Objectives To identify the number and current location of all children receiving long-term ventilation (LTV) in the UK, and to establish their underlying diagnoses and ventilatory requirements.
Design Single time-point census completed by members of the UK LTV working party using an electronic-based questionnaire
Subjects All children in the UK at home or in hospital who, when medically stable, continue to need a mechanical aid for breathing following a failure to wean beyond a 3-month period.
Results 933 children under the age of 17 years in 30 regional centres were identified as receiving LTV. 88 children (9.5%) required continuous positive pressure ventilation by tracheostomy over 24 h, while 658 received ventilation while asleep only. Most children are ventilated by a non-invasive mask (n=704; 75%) or tracheostomy (n=206; 22%). Underlying conditions included neuromuscular disease (n=402; 43%), chronic respiratory (n=343; 37%) and central nervous system conditions (n=168; 18%). 129 (14%) children were aged 16 or over. 844 (91%) children were cared for at home with only 49 children listed as being in acute hospital units (n=34) or paediatric intensive care units/high dependency units (n=15).
Conclusions The last 10 years has seen a very significant increase in the number of children requiring LTV in the UK with an increasing number cared for at home. This reflects both improving technology and increasing clinical expertise in paediatric non-invasive ventilatory support, and a continuing change in attitude towards long-term support, particularly in children with neuromuscular diseases. There are a substantial number of children who soon will require transition to adult services, yet few such services currently exist.
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In 1999, the BMJ published our survey of children with chronic respiratory failure who were receiving long-term ventilatory support in the UK.1 That survey was undertaken in response to an awareness of increasing numbers of ventilator-dependent children and in recognition of the limited and inadequate data available to plan healthcare for this small but high-cost group of children.
Since that survey, there have been further developments in this field. It is now accepted practice that a home environment is preferable to hospital or intensive care for a child with chronic respiratory failure who needs chronic ventilatory support. Packages of care and discharge plans for home ventilation have been developed and widely implemented throughout the UK. In addition, the use of non-invasive ventilation (NIV) with a face or nasal mask as an interface for home ventilation has increased significantly—especially for children with sleep-related breathing disorders such as craniofacial syndromes with upper-airway obstruction or children with hypoventilation/obesity syndromes. Furthermore, the use of NIV for children with neuromuscular weakness has become much commoner after studies have shown convincing benefit in terms of both outcomes and patient acceptability.2,–,5 Finally, technical advances in home ventilators and monitoring equipment more suitable for paediatric use have enabled the successful treatment at home of younger children and those with more complex respiratory problems.
What is already known on this topic
Previous studies have shown an increase in paediatric long-term ventilation (LTV) towards the end of the 1990s. The provision of services for this group of resource-intensive children in the UK is patchy and poorly coordinated. There is an increasing acceptance of the benefits of long-term ventilatory support for children with neuromuscular disorders, especially the use of mask ventilation.
What this study adds
This study provides the first census figures for LTV in children in the UK for a decade. There has been a significant rise in the figures, and the impact of neuromuscular disorders is clearly evident. There is now clarity on the scope of this important area of chronic respiratory failure, which will aid the planning and provision of future healthcare.
Since the 1999 survey, the status of paediatric long-term ventilation (LTV) in the UK has remained unclear, despite calls for such information from healthcare planners, patient support groups and financing bodies within the NHS. All would like to know the numbers of children, types of disorders, support required and age ranges in order to plan for future funding and accommodation of these children within the health service. Despite the clear need, there is no national source of up-to-date information on the numbers of children receiving long-term respiratory support, nor any information on the underlying medical conditions or clinical outcomes. Some of the ethical issues surrounding initiation and continuation of LTV in selected disorders have received recent attention in the press and in the courts.6
Surveying this group of children is notoriously difficult. There is no central register. Definitions are often vague, and no single specialist discipline or professional body cares for all these children. At any one time, a child may be under the care of general practitioners, community teams, intensivists, paediatricians, neurologists, neonatologist, respiratory paediatricians and rehabilitation specialists. Within any particular time period, some children will start ventilation, some will be weaned off support, some will become more (or less) dependent, and some will die from their disease.
The UK paediatric LTV working party is a forum of healthcare personnel involved in the care of childhood LTV in the UK. Formed 13 years ago, the group has grown to include a range of health professionals representing all regions within the country. Meetings are held at least annually, and an email-based discussion group has formed. It is anticipated that now most children starting on LTV or cared for in the community will fall under the care of clinicians within this umbrella organisation.
In order to obtain contemporary data, we approached members of the UK LTV working party to undertake a unique point-census of the status of paediatric LTV in the UK at midnight on the night of the 30 September 2008 using the same definitions as published in 1999 to document changes and provide comparisons over a 10-year period.
Subjects and methods
All members of the LTV group were canvassed by email, and regional representatives were identified and agreed to undertake a census of children receiving LTV and under their care. The date and time were arbitrarily set for midnight on 30 September 2008. This ‘point in time’ census approach resolved the difficulties associated with a longer time frame, wherein the cohort might change, as part of the natural history for this target population.
We defined paediatric long-term ventilatory support as any child below the age of 17 who is medically stable and requires a mechanical aid for breathing either invasively by tracheostomy or by non-invasive mask interface for all or part of the 24 h day.1 Premature infants who were likely to wean from ventilation were excluded, but children with chronic lung disease (CLD) (including the premature baby) where weaning was proving impossible or prolonged beyond 3 months were included.
A questionnaire was designed to collect core data on these children. Care was taken to create a minimal data set to facilitate completion, and provide unambiguous and relevant information. Electronic forms were completed on an online site established by the University of Glasgow. The data were completely anonymised, and no personal information was collected. The individual hospital number was encrypted and stored separately. Duplicate entering could be detected. Data were stored according to the requirements of University of Glasgow under the direction of the Caldicott guardian for the Royal Hospital for Sick Children. In a follow-up process, any missing data items were referred back to the relevant centre for clarification or completion.
The categories of information collected are listed in table 1.
We received replies from all 30 regional centres within the UK. Nine hundred and thirty-three children were thus identified and met the definition of LTV. On recanvassing to obtain complete data sets on all subjects, we failed to receive an adequately completed dataset for 39 children (representing one centre) despite repeated requests. These missing data points are indicated as ‘unknown’ in the tables of results.
Table 2 lists the totals for each aetiological category for the 933 children divided into their principle subgroups and the interfaces used for ventilatory support. In 20 of the 933 children, a classification was not possible with the information available.
Table 3 presents the results for the time the 933 children are dependent on ventilation, their method of ventilatory support and the current location of the child.
The age range of the children as at the time of the census (30 September 2008) is listed in figure 1 by age.
This survey is the first to evaluate the status of children on long-term ventilatory support in the UK since our last report 10 years ago.1 It has used a point-in-time census approach to collect information on children in the UK receiving ventilation. This approach was designed to accommodate the protean nature of paediatric LTV which is under constant flux as children grow, their ventilatory requirements change and the natural history emerges. Data collected in similar studies from other countries7,–,9 have not been able to identify a point prevalence in this way, and interpretation of the data collected over a wide time period is affected by the ever-changing circumstances of the subjects as well as changes in local clinical practice.
The results show a striking increase in the numbers of UK children who receive long-term ventilatory support—from a total of 141 in 1998 to the total from this report of 933 (figure 2). The reasons behind this increase are likely to be the wider availability of improved paediatric home ventilators and a changing ethos towards the benefits of home ventilation. Accumulative survival for this group of complex children is a likely contributor to the increase.
Surveys of this type are bedevilled by incomplete ascertainment. We believe that our approach through the UK LTV forum is likely to have identified the vast majority of the children who receive long-term ventilatory support in the UK within the confines of our definitions. However, we were unable to achieve 100% data collection on all the children, despite considerable efforts.
Within our defined group, there is a spectrum of ventilatory dependency. The number of children at the most ‘complex’ end, that is, children on 24 h tracheostomy ventilation, was 88. This compares with a figure of 33 children in the previous survey in 1998. It is interesting to note that although the absolute numbers of children have more than doubled, the relative contribution of children who are 24 h ventilated has decreased from 1998 (23.4%) compared with the most current figures where 24 h tracheostomy-ventilated children account for under 9.5% of the total group. The underlying conditions for which children require 24 h tracheostomy ventilation have not altered significantly over the last decade. This suggests that with increasing experience, many children can now be supported non-invasively, a fact that represents a considerable simplification in care.
The trend toward home ventilation of children who are technology dependent continues to increase. In 1998, 93 out of a total of 136 children (68.5%) were managed at home. In the present survey, we identified 844 out of 919 (92%) who are currently at home. Figure 3 highlights the wide geographical distribution of this cohort of NIV and trache ventilated children. While it is encouraging that children with complex chronic conditions are increasingly been looked after at home, this has important implications for the children's parents, and the local community and home services that support them. It is also likely to impact on local District General Hospital resources and expertise that provide technical support and care during emergencies and intercurrent illnesses.
The steepest increase in home ventilatory support is for children with neuromuscular conditions. Whereas in 1998, 62 children with neuromuscular disorders were identified, this figure has risen to 402 in the current survey. A similar increase in the use of technological support for respiratory insufficiency in children with neuromuscular disorders has been seen in other centres.9 The widespread adoption of mask interface and suitable bi-level devices for paediatric use has almost certainly contributed to this important change. The introduction of NIV for this group of children has significantly enhanced their respiratory management and in some subgroups has shown evidence of clinical and psychosocial benefits as well as a substantial increase in survival.4 It is anticipated that the use of NIV for paediatric neuromuscular disorders is likely to increase still further in forthcoming years. Although these children generally do not need the complex and expensive packages of care that some of the 24 h ventilated children do, they are still resource-intensive. As the numbers grow, consideration will have to be given to the delivery of home healthcare and local hospital input, as tertiary centres will not be able to deliver the bulk of this service.
Edwards et al10 have previously highlighted that 15 of 27 children discharged home on LTV successfully came off ventilation. Of these, 12 had a combination of CLD and an upper-airway abnormality, and their median age was 4.7 years (2.2–7.5 years). Thus, for these children, ventilation was a long-term but not lifelong treatment. As a point survey, the present study provides no information on the outcome of ventilation. While we suspect that some of the children currently ventilated may come off ventilation in time, only longitudinal follow-up will provide detailed outcome information.10 It is interesting to note, in this current survey, that 247 children (26%) had LTV initiated before 2 years of age.
The ethical issues for some groups of children, for example, those with severe forms of spinal muscular atrophy (SMA) type 1, have received recent media attention. Polarised views within the medical profession on the treatment of these children have also been recorded.6 Despite this background, this survey identified 12 children with SMA type 1 on long-term ventilatory support. Five of them were tracheostomy ventilated, and seven used a mask.
One hundred and twenty-nine children were aged 16 or over. The increased numbers in the older age groups represent a combination of survival through childhood plus a contribution from the children with neuromuscular conditions such as Duchenne muscular dystrophy requiring the initiation of NIV in their teenage years. Programmes addressing the needs of these children, particularly when their care is transferred to adult services, are required. An increasing number of children with disorders, previously considered lethal in childhood, will be transferring to adult physicians unfamiliar with their problems. Transitional programmes and adult service capacity for long-term care must be developed very soon to ensure a smooth transfer to adult services.
Ten-year surveys are self-evidently inadequate for accurate service planning. This census has spearheaded a drive for the development of an ongoing national database of affected children to provide accurate and up-to-date data for the management of this small but growing and resource-intensive group of patients. This survey has not addressed the outcomes of long-term ventilatory support. This is certainly an important area to review for the purposes of healthcare planning and resource allocation. The burden of home ventilation on the families of these children requires study, and future research in this area is planned.
We would like to thank all the members of the LTV working party and in particular the following lead individuals: L Arnavout, Evelina Children's Hospital, London; S Beckley, Lifetime Service, Bath; C Cameron, Royal Aberdeen Children's Hospital; E Chan, Manchester Children's Hospital; M Chatwin, A Ferns, Royal Brompton Hospital, London; J Clarke, Birmingham Children's Hospital; G Connett, Southampton University Hospital Trust; S Cozens, Leeds Teaching Hospitals; P Eckford, Newcastle General Hospital; H Evans, Children's Hospital for Wales; R Evans, Swansea; A Franklin, Royal London Hospital; F Hampton, The James Cook University Hospital, Middlesborough; N Hart, Lane Fox Unit, St Thomas' Hospital; A Laverty, J Perks, Great Ormond Street Hospital, London; L McCarthy, Royal Hospital for Sick Children, Edinburgh; S Nadel, St Mary's Hospital, London; T Ninan, Birmingham Heartlands Hospital, Birmingham; A Prendiville Royal Cornwall Hospital's Trust; R Primhak Sheffield Children's Hospital; R Ross-Russell, Addenbrookes Hospital, Cambridge; G Ruiz, King's College Hospital, London; M Samuels, University Hospital of North Staffordshire; P Seddon, Royal Alexandria Hospital for Children, Brighton; A Selby, Royal Liverpool Children's Hospital; G Simoes, Tadworth Children's Trust; H Smith, Royal Hospital for Sick Children, Bristol; D Thomas, Queen's Medical Centre, Nottingham; A Thomson, John Radcliffe Hospital, Oxford; C Way, St George's Hospital, London; J Watt, Southport and Ormskirk General Hospital; D Widdas, Warwickshire & Coventry PCTs; A Wylie, Royal Hospital for Sick Children, Belfast.
All authors contributed equally to the design of the study, analysis of the results and preparation of the manuscript.
Competing interests None.
Ethics approval Ethics approval was provided by the ICH.
Provenance and peer review Not commissioned; externally peer reviewed.