Home therapy improves life quality for young people with SMA on Spinraza
Respiratory program 'feasible, safe, and effective' at home, per study
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- Receiving respiratory physiotherapy at home was found in a new study to improve life quality for young people with SMA on Spinraza.
- The home-based approach focused on breathing exercises and airway clearance techniques.
- Researchers say the program enhanced physical, emotional, and social well-being, as well as perceived overall health, for these young patients.
A home-based respiratory physiotherapy program focused on breathing exercises and airway clearance techniques can significantly improve quality of life for young people with spinal muscular atrophy (SMA) who are receiving treatment with Spinraza (nusinersen), a small study suggests.
Data showed that lung function among the study’s participants remained largely stable over one year, regardless of whether patients received care at home or in a therapy center. However, participants who followed the at-home program reported greater improvements in perceived overall health than those receiving standard care alone, the researchers noted.
“The most compelling findings were the significant differences in [quality of life] scores observed at follow-up among patients who underwent home-based respiratory physiotherapy” versus clinic-based, the team wrote.
According to the researchers, the results “validate the growing trend of shifting from solely clinic-based interventions to include home-based care.” They also “authenticate the benefits of individualized treatment in a home environment,” the team wrote.
Overall, the study’s findings suggest that “home-based physiotherapy represents a feasible, safe, and effective approach that supports the global trend toward home-centered chronic care and improved patient quality of life,” the researchers wrote.
The study, “Impact of Home-Based Respiratory Physiotherapy in Nusinersen-Treated Patients with Spinal Muscular Atrophy,” was published in the journal Advances in Therapy.
A rare genetic condition, SMA is characterized by progressive muscle weakness and wasting, which often causes problems with breathing. SMA management typically involves disease-modifying therapies such as Spinraza, alongside supportive care like physiotherapy to help maintain motor and respiratory function.
Comparing home therapy to treatment in a clinic
However, physiotherapy for SMA typically is done in specialized centers, which can be burdensome to patients and caregivers who have to travel for such treatment. As such, “there is a growing recognition of the benefits of transitioning to home-based treatment,” the researchers wrote.
Still, the value of adding home-based respiratory physiotherapy to standard multidisciplinary care remains unclear.
To learn more, a team of researchers in Israel evaluated the impact of a yearlong at-home respiratory physiotherapy program among people with SMA types 2 or 3 who were being treated with Spinraza.
The study involved 29 participants altogether: 15 received the home-based intervention in addition to standard care, while 14 continued with standard care alone. The mean age was 16.7 years in the intervention group, and 14.1 years in the control group.
The intervention consisted of weekly one-hour sessions led by respiratory physiotherapists and included individualized breathing exercises and airway clearance techniques, as well as tailored physical conditional exercises such as strengthening, stretching, and mobility exercises.
After one year, lung function remained generally stable in both groups, with no significant differences between them, according to the team.
At-home program also provides benefits for caregivers
Quality of life outcomes, however, were significantly better in the intervention group, the researchers noted, referencing answers from the SF-36 Health Survey, a 36-item questionnaire evaluating eight domains of physical and mental health.
Specifically, compared with those receiving standard care alone, patients in the home-based physiotherapy group had higher scores across multiple SF-36 domains, the data showed. These higher scores were seen for physical functioning, emotional well-being, social functioning, and general well-being. The results also indicated fewer limitations due to physical and emotional problems and less fatigue.
“The observed benefits of the intervention were reported most prominently in physical functioning and emotional well-being and less reported for alleviating pain,” the scientists wrote. Still, “the intervention also led to fewer limitations due to physical and emotional health, better energy levels, and improved overall general health,” the team noted.
Home-based care … enables parents to gain a more comprehensive understanding of their children’s needs, allowing them to organize their living space more effectively and assume a more integral role in caregiving.
Participants in the intervention group also reported greater perceived improvements in overall health on the Global Rating of Change scale. The median score in the intervention group indicated a clinically meaningful improvement, whereas the control group reported only modest changes.
According to the researchers, these perceived benefits may be particularly important for easing caregiver burden as care for chronic conditions overall, as such care is starting to transition more from hospital settings to a person’s home.
“Home-based care also enables parents to gain a more comprehensive understanding of their children’s needs, allowing them to organize their living space more effectively and assume a more integral role in caregiving,” the team wrote.
The researchers noted that this study was small, and called for larger efforts to confirm these findings and to optimize home-based physiotherapy programs for people with SMA.
