Otitis media with effusion in children with cleft palate

Cleft lip and palate are among the most common congenital malformations, with an overall incidence of around 1 in 700 individuals.^1,2 Approximately 90% of children with cleft palate (CP) have a history of non-trivial otitis media with effusion (OME).^1–3 OME (also known as ‘glue ear’) is the accumulation within the middle-ear space of a mucoid or serous fluid. Although the exact mechanism for the development of OME is not fully understood, dysfunction of the Eustachian tube connecting the middle-ear space to the postnasal space is thought to be of fundamental importance. The function of the Eustachian tube is to equalise pressure either side of the tympanic membrane, avoiding the development of negative pressure in the middle ear. In children, the Eustachian tube does not work as efficiently, with the resultant tendency towards the development of negative middle-ear pressure and the accumulation of fluid within the middle-ear space (OME). This tendency towards Eustachian tube dysfunction is further increased in children with CP as a result of dysfunction of the muscles originating from the palate which act to open the Eustachian tube orifice.^4,5

A prospective longitudinal study following children between the ages of 1 and 5 years demonstrated that the overall prevalence of OME was 75% in children with cleft lip and palate compared with 19% in children without clefts.² This difference in prevalence of OME between children with and without clefts was also significant at individual time points throughout the study period. As well as being more common in children with CP, OME is likely to persist longer in children with CP. A retrospective longitudinal study of adolescents with various types of CP has demonstrated a decrease in prevalence of abnormal middle ears over time, with the decline in OME in patients with isolated CP occurring between 13 and 16 years of age.⁶ Other studies have shown a similar decline in the prevalence of OME in late adolescence.^7,8 A questionnaire-based study of the natural history and outcome of middle-ear disease in children with CP reported that ear problems (ear infections and/or hearing loss) were most prevalent in the age range 4–6 years, only settling in adolescence, with 26% of the 13- to 15-years age group reported to have experienced ear problems in the preceding year.⁹ However, 24% were still reported to have below-normal hearing when reaching early adulthood (16 years and above). Therefore, the prevalence of OME from early childhood into adolescence is an important factor when considering the optimum treatment strategy for OME in children with CP.

Otitis media with effusion commonly presents with hearing loss, but may also cause language delay, poor educational progress, recurrent ear infections, otalgia, behavioural deterioration, imbalance, tinnitus and hyperacusis. OME may also have a negative impact on quality of life (QoL) in affected children, with hearing being considered important at key stages in the development of language and behavioural and social relationships.¹⁰

Management options for otitis media with effusion in children with cleft palate

The diagnosis of OME requires a focused history, including information on the clinical features of OME and the general health and developmental status of the child. Clinical examination should include otoscopy, examination of the upper respiratory tract, tympanometry and an age-appropriate hearing test.

There are several possible approaches to the management of persistent OME in children with CP, which can be broadly divided into surgical, non-surgical and combination treatment.⁴ The surgical treatment of persistent OME consists of the insertion of ventilation tubes (VTs, also known as grommets) into the tympanic membrane, which, while patent and in situ, prevent the development of the differential pressure between the surrounding environment and the middle-ear space, thought to be an important factor in the pathogenesis of OME. VTs have recognised complications, which include persistent tympanic membrane perforation, ear infections and early extrusion.¹ Adjuvant adenoidectomy is not recommended in children with CP owing to the risk of velopharyngeal competence. Hearing aids (HAs) provide an alternative non-surgical treatment option for OME, with the aim of amplifying the sound delivered to the middle ear, compensating for the ‘dampening’ of the sound signal as it crosses the middle-ear space to reach the cochlea. HAs may also lead to ear infections and may not be considered cosmetically acceptable by a proportion of children and parents. Compliance with HAs in children with CP with or without cleft lip and OME has been reported to be only 52% (16/31 patients).⁴ However, the same study reported otological complications in 5% (2/44) of children managed non-surgically and 38% of those treated with VTs, with the authors subsequently advocating VTs only in children not compliant with HAs or those who develop recurrent ear infections.⁴ Combination treatment describes the scenario in which the chosen treatment strategy changes between surgical and non-surgical (or vice versa) owing to persistence or recurrence of symptoms.

In a systematic review directed at the early routine insertion of VTs for the management of OME in children with CP, the authors identified 18 eligible studies (case series, retrospective cohorts, prospective cohorts and randomised studies), but only one of these was a randomised clinical trial. This randomised trial had several significant methodological limitations which critically limited interpretation. The authors concluded that the majority of studies were small or of poor quality, with many having no formal sample size calculation, with the resultant risk of being underpowered to demonstrate a clinically important effect of treatment.¹

When we consider outcomes, we see that studies have used diverse measures, mostly selected from clinicians’ point of view and with limited consistency between studies. As OME can impair hearing at stages thought to be important in the development of language and behavioural and social relationships before the start of school, it could be suggested that outcomes relevant to these issues should be used in future studies. It is clear, therefore, that if further research into this treatment is to be commissioned, those outcomes relevant to parents and patients should be considered.

Guidelines for the management of otitis media with effusion in children with cleft palate

In 2008, the National Institute for Health and Care Excellence (NICE) published clinical guideline 60 (CG60), entitled Surgical Management of Otitis Media With Effusion in Children, which included a section specific to children with CP.¹¹ The guideline highlighted the particular problems posed by OME in children with CP, which included early onset, prolonged clinical course and higher rate of recurrence. For children in general, the guideline recommends that

Children with persistent bilateral OME documented over a period of 3 months with a hearing level in the better ear of 25–30 dBHL or worse averaged at 0.5, 1, 2 and 4 kHz (or equivalent dBA where dBHL not available) should be considered for surgical intervention.

The recommendations for children with OME and CP were:

1. 1.8.1 The care of children with cleft palate who are suspected of having OME should be undertaken by the local otological and audiological services with expertise in assessing and treating these children in liaison with the regional multidisciplinary cleft lip and palate team.
2. 1.8.2 Insertion of ventilation tubes at primary closure of the cleft palate should be performed only after careful otological and audiological assessment.
3. 1.8.3 Insertion of ventilation tubes should be offered as an alternative to hearing aids in children with cleft palate who have OME and persistent hearing loss.

The guideline also concluded that the evidence for a benefit of VT insertion in CP was lacking and that the optimal treatment for OME in children with CP had not been determined. In the absence of strong evidence, clinicians were recommended to base the management of OME in children with CP on the needs of the individual. Although the needs of each patient should be central to the decision-making process, there clearly remains a need to determine which treatment strategy is the most appropriate for these children.

Commissioning brief and objectives

The management of Otitis Media with Effusion in children with cleft palate (mOMEnt) study has been funded through a Health Technology Assessment (HTA) programme-commissioned call (project number 09/167/02) to address the uncertainty in the treatment of OME and to address the question ‘What is the most appropriate way to manage OME in children with CP?’ by completing a feasibility study.

Randomised controlled trials (RCTs) provide the highest level of evidence in the evaluation of health care. However, trials are expensive and require considerable additional effort from health-care staff and patients, which may create particularly high barriers to recruitment and successful completion of a study. From evaluating previous surgical trials, it appears that there are several challenges for a potential trial of care of OME in children with CP.¹² These concern feasibility, choice of comparator treatment, selection of relevant outcomes, and surgical compliance and skill. Furthermore, from the patient’s point of view there may be difficulties with equipoise as surgical and non-surgical treatments have different risks.

The aim of our research was to provide information on the feasibility of carrying out a RCT or strong prospective cohort studies of the management of OME in children with CP. The project involved a set of studies and a value of information (VOI) study with the aim of identifying the optimum study design to add to knowledge of the treatment of OME in children with CP.

The study had the following components:

1. Study Advisory Group (SAG) We formed a SAG comprising clinical and methodological experts with nominations from the Craniofacial Society of Great Britain and Ireland. This included audiologists, speech and language therapists, and ear, nose and throat (ENT) surgeons. The SAG had the following specific roles: (i) to regularly provide advice for the study; (ii) to have an input into the design of the clinical survey and Delphi study; (iii) to advise on key parameters to explore the VOI analysis; and (iv) to have full input into the final exercise on feasibility.
2. Clinician surveys Surveys of clinicians were carried out to (i) identify the current UK practice for the treatment of OME in children with CP; and (ii) evaluate the feasibility of performing a RCT, or other relevant type of study, of VTs in comparison with ‘usual methods’ for the treatment of OME in children with CP.
3. A qualitative project The qualitative research project was designed to capture patient and parent opinions on willingness to take part in the trial, and to identify their needs for the content and form of information required to provide or withhold informed consent. Opinions on outcomes were also explored and data collected contributed to the core outcome set (COS) development.
4. The development of a COS A COS for a potential trial was developed. This would reflect the values of both providers and consumers of care.
5. VOI analysis This component was a VOI analysis that provided information on whether or not the extent of existing decision uncertainty about OME care for children with CP justifies the costs of the proposed research.
6. Evaluation The final part of the project was an evaluation of the data collected in the above components, in order to make recommendations on the feasibility of a potential study design.