23. There is some guidance which is relevant to various complaint issues, so we set this out here and will refer to it at the relevant points.
24. GMC guidance says doctors must provide a good standard of practice and care. If they assess diagnose or treat patients, they must:
• adequately assess the patient’s conditions, taking account of their history, views and values and where necessary, examine the patient
• promptly provide or arrange suitable advice, investigations or treatment where necessary
• refer a patient to another practitioner when this serves the patient’s needs.
Complaints about both Trusts regarding the care and treatment provided between January and December 2021: Complaint about medications Chloral Hydrate 25. GMC guidance at paragraph 24 applies here.
26. Although the NPGG statement was not issued until December 2021, it provides some explanation of the use of chloral hydrate up to that point. The statement is endorsed by multiple reputable organisations.
27. It explains chloral hydrate has been widely used in paediatric practice for many years. It has been used for a range of issues including night-time sedation, management of dystonia and other movement disorders.
28. The NPGG statement says use of chloral hydrate for management of dystonia and other movement disorders is off label. This means although none of the chloral hydrate products in the UK are licensed for these uses, it remains a valuable tool in their management.
29. It says it may be appropriate to use chloral hydrate off-label to manage distressing symptoms in patients with movement and motor disorder when all other therapies have failed, or rapid stabilisation of symptoms is required.
30. The records show doctors prescribed chloral hydrate to P when he was admitted to hospital due to dystonia. They continued to prescribe it during 2021, to be used as a rescue medication for when P experienced significant discomfort from dystonia for an hour.
31. The BNFC guidance sets out the dose for a child of P’s age was 30-50mg per kg. The records show during this time, P’s weight was around 22.6kg. Doctors prescribed 500mg which is 22mg per kg. This is lower than the standard dose. Our neurology adviser said it is appropriate to exercise caution in disabled children with a chronic illness because they may be sensitive to the effects of drugs.
32. Doctors prescribed chloral hydrate on an as required rather than routine basis. This was in line with the GMC guidance and the BNFC, and as such we have not found a failing in its use for P.
Levetiracetam
33. Article 1 is a global statement by experts in Batten Disease. It says in Batten Disease, drug management generally follows the accepted principles for epilepsy which are set out in NICE epilepsy guidance. Anti-epileptic drugs are the mainstay of seizure management and levetiracetam is one of the common first-line options.
34. The NICE guidance recommends levetiracetam as an anti-epileptic drug for children. The BNFC guidance says the starting dose is 10mg per kg once daily, then increased in steps of 10mg per kg up to maximum of 30mg per kg twice daily.
35. Although the records we have seen do not show when doctors first prescribed this to P, we can see they prescribed it throughout 2021. This is because P was experiencing seizures.
36. During that time, doctors prescribed it at 250mg twice daily and then increased it to 400mg twice daily. There is evidence in the records P’s weight was 22.6kg in June. This means the amounts prescribed were in line with the BNFC.
37. We acknowledge that on occasions from August 2021, Miss C had concerns this medication was overstimulating P and that a paediatrician at Trust one advised her to continue it to prevent another epilepticus state. This is where seizures are prolonged or there are multiple seizures without recovery between.
38. Article 1 says the overarching goal of seizure management is to achieve sufficient seizure control to support social function while balancing the side effects. The aims are to minimise the impact of seizures on the child’s well-being, diminish the most disabling and life threatening seizures, and maintain quality of life.
39. We consider the Trust was acting in line with NICE guidance and Article one when it prescribed levetiracetam to P to try to control his seizures and therefore to minimise the impact of these on him. We have not found a failing here.
Diazepam
40. Article 1 explains that children with Batten Disease typically present with different types of movement disorders. Dystonia is common. These neurological symptoms are difficult to control. It explains that benzodiazepines (of which diazepam is one) are commonly used to treat dystonia.
41. The BNFC guidance says the dose for a child of P’s age is 5mg twice a day. This equals a total daily dose of 10mg. As in paragraph 30, our neurology adviser said prescribing to P cautiously was appropriate.
42. The records show doctors at Trust two prescribed diazepam in December 2020 because P was experiencing dystonia. They prescribed it at 2.5mg three times per day. This is a total daily dose of 7.5mg per day, which is slightly lower than standard and was split over three doses.
43. On discharge, there was a plan in place to reduce the dosage and ultimately stop taking diazepam. Over the next few months, Miss C gradually reduced P’s dosage of diazepam. Although this was increased temporarily when he was admitted to hospital in June, by September Miss C had been able to wean P off it.
44. Prescribing diazepam to P was in line with the guidance at paragraph 39 and 40.
Epidiolex
45. The NHS website explains Epidiolex is a highly purified liquid containing cannabidiol (CBD). CBD is a chemical substance found in cannabis that has medical benefits. It does not contain tetrahydrocannabinol (THC), which is the chemical compound in cannabis that gives a euphoric effect (makes them high).
46. GMC guidance says doctors must provide effective treatments based on the best available evidence.
47. NICE guidance one approved cannabidiol for treating seizures associated with Dravet syndrome which is a rare form of epilepsy and Lennox-Gastaut syndrome which is a severe form of epilepsy. NICE guidance two says Epidiolex is licensed specifically for those conditions. All other cannabis-based medicinal products are unlicensed for epilepsy. It explains healthcare professionals are not prevented from considering the use of unlicensed cannabis-based medicinal products where it is clinically appropriate in an individual case.
48. However, it set out that cannabis products can be used off licence if a specialist neurologist considers it appropriate, when balancing the risk and benefit.
49. In its complaint response, Trust two said in the absence of high quality evidence in P’s condition and the risk-benefit ratio would not support the use of Epidiolex.
50. We have considered what evidence there was available regarding Epidiolex. Article 2 sets out that cannabis oil was used to successfully treat a 25 year old man with Batten disease and status epilepticus. Our neurology adviser said this patient had a different type of Batten disease to P (CLN6 rather than CLN2).
51. Also, in that case, the cannabis product used was different from Epidiolex. It was not a pure product because it contains THC, whereas Epidiolex does not contain this component.
52. The difference in type of Batten disease and the product used means it cannot be directly applied to P’s case.
53. Article 3 is a poster presentation which describes cannabidiol being used for therapy-resistant epilepsy in eight patients with Batten Disease. At least one of them had the same type of Batten Disease as P. The conclusion was that it was well tolerated. However, further information was not available.
54. Our neurology adviser said there was insufficient evidence to justify using Epidiolex in P’s case.
55. We acknowledge Miss C wanted doctors to try Epidiolex for P. We know the NICE guidance allows neurologists to prescribe cannabidiol off licence if they consider it appropriate. However, this does not mean that they had to do so in P’s case. As set out in paragraph 24, the GMC guidance expects doctors to provide effective treatments which are based on good evidence.
56. The effectiveness of the medication in treating seizures in Batten disease was not known. For those reasons, and considering the views of our neurology adviser, we do not consider there was a failure by the Trusts in not prescribing this medication to P.
Hormone Blockers
57. The GMC guidance about prescribing from paragraph 24 applies here.
58. We are not aware of any recommendations for using hormone blockers in children with Batten disease. Our neurology adviser said they are not used to treat epilepsy in male children, with or without Batten disease.
59. Therefore, we have not found a failing in the Trusts not prescribing these.
Complaint about the lack of response to smell of ammonia
60. There is no specific guidance relevant to this issue. The GMC guidance at paragraph 24 applies here.
61. Our neurology adviser said urine can smell abnormal. This can be due to things such as the consumption of certain foods, drinks, medicines, or vitamin supplements. They said medical conditions which could potentially cause this smell include urinary tract infection, type 2 diabetes or dehydration.
62. On 17 November, a paediatrician at Trust one spoke to P’s father and told him the ammonia smell was not likely to be significant. There is no indication P had any other symptoms to suggest there may be a medical cause for the smell.
63. In early December, Miss C explained to dieticians at Trust two that P’s GP was arranging for him to have blood tests to check for infection, as she had raised concern with the GP about the smell.
64. The dieticians offered to complete these blood tests as part of others they were doing, to save P having unnecessary bloods taken. They would forward the results to the GP.
65. The tests were subsequently not done as P’s parent was unwell so unable to take him to the appointment. P was then admitted to hospital before the subsequent appointment took place.
66. We know Miss C is concerned that the ammonia smell was due to P being dehydrated. We would like to assure Miss C that when P was admitted to hospital, his blood tests indicated he was not showing any biochemical signs of dehydration.
67. We consider no further action was necessary by the Trusts in relation to this issue. The Trusts actions were in line with GMC guidance.
Complaint about Trust one only – the use of phenytoin
68. APLS guidance says status epilepticus is a medical neurological emergency. It recommends phenytoin in a convulsing child if benzodiazepines have not stopped the seizure. Article 1 says some anti-epileptic drugs such as phenytoin should be used with caution, as they may exacerbate myoclonus (a type of seizure).
69. Our neurology adviser said that P was not experiencing myoclonus seizures. The records show he had been experiencing generalised tonic clonic seizures, which are another type of seizure.
70. In any event, article 1 only says drugs such as phenytoin should be used with caution, not that they should not be used. The BNFC guidance allows for twice daily doses to be given after the initial loading dose.
71. The records show before arrival to hospital paramedics had given P midazolam, which is a type of benzodiazepine. In the emergency department, doctors gave P one dose of phenytoin at 5.35pm in an attempt to stop his seizures.
72. The prescription and administering of this medication was in line with the guidance. Therefore, we have not found a failing.
Complaint about Trust two only
Complaint about COVID-19 diagnosis and treatment
73. The GMC guidance at paragraph 24 applies here.
74. P tested negative for COVID-19 at Trust one in the evening on 4 January. On 5 January at Trust two, he tested positive for COVID-19.
75. P was experiencing respiratory problems and had signs of infection, specifically a fever. P’s father, with whom he lived, had COVID-19. Our paediatric adviser said given these factors, and in the absence of other obvious causes, diagnosing and treating P as having COVID-19 was a reasonable clinical decision.
76. There is no failing here. Although later testing negative for COVID-19, when diagnosing P with COVID-19 on 5 January, the Trust acted in line with GMC good medical practice.
77. Turning to the treatment provided to P, a COVID-19 guideline was published in March 2021. Although this was published shortly after the events we are considering, the guidance reflected what was known at that time in terms of treatment for COVID-19. We therefore use it to assist us in considering what would be considered good clinical care and treatment, in line with the GMC guidance.
78. The guidance recommends dexamethasone for people with COVID-19 who need supplemental oxygen to meet their prescribed oxygen levels. It also recommends LMWH for patients who are having oxygen support including invasive mechanical ventilation.
79. As P was on ventilation, Trust two acted in line with this guidance when it prescribed and administered this to P. Therefore, we have not found a failing here.
Complaint about blood transfusion
80. The blood transfusion guidance recommends that in paediatric intensive care, transfusions are indicated for children with a haemoglobin level of below 70. Haemoglobin is a protein in red blood cells which carries oxygen from the lungs to the body’s tissues.
81. NICE decision making guidance says when a person lacks capacity to make a particular decision at the time the decision needs to be made, all actions and decisions taken must be in the person’s best interests. GMC 0-18 guidance says doctors should consider the views of the child so far as they can express them and the views of parents.
82. The records show on 5 January, P’s haemoglobin level was 66, so the same day staff gave him a blood transfusion. Our paediatrician adviser said this was an essential treatment.
83. We consider, in giving this transfusion, Trust two acted in line with the blood transfusion guidance.
84. The records do not show staff discussed blood transfusion with Miss C in advance. In normal circumstances, P’s parents would have been present at the hospital when the blood transfusion was given. If they had been present, this would have enabled discussion between doctors and the parents about the transfusion.
85. Unfortunately, due to the unique circumstances of COVID-19 restrictions, such discussions could not always take place. Considering the circumstances at the time, Trust two acted in P’s best interests by deciding to give a transfusion without discussing it with his parents. We have not found a failing.
86. We acknowledge Miss C would have preferred P to have blood donated from a person who had not been vaccinated for COVID-19. We offer the following information as explanation to Miss C.
87. The NHS blood and transplant website explains that for a person to donate blood, they must meet certain criteria, such as age and weight. If people have certain health conditions, they are excluded from donating blood, and blood tests will be done for certain conditions.
88. Donors are not asked about their vaccination status. Our paediatric adviser said this is because it has no clinical bearing on the blood. Therefore, doctors would not have known whether the donated blood was from someone who had, or had not, been vaccinated.
Complaint about drug screening
89. Generally, GMC guidance at paragraph 24 is relevant here.
90. The BMJ guidance outlines the broad circumstances in which toxic drug screening might be done. This includes circumstances such as where there is unexplained acute decrease in a patient’s conscious level, recent history of sexual assault, or if there is intentional intoxication. The BNFC says there should be therapeutic drug monitoring for phenobarbitone (an anti-convulsant medication).
91. Our neurology adviser explained that drug toxicity can occur if a patient’s liver and/or kidneys are not working properly. This is because it is the liver and kidneys which remove drugs from the body. In our view, it would be in line with GMC guidance to monitor P’s liver and kidney function.
92. We can see that during his admission, P did not meet any of the listed criteria in paragraph 90. The records show P also had almost daily measures of his liver and kidney functions and there was never any evidence of liver or kidney dysfunction. Doctors also regularly measured P’s phenobarbitone levels and these were never above the upper limit of normal.
93. Our neurology adviser said there was nothing within P’s presentation to suggest drug toxicity during his admission.
94. Trust two appropriately monitored P’s liver and kidney function in line with the guidance. There was no indication it needed to screen P for drugs.
Complaint about screening for vitamin D and zinc deficiency, and immunoglobulin (antibodies produced by the immune system).
95. We acknowledge there is interest and research into the role of vitamins and minerals in seriously ill patients. However, we are not aware of any guidance which says screening for vitamin D and zinc deficiency should be done. Our paediatric adviser said there is nothing in P’s history which would suggest he had a deficient immune system.
96. Based on this, we are unable to say that by not doing such screenings or immunoglobin tests, the Trust acted against the GMC guidance at paragraph 24. We have not found a failing here.
97. We acknowledge Miss C’s concern that these screenings might have made a difference to P. Our paediatric adviser said they are not aware of any research or evidence which would suggest that if P’s levels of vitamin D or zinc had been low, it would have affected the outcome for him. We hope this reassures Miss C.
Complaint about ventilation
98. The APLS guidance explains the rate at which respiratory function can deteriorate in children is particularly high. Management of airway and breathing has priority in patients of all ages.
99. It sets out that where anti-epileptic drugs have been unable to stop the seizures, the child should be transferred to the PICU. Our neurology adviser explained this would result in a child requiring a ventilator to assist with their breathing.
100. There is no specific guidance for when to take a child off a ventilator. GMC guidance at paragraph 24 applies. Our neurology adviser said the timing of when to take a child off ventilation is determined by the general health of the individual child and whether their seizures are thought to be stable.
101. P was transferred to PICU at Trust 2 on 5 January and placed on a ventilator. This was due to frequent seizures which did not settle despite multiple attempts with different drugs.
102. His seizures improved initially so staff took him off the ventilator on 6 January. Staff transferred him to high dependency unit (HDU) on 8 January. However, the same day there was an increase in his seizures which could not be controlled with drugs. To regain control of his seizures and protect his airways staff had to intubate him again.
103. On 12 January, staff extubated P but unfortunately this failed as he was finding it difficult to breathe and required increased amounts of oxygen. Staff therefore put him back on ventilation the same day.
104. In placing P on ventilators on each occasion and attempting to take him off these, the Trust was acting in line with the GMC guidance.
105. Turning to the final time staff took P off ventilation, GMC end of life guidance is relevant. This says the starting point for reaching good decisions in end of life care is careful consideration of the patient’s clinical situation. Doctors must carry out a thorough assessment of the patient’s condition and consider the likely prognosis. It says doctors should work in partnership with parents when considering decisions about their child’s treatment.
106. Our neurology adviser said P had an incurable, neurodegenerative disease and attempts at extubating had failed because it was not possible to stop P’s seizures from impairing his ability to breathe.
107. The records show, that in line with the GMC end of life guidance, on multiple occasions over a week, clinical staff discussed and considered P’s condition and prognosis in detail with his parents. The decision that he would be taken off ventilation on 21 January and moved to comfort care was made jointly between them.
108. We have not seen any evidence that during those discussions staff were acting in anything other than the best intentions towards P and his parents when he was sadly at the end of his very short life. We have not found any failings here.
109. Despite his clinical prognosis, we do not doubt just how difficult it was for his parents to make such a heartbreaking decision to let P go. We acknowledge that the three days after P was taken off ventilation were traumatic for his parents.
Complaint about lack of anti-seizure medication after ventilator turned off 110. There is no specific guidance regarding this issue but the more general guidance from paragraph 24 is relevant.
111. The records show staff explained to P’s parents that they would only intervene in the natural dying process to ensure he was comfortable, if they thought he was having symptoms.
112. Our neurology adviser said the aim is to minimise distress whilst withdrawing life-sustaining treatment. They said P’s seizures were drug resistant and staff acted appropriately after turning off his ventilator. Once it became apparent P was not going to die quickly and he began to have seizures, they appropriately restarted his antiseizure medication to try to relieve his symptoms.
113. The records show that after turning off the ventilator, P was initially not experiencing any seizures. His parents were understandably relieved by this. In the morning of 22 January, P started having more seizures, for which staff gave him levomepromazine (a medication to ease symptom in palliative care). This was unsuccessful.
114. Staff then administered phenobarbitone that afternoon, but seizures continued. They then recommenced levetiracetam, sodium valproate, phenobarbitone and commenced ketamine infusions (an anaesthetic medication). This subsequently gave better control of his seizures.
115. Staff were acting in line with GMC guidance to provide treatment where necessary. We are pleased that staff were able to control P’s seizures, so his final hours spent with his family were more peaceful.
Complaint that P was not discharged home for end-of-life care
116. The NICE end of life guidance says staff should agree the preferred place of care and place of death with children and their parents or carers. This should take into account their wishes and values, the view of relevant and experienced healthcare professionals, and the safety and practicality.
117. It also says health professionals should explain that the place of care or death may change. For example, if the child and parents change their mind, for clinical reasons or due to problems with service provision.
118. The records show that from 13 January staff had conversations with P’s parents about their preferred place of care and death for P. His parents initially expressed their wishes for P to be at home with them and his brother, and so other relatives could visit.
119. They wanted to spend time at home with him before he was extubated. Staff explained that, unfortunately, it was not possible for P to be on a ventilator at home. Trust two has explained there was not the service provision for P to be transported home while he was intubated.
120. However, the records show that staff explained that once he was extubated in hospital, if he remained stable enough, he may be able to transfer home. Staff also discussed hospice options with P’s parents, but they declined this.
121. Subsequently, his parents were worried that P may not survive the journey home and that they would not all be with him at the time. Trust two could not provide guarantee this would not happen. His parents decided they did not want to take the risk of this happening.
122. Being with P in his final moments was understandably the priority for his parents, rather than the location. We are pleased that although P was not at home, Trust two was able to facilitate an ‘P day’ for the family. This meant they could spend time with him without medical reviews and tried to make his environment less clinical, and that relatives were able to visit him.
123. Once P had been extubated, in the days that followed, his parents expressed that they would not wish to be at home with P in those circumstances as it would be too traumatic.
124. Having considered this part of Miss C’s complaint, we have seen no evidence Trust two was not trying to facilitate his parents’ wishes around P’s place of care and death. We have found the Trust acted in line with the NICE end of life guidance.
125. We do not underestimate the immense pain it caused P’s parents and the family saying goodbye to P. We offer our heartfelt condolences they had to go through this and for the loss of their much loved son.