Parents and caregivers of children in the CAS program described their experiences managing their children’s chronic health condition having implications for managing uncontrolled seizure activity, status epilepticus, unsuccessful pharmacological, dietary and medical interventions and the impact on family, social and economic participation. Parents and caregivers reported that CBD treatment had benefits in relation to reducing the severity and frequency of seizure activity for some of the children, but not all experiencing refractory epilepsy. At the time of the study, 2 participants were weaning off the CBD and 5 of the families (37%) had withdrawn from the CAS program. Reasons for withdrawalthat were provided were the lack of improvement in seizure activity for their child and side effects that included daytime sedative effect with nocturnal insomnia and increased dribbling.For other patients, benefits included improved social engagement, wakefulness and a reduction of side effects related to a reduction of conventional medication dosage.The opportunity for participants to provide unstructured responses yielded novel perspectives about the program delivery in the context of normal hospital clinic service delivery. A summary of the results is shown below in Table
Theme 1: Seizure activity
Coding: refractory epilepsy, uncontrolled, ‘failure’ of other treatment modalities.
Families described their fears and concerns for their child, their disappointment with many failed treatment options, and the difficulty for their children to engage with mainstream and modified education programs. Participants explained their children had complex seizure patterns with frequent and often protracted seizure activity. Descriptions included occasions when their children experienced periods of status epilepticus, requiring hospitalisation and rescue medications as shown in Extract 1 and 2.
“There was one time where she was continually having seizures and the Midazolam had done nothing, so they gave her Clobazam, and she was non-responsive for quite a while”
“She was having periods of hypoxia between her tonic-clonics at night. That was so scary. Basically, I felt like it was an all-night tonic-clonic, not breathing, into a tonic-clonic not breathing, into a tonic-clonic kind of cycle all night long… I was very concerned about her, because her speech started to slur, and she was just lacking in energy. She had a big change there from how she had been a few months earlier, she was full of life, to this…”
Theme 2: Social engagement
Coding: diminished participation in family activities, diminished participation in school and learning.
Extract 3 demonstrates how seizure activity is not only disruptive for planned and structured activity such as schooling or social and community activities, it also has significant impact for the expectation of usual patterns of child growth and development, as a significant risk of hypoxia may therefore impair the expected trajectory of child and adolescent development.
“He would have 60+ seizures a day, would be just fatigued and really unable to do anything. You just had to put a line through your day. We would have to go back to bed and wait it out and he may or may not get kindergarten that morning.”
Families described their desire to find any treatment option that provided some form of improvement, either in reduced seizure activity, quality of life, or improvement associated with the reduction in symptoms or side effects associated with pharmacological interventions as shown in Extract 4.
“I had to resign from my work because of my son’s epilepsy and to care for him, so that I am his full-time carer. We would have done anything. At one stage we thought we would even consider going overseas to access cannabis if that was the way we had to do it. We were very aware that we couldn’t travel because he was so unstable with his epilepsy, that we wouldn’t have been travelling anyway.”
Theme 3: Drug safety
Coding: knowledge of drug trials, legal access to Cannabidiol.
Extract 5 is an example for about a quarter of those interviewed who described accessing unregulated cannabis products prior to starting on the CAS program. The participant refers to a coastal town in New South Wales, Australia. Concerns about accessing unregulated cannabis products ranged from the drug being illegal, lack of ability to accurately identify dosing quantities and administration techniques as well as having experience of adverse side effects or a fear of unknown, adverse side effects. As shown in Extract 6, having legal access to an experimental cannabinoid product that may help their child was therefore identified as a benefit of the CAS program.
“I had nipped down to “a coastal town (sic)” and gotten a little something, and it did nothing… I stopped because I was too frightened to go down and get more.”
“We were desperate for something that would work. We had no problem with the use of Cannabis in a medical situation. We weren’t worried about what people thought or any stigma or anything. We had good support from people around us in (sic) approaching that sort of treatment. So really, we liked the idea that hopefully, that it was a plant-based sort of product, and felt very lucky that we had medical supervision while using it.That was a big thing for us, so before we even knew the trial was coming, we had been, sort of agitating the Epilepsy Team.We were saying: ‘is something coming? Please consider our son for that.’ We just felt it was a very great opportunity to try treatment that was a bit different from the ones that we tried and that weren’t working.”
Theme 4: Drug efficacy
Coding: varied results, “some benefit” or “no benefit”.
Whilst families’ perceptions showed the use of CBD did not provide a therapeutic reduction in the seizure activity for all patients diagnosed with refractory epilepsy, Extract 7 and Extract 8 are examples of how it’s use as an adjunctive, safe pharmacological agent was perceived to provide other benefits by some patient’s families.
“He’s been a lot better, all round I think a happier child. Sleeping better, more alert and more interactive with us as well. You can tell he’s more there.”
“She had a period of almost no seizures and then the seizures started to very gradually increase again.”
Theme 5: Clinical support
Coding: health service provision, clinical trial administration
Parents and caregivers’accountswere similar regarding having no concerns about the safety of the drug being provided by the hospital pharmacy. Extract 9 illustrates how they felt they were supported through the information and explanations provided by their treating clinicians and pharmacists.
“The side effects were (explained). I remember going to the appointments and getting the information about it, what to do with it, talking to the pharmacist for a couple of hours. I felt like I knew everything I needed to know and then we just got it.. To go with it and see what happens. The pharmacist was wonderful.”
Having a dedicated registered nurse allocated to the CAS program was described as importantby families as important.Extract 10 and 11 are examples of comments made by parents and caregivers. Families were contacted by the same person each time for follow up and monitoring, in this way families developed rapport and felt comfortable to provide information and data throughout the program. Participants were also able to make contact with the trial nurses when they had questions.
“The clinical nurse consultant is very approachable and we’re able to access her quickly if we need to. If we have issues in terms of her script or the pharmacy prescription, they’re managed within 24 hours and always with confidence and efficiency, and we’re kept in the loop.”
“We’ve had a great experience. In regard to the doctors, her doctor who is doing it with her, the nurses that are involved in it, we haven’t had a bad experience at all and I could not complain about one person that’s been involved… Being part of a Facebook group, you see what happens outside of Australia in other countries, I am really grateful for what we have here because there are a lot of countries that are way worse off than what we are so it does give you that really good perspective of how we are treated very well.”
After being accepted on the program, most families described their participation in the scheme as being straightforward and unproblematic. However,Extract 12 is an example of reported instances of difficulties for families travelling interstate, and not being confident about taking the CBD ‘across the border’.
“If I do have an issue, it’s the lack of ability to get it from anywhere else. So when we had episodes a few months back when we had status (epilepticus), we were in the Lismore Base Hospital. We couldn’t get any supplies. We were down there for a funeral, and I wasn’t sure of the rule for transporting it over into new South Wales, so I didn’t take my Epidiolex with me, we were coming back the same day. We ended up in hospital all weekend, and couldn’t get Epidiolex. So that’s probably the only real issue I can say that I’ve had, is accessing it outside of the Queensland Children’s Hospital.”
For some families, they felt that being part of the program meant that they had added reassurance that their child had additional clinical review time with their Neurology team. Extract 13 shows how families felt they were able to monitor their child’s overall condition more closely. Extra appointments and pathology requirements for blood tests were managed through the usual hospital booking system, and clinical service requesting pathways.
“We felt that we weren’t managing a lot of his illness on our own without enough medical support.More frequent appointments was actually quite attractive to us. We wanted access to the Neurologists and if that meant that we could get more access during the trial and closer follow-up then that was a bonus for us.”
For clinicians, combining regular planned patient clinical review meetings with CAS program reviews allowed for the program to be integrated without considerable extra clinic bookings and resourcing as shown in Extract 14.
“Before we even were kind of fully accepted onto the trial and started Epidiolex, we’d had meeting with our daughter’s Neurologist who kind of took us through all the risks and potential benefits and the procedures for the trial and that kind of thing, so we were well informed before we even started her on Epidiolex.”
Theme 6: Social acceptance of drug therapy
Coding: personal choice of family, positive generally, “whatever works”.
Families discussed the perceptions of their families and friends of participating in a trial for CBD as being generally positive. Similar reports to Extract 15 were described by participants of their families and friends adopting a ‘whatever works’ attitude for the management of refractory epilepsy.
“They have all been super supportive, you get the classic jokes of: ‘can I have some’ type of thing, but they have all seen the change in her, so even if anyone was sort of ’on the fence’ initially, they have seen the difference in her now, and they would never say it was a bad thing for her.”
Theme 7: Program delivery
Results from the interviews with some families highlighted their determination to advocate for the CAS program and for their child’s participation. Extract 16 demonstrates the commitment expressed by many families to participating in the CAS program.
“Our son’s life was on the line, we are lucky he was a fighter, or strong enough child to the point where he could trial. What about the families that don’t get that? I thinkrefractory (epilepsy) is – you have exhausted medications, the likelihood of them not working, so how many is too many?”
As the program had a limited number of places, the findings suggested that those families not meeting the inclusion criteria may have experienced disappointment. Extract 17 highlights the challenges of delivering a program for a novel treatment in the context of usual care health services and the perception of ‘gaps’ in the service delivery model.
“I’m not sure if there was an appreciation of the emotional investment families had in attempting to access a product, and then had a lack of support if they were rejected. Maybe a better recognition by clinicians, whether that is more training, or maybe… some sort of support services for families to assist them through that process, of whether they got on it, or not or had to come off it or something like that, more of the holistic, not just the medical (support). I think that was really underestimated, just how people had been, us included, hoping that this would be the answer for years, and I don’t know that people realised. Some of the clinicians would have, maybe the clinicians involved in the trial realised just how devastating it was to some families to not be given access.”
The CAS program was a new service model at the Queensland Children’s Hospital, evolving over the course of the CAS program delivery period. There were new processes implemented by clinicians, and at times improvements to the early model of care. Extract 18 is an example of how families contributed to the development and enhancement of the CAS program model.
“We had gone in hoping that there would be really good data coming from this, about who benefitted and what happened, and things were quite disorganised. We weren’t the first, but we were maybe a bit early into it, and the paperwork and everything was quite disorganised, and the systems and the protocols were quite disorganised. I think we were a bit disappointed with that aspect. It seems the trial had started before the systems and protocols were in place, possibly, so we felt that some of the experiences that we were seeing probably weren’t documented and probably weren’t being collated across other people who were participating. We were disappointed that it was a missed opportunity”.