NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE 1 Guideline title SCOPE Motor neurone disease: the use of non-invasive ventilation in the management of motor neurone disease 1.1 Short title Motor neurone disease non-invasive ventilation 2 The remit The Department of Health has asked NICE: To produce a short clinical guideline on the use of non-invasive ventilation in the management of motor neurone disease. 3 Clinical need for the guideline 3.1 Epidemiology a) Motor neurone disease (MND) is a neurodegenerative disorder characterised by signs of degeneration of upper and lower motor neurones (nerves that control movement). The degeneration leads to progressive weakness of muscles in the throat that are used in speaking, breathing and swallowing (bulbar muscles), and of muscles in the limbs, thorax (chest) and abdomen. It is also known as amyotrophic lateral sclerosis (ALS). b) The annual incidence of MND is approximately 2.9 per 100,000 people and men are slightly more commonly affected than women (1.7 to 1 ratio). The incidence increases with age, with a mean age of onset of 63 years. It is estimated that there might be up to 5000 people with MND in England and Wales. Motor neurone disease non-invasive ventilation final scope October 2009 Page 1 of 8
c) MND causes significant morbidity. People with MND have symptoms that are directly related to the disease process (for example, weakness, wasting, muscle cramps, difficulty in communicating, breathlessness, difficulty swallowing, excessive salivation and mood swings) and symptoms that are indirectly related (for example, depression, anxiety, fatigue and constipation). MND is a progressive condition: after the onset of symptoms about 50% of people live for 3 years and 10% of people for 5 years, although some live beyond 10 years. 3.2 Current practice a) The care of people with MND is provided by multidisciplinary teams that may involve primary, secondary or tertiary care staff. The team provides diagnostic and management services and promotes continuity of care. People newly diagnosed with MND need support to help them to come to terms with the diagnosis. As the disease progresses they must cope with significantly impaired function and increasing disability. Palliative care, including end of life care, is an important component of managing MND. Advance directives are also widely used. The involvement and support of family and carers is important in all of these stages. b) Symptom management is a core component of care. Symptoms are treated as they become significant in individual patients. A wide range of symptoms directly and indirectly associated with the disease process needs to be managed, notably: breathlessness and respiratory impairment, swallowing difficulties, eating problems, excessive salivation and musculoskeletal pain. c) Respiratory muscle weakness leading to respiratory impairment is a major feature of MND and can present with a range of symptoms and signs. Respiratory muscle strength could also be a strong predictor of quality of life for people with MND. Symptoms of respiratory impairment include shortness of breath (on exertion, at rest and on lying flat), difficulty clearing secretions, unrefreshing Motor neurone disease non-invasive ventilation final scope October 2009 Page 2 of 8
sleep, excessive daytime sleepiness and fatigue. Difficulties in breathing during sleep (sometimes called 'sleep-disordered breathing') can also cause poor appetite and weight loss, difficulty in communication and headaches. Signs include an increased breathing rate and using extra muscles when breathing in and out. If a person is thought to have difficulty breathing, further investigations can be carried out to confirm and determine the extent of the impairment. Tests that may be used include forced vital capacity (FVC), sniff nasal inspiratory pressure (SNIP), pulse oximetry and morning arterial blood gases. Non-invasive positivepressure ventilation (NIV) and, less commonly, invasive mechanical ventilation via tracheostomy (TV), are used in people with MND and breathing difficulties to increase blood oxygen levels and to alleviate respiratory symptoms, improve quality of life and prolong survival. d) NIV uses interfaces such as a mask or similar device to provide ventilator support. It is initially used for intermittent support to relieve symptoms of hypoventilation at night. As respiratory muscle strength declines, daytime NIV may become an option and some people may require continuous NIV. There is an expanding evidence base on the clinical effectiveness of NIV. Observational studies and a recently published UK-based randomised controlled trial (RCT) suggest that NIV can improve both survival and quality of life. e) NIV can be delivered through nasal masks, oronasal masks (masks that cover the mouth and nose) or mouthpieces, and can be controlled by a pressure-limited ventilator (bilevel positive airway pressure ventilator), a volume limited ventilator or a newer noninvasive ventilator such as a proportional assist ventilator (PAV). Bilevel positive airway pressure ventilator devices are commonly used by people with MND in the UK. The ventilators are available Motor neurone disease non-invasive ventilation final scope October 2009 Page 3 of 8
as small portable units with battery back-up and can be used at home and outside the home. f) The National Service Framework for Long Term Neurological Conditions recommends that people with long term neurological conditions should receive timely, appropriate assistive technology/equipment (such as NIV) to support them to live independently, help them with their care, maintain their health and improve their quality of life. However, access to NIV across England and Wales is variable and NIV is available to only a minority of people with MND. It is estimated that only around 5.5% of people with MND are currently receiving NIV. Not all people with MND will benefit from or want NIV, but it is thought that there is currently an unmet demand. g) There is currently no evidence-based clinical guideline for use in England, Wales and Northern Ireland that addresses the use of NIV in people with MND. 4 The guideline The guideline development process is described in detail on the NICE website (see section 6, Further information ). This scope defines what the guideline will (and will not) examine, and what the guideline developers will consider. The scope is based on the referral from the Department of Health. The areas that will be addressed by the guideline are described in the following sections. 4.1 Population 4.1.1 Groups that will be covered a) Adults (aged 18 and over) with a diagnosis of MND. Motor neurone disease non-invasive ventilation final scope October 2009 Page 4 of 8
b) The guideline will specifically consider the assessment of respiratory function and response to NIV in people with MND who have moderate or severe bulbar impairment. c) The guideline will also specifically consider assessment of respiratory impairment in people with MND who have severe cognitive impairment or dementia. 4.1.2 Groups that will not be covered a) Children (17 or younger) with a diagnosis of MND. b) Adults with other neurodegenerative disorders. c) Adults with respiratory impairment not related to MND. 4.2 Healthcare setting a) Primary care and community settings, secondary care and tertiary care. 4.3 Clinical management 4.3.1 Key clinical issues that will be covered a) Identification and assessment of respiratory impairment to determine who should be offered NIV and when they should be offered it. This will include an assessment of the following: Clinical symptoms and signs to predict and monitor respiratory impairment. Specific investigations to confirm, assess and monitor the severity of respiratory impairment. The following investigations will be considered: forced vital capacity (FVC) sniff nasal inspiratory pressure (SNIP) overnight/nocturnal pulse oximetry arterial blood gases venous bicarbonate and chloride Motor neurone disease non-invasive ventilation final scope October 2009 Page 5 of 8
peak cough expiratory flow Epworth Sleepiness Score/Questionnaire measurement of inspiratory (Pimax) and expiratory (Pemax) pressure transcutaneous PCO 2. Performance of screening and assessment tools for respiratory impairment that have been developed and/or validated in people who have MND. b) Clinical and cost effectiveness of NIV for respiratory impairment compared with standard care without NIV. c) Comparisons of the clinical and cost effectiveness of different masks and mouthpieces for NIV and different types of ventilator, if evidence is available. d) Ongoing management of the use of NIV for people who have difficulty breathing, decisions on the continuation or withdrawal of NIV including during end of life care. e) Specific information and support needs of people with MND and their carers relating to the use of NIV. 4.3.2 Clinical issues that will not be covered a) Diagnosis of MND. b) Management of other symptoms of MND. c) Management of adverse effects of NIV or its interfaces. d) Other forms of management of respiratory impairment including TV, unless they are being used as comparators for NIV. 4.4 Main outcomes a) Health related quality of life of people with MND and their carers. Ideally this will include data from validated generic instruments such as the EQ-5D that are able to provide a single index value of Motor neurone disease non-invasive ventilation final scope October 2009 Page 6 of 8
health status (on a scale of 0 to 1). Generic health survey questionnaire data, such as from the Short Form 36, may also be appropriate. b) Morbidity: this will include symptom relief, such as sleep disturbance and respiratory function. c) Tolerance and adherence. d) Adverse events of NIV. e) Survival. f) Frequency and duration of hospitalisation. g) Resource use and costs. 4.5 Economic aspects Developers will take into account both clinical and cost effectiveness when making recommendations involving a choice between alternative interventions. A review of the economic evidence will be conducted and analyses will be carried out as appropriate. The preferred unit of effectiveness is the quality-adjusted life year (QALY), and the costs considered will usually only be from an NHS and personal social services (PSS) perspective. Further detail on the methods can be found in 'The guidelines manual' (see Further information ). 4.6 Status 4.6.1 Scope This is the final scope. 4.6.2 Timing The development of the guideline recommendations will begin in October 2009. Motor neurone disease non-invasive ventilation final scope October 2009 Page 7 of 8
5 Related NICE guidance Continuous positive airway pressure for the treatment of obstructive sleep apnoea/hypopnoea syndrome. NICE technology appraisal guidance 139 (2008). Available from www.nice.org.uk/ta139 6 Further information Information on the guideline development process is provided in: How NICE clinical guidelines are developed: an overview for stakeholders the public and the NHS The guidelines manual. These are available from the NICE website (www.nice.org.uk/guidelinesmanual). Information on the progress of the guideline will also be available from the NICE website (www.nice.org.uk). Motor neurone disease non-invasive ventilation final scope October 2009 Page 8 of 8