New England Institute for Neurology and Headache

Goal concordance underpins high-quality care for patients with serious illness and older adults with multimorbidity and geriatric syndromes; however, a criterion standard for measurement is lacking. To identify and describe patient- and caregiver-reported measures of goal concordance for patients with serious illness, geriatric syndromes, and multimorbidity to create a conceptual model of goal-concordance measurement. This scoping review included an electronic search across MEDLINE, Embase, Web of Science Core Collection, the CINAHL, and PsycINFO through September 2024. Search terms included goals, priorities, preferences, value, expectations, concordance, discordance, match, attain, achieve, align, congruence, dissonance, respect, and terms related to multimorbidity, geriatric syndromes, and serious illness. The review included randomized clinical trials and quasiexperimental, observational, cohort, cross-sectional, mixed methods, quality-improvement, and qualitative studies, plus secondary analyses that either included a patient- and/or caregiver-reported measure of goal concordance as an outcome or as part of a measure development or validation study. Two reviewers extracted data and resolved disagreements via discussion. Study and measure characteristics were summarized and cross-tabulated to identify measurement framework patterns and to propose a conceptual model of measurement. Of 4801 articles screened, 664 full-text articles were assessed for eligibility. A total of 63 studies met inclusion criteria, yielding 67 measures; 44 measures were unique. Most studies were secondary data analyses (17 studies [27.0%]) and included patients with serious illness (44 studies [69.8%]). Measures used 7 of 10 available goal concordance frameworks, with most adopting framework 7 (40 measures [59.7%]), which matched patient preference for an outcome with degree of desired outcome achievement. Four framework components were identified and used to create a conceptual model for goal-concordance measurement: (1) patient preference for an outcome or treatment, (2) treatment undergone or treatment intention, (3) degree of treatment alignment with preference, and (4) degree of desired outcome achievement. This scoping review of patient- and caregiver-reported measures of goal concordance for patients with serious illness, geriatric syndromes, and multimorbidity describes existing measurement frameworks and proposes a conceptual model of measurement that can be used to guide future goal-concordance measure development in geriatrics and palliative care.

BackgroundThis study aims to improve the understanding of the patient journey via the perspectives of patients with migraine.MethodsThe questionnaire was created by patients. It was based on the experiences of 24 patients with migraine and 4 members of the patient advocacy organization La Voix des Migraineux. Two focus groups were held to assess the various dimensions of the patient journey. This survey was accessible on the MoiPatient online platform between March 19th and May 17th, 2022.ResultsOut of the 683 participants (average age: 41.8 years; female: 92.4%; average age at first migraine attack: 16.2 years), 95.9% had received a formal migraine diagnosis from a physician. Migraine had a significant impact on most participants (96.0% had a severe HIT-6 score and 70.7% had severe disability on the MIDAS). Unmet patients’ needs highlighted in this study included diagnosis delay (mean 7.5 years), treatment delay, limited access to neurologists and/or headache specialists, long trial-and-error process of different medications (participants had tried an average of 5.6 acute treatments and 5.0 preventive treatments), numerous (7.2 side effects per participants on average) and often inadequately addressed side effects, suboptimal patient education, and the need for a therapeutic, empathic, and supportive relationship between patients and healthcare professionals. Participants had a negative perception of the care journey and experience with healthcare professionals: 36.2% described their care journey as an uphill battle (“parcours du combatant”). More than half of patients did not feel heard nor supported by healthcare professionals.ConclusionPatients with migraine have to face a multitude of complex obstacles trying to get the care they deserve. The burden of disease is amplified by the complexity of the migraine patient’s journey, both in terms of diagnosis and treatment. This study highlights specific areas in need for improvement.

The therapeutic efficacy remaining from prior treatments with botulinum toxins (BoNTs) when cervical dystonia (CD) patients prefer to be re-treated has not been well characterized. Here, we assessed the residual therapeutic efficacy of BoNT injections at the time of a patient-desired re-treatment. In pivotal trials for daxibotulinumtoxinA (DAXI) in CD, subjects could request re-treatment before returning to pre-treatment symptom levels (defined as ≤20% of peak efficacy remaining). In this post hoc analysis of the Phase 3 ASPEN-OLS trial, the median percent efficacy remaining (based on change in TWSTRS total score) was determined in subjects who requested re-injection before returning to pre-treatment symptoms. Dysphagia and muscle weakness were evaluated in patients requesting re-treatment with efficacy remaining, relative to those waiting to return to baseline. There were 264 (28.7% of 920 total treatments) patient requests for re-treatment before returning to pre-treatment status across the study. The median percent efficacy remaining at the time of requested re-injection was 45.5%, which corresponded to a median of 16.0 weeks (range 10.9-40.3) post-treatment. The rates of dysphagia (≤4.9%) and muscle weakness (≤6.8%) were low and were not significantly different in those who waited for return to pre-treatment symptom status versus subjects who requested re-injection with efficacy remaining. A significant proportion of CD patients wished to be re-treated with efficacy still remaining from prior BoNT injections as early symptoms re-emerged. With the overall clinical profile of DAXI, physicians can safely provide individualized treatment regimens based on the treatment goals or symptomatic needs of their patients.

Traumatic brain injury (TBI) is frequently characterized by chronic motor deficits. Therefore, this clinical trial assessed whether intracranial implantation of allogeneic modified mesenchymal stromal (SB623) cells can improve chronic motor deficits after TBI. Post hoc analysis of the double-blind, randomized, prospective, surgical sham-controlled, phase 2, STEMTRA clinical trial (June 2016 and March 2019) with 48 weeks of follow-up was conducted. In this international, multicenter clinical trial, eligible participants had moderate-to-severe TBI, were ≥12 months postinjury, and had chronic motor deficits. Participants were randomized in a 1:1:1:1 ratio to stereotactic surgical intracranial implantation of SB623 cells (2.5 × 106, 5.0 × 106, 10 × 106) or surgical sham-controlled procedure. The prespecified primary efficacy end point was significantly greater change from baseline of the Fugl-Meyer Motor Scale (FMMS) score, a measure of motor status, for the SB623 pooled vs control arm at 24 weeks. A total of 211 participants were screened, 148 were excluded, and 63 underwent randomization, of which 61 (97%; mean age, 34 [SD, 12] years; 43 men [70.5%]) completed the trial. Single participants in the SB623 2.5 × 106 and 5.0 × 106 cell dose groups discontinued before surgery. Safety and efficacy (modified intent-to-treat) were assessed in participants who underwent surgery (N = 61; SB623 = 46, controls = 15). The primary efficacy end point (FMMS) was achieved (least squares mean [SE] SB623: +8.3 [1.4]; 95% CI 5.5-11.2 vs control: +2.3 [2.5]; 95% CI -2.7 to 7.3; p = 0.04), with faster improvement of the FMMS score in SB623-treated groups than in controls at 24 weeks and sustained improvement at 48 weeks. At 48 weeks, improvement of function and activities of daily living (ADL) was greater, but not significantly different in SB623-treated groups vs controls. The incidence of adverse events was equivalent in SB623-treated groups and controls. There were no deaths or withdrawals due to adverse events. Intraparenchymal implantation of SB623 cells was safe and significantly improved motor status at 24 weeks in participants with chronic motor deficits after TBI, with continued improvement of function and ADL at 48 weeks. Cell therapy can modify chronic neurologic deficits after TBI. ClinicalTrials.gov Identifier: NCT02416492. Submitted to registry: April 15, 2015. First participant enrolled: July 6, 2016. Available at: classic.clinicaltrials.gov/ct2/show/NCT02416492. This study provides Class I evidence that intracranial implantation of allogeneic stem (SB623) cells in adults with motor deficits from chronic TBI improves motor function at 24 weeks.

This post hoc analysis of the PREVAIL study explored the effectiveness of eptinezumab for up to 2 years of open-label treatment in the subgroup of patients with chronic migraine who had a confirmed diagnosis of medication-overuse headache (MOH) at screening. MOH is a disabling and costly secondary headache disorder characterized by increased headache frequency and/or severity with increased acute headache medication use. Eptinezumab, an anti-calcitonin gene-related peptide monoclonal antibody, reduces headache frequency, severity, and associated disability and improves functioning and health-related quality of life as a preventive migraine therapy; short-term benefits in patients with concurrent MOH have also been reported. Participants received up to eight quarterly intravenous infusions of eptinezumab 300 mg in the phase 3, single-arm, open-label PREVAIL study. Safety and patient-reported outcome measures (Migraine Disability Assessment [MIDAS], 6-item Headache Impact Test [HIT-6], patient-identified most bothersome symptom [PI-MBS], Patient Global Impression of Change [PGIC], and 36-item Short-Form Health Survey [SF-36]) were conducted at predefined intervals. Patients were observed up to 20 weeks after their last infusion (Week 104). A total of 49/128 (38.3%) patients enrolled in PREVAIL had an MOH diagnosis at screening. In the MOH subgroup, long-term eptinezumab treatment was associated with reductions in headache frequency (43/49 [87.8%] patients reported ≥50% reduction in MIDAS-derived headache days at ≥1 visit), severity (2.2-point reduction [on a 10-point scale]), disability (mean MIDAS total score reduction of 51.9 points), and impact (mean HIT-6 total score reduction of 9.7 points) at Week 104. Most patients described a "much improved" or "very much improved" status by Week 48 (PI-MBS, 31/46 [67.4%]) and Week 104 (PGIC, 31/36 [86.1%]). Health-related quality of life improvements in the SF-36 were also observed. Eptinezumab preventive therapy in patients with chronic migraine showed benefits that extended to the subset of patients with concomitant MOH.

To assess the residual therapeutic efficacy of prior botulinum toxin (BoNT) injection at the patient-desired retreatment interval in cervical dystonia (CD).