Preference for Inebilizumab Home Infusion Among People with Neuromyelitis Optica Spectrum Disorder
Gerome Vallejos, MD Harvard Medical School Division of Neuroimmunology and Neuroinfectious Disease, Department of Neurology
Gabriela Romanow Harvard Medical School Division of Neuroimmunology and Neuroinfectious Disease, Department of Neurology
Michael Levy, MD, PhD Harvard Medical School Division of Neuroimmunology and Neuroinfectious Disease, Department of Neurology
Abstract
Objective
To determine whether people with neuromyelitis optica spectrum disorder (NMOSD) prefer to receive inebilizumab infusions at home versus in an infusion center or hospital, and to assess how that choice affects their quality of life.
Background
Recently, inebilizumab was approved for treating NMOSD and demonstrated a low risk of infusion reactions. In this study, we collected data on patients’ preferences for home infusions of inebilizumab and its impact on their quality of life.
Design/Methods
This was an observational study of 9 patients with a diagnosis of NMOSD confirmed by a positive AQP4 antibody test. Participants completed a preference survey and the short-form quality of life (SF-36) questionnaire at baseline, and at 1 week after the first and second home infusions. Descriptive statistics for the baseline characteristics were calculated and a linear regression analysis was conducted to identify whether the first or second home infusion was associated with statistically significant improvements or declines in these subdomains.
Results
Nine AQP4+ NMOSD patients met the inclusion criteria, with an average age of 60 ± 12 years (8 females, 1 male). Post-home infusion data showed that 8 out of 9 patients preferred home, felt “very safe” and that it was “much more convenient.” Lastly, all 9 participants strongly preferred future infusions to be at home. Although no significant difference was noted, 2 SF-36 subdomains are highlighted. Physical functioning demonstrated a slight improvement, with a 7.7 increase (95% CI:-21.9-37.5. p = 0.59) following the first home infusion and 3.3 increase (95% CI:-26.4-33.0, p = 0.82) after the second. Also, social functioning showed a slight improvement throughout the home infusion period, reflecting an increase score of 4.2 (95% CI:-26.5-34.9, p = 0.78) after the first infusion and 0 (95% CI:-30.7-30.6, p = 1.00) after the second.
Conclusion
Results suggest that home infusion may be a feasible and preferred option among patients who use inebilizumab to manage their NMOSD, potentially improving their quality of life.
Introduction
Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune disorder affecting 1-5 people/100,000 associated with the presence of aquaporin 4 (AQP4) antibodies which commonly presents with optic neuritis (ON) and/or longitudinally extensive transverse myelitis (LETM).1 Although there is no cure for NMOSD yet, there are medications that work to suppress attacks, which are the drivers of disability in this disease. The impact of this physically debilitating disease is highly dependent on early diagnosis, proper treatment, and slowing of disability progression. B-cell depletion therapy with rituximab has been the off-label mainstay of therapy for prevention of relapses in NMOSD over the past 15 years.2 Due to the high number of infusion-related reactions, rituximab infusions have been largely confined to infusion centers with close observation.3
In 2020, a new B-cell depletion therapy, inebilizumab (Uplizna®, Amgen), was approved for use in NMOSD. The trial compared inebilizumab to placebo (saline) to prevent relapses over the course of 6.5 months and demonstrated a risk reduction of 73%. The safety profile was nearly the same as placebo with only a slight increased incidence of arthralgia and declining IgG levels.4,5
Home-based infusion therapy has emerged as a useful approach in the management of chronic conditions such as cancer and multiple sclerosis (MS), which not only provides patients with greater flexibility and comfort but also reduces the need for frequent hospital visits.6,7 In a pilot study, significant improvements in quality of life were noted among cancer patients receiving home infusion.8 Similarly, for individuals with MS, home therapy options, such as self-administration of medications and remote monitoring, empower patients to take an active role in their care, which ultimately contributes to better health outcomes.9
While inebilizumab was considered safe for home administration due to its low risk of infusion-related reactions in the pivotal trial, there is currently no data indicating whether NMOSD patients prefer home-based infusions. The chronic nature of NMOSD, with its wide range of symptoms such as depression, disability, pain, and fatigue, underscores the need for ongoing research to improve quality of life. Factors like economic stability and access to health care play a significant role in this. Through surveys of NMOSD patients who undergo home infusions, this study explores patient preferences for home-based versus infusion-center based treatments, and to examine how these preferences impact their quality of life.
Methods
Study Design
This was an observational study of 9 participants with NMOSD who were prescribed inebilizumab home infusions for preventive care by their neurologist. Participants were recruited remotely by the study coordinator with cooperation from local neurologists. All consents were conducted by telemedicine visits.
Participants
Inclusion criteria required that patients be at least 18 years of age with a diagnosis of NMOSD confirmed by a positive AQP4 antibody test. Patients either had started to receive inebilizumab at home as part of routine clinical care, or had agreed to start home infusions, and were willing and able to participate. All participants received home infusions facilitated by a certified and trained provider of inebilizumab infusions, according to a standardized protocol. Any infusion-related reactions were thoroughly monitored through nurses’ infusion reports and promptly addressed in accordance with established guidelines. The outcomes were subsequently communicated to our research team for analysis.
Data Collection
Baseline characteristics such as age, sex, and race were collected, as well as clinical data on diagnosis, serostatus, previous and concurrent treatment and comorbidities. We developed a simple 5-question survey for participants. We defined its purpose, identified the audience, designed clear questions and tested the draft. The survey was developed to assess patient preferences regarding inebilizumab infusion locations, specifically comparing home infusions to those at infusion centers or hospitals (Supplementary). Participants were asked about their preferred setting for infusions, their sense of safety during home infusions, the convenience of home versus center-based infusions, and perceived cost differences. They also indicated their preferences for future infusions.
SF-36 is a validated questionnaire designed in 1992 that evaluates health-related quality of life across 8 dimensions: Physical Functioning (PF), Role Physical (RP), Role Emotional (RE), Social Functioning (SF), Bodily Pain (BP), Vitality (VT), Mental Health (MH), and General Health (GH).10 Participants provided responses reflecting their health status after home infusions, which were then scored on a scale from 0 to 100, with higher scores indicating better quality of life.
These surveys were administered at 3 key time points: at the start of the study, after the first home infusion, and after the second home infusion, providing patients with an opportunity to assess preference and quality of life at these junctures. No adverse effects were collected during or after the infusions, as well as during followup assessments.
Statistical Analysis
The raw scores for each of the 8 SF-36 dimensions were obtained by adding the item scores and then transforming them into a scale from 0 to 100, with 0 indicating the poorest health state and 100 representing the optimal health state as assessed by the questionnaire.
The demographics of patients, clinical disease characteristics, treatment variables as well as adverse events were summarized using descriptive statistics. The results were reported as either the mean ± standard deviation (SD) or, for categorical data, as percentages of the overall group. To evaluate the mean score differences across the 3 timepoints, an ANOVA was conducted to assess whether there were statistically significant differences in the means of these 8 SF-36 subdomains over time, accounting for the correlation between repeated measures within patients.
Linear regression analyses were also conducted to evaluate the impact of home infusions across the 8 SF-36 subdomains. Specifically, the analyses aimed to identify whether the first or second home infusion was associated with statistically significant improvements or declines in these subdomains.
A p-value of less than 0.05 was regarded as statistically significant. All analyses were conducted using R version 4.3.2.
Results
A total of 9 AQP4+ NMOSD patients met the inclusion criteria and were included in the study (Table 1). The average age of the participants was 60 ± 12 years. In terms of gender distribution, the cohort was predominantly female, with 8 women (88%) and 1 male (11%), which is consistent with the overall female preponderance in NMOSD.11 Regarding racial demographics, the majority of patients were White (n=6), followed by Black patients (n=2), with 1 patient’s race being either unknown or not reported. Additionally, 8 out of the 9 patients (89%) identified as non-Hispanic. The patients’ prior treatments were also documented: rituximab was the most commonly-used prior therapy, administered to 5 out of 9 patients (56%). Two patients (22%) had been treated with steroids. Comorbidities were prevalent among the study population, with osteoarthritis and hepatitis B being the most frequently reported, each affecting 2 patients. Other notable conditions included sickle cell anemia and Hashimoto’s thyroiditis, each diagnosed in 1 patient.
In the preference survey, 5 key questions were asked before the first home infusion, after the first home infusion, and after the second home infusion. Figure 1 shows the results regarding patient preferences for either home or hospital setting infusions. The results indicate a significant shift in patient preference towards home infusion of inebilizumab, particularly following the first and second home infusion experiences. Prior to receiving home infusions, fewer patients (6 out of 9) expressed a preference for this option. However, post-infusion data show a substantial increase (8 out of 9) in the proportion of patients who “much prefer home,” suggesting a positive reception for home-based administration. No patient reported a preference for infusion center treatment; however, 1 out of 9 expressed an equal preference for both settings across the 3 different time points.
Figure 2 illustrates the results of patient perceptions of safety regarding home-based inebilizumab infusions across different time points. Prior to home infusion, a notable proportion of patients expressed uncertainty, with several selecting “No answer” (3 out of 9) or “I don’t know” (5 out of 9). One patient expressed mild nervousness before the home infusion. However, following the first home infusion and especially after the second, there was a marked increase in the number of patients reporting that they felt “very safe” with 6 out of 9 after the first infusion and 8 out of 9 at the end of the second home infusion. This indicates a positive shift in perceived safety. No patients reported feeling unsafe.
Next, Figure 3 illustrates that the majority of respondents (7-8 (depending on which infusion)) perceived home administration of inebilizumab as “much more convenient” compared to receiving infusions at a hospital or infusion center, which was consistent across all time points. However, prior to home infusion, 1 out of 9 participants reported that home infusions were “a little more convenient,” whereas 1 participant (after the first home infusion) found home infusions to be less convenient.
Figure 4 shows that most respondents were uncertain about cost differences, with the majority selecting “I don’t know,” particularly after the first home infusion (8 out of 9). Across the 3 phases, 1-3 patients did not provide an answer regarding whether they believed there was a cost difference between home and hospital infusions. Additionally, 1 participant perceived no cost difference between the 2 settings.
Lastly, Figure 5 illustrates preferences for the location of the next inebilizumab infusion, comparing responses across 3 phases: before, after the first, and after the second home infusion. Before or after the first home infusion, 6-8 participants expressed a preference for infusions at home, while very few indicated they would “probably” prefer home or were indifferent (“anywhere is fine”) After the second home infusion, all respondents preferred to have future infusions “at home for sure.”
Table 2 provides a summary of the mean scores across the various subdomains of the SF-36. The GH subdomain scores, showed a slight decline over time. The mean GH score was 32 (22) before the home infusion, decreasing to 30 (20) after the first home infusion, and further to 29 (28) after the second home infusion.
Linear regression analysis, as seen in Figure 6, revealed no statistically significant changes in GH scores across time points. Compared to the reference (before home infusion), the change in GH was -2.2 (95% CI: -25.5- 21.1, p = 0.85) after the first home infusion, and -2.8 (95% CI: -26.1-20.5, p = 0.81) after the second home infusion. The PF results show a modest improvement after the first home infusion, followed by a slight decline after the second infusion. Before home infusion, the mean PF score was 26 (29), which increased to 34 (31) after the first home infusion, but slightly decreased to 30 (30) following the second home infusion (Table 2).
However, regression analysis indicates that these changes are not statistically significant. Compared to the reference, there was a 7.7 increase in PF (95% CI: -21.9-37.5, p = 0.59) after the first home infusion, and 3.3 (95% CI: -26.4-33.0, p = 0.82) after the second home infusion (Figure 7). The RP scores showed a decline following home infusions. Before home infusion, the mean RP score was 36 (37), which decreased to 22 (20) after the first home infusion and slightly improved to 25 (21) after the second home infusion.
As illustrated in Figure 8, although not statistically significant, regression analysis shows that the change in RP was -13.9 (95% CI: -50.1- 22.3, p = 0.44) after the first home infusion and -11.1 (95% CI: -47.3 -25.1, p = 0.53) after the second home infusion when compared with before the home infusion. Similarly, the RE scores show a gradual decline following home infusions.
The mean RE score before home infusion was 62 (39), which decreased to 55 (47) after the first home infusion and further to 48 (44) after the second home infusion. As shown in Figure 9, there are no statistically significant changes in RE scores across time points, despite the decline of -7.3 (95% CI: -49.8 -35.2, p = 0.72) after the first home infusion and -14.8 (95% CI: -57.3 – 27.2, p = 0.48) after the second.
The SF scores also showed minimal changes throughout the course of home infusions. Before the home infusion, the mean SF score was 50 (31), which slightly increased to 54 (26) after the first home infusion but returned to 50 (35) after the second (Table 2). Additionally, compared to the pre-home infusion scores, there is a 4.2 (95% CI: -26.5-34.9, p = 0.78) increase in SF after the first home infusion and 0 (95% CI: -30.7-30.6, p = 1.00) after the second home infusion (Figure 10).
Throughout the study, fluctuations were observed in the BP scores. Before the home infusion, the mean BP score was 41 (31), which decreased to 34 (30) after the first home infusion but increased to 43 (21) after the second. However, these changes were not statistically significant. Compared to the reference, the change in BP was -7.1 (95% CI: -34.5- 20.3, p = 0.59) after the first home infusion and 2.1 (95% CI: -25.3- 29.5, p = 0.87) after the second home infusion (Figure 11).
There is also a slight decline on VT scores in between time points. Prior to home infusion, the mean VT score was 31 (19), which dropped to 24 (18) following both the first and second home infusions, with no additional improvement noted. Linear regression analysis reveals that these changes are not statistically significant. The change in VT, as seen in Figure 12, was -6.7 (95% CI: -26.8 -13.5, p = 0.50) after the first home infusion and -7.8 (95% CI: -27.9 -2.3, p = 0.43) after the second home infusion.
Finally, the MH scores show a gradual decline following home infusions. Before the first home infusion, the mean MH score was 63 (23) After the first home infusion, this score dropped to 59 (23) and decreased further to 56 (21) after the second home infusion (Table 2). However, these changes are not statistically significant across the different time points. Which was also on par with the regression analysis, as seen in Figure 13, showing a -4.0 (95% CI: -26.1-18.1, p = 0.71) after the fisrt home infusion and -7.1 (95% CI: -29.2-14.9, p = 0.51) after the second home infusion.
Discussion
Home infusion has been implemented for several chronic inflammatory diseases, including infliximab for Crohn’s disease, subcutaneous rituximab for B-cell malignancies, and ocrelizumab for multiple sclerosis.7,12,13 To date, no studies have focused on the feasibility or preference for home infusions for patients with NMOSD. Therefore, we designed a prospective study to determine whether NMOSD patients on inebilizumab would prefer to get their infusions at home, and if so, whether that choice might impact their quality of life.
In our study, the majority of patients reported that home infusion is a safe and convenient option for their treatment. Additionally, all participants expressed a strong preference for continuing home-based infusions for future treatments. However, one area of uncertainty is the cost impact of home infusion, as many patients remain uninformed about whether it is truly costeffective. Several patients are unaware of the potential financial benefits of home-based treatment. Previous studies have indicated that nursing costs for home infusion tends to be significantly less expensive than hospital-based alternatives.14 Overall, when it comes to patient preferences, home infusion appears to be a widely accepted and favorable method of treatment for individuals with NMOSD.
Quality of life is a multifaceted concept that includes various dimensions, such as physical health, social interactions, emotional well-being, and mental functioning. In our study we used the SF-36 questionnaire to assess the overall quality of life in participants. We gathered data at 3 distinct time points, enabling us to track changes and trends over time, thereby improving our understanding of how home infusion may influence quality of life throughout the treatment journey.
Our findings indicate that home-based infusion has a positive impact on physical functioning (PF). The PF section of the SF-36 evaluates limitations in essential physical activities, such as climbing stairs, walking distances, bending, lifting, and participating in moderate to vigorous exercise. We observed an increase in mean PF scores following the first home infusion, consistent with a study by Polinski, where patients reported enhanced physical functioning.14 This improvement may be attributed to several factors, including reduced travel fatigue and the comfort of a familiar environment. Previous research suggests that home settings can boost patients’ physical functioning,15 as changes in the physical environment such as in-hospital settings can induce stress, ultimately leading to poorer health outcomes.16 Another noteworthy finding relates to social functioning. This section of the questionnaire assesses how physical or emotional difficulties impact an individual’s ability to participate in social activities, such as spending time with friends and attending events. Individuals with NMOSD often face challenges in engaging in social activities over time due to physical disabilities and visual impairments.17 In our study, while the increase in the social functioning domain during the course of home infusion treatments was not statistically significant, the slight trend towards improvement may be linked to various factors, including a reduction in feelings of stigmatization, enhanced psychosocial well-being, greater autonomy, and improved dignity, elements that were effectively seen in a previous review of patients’ and caregivers’ perceptions regarding hospital-at-home services.18 While these 2 domains stand out in this study, it is important to note that the mean scores for the GH, RP, RE, BP, VT, and MH domains declined after the first home infusion. One possible explanation for this observed decline is that patients may have experienced a range of physical and emotional reactions while adjusting to care in a new setting, which could lead to significant emotional stress and heightened expectations. Additionally, although the majority of patients in this study did not report adverse effects from the infusions, the treatment itself may still produce effects that could result in short-term declines in these domains.
The study has several strengths that significantly contribute to the field. First, it focuses on an important and emerging topic of home infusion therapy, offering valuable insights into this treatment modality within neuroimmunology. By prioritizing patient preferences and experiences, our research takes a patient-centered approach, which is essential for enhancing the quality of care. Additionally, employing validated assessment tools, such as the SF-36 questionnaire, strengthens the reliability of the findings related to quality of life measurement. Lastly, collecting data at 3 distinct time points provides a thorough understanding of how home infusion affects patients over time, uncovering trends and shifts in patient quality of life.
Several limitations of this study merit consideration. Firstly, the small sample size (n=9) limits the generalizability of the results and significantly decreases the statistical power to identify meaningful differences. Additionally, the lack of a control group complicates the ability to directly attribute observed changes to home infusion therapy. Moreover, reliance on self-reported measures can introduce bias, as participants’ perceptions of their quality of life may be shaped by subjective influences, including personal expectations and on how they interpret any improvements. Variability in patient’s home environment and support systems may also impact their experiences and outcomes, leading to inconsistencies not captured in the analysis. Also, as this study focused solely on patient perceptions, a cost-effectiveness analysis comparing home infusion to in-hospital administration was not conducted, leaving the financial and economic impact of inebilizumab home infusion unanswered. Furthermore, the study did not utilize a comprehensive array of assessment tools to adequately cover all relevant dimensions of health-related quality of life. Lastly, recall bias is another concern, as participants may find it difficult to accurately remember and recount their experiences over the previous week.
Conclusions
In summary, this study offers important insights into the perceptions and experiences of NMOSD patients concerning home infusion therapy of inebilizumab. Although there are limitations, the results suggest that home infusion can serve as a viable and acceptable option for NMOSD patients, potentially enhancing their quality of life.
Supplementary
References
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