Parents of children with spinal muscular atrophy (SMA) reported different perspectives and concerns about Spinraza (nusinersen) therapy in an interview-based study, done to further understand the decision-making process involved in choosing whether to try the new treatment.
The findings showed that, while many parents were focused on battling the disease and improving their child’s quality of life, some were more concerned that the new treatment might not work effectively for their child and could potentially cause further pain and discomfort.
Life expectancy, slowing deterioration, and improving quality of life were the perceived benefits of the therapy, and parents’ decisions to treat their children were facilitated by open communication with clinicians, the results showed.
The study, “Parents’ perspectives on nusinersen treatment for children with spinal muscular atrophy,” was published in the journal Developmental Medicine & Child Neurology.
Spinraza is a disease-modifying treatment that has been shown to stimulate physical improvements and prolong life in people with SMA. Repeated injections — most patients receive treatment three times per year, about every four months — administer the therapy into the space surrounding the spinal cord.
Parents caring for a child with early onset SMA face many uncertainties regarding prognosis. They must decide whether or not to choose Spinraza treatment by weighing the therapy’s potential benefits against safety and effectiveness concerns. They also must decide if Spinraza or another available treatment is the right choice for their child.
With a relatively new medical treatment like Spinraza, it is essential to understand these parents’ perceptions and expectations to support their decision‐making process.
To address this issue, researchers at the Utrecht University, in the Netherlands, conducted interviews with parents of children with SMA to gain insight into their perspectives on the need for and concerns about Spinraza and investigate factors that influenced their decision‐making process.
The Dutch national SMA database was sourced to find parents with SMA children — ages 9 years 6 months or younger — with SMA types 1 to 3 who were offered nusinersen treatment.
Of note, SMA type 1 (infantile-onset SMA) may be present at birth or appear within the first few months of life, and children will have developmental delays. SMA type 2 develops between 7 and 18 months of age, with most children needing help to stand or walk, whereas SMA type 3 (juvenile SMA) appears before age 3, but also in the teenage years, is generally milder, and may require help as the patient ages.
Single face‐to‐face interviews were conducted, which were audio‐recorded and transcribed word-for-word.
A total of 13 interviews were conducted with 19 parents of 16 children. Of these, six included both parents, while the remaining seven interviews were done with the mother only. Three of the 13 families represented had two or more children with SMA, in which one child in each family was offered treatment.
Regarding parents’ perspectives on Spinraza, the study found that their main focus was to manage the physical symptoms of SMA while doing everything to provide a good life for their child. Spinraza gave them that opportunity, many said. Parents felt that rejecting the treatment felt like giving up on their child, or allowing the child to deteriorate.
“You know what will happen if you don’t do anything… It will get worse, and he will soon end up in a wheelchair or worse,” said the mother of a child with SMA type 3.
Some parents were eager to get Spinraza for their child, even before it was available in the Netherlands. These parents kept a close eye on research developments, and many registered their child at the national center of SMA expertise to maximize their chances of access.
Based on the wide range of perspectives from parents, a spectrum was identified between aiming for a biochemical cure to a more holistic good quality of life. At various time points, parents varied in their perspectives and position within this spectrum.
Overall, the parents’ most important needs included life expectancy, stopping deterioration, and increasing independence. On life expectancy, all parents said they hoped Spinraza would prolong their child’s life, and for many, this was the most important reason for treatment, which did not feel like a choice.
“You’re going to see your child deteriorate; more and more things will happen. You’re going to see your child being able to do less and in the end… it’s over. So, what do you want? To be able to postpone it,” said the father of a child with SMA type 2.
Parents of SMA type 1 children mainly said they hoped to stop their child’s deterioration. In contrast, parents of children with SMA types 2 or 3 indicated that stabilizing the child now might lead to an independent future. Most suggested that increased independence would improve their child’s ability to live a “normal life” with increased social participation, meaning fewer caregiving demands.
Regarding perceived concerns, parents worried about treatment complications, quality of life, access to Spinraza, and increased caregiving demands. Some parents were afraid the treatment would cause too much suffering and effort, and perhaps not lead to positive results.
“My child has to have an anaesthetic, it is a spinal tap. Maybe it will cause a lot of pain or it might actually make my child tired. If you don’t think there will be any benefit, as in: progress or stability, then you have to ask yourself: ‘what are you doing?’” said the mother of an SMA type 1 child.
Parents first considered whether treatment could add to their child’s quality of life, especially regarding treatment intensity or suffering issues. Others had concerns about the intensity of the therapy and side effects that might impact their child’s daily living; however, this was not a reason to reject Spinraza.
In two cases, parents of SMA type 1 children rejected the treatment. These parents said Spinraza therapy could not guarantee a good quality of life and they did not want their child to experience any additional suffering or a poor quality of life.
“In my opinion, it’s a very selfish choice because the only thing you want to achieve is to keep your child with you. And then, you are putting your child through this. Well, I can’t reconcile this,” said the mother of a child with SMA type 1.
Overall, most stated that although the effects of Spinraza could not be guaranteed, they gave the new treatment the benefit of the doubt.
The increased intensity of caregiving demands was a concern, with a few parents unsure whether they could handle any additional demands while maintaining a healthy physical and mental state themselves. “And also for ourselves: we can’t give 24/7 care. Really it was nearly killing us,” said the father of an SMA type 1 child.
During the interviews, the parents described what helped or hindered their treatment decision, naming factors such as communication with their child’s physician and the availability of information about the therapy. For those parents with more than one child with the disease, unequal availability of Spinraza within the family also was a factor.
All of the parents said the physician played a significant role in their decision‐making process, with doctors cited as the parents’ primary source of up‐to‐date information on the new therapy. Many parents reported that trust in their physician’s expertise helped their decision, which was strengthened by an equal and open conversation. Meanwhile, some other parents said making their own choice was aided by physicians remaining neutral, making them feel in control.
This was very important to the parents of one child with SMA type 2. This child’s mother said, “No, the physicians certainly did not put pressure on us. They involved us at every stage,” while the father added, “The physician was very reticent. … I appreciated that. I think that responsibility for making the decision should rest with the parents.”
Internet searches gave some parents additional information and insights on Spinraza. Some mothers and fathers reported finding data from medical studies. Others found forums and videos in which parents of other children with SMA shared their experiences with Spinraza treatment. These parents said that videos of children already receiving Spinraza were hopeful.
Notably, some parents reported that searching for data was difficult because the information was scattered. They noted that such difficulty was an obstacle in making an informed decision.
Finally, three families had several children with SMA; thus, some children were offered the medicine, whereas the others were not eligible. This made a few parents feel conflicted during and after the decision‐making process.
Overall, “parents reported a wide variety of views about nusinersen treatment, which varied across a spectrum from a biomedical to a holistic perspective on the situation of their child; this might vary at different points in time,” the researchers concluded.
“It is important to take these differences in parental perspectives into account when presenting a new treatment option,” the wrote.
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