Amyotrophic Lateral Sclerosis
Physician Update, Winter 2004
by Leo
McCluskey, MD
Medical Director, ALS Assn. Center, Penn Neurological
Institute,
Assistant Professor of Neurology, University
of Pennsylvania
Introduction
Amyotrophic Lateral Sclerosis (ALS) is a progressive
neuromuscular disorder that results from the
variable loss of motor neurons in the precentral
gyrus (upper motor neurons) of the frontal
lobe, some brainstem nuclei (lower motor neurons)
and the anterior grey matter of the spinal
cord (lower motor neurons). Heralding symptoms
of the disease depend upon the nervous system
segment of onset (cranial, cervical, thoracic
or lumbosacral) and the relative proportion
of upper and lower motor neuron cell loss.
The
overall clinical course is determined by the
pattern and rapidity of segment-to-segment
spread. While ALS nearly inevitably results
in neuromuscular respiratory failure, malnutrition
and dehydration, or both, patients with initial
or early involvement of bulbar or respiratory
motor neurons face life-threatening symptoms
before the development of significant limb
weakness.(1) Gastrostomy
tube insertion can ameliorate nutritional and
hydration needs while relieving suffering associated
with dysphagia.
Those electing to forego gastrostomy
insertion may succumb to malnutrition and dehydration.
Non-invasive positive pressure ventilation
(NIPPV) can temporarily relieve dyspnea, orthopnea
and symptoms of sleep-disordered breathing.
However, progressive respiratory muscle weakness
eventually causes nearly all ALS patients to
face a choice between tracheostomy and mechanical
ventilation or comfort care. The majority of
ALS patients in the United States exercise
the latter option.
The 1990's in the United States was characterized
by both an increase in the number of hospices
and the number of patients of all types utilizing
hospice services. At our center, the ALS Association
Center at the Penn Neurological Institute, nearly
all patients who elect palliative care at the
end of life enroll in hospice. Nationally, it
is uncertain how many ALS patients utilize the
hospice benefit. The patients who participated
in the ALS Patient CARE database, a self reporting
database of patients and caregivers, utilized
hospice 50-60% of the time.(2,
3) Others, however, have commented on the
underutilization of hospice services by ALS patients
and the need to increase awareness of both patients
and clinicians regarding the effectiveness of
hospice care for advanced ALS patients. This
is particularly important since the variable
presentation and progression of ALS results in
an array of end-of- life problems that is particularly
well suited to the interdisciplinary, multi-modality
setting of comprehensive hospice care.(4)
A Need for Change?
Why some ALS patients fail to take advantage
of hospice care is uncertain. The Medicare
ALS guidelines for hospice care may be one
reason. To be eligible for hospice ALS patients
must [1] demonstrate critically impaired breathing
or exhibit rapid progression and [2] have critical
nutritional impairment or exhibit rapid progression
and [3] have life threatening complications.
In our opinion hospice eligibility criteria
should accurately reflect disease specific
end of life physiology and allow for a survival
of six months or less. For ALS, such eligibility
guidelines should emphasize symptoms indicative
of impending neuromuscular respiratory failure
and/or dehydration and malnutrition while allowing
most if not all ALS patients deemed appropriate
for hospice enrollment by an
experienced clinician eligible for hospice services.
To gauge the accuracy of the present Medicare
guidelines we recently undertook a retrospective
review of 97 consecutive ALS patients referred
to hospice care from our ALS center (1999-2002).
Patients were referred to hospice if they agreed
to hospice care, had one or more permanent caregivers,
manifested critically impaired respiratory dysfunction
and did not elect for mechanical ventilation
via tracheostomy or suffered from severe nutritional
deficiency. Use of NIPPV and a gastrostomy tube
did not affect referral. We defined severe nutritional
deficiency as dysphagia with progressive weight
loss of at least five percent with or without
election for gastrostomy tube insertion. The
characteristics of each patient on date of hospice
enrollment or on the date of the most proximate
clinic visit were used to assess if patients
met Medicare criteria.
Only five (5.2%) of the 97 ALS patients met
Medicare hospice guidelines at the time of enrollment.
The mean number of days on hospice was 84 (minimum
1, maximum 534). Eighty-eight patients (91%)
stayed in hospice for less than 180 days.
Two patients met criterion 1. While 52% of patients
had dyspnea at rest, 41% had an FVC <30% and
nearly all elected not to pursue invasive ventilation,
91% did not use supplemental oxygen. Lack of
supplemental oxygen use was the most common reason
why the patients did not meet Medicare criteria
for critically impaired breathing capacity.
Two of our patients met criterion 2. To earn
the label of rapid progression, patients must
manifest progression from independent ambulation
to wheelchair or to bed bound status, progression
from normal to barely intelligible or unintelligible
speech, progression from a normal to a pureed
diet and progression from independence in most
Activities of Daily Living to needing major assistance
by a caretaker in most or all ADLs. While one
or more of these phenomena were present in any
one individual, it was unusual for all to be
present. As a result, many of our patients did
not fulfill Medicare's definition for rapid progression.
All but two patients who did meet the guidelines
for rapid progression failed to meet the definition
of critical nutritional impairment necessary
to meet the second criterion. Medicare's guidelines
for nutritional impairment are weighted in favor
of pre-terminal patients. While 75% of our patients
at the time of hospice enrollment were losing
weight, only 20% were considered to have insufficient
oral intake to sustain life, 86% were not dehydrated
and 51% were using a gastrostomy tube.
To fulfill Medicare's third category, ALS patients
must manifest both rapid progression (discussed
above) and life threatening complications. While
three of our 97 patients developed a single episode
of aspiration pneumonia only one experienced
a recurrence. No patients developed any other
life threatening complications.
Our experience indicates that while one or
more of the features of each of the three Medicare
hospice criteria may be present in an ALS patient
it is distinctly unusual for all to be represented.
The Medicare requirement that patients fulfill
each one of multiple respiratory guidelines or
guidelines defining both rapid progression and
critical nutritional impairment or guidelines
defining both rapid progression and life-threatening
complications places the bar for hospice eligibility
too high. While unfortunate, loss of ability
to ambulate and to speak, loss of the ability
to independently perform ADLs, or loss of the
ability to eat anything but a pureed diet are
not life threatening features of ALS. Sepsis,
upper urinary tract infection and recurrent aspiration
are clearly life threatening but, in our experience,
extraordinarily rare. Patients who are unable
to take in nutrients and fluids sufficient to
sustain life, who manifest dehydration and hypovolemia
and who do not have a gastrostomy tube or other
feeding method are clearly in a terminal phase.
This criterion clearly tilts towards those nearing
death within days to weeks rather than months.
Symptom Management
Progressive loss of motor neurons produces a
panoply of weakness related symptoms that require
management at the end of life. While it is
possible to effectively manage ALS related
symptoms and thereby improve function and quality,
a mixed picture emerges from review of studies
dealing with management of symptoms at the
end of life. Bradley reported that although
89% of ALS patients within the ALS Care Database
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.(2) Ganzini
reported that the most common symptoms in the
last month of life included difficulty communicating
(62%), dyspnea (56%), insomnia (42%), and discomfort
other than pain (48%). Two-thirds of patients
were enrolled in hospice and these patients
were significantly more likely to: 1) die in
their preferred location; 2) die outside the
hospital; and 3) receive morphine.(5) Mandler's
study of 1014 American and Canadian patients
with ALS who died while participating in the
ALS Patient Care Database during a four year
period found that most patients died peacefully
(90.7%) and 62.4% died in a hospice-supported
environment. Only 67 patients exhibited distress
in the dying process with symptoms that included
breathing difficulties (82.1%), fear/anxiety
(55.2%), pain (23.9%), insomnia (14.9%), and
choking (14.93%). Oxygen was given to 52.6%
of patients, and pain medications were given
to 74%.(3)
Table 1 summarizes available treatments for
the various difficulties that ALS can produce.
Since ALS is such a variable process, individual
patients may require unique combinations of interventions.
ALS and the End of Life
At present it can be surmised that the current
guidelines, if strictly applied, will pose
a significant obstacle to hospice admission
for ALS patients. However, despite the potential
difficulties posed by the present Medicare
criteria, it has been our experience that Philadelphia
area hospices are willing to accept ALS patients
whom we have deemed hospice appropriate.
References
1.Rowland, L.P. and N.A. Shneider,
Amyotrophic lateral sclerosis. [see comments.].
[Review] [148 refs]. New England Journal of
Medicine. 2001 May 31;344(22):1688-700.
2.Bradley,W.G., et al., Current management
of ALS: comparison of the ALS CARE Database
and the AAN Practice Parameter. The American
Academy of Neurology. Neurology. 2001 Aug
14;57(3):500-4.
3.Mandler, R.N., et al., The ALS Patient
Care Database: insights into end-of-life
care in ALS. Amyotrophic Lateral Sclerosis & Other
Motor Neuron Disorders. 2001 Dec;2(4):203-8.
4.Carter, G.T., et al., Expanding the role
of hospice care in amyotrophic lateral sclerosis.
[see comments.]. [Review] [22 refs]. American
Journal of Hospice & Palliative Care.
1999 Nov-Dec;16(6):707-10.
5.Ganzini, L., W.S. Johnston, and M.J. Silveira,
The final month of life in patients with
ALS. Neurology. 2002 Aug 13;59(3):428-31.
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