A new study addresses the balancing act that doctors and families must master when it comes to meeting expectations at a time of great scientific advances in spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).
Advances in the therapy of neuromuscular diseases have brought within sight what once seemed impossible: curative therapy. However, for parents of children with diseases such as Duchenne muscular dystrophy (DMD) and type 2 spinal muscular atrophy (SMA) scientific advances have also raised new questions about managing expectations and communicating expectations with their children.
These thorny questions are the subject of a new article published in the magazine this month care request by Annette F. Mahoney, PhD, from the Danish National Rehabilitation Center for Neuromuscular Diseases, and Charlotte Handberg, PhD, from Aarhus University.
Mahoney and Handberg found that both SMA and DMD are debilitating diseases in which children grow up with high levels of dependency on other people. Although both diseases are not yet believed to be curable, new advances have raised hopes of slowing, halting, or possibly even reversing the disease’s progression. In patients with the most common type of SMA (type 1), treatment before the onset of symptoms can allow children to have near-normal motor development, the authors noted.
However, the therapies do not have the same effect on patients with stage 2 or 3 SMA, and even to the extent that the therapies may improve, they also come with high prices that increase the cost of the drugs for some patients can make unaffordable .
As such, parents of children with SMA must live in a space of hope, but still with many potential hurdles. To better understand how parents deal with this ambiguity, Mahoney and Handberg used a variety of methods, including participant observation, semi-structured interviews, and analysis of internet data and social media, to learn more about the hopes and expectations of families of children with SMA and DMD. A total of 6 Danish families with a total of 12 people were interviewed.
What investigators found was that the new drugs were the source of controversy and that families tended to fall into one of two camps.
“Based on the analysis of the data, we identified patterns from roughly two groups of parents: one who spent much of their time advocating pharmaceutical treatment for their children: ‘the healing optimists’, and one who had a more hesitant approach : ‘heal the pragmatists,’” said Mahoney and Handberg.
How these attitudes trickle down to children can have a significant impact on the child’s perspectives and experiences, they found.
“The healing optimists in the present study appeared to have no ethical concerns about actively involving their children in their hopes for the future and using hope as a motivation for exercise or as a comfort when the child was saddened by his or her physical deficiencies,” they wrote.
The optimists are also willing to let their children participate in clinical trials, even if it means long trips and numerous absences from school, they said.
“Healing Pragmatists” were more concerned with giving children false hope or making their children feel like they weren’t good enough.
The authors said one approach to decision-making is to actively involve children in the decision-making process. They said some existing research suggests that children with DMD rate their quality of life higher than their caregivers.
All of this data and viewpoint complicates the role of healthcare professionals who must counsel parents, even though the drugs in question are new and their long-term effects may not be known. In addition, the authors say, physicians sometimes face challenges in matching parents’ expectations to reality.
“Thus, to avoid false hope and therapeutic misunderstandings in parents, it is important that health professionals help parents understand the long and complicated process of developing new drugs and the many uncertainties involved,” they wrote.
They said doctors can also play a role in encouraging positive self-identity in patients, including by connecting them with older mentors who have the same health condition.
The authors concluded that while their sample size was small, the concerns expressed should play a large role in the thinking of clinicians and the families they serve.
“Although our study represents only a small sample, we believe that the dilemmas presented and discussed are applicable to families with other chronic diagnoses, for whom new developments in bioengineered medicine may lead to similar moral dilemmas about what is best for them children,” they wrote.
Mahoney AF, Handberg C. New medicine for neuromuscular disease: An evolving paradox for patient and family hopes and expectations. Nurs Inq. Published online September 17, 2022. doi:10.1111/nin.12527