident that many of the patients entered at the beginning of the study in 1996–1997 were prevalence cases; that is, they were patients who were already under the care of the neurologist and were enrolled some time after diagnosis. In 1998 and early 1999, there were more incidence cases; that is, they were enrolled at or soon after diagnosis. This change in the duration of the disease in the enrolled patients from the beginning to the end of our period of analysis might potentially have introduced bias, although in the data presented here we have not found any significant temporal trends. Not all the ALS patients of a neurologist are recorded, and hence the ALS CARE Database does not represent an unselected consecutive series of patients.
The ALS Association, the Muscular Dystrophy Association, and “Other” (which may include the neurologist, the ALS Center, the Internet, etc.) were recorded to be the most important sources of information for patients. The database was initiated before the major explosion of the amount of information about ALS on the Internet. It will be important to add additional categories describing the source of information about ALS in future data collecting forms used by the ALS CARE Database. It will be interesting to determine the trend over the next few years as yet more people use the Internet and as specialized ALS Center Web sites proliferate.
Only approximately half of patients received medications to control moderate to marked sialorrhea.6 Although side effects like excessive dryness of the mouth and constipation may have resulted in some patients not taking medications for drooling, this is a surprisingly low figure. The AAN ALS Practice Parameter should help raise awareness of the availability of useful treatments for this symptom, particularly glycopyrrolate and atropine as well as radiation therapy and botulinum toxin injections directed to the salivary glands.
Less than one-third of patients with emotional lability received antidepressant medication. Again this suggests the possibility of improving the care of patients with ALS through professional education about the evidence-based recommendations in the AAN ALS Practice Parameter.
We found that 16% of patients were moderately to severely depressed according to the graded response to the question on the Sickness Impact Profile. The reported prevalence of moderate to severe depression in ALS patients ranges from 10 to 75%; the more recent studies have reported the lower prevalences.7-12⇓⇓⇓⇓⇓ In our study, fewer than half of patients with depression were taking antidepressant medications.
Only 30% of patients with significant dysphagia had a gastrostomy tube. Only 12% of those with FVC <50% were using a gastrostomy tube. The practice parameter recommends placement of a gastrostomy tube early after onset of dysphagia and before FVC falls below 50%. This indicates that there may be additional benefit from public and professional education of the benefits of maintaining nutrition in ALS with a gastrostomy tube, as well as the critical issues related to timing of gastrostomy tube placement and safety. The risk of gastrostomy tube placement increases when the FVC falls below 50% and rises markedly when the FVC is below 30%.
Less than 10% of patients with an FVC less than 40% predicted, and only 28% of those with severe dyspnea, were using NIPPV support. The practice parameter recommends instituting NIPPV support when FVC falls below 50% of predicted. This discrepancy between the AAN Practice Parameter recommendations and the observed practice patterns in the ALS CARE Database with respect to managing this aspect of respiratory care is probably the most significant finding of this study. This low figure may result from some patients not tolerating NIPPV. However, there appears to be a need for more education of clinicians and patients about the benefits of NIPPV support earlier in the course of the disease. Three studies have now indicated a lengthening of survival with the use of NIPPV support in ALS, as well as an improvement in quality of life.13-15⇓⇓
The finding that less than 4% of patients were using a ventilator and/or a tracheostomy reflects the relatively small fraction of patients who opt to prolong life in ALS with invasive ventilation when respiratory failure develops. The reasons for this may be many, including the patient’s concerns about quality of life and the great financial and overall burden placed on the caregiver, as well as whether the patient has insurance and is provided with the information to select ventilator support. Anecdotal evidence suggests that the attitude of the counseling neurologist may play a significant role in influencing this outcome, and there is a need for a systematic study of this factor. The finding from the ALS CARE Database that only a small proportion of patients with ALS use a ventilator with tracheostomy supports the recent report of Albert, Murphy, Del Bene, et al.16
The AAN Practice Parameter for ALS recommends referral to hospice services in the terminal stages. We found that approximately half of patients in the ALS CARE Database who died did so at home, whereas only approximately half received home hospice services. Compared with the report that 29% of all patients dying in the United States receive hospice services,17 it appears that patients with ALS may be receiving better than average access to hospice care. However, there is still a role for public and professional education about the availability and benefits of home hospice services for patients with ALS.
The use of advance directives appears to be widespread and appropriate in the care of patients with ALS. A recent survey showed that 75% of ALS patients of neurologists specializing in ALS received advance directives, compared with only 55 to 57% for patients of general neurologists and neuro-oncologists.18
In the current era, the public is becoming comfortable about discussing “peaceful dying.”19 It appears that the goal of a peaceful death was achieved for most of the patients in the ALS CARE Database: 89% of patients were recorded as having died peacefully. However, a review of the symptoms recorded by the surviving caregivers indicates that there may be room for improvement. The AAN ALS Practice Parameter recommends the use of anxiolytics, oxygen, and where necessary morphine by enteral or parenteral routes to control the symptoms of dying with ALS. Two-thirds of those who died in the ALS CARE Database received medications to control pain and distress. Oxygen was received by 37% in the terminal phase. It will be interesting to determine whether dissemination of the AAN Practice Parameter for the management of ALS reduces the proportion of patients dying with respiratory difficulty, anxiety, fear, and pain.
We have compared the management of patients with ALS in North America in the 3-year period before the publication of the recommendations of the AAN ALS Practice Parameter using data extracted from the ALS CARE Database (see the table on the Neurology Web site; go to www.neurology.org). Information is not available in the database regarding some important issues in the practice parameter. There is a need to expand the questionnaires of the database program to include more of the relevant items, and this revision is under way. Nevertheless, comparison of the data that we have already extracted with the data collected over the next few years will allow evaluation of changes in the management of ALS in North America that result from dissemination of the AAN ALS Practice Parameter.
Appendix
The ALS CARE Study Group: Robert G. Miller, MD (Chair); Frederick Anderson, PhD; Walter G. Bradley, DM, FRCP; Mark B. Bromberg, MD; Benjamin R. Brooks, MD; Neil Cashman, MD; Lora Clawson, MSN, CRNP; Merit Cudkowicz, MD; Dallas A. Forshew, RN, BSN; Michael Graves, MD; Yadollah Harati, MD; Terry Heiman–Patterson, MD; Mary Lyon, RN, MN; Raul N. Mandler, MD; Hiroshi Mitsumoto, MD; Dan H. Moore, PhD; Steven P. Ringel, MD; Jeffrey Rosenfeld, MD, PhD; Mark A. Ross, MD; Michael J. Strong, MD; Robert L. Sufit, MD.
Acknowledgments
Acknowledgment
The authors thank Darleen Lessard, MS, Colleen Singleton, RN, MPH, and Matthew Davidson, PhD, for assistance with database management and statistical analyses.
Footnotes
-
Additional material related to this article can be found on the Neurology Web site. Go to www.neurology.org and scroll down the Table of Contents for the August 14 issue to find the title link for this article.
-
↵*See the Appendix on page 504 for a complete list of the investigators in the ALS CARE Study Group.
- Received October 30, 2000.
- Accepted March 27, 2001.
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