U.K. consensus sets new guidance for hip care in children with SMA
Experts call for individualized monitoring and treatment decisions
Written by |
- U.K. guidance recommends earlier, consistent monitoring for hip problems in children with SMA.
- Hip displacement and pain can occur in children with SMA; care should be proactive and individualized based on motor abilities.
- SMA alone does not rule out hip surgery, but more long-term data and registry tracking are needed.
A panel of experts has developed the first U.K. national consensus recommendations for preventing and managing hip problems in children with spinal muscular atrophy (SMA), aiming to support earlier monitoring and more consistent care in the era of disease-modifying treatments.
The recommendations emphasize a more proactive and individualized approach to hip health, while also noting that long-term data are still limited and more research is needed.
The study, “Hip displacement management in spinal muscular atrophy in the era of disease modifying therapies: a Delphi consensus study in the UK,” was published in eClinicalMedicine.
New treatments have changed SMA care
Most SMA cases are caused by mutations in the SMN1 gene, leading to a deficiency in the SMN protein that’s essential for the functioning and maintenance of motor neurons, the nerve cells responsible for muscle movement. Without enough SMN, muscles become progressively weaker and waste away, affecting movement as well as functions such as swallowing and breathing.
Outcomes for children with SMA have improved significantly with the availability of disease-modifying therapies such as Spinraza (nusinersen), Evrysdi (risdiplam), and Zolgensma (onasemnogene abeparvovec). These treatments can extend survival and help some children reach motor milestones that were previously unlikely, especially when started before symptoms appear or early in the disease course.
However, as children live longer, clinicians are seeing new challenges in SMA management, including orthopedic complications such as hip displacement — where the ball of the hip joint gradually moves partly or fully out of its socket — and hip pain. These issues often begin early in life and may lead to reduced mobility.
While some recent studies have suggested taking a more proactive approach to managing hip problems, there has been little agreement on the best strategies for monitoring and treatment.
To address this, researchers in the U.K. used a Delphi consensus exercise — a structured method where experts review evidence and vote in multiple rounds to reach an agreement on recommendations — to develop national guidance for preventing and managing hip problems in children with SMA.
Delphi process included experts from 19 U.K. centers
The effort was funded by Novartis, Roche, and Biogen, which market Zolgensma, Evrysdi, and Spinraza, respectively. The consensus process included participants from 19 U.K. pediatric neuromuscular centers, including pediatric neurologists, orthopedic surgeons, neuromuscular or orthopedic physiotherapists, and patient input.
The exercise unfolded in two rounds: first through an online questionnaire, then through a combined online and in-person meeting with anonymous voting. Consensus was defined as at least 75% of participants rating a statement as critical for inclusion.
After the two rounds of voting and discussion, the group agreed on 13 key recommendations.
Overall, the panel supported annual hip X-ray surveillance starting early in life, ideally between 6 and 18 months of age, to track changes over time. The goal is to detect problems sooner and guide decisions about care.
The recommendations also stressed that treatment should be tailored to each child, particularly taking into account their motor abilities and potential. For example, efforts to prevent hip dislocation may be more relevant in children expected to achieve higher motor abilities, such as standing or walking.
SMA alone should not rule out hip surgery
For families considering treatment options, the panel concluded that having SMA alone should not automatically rule out hip surgery. Instead, decisions should be made case by case, weighing each child’s potential risks and benefits. At the same time, they highlighted the need for more long-term evidence to better understand the outcomes of both surgical and non-surgical approaches.
Ongoing data collection in a national registry, such as the SMA REACH UK registry, will be critical to strengthen future guidance and improve care, the researchers noted.
“By addressing gaps in clinical practice, the consensus recommendations provide a foundation for consistent, evidence-based care while promoting research and audit initiatives,” the team wrote.
“Future multicentre prospective studies and standardised registries are needed to evaluate long-term clinical, functional, and surgical outcomes and to develop evidence-based guidance for hip management in children with SMA,” they concluded.
