Extrapolation of Impact on Individual Patient Outcomes in Neuromyelitis Optica Spectrum Disorder (NMOSD) Due to Relapse Reduction From Ravulizumab and Other Novel Biologic Treatments
Author(s)
Paulus S. Rommer, MD1, Martin Kleman, MSc2, Jeffrey C. Yu, PhD, MHS3, Banu Kilic Taskopru, MD, PhD, MBA4, Giorgio Castellano, MSc5, Emily Trenholm, BS5, Zoltan Nemeth, MSc6.
1Medical University of Vienna, Vienna, Austria, 2Alexion, AstraZeneca Rare Disease, Baar, Switzerland, 3Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 4Alexion, AstraZeneca Rare Disease, Barcelona, Spain, 5Adelphi Real World, Bollington, Cheshire, United Kingdom, 6Quantify Research, Stockholm, Sweden.
1Medical University of Vienna, Vienna, Austria, 2Alexion, AstraZeneca Rare Disease, Baar, Switzerland, 3Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 4Alexion, AstraZeneca Rare Disease, Barcelona, Spain, 5Adelphi Real World, Bollington, Cheshire, United Kingdom, 6Quantify Research, Stockholm, Sweden.
Presentation Documents
OBJECTIVES: Extrapolate the long-term impact of ravulizumab, satralizumab, and inebilizumab on individual outcomes resulting from relapse reduction in patients with anti-aquaporin-4 antibody-positive (AQP4-Ab+) NMOSD. Data are limited on the long-term consequences of relapse reduction in patients with AQP4-Ab+ NMOSD receiving biologic therapies.
METHODS: Data were sourced from a published indirect treatment comparison of these therapies and from the 2023 Adelphi NMOSD Disease Specific Programme™, a cross-sectional, retrospective survey of physicians and their patients in France, Germany, Italy, Spain, and the United Kingdom. Based on the relapse risk from a network meta-analysis of these therapies, a Markov cohort model was utilized to extrapolate the average risks of individual relapse-associated symptoms/outcomes over a 5-year time horizon.
RESULTS: Of 433 patients with AQP4-Ab+ NMOSD, 18.9%, 5.5%, and 5.1% had 1, 2, and 3+ relapses since their initial NMOSD attack, respectively. The most common symptoms/outcomes (occurring in >10% of patients) were decreased visual acuity (50.3%), bladder control deficit (30.9%), tactile deficit (15.2%), and nociceptive deficit (10.6%). All 5-year modeled symptom/outcome increases from baseline were lowest with ravulizumab versus satralizumab, inebilizumab, or placebo/best supportive care (blindness in one/both eyes [incidence]: 0.002, 0.073, 0.062, 0.256, respectively; nociceptive deficit [incidence]: 0.003, 0.047, 0.042, 0.178; bowel control deficit [incidence]: 0.001, 0.027, 0.023, 0.148; bladder control deficit [incidence]: 0.007, 0.099, 0.089, 0.265; tactile deficit [incidence]: 0.004, 0.050, 0.046, 0.146; symptoms experienced [number]: 0.034, 0.658, 0.574, 2.771; activities of daily living requiring assistance [number]: 0.033, 0.431, 0.385, 1.806; worse than mild pain [proportion of patients]: 0.002, 0.067, 0.057, 0.276; symptomatic treatments [number]: 0.009, 0.148, 0.132, 0.483).
CONCLUSIONS: This research estimates the consequence of relapses experienced by patients with AQP4-Ab+ NMOSD for 3 approved treatment options and placebo/supportive care, illustrating a substantial risk of permanent disability even with biologic treatment. Timely treatment with the most effective preventive therapy may avoid irrevocable deterioration in NMOSD symptoms.
METHODS: Data were sourced from a published indirect treatment comparison of these therapies and from the 2023 Adelphi NMOSD Disease Specific Programme™, a cross-sectional, retrospective survey of physicians and their patients in France, Germany, Italy, Spain, and the United Kingdom. Based on the relapse risk from a network meta-analysis of these therapies, a Markov cohort model was utilized to extrapolate the average risks of individual relapse-associated symptoms/outcomes over a 5-year time horizon.
RESULTS: Of 433 patients with AQP4-Ab+ NMOSD, 18.9%, 5.5%, and 5.1% had 1, 2, and 3+ relapses since their initial NMOSD attack, respectively. The most common symptoms/outcomes (occurring in >10% of patients) were decreased visual acuity (50.3%), bladder control deficit (30.9%), tactile deficit (15.2%), and nociceptive deficit (10.6%). All 5-year modeled symptom/outcome increases from baseline were lowest with ravulizumab versus satralizumab, inebilizumab, or placebo/best supportive care (blindness in one/both eyes [incidence]: 0.002, 0.073, 0.062, 0.256, respectively; nociceptive deficit [incidence]: 0.003, 0.047, 0.042, 0.178; bowel control deficit [incidence]: 0.001, 0.027, 0.023, 0.148; bladder control deficit [incidence]: 0.007, 0.099, 0.089, 0.265; tactile deficit [incidence]: 0.004, 0.050, 0.046, 0.146; symptoms experienced [number]: 0.034, 0.658, 0.574, 2.771; activities of daily living requiring assistance [number]: 0.033, 0.431, 0.385, 1.806; worse than mild pain [proportion of patients]: 0.002, 0.067, 0.057, 0.276; symptomatic treatments [number]: 0.009, 0.148, 0.132, 0.483).
CONCLUSIONS: This research estimates the consequence of relapses experienced by patients with AQP4-Ab+ NMOSD for 3 approved treatment options and placebo/supportive care, illustrating a substantial risk of permanent disability even with biologic treatment. Timely treatment with the most effective preventive therapy may avoid irrevocable deterioration in NMOSD symptoms.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO105
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment, Clinician Reported Outcomes, Relating Intermediate to Long-term Outcomes
Disease
SDC: Neurological Disorders, STA: Biologics & Biosimilars, STA: Multiple/Other Specialized Treatments