Quality of Life and Psychosocial Issues in Ventilated Patients with Amyotrophic Lateral Sclerosis and Their Caregivers

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1 890 Journal of Pain and Symptom Management Vol. 26 No. 4 October 2003 Original Article Quality of Life and Psychosocial Issues in Ventilated Patients with Amyotrophic Lateral Sclerosis and Their Caregivers Dagmar Kaub-Wittemer, MD, Nicole von Steinbüchel, PhD,* Maria Wasner, MA, Gerhard Laier-Groeneveld, MD, and Gian Domenico Borasio, MD Interdisciplinary Palliative Care Unit and Department of Neurology (D.K.-W., M.W., G.D.B.) and Institute of Medical Psychology (N.v.S.), Ludwig-Maximilians University, Munich; and Department of Internal Medicine (G.L.-G.), Helios Hospital, Erfurt, Germany Abstract Non-invasive ventilation (NIV) is an efficient palliative measure for symptoms of chronic hypoventilation in patients with amyotrophic lateral sclerosis (ALS), and can also lengthen survival. A subset of ALS patients undergoes tracheostomy ventilation (TV) for life prolongation. We investigated the quality of life (QOL) and psychosocial situation of 52 home ventilated ALS patients and their caregivers.the battery included sociodemographic, generic, and disease-specific variables, as well as the Profile of Mood States and the Munich Quality of Life Dimensions List. Data were compared between the NIV (n 32) and the TV (n 21) groups. Mean ventilation time was 14 months for NIV and 35 months for TV. Eighty-one percent of TV patients had been tracheotomized without informed consent. The data show a good overall QOL for both NIV and TV patients, but a very high burden of care for TV caregivers, 30% of whom rated their own QOL lower than their patient s QOL. Sexuality was an important issue. Thus, any assessment of QOL in a home palliative care situation should include the primary caregivers. J Pain Symptom Manage 2003;26: U.S. Cancer Pain Relief Committee. Published by Elsevier Inc. All rights reserved. Key Words Amyotrophic lateral sclerosis, motor neuron disease, quality of life, non-invasive ventilation, tracheostomy, caregivers, sexuality Introduction Amyotrophic lateral sclerosis (ALS) can be considered a paradigm disease for nononcolog- Address reprint requests to: Gian Domenico Borasio, MD, Interdisciplinary Palliative Care Unit and Department of Neurology, Munich University Hospital Grosshadern, D Munich, Germany. *Present address: Neurogerontopsychology Unit, University of Geneva, Switzerland. Accepted for publication: March 1, U.S. Cancer Pain Relief Committee Published by Elsevier Inc. All rights reserved. ical palliative care. 1 It is the most common degenerative motor neuron disorder in adulthood, with an incidence of around 1.5 2/ 100,000/year and a prevalence of around 6 8/100, Progressive paralysis of all voluntary muscles, coupled with muscular atrophy and spasticity, results in an increasing need for care. Patients suffer from loss of ambulation, dysarthria, and dysphagia, and eventually die of respiratory failure within 3 to 5 years from onset. There are no sensory, autonomic, or cognitive /03/$ see front matter doi: /s (03)

2 Vol. 26 No. 4 October 2003 QOL in Ventilated ALS Patients and Their Caregivers 891 disturbances. 3 Thus, patients witness their unstoppable physical decline with a fully clear mind. The timing and expression of the symptoms is quite variable. Almost all ALS patients develop respiratory symptoms during the course of their disease. The most common cause of death is terminal respiratory insufficiency secondary to diaphragmatic paralysis. 4 However, ALS patients suffer long before death from symptoms of chronic hypoventilation, which can severely hamper their quality of life (Table 1). These symptoms can be efficiently relieved by noninvasive intermittent mechanical ventilation via mask (NIV). 5,6 This palliative measure is now recognized as a standard in ALS care, 7 and is also used in several other neurological and pulmonological disorders. A number of ALS patients undergo tracheostomy for life prolongation when they reach the stage of terminal respiratory failure. 8 Several studies have reported increased survival with mechanical ventilation in ALS. 9,10 However, there are few data available on the quality of life (QOL) of ventilated ALS patients. 11,12 QOL assessment in ALS poses a particular challenge, because recent studies have shown that the individual, patient-perceived QOL of ALS patients does not correlate with physical function 13,14 and is, therefore, unlikely to be accurately reflected by scales which focus on the general health status of the patient, such as the ALS Assessment Questionnaire 40 (ALSAQ-40). 15 A disease-specific individual QOL instrument for ALS is not available. Importantly, at earlier disease stages, distress levels of patients with ALS and their caregivers show a Table 1 Symptoms of Chronic Hypoventilation in ALS Difficulty falling asleep, frequent awakenings, nightmares Daytime fatigue and sleepiness Morning headache Concentration problems Nervousness, hyperhidrosis, tremor Depression, anxiety Tachycardia and tachypnea Visible efforts of auxiliary respiratory muscles Dyspnea, voice disorder Thick mucous secretions Recurrent or chronic upper respiratory tract infections Reduced appetite, weight loss Edemas Diffuse pain in head, neck and extremities Vision disturbances, dizziness, syncope Cyanosis close association. 16 In this study, we investigated the QOL of ventilated ALS patients and their caregivers using a cross-sectional approach. Methods Two separate disease-specific questionnaires were developed, one for the patient and one for the primary caregiver. The patients battery consisted of 584 items covering demographics, disease state and symptoms, ventilation type and time course, home and professional situation, partnership, and social life. In addition, two validated QOL instruments were used: the Profile of Mood States (POMS) 17 and the Munich Quality of Life Dimensions List (MLDL). 18 The POMS assesses feelings and emotional states in 34 items, which are summarized into four domains (depression, fatigue, vigor, anger). The self-rating scale ranges from 0 (not at all) to 4 (very strong). The MLDL assesses the satisfaction with generic aspects (physical condition, psyche, social life, everyday life) of QOL with regard to the week prior to the date of survey. The answer scale ranges from 1 (not satisfied at all) to 10 (very satisfied). The battery for the primary caregivers contained 274 items on the subjects of ventilation and patient care, home and personal situation, partnership, psychological and physical burden, as well as the POMS and the MLDL. At the end of both questionnaires, the respondents were asked to assess on a numeric analogue scale (0 10) their own overall QOL and the QOL of the respective other. The questionnaires were mailed by the German Association for Neuromuscular Disease (DGM) to ALS patients all over Germany who were recorded on file to be ventilated. In addition, they were distributed by several centers in Germany administering ventilation to their ALS patients on follow-up visits. Ventilation was defined as either non-invasive positive pressure ventilatory support via nasal or nasofacial mask (NIV) or tracheostomy ventilation (TV). The survey was anonymous, the questionnaires were coded and the names and addresses of the patients were not known to the investigators. The patients had the option to indicate their name and address on the questionnaire if they so wished. A telephone number

3 892 Kaub-Wittemer et al. Vol. 26 No. 4 October 2003 for queries was provided. Patients and caregivers were advised to complete their questionnaires independently from each other. The study was conducted in accordance with German data protection laws and the regulations of the Ethical Committee of the University of Munich. The data were analyzed using a combined Access, Excel, and SPSS database. Patients were divided into two groups according to the mode of ventilation: non-invasive ventilation (NIV) or invasive (tracheostomy) ventilation (TV). For statistical comparisons the Mann Whitney U-test, the Chi square test, and the Fisher test were employed where appropriate. Results A total of 52 of questionnaire pairs (plus one single questionnaire from a tracheotomized patient, which was included in the analysis) out of 102 sent out were completed and returned (response rate 52%). No independent verification of the diagnosis of ALS could be performed in the respondent population due to the anonymous nature of the survey. The detailed symptoms reported by the patients in the questionnaire were consistent with the diagnosis of ALS 19 in all cases. All patients had been on ventilatory support for at least one month at the time of completion of the survey. Thirty-two patients were ventilated via NIV, 21 via TV. The demographics of the two groups are shown in Table 2 and correspond to the data expected for a population of patients with advanced ALS. All caregivers were spouses except for one daughter, and all took care of the patient at home. There was a marked predominance of male patients (19/21) in the TV Table 2 Demographics TV NIV (n = 21 pts) (n = 32) (n = 20 caregivers) Patients Age (years) 60.0 (46 74) 61.6 (47 82) Sex 23 M (72%) 19 M (91%) Months on 13.8 (1 52) 34.6 (1 108) P 0.01 ventilation Caregivers Age (years) 57.8 (35 77) 55.0 (35 69) Sex 24 F (75%) 18 F (90%) Relationship 31 spouse 20 spouse to patient 1 daughter group. All but one TV patients (vs. 6% of the NIV patients) were ventilated 24 h/day, and only one TV patient (5%) reported problems with the ventilator. Of the NIV patients, 88% used a nasal mask, and 9% a combined nasofacial mask. One NIV patient reported technical problems with the ventilator, and five (16%) reported problems with the mask. There was a significant difference between the two groups with regard to information prior to the beginning of ventilation: only 33% of the TV patients had been informed in advance about the impending respiratory failure, compared with 84% of the NIV patients (P 0.001). Eighty-one percent of the TV patients underwent tracheotomy without prior informed consent. Even among NIV patients, 34% deemed their information status on the disease to be insufficient. At the time of the survey, one-third of the NIV patients had already made a decision regarding future treatment escalation, and all except one had decided against progression to TV. The foreseeable course of the terminal phase had been discussed with 56% of the NIV and 29% of the TV patients. The scales used to assess QOL did not show any differences between the NIV and the TV groups. Both in the POMS (Figure 1) and the MLDL (Figure 2), the values were very similar between groups for both patients and caregivers. When asked directly, 94% of NIV patients and 81% of TV patients would choose ventilation again and over 80% (NIV 88%, TV 81%) would advise other patients to do the same. On the other hand, 97% of the NIV caregivers would advise their patient to choose ventilation again, but only 75% of the TV caregivers would do so (P 0.008). If faced with the same decision for themselves, 94% of the NIV caregivers would opt for ventilation, compared with 50% of the TV spouses (P 0.001). Strikingly, 30% of the TV caregivers rated their own overall QOL lower than that of their patient. Several other data indicating the huge burden of care for the primary caregivers of ventilated ALS patients are summarized in Table 3. Of those caregivers attending support groups, the vast majority perceived them as helpful (NIV 12/13, 92%; TV 6/9, 67%). Sexuality is still an important issue for a high proportion of ventilated ALS patients (NIV 56%, TV 43%), less so for the caregivers (NIV 41%, TV 20%). Sexual activity is reported to be still

4 Vol. 26 No. 4 October 2003 QOL in Ventilated ALS Patients and Their Caregivers 893 Fig. 1. POMS data from ventilated ALS patients and their caregivers. Note the low levels of self-reported depression and anger for both patients and caregivers. NIV non-invasive ventilation; TV tracheostomy ventilation. possible by about one-third (31%) of NIV couples, but only by three TV patients and one TV caregiver. Financial considerations were not a huge issue: only a minority of patients reported outof-pocket expenses for home care (NIV: four patients, 230 1,900 Euros/US $ per month; TV: five patients, 200 5,000 Euros/US $ per month). All patients were eligible for the federal nursing care insurance which is mandatory in Germany. Discussion In this cross-sectional survey, generic and disease-specific aspects of the QOL of ventilated ALS patients and their primary caregivers were assessed. The sex distribution showed a very high proportion of men in the TV group (90%). Because most tracheotomies were performed in an emergency situation, this seems to indicate that heroic life-prolonging measures are more likely to be initiated in men. Interestingly, Ganzini and co-workers showed a male preponderance among ALS patients who would consider undergoing physician-assisted suicide. 20 A possible reason for the higher frequency of tracheostomies in men could be that women are generally more willing caregivers. At the time of survey, the mean duration of ventilation was considerably higher for TV patients (35 months vs. 14 months for NIV, P 0.01). NIV, although proven to increase survival, 9,10 is usually initiated well in advance of the terminal phase as a palliative treatment for symptoms of chronic hypoventilation, and the patient decides on frequency and duration of use. On the other hand, TV is usually initiated at the onset of terminal respiratory insufficiency as a life-prolonging treatment. A voluntary Fig. 2. MLDL data from ventilated ALS patients and their caregivers. As for the POMS, there were no significant differences between the NIV and TV groups. NIV non-invasive ventilation; TV tracheostomy ventilation.

5 894 Kaub-Wittemer et al. Vol. 26 No. 4 October 2003 Table 3 Caregivers Burden of Care NIV (n 32) TV (n 20) Time spent 12.6 hr/ 14.4 h/ on care day (2 24) day (2 24) Wakings/night 2.3 (0 8) 2.4 (0 15) Health 63% 70% problems Quit working 19% 60% P due to ALS ending of TV is still a problem in clinical practice. 21 This may partly explain the difference in ventilation time. Interestingly, only two of the TV patients had originally progressed from NIV to TV, and conversely, of those NIV patients who had already made a decision on future treatment, all but one had opted against progression to TV. This is in accord with published data showing that, when informed in advance about the available treatment options for the terminal phase, the vast majority of ALS patients on NIV refuse tracheostomy. 5 The general level of patient information in our study was poor, particularly in the TV group. Information on the terminal phase was given only to slightly more than half of the NIV and one-quarter of the TV patients. These data are consistent with a recent survey on standards of palliative care in ALS in Europe. 22 We have previously shown that the natural course of the terminal phase in ALS is a peaceful one, and that fears of choking to death are unwarranted. 4 This information should be available to ALS patients at the time when ventilatory options are discussed. 23 The subject of QOL assessment in palliative care has received considerable attention in the last years. We have recently shown that ALS patients prefer measures looking at QOL domains generated by the individual patient, such as the Schedule for the Evaluation of Individual Quality of Life - Direct Weighting (SEIQoL-DW) over traditional, health-related QOL scales. 13 Because the SEIQoL-DW is administered as a structured interview, this approach was not feasible for a nationwide, anonymous survey. The scales we chose (POMS and MLDL) look at several different domains of QOL, with an emphasis on psychological factors and subjective satisfaction with QOL. Interestingly, although no differences between the NIV and the TV group were detected with these generic scales for either the patients or the caregivers, additional disease-specific questions indicated a significantly greater burden of care for TV spouses, which is consistent with published data. 12 This raises the issue of social desirability bias when caregivers complete scales asking for self-rating of their psychological state and QOL. The higher burden for TV caregivers which emerges from the additional questions could be due to several factors, including 1) the longer mean ventilation period, 2) the more severe disease state of the patient, and 3) the feeling of having been trapped into a hopeless situation without any choice. The high percentage of ventilated ALS patients for whom sexuality was still an important issue was unexpected. This is still an understudied and possibly underestimated area in neurological palliative care. We are, therefore, currently conducting a prospective study on sexuality in ALS. This study has several limitations. Judged in retrospect, the number of items in the questionnaires was too high. The response rate was actually quite good (52%), yet the possibility of a bias is present. We hypothesize that the burden of care may be higher and the overall QOL may be lower for the non-respondent families. This needs to be addressed in a prospective study. In addition, the absolute number of patients in both groups was quite small, which reflects the limited availability of NIV and TV for ALS patients, both in Germany and worldwide. 24 Moreover, the QOL scales used may not have been sensitive enough to detect differences between groups given the small sample size. Finally, this study was limited to German patients who have good access to nursing care and drug reimbursement compared, for example, to most patients in the U.S. 22,25 It is likely that financial considerations may play a higher role in other countries. Despite these limitations, we feel that our data provide some insights into the situation of ventilated ALS patients and their caregivers. This is an example of a palliative care situation in neurology requiring high-tech intervention as well as skilled nursing care, both of which are not easily available. We draw the following conclusions from our data: 1. NIV a measure aimed primarily at relieving symptoms, which can also lengthen survival shows good acceptance for both

6 Vol. 26 No. 4 October 2003 QOL in Ventilated ALS Patients and Their Caregivers 895 patients and caregivers, and both groups rate their QOL relatively high. 2. TV, which is most often initiated in an emergency situation without the patient s prior informed consent, seems to result in a good generic QOL for the patients, but at the price of a very high burden for their caregivers. 3. Given the foreseeable course of the disease, improvement of patient information through physician education and increased use of advance directives/health care proxies are of paramount importance in ALS. We advocate a stepwise approach to ventilation, offering NIV early and progressingtotvonlyinselectedcasesafter careful discussion. 4. A concise, disease-specific QOL measure for ALS needs to be developed which does not focus solely on health status. 13 More generally, any assessment of QOL in a palliative care situation where the patients are cared for at home should include the primary caregivers in order to get the full picture. Acknowledgments The authors thank the German Society for Neuromuscular Disorders (DGM), as well as Dr. M. Winterholler, for help in the distribution of the questionnaires. Their warmest thanks also go to the patients and their caregivers for their willingness to participate in the study. References 1. Borasio GD, Voltz R, Miller RG. Palliative care in amyotrophic lateral sclerosis. Neurol Clin 2001; 19: Brooks BR. Clinical epidemiology of amyotrophic lateral sclerosis. Neurol Clin 1996;14: Borasio GD, Miller RG. Clinical characteristics and management of ALS. Sem Neurol 2001;21: Neudert C, Oliver D, Wasner M, Borasio GD. The course of the terminal phase in patients with amyotrophic lateral sclerosis. J Neurol 2001;248: Schlamp V, Karg O, Abel A, et al. Non-invasive intermittent home mechanical ventilation as a palliative treatment in amyotrophic lateral sclerosis. Nervenarzt 1998;69: Cazzolli PA, Oppenheimer EA. Home mechanical ventilation for amyotrophic lateral sclerosis: nasal compared to tracheostomy-intermittent positive pressure ventilation. J Neurol Sci 1996;139(Suppl): S123 S Lyall R, Moxham J, Leigh N. Dyspnea. In: Oliver D, Borasio GD, Walsh D, eds. Palliative care in amyotrophic lateral sclerosis (motor neurone disease). Oxford: Oxford University Press, 2000: Gelinas DF. ALS and invasive ventilation. In: Oliver D, Borasio GD, Walsh D, eds. Palliative care in amyotrophic lateral sclerosis (motor neurone disease). Oxford: Oxford University Press, 2000: Aboussouan LS, Khan SU, Meeker DP, et al. Effect of noninvasive positive-pressure ventilation on survival in amyotrophic lateral sclerosis. Ann Intern Med 1997;127: Kleopa KA, Sherman M, Neal B, et al. Bipap improves survival and rate of pulmonary function decline in patients with ALS. J Neurol Sci 1999;164: Lyall RA, Donaldson N, Fleming T, et al. A prospective study of quality of life in ALS patients treated with non-invasive ventilation. Neurology 2001;57: Gelinas DF, O Connor P, Miller RG. Quality of life for ventilator-dependent ALS patients and their caregivers. J Neurol Sci 1998;160(Suppl 1):S134 S Neudert C, Wasner M, Borasio GD. Patients assessment of quality-of-life instruments: a randomized study of SIP, SF-36 and SEIQoL-DW in patients with amyotrophic lateral sclerosis. J Neurol 2001;191: Simmons Z, Bremer BA, Robbins RA, et al. Quality of life in ALS depends on factors other than strength and physical function. Neurology 2000;55: Jenkinson C, Fitzpatrick R, Brennan C, et al. Development and validation of a short measure of health status for individuals with amyotrophic lateral sclerosis/motor neuron disease: the ALSAQ-40. J Neurol 1999;246(Suppl 3):S16 S Rabkin JG, Wagner GJ, Del Bene M. Resilience and distress among amyotrophic lateral sclerosis patients and caregivers. Psychosom Med 2000;62: McNair D, Lorr M, Droppleman LF. The Profile of Mood States. San Diego: Educational and Industrial Testing Services, von Steinbüchel N, Bullinger M, Kirchberger I. Die Entwicklung und Prüfung eines Verfahrens zur krankheitsübergreifenden Erfassung der Lebensqualität. Zschr Med Psychol 1999;3: Brooks BR. El Escorial World Federation of Neurology criteria for diagnosis of amyotrophic lateral

7 896 Kaub-Wittemer et al. Vol. 26 No. 4 October 2003 sclerosis. Subcommittee on Motor Neuron Diseases/ Amyotrophic Lateral Sclerosis of the. World Federation of Neurology Research Group on Neuromuscular Diseases and the El Escorial Clinical limits of amyotrophic lateral sclerosis workshop contributors. J Neurol Sci 1994;124(Suppl):S96 S Ganzini L, Johnson WS, Mc Farland BH, et al. Attitudes of patients with amyotrophic lateral sclerosis and their caregivers towards assisted suicide. New England J Med 1998;339: Borasio GD, Voltz R. Discontinuation of mechanical ventilation in amyotrophic lateral sclerosis. J Neurol 1998;245: Borasio GD, Shaw PJ, Hardiman O, et al. Standards of palliative care for patients with amyotrophic lateral sclerosis: results of a European survey. Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders 2001;2: Borasio GD, Sloan R, Pongratz DE. Breaking the news in amyotrophic lateral sclerosis. J Neurol Sci 1998;160(Suppl 1):S Borasio GD, Gelinas DF, Yanagisawa N. Mechanical ventilation in ALS: a cross-cultural perspective. J Neurol Sci 1998;160(Suppl 2):S7 S Moss AH, Casey P, Cazzolli PA, et al. Patients with amyotrophic lateral sclerosis receiving long-term mechanical ventilation. Advance care planning and outcome. Chest 1996;110: