88% of clinicians in survey prescribe approved NMOSD treatments
Insurance, cost main barriers for US patients to switch from off-label therapies
Nearly all neuroimmunologists treating patients with neuromyelitis optica spectrum disorder (NMOSD) who test positive for anti-AQP4 antibodies have prescribed one of the three currently approved therapies: Soliris (eculizumab), Uplizna (inebilizumab-cdon), and Enspryng (satralizumab-mwge).
These are the findings of a U.S. survey that also found most responding clinicians have not switched patients who are stable on off-label immunosuppressants to the approved therapies, mostly due to insurance and cost issues.
The study, “Utilization of FDA approved treatments for neuromyelitis optica spectrum disorder in clinical practice: A survey study of academic neuroimmunologists,” was published in the journal Multiple Sclerosis and Related Disorders.
NMOSD is an autoimmune disease marked by inflammation and damage to healthy parts of the brain and spinal cord. In about 75% of patients, this damaging inflammation is driven by self-reactive antibodies against a protein called AQP4.
Until recently, there were no approved NMOSD therapies, and patients mainly received immunosuppressive treatments that were used off-label, meaning they were approved for indications other than NMOSD.
3 NMOSD treatments approved in US, Europe since 2019
Since 2019, three treatments were approved in the U.S. and Europe for NMOSD patients testing positive for anti-AQP4 antibodies: Soliris, Uplizna, and Enspryng. Despite having different mechanisms of action, all three medications work by suppressing immune responses and reducing inflammation.
Earlier this year, an international team of experts published evidence-based recommendations about the appropriate use of these therapies for people with AQP4-positive NMOSD.
Still, “there has been limited information on the utilization of these treatments in clinical practice, as well as the barriers to their use,” the researchers wrote.
To better understand current treatment practices, a team of researchers at Cooper University Healthcare, in New Jersey, sent a 14-question electronic survey to 381 neuroimmunologists associated with U.S.-based academic hospitals in 43 states.
Clinicians were asked about their experience with NMOSD, the patients they treated, the percentage of those started on one of the three approved therapies in different clinical scenarios, and treatment barriers.
Of the 33 respondents practicing in 18 U.S. states, 28 (85%) had completed training in neuroimmunology. Beyond training, the clinicians had been in independent practice for a median duration of eight years, with a median of 15 NMOSD patients. More years in practice was significantly associated with more treated patients.
Survey respondents “represented a geographically diverse group with reasonably large NMOSD patient panels considering the rarity of the condition,” the team wrote.
All clinicians surveyed felt comfortable prescribing NMOSD treatments
All respondents said they were comfortable prescribing the approved therapies, referred to as novel NMOSD treatments (NNTs), and all but four (88%) reported having prescribed them in clinical practice.
While there was a good correlation between the comfort level with one therapy and the others, the number of clinical practice years was not related to these comfort levels.
Of the 26 clinicians who prescribed NNTs to newly diagnosed patients, half (50%) said 25% of these patients were prescribed NNTs, while one-fifth (19%) used them in more than 75% of the patients.
“The variability of use of NNTs among neuroimmunologists treating newly diagnosed patients in our study may highlight the need for future trials specifically evaluating NNT use in treatment-naïve patient populations to help guide management decisions,” the researchers wrote.
Answers from 25 clinicians showed most (84%) used NNTs in patients who relapsed while receiving off-label B-cell depleting therapies or other steroid-sparing agents. B-cells are the immune cells responsible for producing antibodies. The majority of these neuroimmunologists (71%) switched to approved therapies in more than 75% of their relapsing patients.
The likelihood of a clinician switching to an NNT following a patient relapse was not dependent on the length of clinical practice or the number of treated patients.
69% did not switch patients on stable off-label treatment to approved therapies
Twenty-two of 32 respondents (69%) said they had not switch their patients who were stable on off-label treatment to the approved NMOSD therapies. Of those who did, seven out of 10 (70%) did so in up to 25% of their stable patients.
These findings are in line with “the expert consensus statement that for patients who are relapse-free and tolerating an off-label immunosuppressant treatment, there is no need to initiate an NNT,” the team wrote.
Five of 30 neuroimmunologists (16%) said they used NNTs off-label for NMOSD patients without anti-AQP4 antibodies. However, most respondents (76%) would use them if approved for this patient population.
Nearly half of respondents (48%) said they wanted to start patients on NNTs but could not, with insurance coverage or therapy cost as the most frequently reported barrier for all three therapies. A lack of availability or experience with the medication, and patient refusal were among other reported obstacles to NNT treatment.
“Future studies are needed to better ascertain adoption and utilization of NNTs among a larger, more diverse sample of neurologists, including those in other practice settings than academic institutions,” the researchers concluded.