Saudi Arabian doctors need to be more aware of consensus guidelines for managing patients with spinal muscular atrophy (SMA) and their role in the variability of care for these children, a study says.
The study, “Effect of new modalities of treatment on physicians management plan for patients with spinal muscular atrophy,” appeared in the journal Neurosciences.
Before the launch of Biogen’s Spinraza (nusinersen) in 2016, management of patients with SMA was limited to supportive measures meant to provide adequate nutrition and respiratory assistance, and to treat or prevent complications of muscle weakness. Many of these practices were based on the clinician’s or institution’s experience or from small studies.
Choosing the best way to manage an individual with SMA and provide the appropriate support to their family based on a single institution and a doctor’s personal experience “is difficult and scientifically not appropriate,” the researchers note.
The approval of Spinraza, the first medicine indicated for patients with SMA, has changed the way doctors manage care for these patients.
In this context, researchers at King Khalid University Hospital, in Saudi Arabia, were interested in determining neurologists’ knowledge and practices regarding the acute and long-term management of children with SMA, especially with the appearance of new treatment options.
They surveyed 169 physicians, mostly pediatric and adult neurologists, who were attending the 2nd Saudi pediatric neurology society conference in 2017.
Results showed that nearly half of the participants (49.7%) were aware of the international guidelines for SMA standard of care. The most recent guidelines were published in 2018, spanning diagnosis, rehabilitation, orthopedic and nutritional care (part 1) and medications, supplements, other organ systems and ethics (part 2).
Pediatric neurologists were most aware of the guidelines, when compared with intensive unit and general pediatricians.
When asked about Spinraza, half of the physicians (52.1%) were uncertain, 34.3% were aware of it, and a few (13.6%) were not aware of the new treatment.
About half (53.3%) were uncertain about the benefits of Spinraza. While 32% believed the therapy could improve patient outcomes, 14.8% did not.
Given the promising benefits of Spinraza, doctors were asked if they would reverse a do-not-resuscitate (DNR) order of an end-stage SMA patient. A DNR order instructs health care providers not to do cardiopulmonary resuscitation if a patient’s breathing or heart stops.
Half (52.1%) of the doctors were hesitant to reverse a DNR order, while 23.1% agreed to do it, and 24.9% did not. Younger doctors were considerably more willing to reverse a DNR order than more experienced physicians.
Also, opinions varied on whether it was acceptable to intubate an SMA patient with impending compromised respiration. Half of the doctors (50.3%) agreed it was acceptable, 33.7% were unsure, and another 16% disagreed.
More than half of the doctors (53.7%) also indicated that DNR and “no intubation” orders were implemented at their hospital, followed by full supportive care (32.9%), tracheostomy and home ventilation (30.5%), and non-invasive ventilation therapy (26.8%).
The results suggest that clinical practices vary widely for patients with SMA, demonstrating “a need for increased awareness of consensus guidelines for standard of care,” the researchers wrote.
“Furthermore, physicians’ attitudes towards and knowledge of novel modalities of treatment were found to be a challenge in the management of SMA,” they concluded.