Patient-reported Improvement Research Articles

Quality of life measures with etripamil self-administration for acute episodes of paroxysmal supraventricular tachycardia in a medically unsupervised setting: patient-reported outcomes from NODE-303

Abstract Background Etripamil nasal spray (NS) is a fast-acting, self-administered calcium-channel blocker in development for the conversion of AV-nodal dependent paroxysmal supraventricular tachycardia (PSVT) in a medically unsupervised setting. Prior studies show favorable safety and efficacy in converting single episodes of PSVT to sinus rhythm (SR). Purpose In NODE-303, patient-reported outcome (PRO) instruments assessed quality of life (QoL) over multiple etripamil-treated episodes of PSVT. Methods NODE-303 was an event-driven, multi-center, open-label Phase 3 study in North and South American patients with documented PSVT that evaluated the safety and efficacy of etripamil in PSVT termination, over multiple episodes and without the need for prior test dosing. Enrolled patients who perceived PSVT symptoms applied an ambulatory ECG cardiac monitoring system (CMS), performed a pre-trained vagal maneuver and, if symptoms persisted, self-administered etripamil NS 70 mg. A study amendment allowed a repeat dose (etripamil NS, 70 mg) if symptoms persisted 10 min after the first dose. Each patient could self-treat up to 4 episodes. Safety was assessed through adverse events, clinical data, and ECG CMS recordings. The Treatment Satisfaction Questionnaire for Medication (TSQM; validated for acute episodic assessments) and other PROs were administered, and healthcare utilization data were acquired. Patients completed PROs at baseline (BL), monthly, and per PSVT episode, which collected information on symptoms, episode characteristics, rescue medications, and emergency room visits. Additional PROs were the Brief Illness Perception Questionnaire (BIPQ), Cardiac Anxiety Questionnaire (CAQ), and 36-Item Short Form Health Survey (SF-36). Results There were higher proportions of patients reporting mild, minimal or no disease severity, every 6 months, than at BL; extremely severe, severe, and moderate disease severity was reported by smaller proportions of patients than at BL, though incomplete surveys at later visits may limit this analysis. Anxiety and stress about future PSVT episodes were consistently reduced from BL. Measures of treatment satisfaction, effectiveness, and convenience were consistent across multiple episodes, and were at levels considered favorable for the TSQM instrument (Table 1). Rates of emergency-room visits and hospital admissions across multiple, treated PSVT episodes were similar to those observed in trials assessing single episodes (Table 2). The BIPQ, CAQ or SF-36 had no notable changes from BL. Conclusions Etripamil self-administration was associated with patient-reported improvements in degree of disease severity, decreased anxiety and stress over future PSVT episodes, and levels of treatment satisfaction, effectiveness, and convenience considered favorable for respective PRO instrument. Using a symptom-prompted, self-administered treatment for PSVT could potentially improve QoL measures and reduce healthcare resource utilization.

Exploring Dual-Targeted Therapy in the Management of Moderate to Severe Inflammatory Bowel Disease: A Retrospective Study

Abstract Background Inflammatory bowel disease (IBD), including ulcerative colitis (UC) and Crohn's disease (CD), often results in significant morbidity among patients with moderate to severe forms. While biologics and small molecules are effective in inducing remission, many patients experience refractory disease or extra-intestinal manifestations. This study assesses the safety and efficacy of dual-targeted therapy in IBD patients treated at the Inflammatory Bowel Disease Center. Methods This retrospective cohort study examined 79 patients with UC or CD who received dual-targeted therapy at the University from October 2018 to August 2023. Data collected included demographics, disease characteristics, previous treatments, and clinical outcomes. Primary outcomes were endoscopic, radiographic, and patient-reported clinical improvements, with secondary outcomes focusing on safety profiles. Results Among the 79 patients (42 UC, 37 CD), 97 dual-targeted therapy cases were analyzed, primarily involving a biologic combined with a JAK inhibitor (90.7%). The median therapy duration was 39.1 weeks. Endoscopic improvement occurred in 69% of matched samples, with significant differences between pre- and post-dual-targeted therapy Mayo scores for UC (p = 0.002) and SES-CD scores for CD (p = 0.018). The median pre- and post-dual-targeted therapy Mayo scores across matched samples were 3 (range 1-3) and 1 (range 0-3) respectively, and for SES-CD scores were 12 (range 0-36) and 4 (range 0-20) respectively. Clinical improvement was reported by 73.2% of patients, with notable reductions in ESR (median 19 [range 2-124] mm/hr to 9 [range 0-116] mm/hr, p = 0.006), CRP (median 8.0 [range 0.2-78.5] mg/L to 3.0 [range 0.2-68.2] mg/L, p <0.001) , and albumin levels (4.0 [range 2.2-4.9] mg/dL to 4.2 [range 3.4-5.2], p < 0.001). Non-obesity was associated with both more endoscopic improvement (p = 0.002) and clinical improvement (p = 0.007). Adverse events occurred in 37 cases, predominantly upper respiratory tract infections and dermatologic issues, with no thromboembolic events reported. Discussion Dual-targeted therapy demonstrated efficacy in improving clinical and endoscopic outcomes in patients with severe, refractory IBD, and exhibited an acceptable safety profile. Despite the promising results, further research is needed to confirm these findings and determine optimal therapy combinations.

Midterm Outcomes After Osteochondral Allograft Transplantation in the Knee Using High–Chondrocyte Viability Grafts

Background: Osteochondral allograft transplantation (OCAT) has become a standard-of-care treatment option for patients with large symptomatic articular defects. Recent advances in allograft science and OCAT protocols have been reported to result in consistently robust outcomes after OCAT in the knee. However, only short-term comparisons have been reported, and analyses are lacking for treatment failure risk factors that account for confounding variables. Hypothesis: Midterm functional graft survival rate would exceed 80% for all OCATs combined, with consideration of risk factors for lower survivorship including older patient age, higher body mass index (BMI), tibiofemoral bipolar OCAT, and nonadherence to prescribed postoperative rehabilitation protocols. Study Design: Case series; Level of evidence, 4. Methods: Patients with outcome data available at ≥5 years after primary OCAT using high chondrocyte–viability (HCV) osteochondral allografts were analyzed according to 2 clinically relevant definitions: (1) initial treatment failure, defined by revision or arthroplasty surgery performed for the primary OCAT at any time point during the study period; and (2) functional graft failure, defined by documented conversion to arthroplasty after primary or revision OCAT at any time point during the study period. Analyses were used to assess outcomes for each definition, separately for age group, sex, obesity status, tobacco use, type of OCAT surgery, osteotomy status, concurrent ligament surgery status, and adherence to postoperative protocols. Kaplan-Meier analyses were used to assess differences in survival rates, and Cox proportional hazards models were used to assess risk factors and multivariable relationships with survival. Patient-reported outcome measures for pain, function, mobility, and satisfaction were also analyzed. Results: Analysis included 137 primary knee OCATs performed in 134 patients with a mean follow-up of 66 months (59 female, 75 male; mean age, 37.8 years; mean BMI, 28.5). The midterm (5- to 8-year) functional graft survival rate for patients undergoing primary OCAT in the knee using HCV grafts was 82% for all cases combined, ranging from 69% for tibiofemoral bipolar HCV OCATs to 89% for patellofemoral bipolar, 94% for multisurface unipolar, and 97% for single-surface unipolar. Initial treatment failure rates (revision or arthroplasty after primary OCAT) and OCAT nonsurvival rates (arthroplasty after primary or revision OCAT) were greater for older patient age, concurrent ligament reconstruction, tibiofemoral bipolar OCAT, and nonadherence to the prescribed postoperative rehabilitation protocols. When adjusted for patients’ age, BMI, and tobacco use status, different surgery types did not demonstrate an increased risk for failure, while concurrent ligament reconstruction and nonadherence did. Patients who experienced functional graft survival after primary OCAT reported significantly greater improvements in PROMIS Physical Function and Mobility (Patient-Reported Outcomes Measurement Information System), International Knee Documentation Committee questionnaire, and Single Assessment Numeric Evaluation scores such that they were significantly higher at final follow-up as compared with patients who required arthroplasty. Patient-reported improvements in pain, function, and mobility exceeded minimal clinically important differences for ≥5 years after primary OCAT. When asked if they were satisfied with primary OCAT surgery, 76.2% of patients were very satisfied or satisfied with their results, while 8.5% were neutral and 15.4% were unsatisfied or very unsatisfied. Conclusion: With use of HCV osteochondral allografts, midterm (5- to 8-year) functional graft survival rates for patients undergoing primary OCAT in the knee were notably higher than previously reported midterm rates for traditional OCATs. When adjusted for patient characteristics, risk factors for nonsurvival included concurrent ligament reconstruction for knee instability and nonadherence to the prescribed postoperative rehabilitation protocols. Patients who experienced functional graft survival for ≥5 years after primary OCAT reported statistically significant and clinically meaningful improvements in pain, function, and mobility.

Preoperative Psychopharmacological Treatment Is Not a Risk Factor for Poorer Patient-Reported Improvements 12 months After Hip or Knee Arthroplasty: A Multicenter Registry-Based Cohort Study of 7,247 Procedures

BackgroundPreoperative psychopharmacological treatment (PPT) has been associated with increased hospital length of stay and readmission rate after hip and knee arthroplasty. However, little is known regarding the association between PPT and improvements in patient-reported outcomes postoperatively in a multicenter fast-track setting. Thus, the primary objective was to investigate whether PPT is a risk factor for poorer patient-reported improvements 12 months after surgery. Secondary objectives included assessment of additional time points and subgroups of PPT. MethodsThis multicenter registry-based cohort study included 4,021 primary hip and 3,226 primary knee arthroplasties performed from 2016 to 2020 at three fast-track departments in Denmark due to primary osteoarthritis. The Oxford Hip Score/Oxford Knee Score (OHS/OKS), EuroQol-5 Dimensions-3 Levels/EuroQol-5 Dimensions-5 Levels, and EuroQol visual analog scale were collected at baseline and 3, 6, 12, and 24 months after surgery. Exposure status was assigned using the Danish National Prescription Registry. Marginal mean differences (MD) with 95% confidence intervals (CIs) were estimated using multilevel Tobit regression and adjusted for age, sex, and the Charlson Comorbidity Index obtained from the Danish National Patient Register. ResultsNo associations were found between PPT and improvements in OHS (MD −0.5, CI −1.4 to 0.4) or OKS (MD −0.3, CI −1.2 to 0.5) after 12 months. However, PPT was associated with lower baseline OHS (MD −1.4, CI −2.2 to −0.6) and OKS (MD −2.1, CI −2.9 to −1.3), and 12 months follow-up OHS (MD −1.9, CI −2.8 to −1.1) and OKS (MD −2.4, CI −3.2 to −1.6). Similar findings were observed at other time points, using EuroQol-5 Dimensions-3 Levels/EuroQol-5 Dimensions-5 Levels or EuroQol visual analog scale, and when evaluating PPT subgroups. ConclusionsIn hip and knee arthroplasty, PPT was not a risk factor for poorer patient-reported improvements 12 months after surgery. However, PPT was associated with marginally poorer baseline and follow-up scores. Thus, arthroplasties remain effective treatments despite PPT from a patient-centered perspective.

Efficacy and safety of ritlecitinib, an oral JAK3/TEC family kinase inhibitor, in adolescent and adult patients with alopecia totalis and alopecia universalis.

This post-hoc analysis of the ALLEGRO phase 2b/3 study (NCT03732807) evaluated the efficacy and safety of ritlecitinib, an oral Janus kinase 3/TEC family kinase inhibitor, in patients with alopecia totalis (AT) and alopecia universalis (AU). Patients aged ≥ 12 years with alopecia areata (AA) and ≥50% scalp hair loss received once-daily ritlecitinib 50 or 30 mg (± 4-week 200-mg loading dose) or placebo for 24 weeks. In a subsequent 24-week extension period, the ritlecitinib groups continued their doses and patients initially assigned to placebo switched to ritlecitinib (200/50 or 50 mg daily). In this analysis, clinician- and patient-reported hair regrowth outcomes were assessed at weeks 24 and 48 in four AA subgroups: AT/AU, AT, AU, and non-AT/AU. Safety was monitored throughout. Of the 718 randomized patients, 151 (21%) and 147 (20%) were defined as having AT or AU, respectively. At week 24, Severity of Alopecia Tool (SALT) score ≤20 (≤20% scalp hair loss) response rates were higher in the ritlecitinib-treated AT/AU, AT, and AU groups (7%-14%, 7%-21%, and 4%-10%, respectively) vs the placebo group (0% in the AT/AU, AT, and AU groups). The proportions of patients with a SALT score of ≤20 increased through week 48 (AT/AU, 13%-31%; AT, 11%-27%; AU, 6%-41%). Additionally, at week 24, 25%-43%, 32%-42%, and 12%-50% of patients with AT/AU, AT, and AU, respectively, who received ritlecitinib achieved a moderately or greatly improved response based on the Patient Global Impression of Change scale. Response rates generally increased through week 48 and were similar across AA subgroups. In patients with AT/AU, ritlecitinib was well tolerated with a safety profile consistent with that of the overall AA population. Ritlecitinib demonstrated clinical efficacy, patient-reported improvement, and an acceptable safety profile in patients with AT and AU through week 48. A plain language summary of this study is available at https://doi.org/10.25454/pfizer.figshare.26879161. Clinicaltrials.gov: NCT03732807.

Utilization frequency and patient-reported effectiveness of symptomatic therapies in post-COVID syndrome

BackgroundTo date there is no causal treatment for post-COVID syndrome, leaving symptomatic treatments as the primary recourse. However, the practical implementation and effectiveness of these interventions remain underexplored. This study aimed to investigate the utilization frequency of symptomatic therapies and patient-reported effectiveness across various treatment modalities at a German post-COVID center.MethodsAs the baseline investigation we conducted a single-cohort retrospective study to analyze the frequency of symptomatic therapies among post-COVID patients who attended the post-COVID center of the University Hospital of Erlangen, between December 2022 to July 2023. Additionally, we administered a follow-up at least 3 months after the initial presentation, using a questionnaire to assess patient-reported improvements in post-COVID symptoms associated with the symptomatic therapies received.ResultsOur study included 200 patients (mean age: 44.6 ± 12.6 years; 69.0% women; mean duration since acute infection: 15.3 ± 8.3 months). Pharmacotherapy was the predominant symptomatic treatment (79.5%), with psychotropic drugs (32.5%) and analgesics (31.5%) being the most frequently prescribed. Over half of the patients (55.5%) utilized vitamins and nutritional supplements. Hospital admission rates to acute care occurred in 35.5% of cases; 33.0% underwent inpatient rehabilitation and 31.0% pursued outpatient psychotherapy. Cardiologists (76.5%), pulmonologists (67.5%), and neurologists (65.5%) were the most consulted specialists. Therapies involving medical devices were infrequently employed (12.0%). In a follow-up questionnaire (response rate: 82.5%, 6.3 ± 2.2 months post-baseline), beta-blockers were the most effective pharmacological intervention with 31.5% of patients reporting strong to very strong symptom improvement, followed by antibiotics (29.6%). Furthermore, 33.0% of the patients perceived plasmapheresis to strongly alleviate symptoms. Only a small proportion of the sample attributed a strong or very strong symptom improvement to outpatient psychotherapy (11.0%).ConclusionThis study provides initial insights into symptomatic therapy utilization and patient-reported symptom improvement in post-COVID syndrome. Further research into symptoms clusters and interdisciplinary collaboration are warranted to comprehensively address the multifaceted physical and psychological symptomatology.Trial registrationThe study was registered at the German Clinical Trials Register (DRKS-ID: DRKS00033621) on March 20, 2024.

Safety and Efficacy of Balloon-Assisted Kyphoplasty Followed by Stereotactic Body Radiation Therapy for Pathological Fractures.

In patients experiencing pain secondary to pathological compression fractures, balloon-assisted kyphoplasty and subsequent stereotactic body radiation therapy (SBRT) may allow for restoration of vertebral height and irradiation of the underlying malignancy to control local disease progression. The aim of this study was to evaluate the safety and efficacy of kyphoplasty treatment before SBRT in patients with spinal metastases and benign tumors. An analysis of a prospectively collected database of 70 patients and 75 metastatic and benign spinal lesions that underwent kyphoplasty before SBRT at a single institution (2002-2023) was conducted. Patient characteristics were recorded, including treatment history, dosimetry and fractionation schedule, pain outcomes, local control (LC), and overall survival. The Bilsky score and Spinal Instability Neoplastic Score were calculated to assess epidural involvement and spinal stability, respectively. The median time from kyphoplasty to SBRT was 29 days (range: 2-159). Seventy-two lesions (96%) were managed with single-fraction SBRT and 3 lesions (4%) with a multifraction regimen. The median single-fraction prescription dose was 20 Gy (range: 12-25) delivered to a median tumor volume of 35.1 cc (range: 2.2-160). After a median follow-up period of 9 months (range: 1-201), 6 lesions (8%) locally progressed. Pain was reported to improve or remain stable for most patients (88%). The LC rate was 88% at 6 months, 1 year, and 2 years. No prognostic factors were significantly associated with LC. The median overall survival was 11 months (range: 1-201) after radiosurgery. There were no reports of cement extravasation or radiation-induced neurological deficit. Two acute pain flares (3%) were reported 1 and 3 months after radiosurgery. The combined kyphoplasty and SBRT treatment paradigm can be used to treat patients with painful pathological compression fractures. Long-term LC and patient-reported improvement in pain were observed without the morbidity associated with open surgery.

Response to Upadacitinib in Patients with Inflammatory Bowel Disease Previously Treated with Tofacitinib.

Upadacitinib is an oral selective Janus kinase (JAK) inhibitor approved in the United States for ulcerative colitis (UC) and Crohn's disease (CD). However, data regarding its use following prior treatment with the JAK inhibitor tofacitinib is sparse. As such, we aimed to evaluate the effectiveness of upadacitinib therapy following tofacitinib exposure. This is a multicenter retrospective study of patients with confirmed diagnosis of UC or CD who received upadacitinib after prior treatment with tofacitinib. The primary outcome of interest was patient-reported clinical improvement at first follow-up. Secondary outcome included discontinuation of corticosteroids, change in Mayo Endoscopic Score (MES) and change in inflammatory marker levels. A total of 31 patients met the inclusion criteria. Following upadacitinib initiation, 80.6% (25/31) of patients had clinical improvement, including 92.3% (24/26) of those with UC and 20% (1/5) of those with CD. Of the patients initially requiring systemic corticosteroid therapy, 80% (12/15) were able to discontinue corticosteroids. Individual mean change of fecal calprotectin was a decrease of 501.5mcg/g ± 608.6 (P value = 0.01) while C-reactive protein decreased on average by 14.8mg/L ± 25.3 (P value = 0.02) compared to when patients were on tofacitinib, with significant changes observed in the UC cohort. In patients with UC, individual MES after initiating upadacitinib decreased compared to prior to tofacitinib discontinuation (P value = 0.04). Our study demonstrates that upadacitinib therapy in patients with prior tofacitinib exposure is associated with clinical improvement and a decrease in objective markers of inflammation in patients with UC.

Retrospective study of the efficacy of methylprednisolone vs. triamcinolone in lumbar epidural steroid injections for the treatment of low back pain due to degenerative disc disease

ObjectiveA common low back pain treatment is epidural injection of corticosteroids. The nominal target of anti-inflammatory corticosteroid drugs is the glucocorticoid receptor (GR). In vitro studies show many clinically used steroids also activate the mineralocorticoid receptor (MR) with substantial potency. Based on preclinical studies, this may have pro-inflammatory and pro-nociceptive effects that counter the desired GR effects. Of two outpatient pain clinics associated with the University of Cincinnati Department of Anesthesiology, one primarily used methylprednisolone while the other used mainly triamcinolone for epidural steroid injections. We hypothesized that triamcinolone would give better outcomes because in vitro, ratio of MR/GR potency is about 10 fold less favorable for methylprednisolone.MethodsWe conducted a retrospective chart review of adults receiving lumbar epidural steroid injection for low back pain due to degenerative disc disease at the two pain clinics. For subjects treated at the first clinic, we obtained basic demographics, smoking history, 2 primary outcomes (patient-rated percent improvement in pain levels, and injection outcome rated as poor, partial, or good), and pain ratings (0–10 scale) before and after injection. For analysis, a subset of subjects from the second clinic was matched as closely as possible (sex, age, race, and ethnicity) to those from the first clinic.ResultsEighty-six subjects from the first clinic were identified, of whom fifty-five met inclusion criteria. Review of 83 potentially matched subjects from the second clinic yielded 37 subjects. From this combined set of subjects, 44 receiving triamcinolone and 48 receiving methylprednisolone were obtained. Matching was effective in avoiding significant differences between the two drug groups in age, weight, sex, race, and body mass index, however, the incidence of smoking (current and former) was significantly higher in the methylprednisolone group (who were primarily from clinic 1). The injection responses codified on a 0–2 scale, where 0 indicated a poor response, 1 a partial response with a second injection recommended, and 2 a good response where no further treatment was recommended at the 1 month follow up point, were not significantly different between the groups (Mann–Whitney, p = 0.44) although the triamcinolone group overall had slightly better responses. However, the patient-reported percent improvement after the injection was significantly better for the triamcinolone than for methylprednisolone (60% ± 5.3 vs. 42% ± 4.9), as was the pain ratings (0–10 scale) after the injection (5.0 ± 0.5 vs. 6.3 ± 0.3). A marked demographic difference between the two clinics in smoking rates was not controlled for in subject matching but accounting for smoking status did not affect the observed differences between the two steroids.ConclusionsDifferences in the two primary outcomes, patient-reported percent improvement and pain ratings after epidural steroid injection, were consistent with the hypothesis that more GR-selective steroids may give better outcomes though the differences were modest. We propose that one factor in choosing steroids should be their relative potency in also activating the pro-inflammatory mineralocorticoid receptor.Graphical

External Validation Demonstrates Machine Learning Models Outperform Human Experts in Prediction of Objective and Patient-Reported Overactive Bladder Treatment Outcomes

ObjectivesTo predict treatment response for overactive bladder (OAB) for a specific patient remains elusive. We sought to develop accurate models using machine learning for prediction of objective and patient-reported treatment response to intravesical botulinum toxin (OBTX-A) injection. We sought to validate the models in a challenging setting using an external dataset of a markedly different patient cohort and dosing regimen. We hypothesized the model would outperform human experts and top available algorithms. MethodsAlgorithms using “operator splitting” designed for accuracy and efficiency even in small training data sets with variable completeness, were trained to predict objective response and patient-reported symptomatic improvement using the ROSETTA trial cohort and validated using the ABC trial cohort of patients who underwent OBTX-A. Areas under the curve (AUC) of algorithms were compared to the top publicly-available machine learning classifier XGBoost, logistic regression with cross validation, and human expert predictions in the external validation set. ResultsIn the validation set, the operator splitting neural network had AUC 0.66 and outperformed XGBoost with DART (top available machine learning classifier, AUC 0.58), logistic regression (AUC 0.55), and human experts (AUC 0.47 – 0.53) for prediction of clinical responder status. It was similarly accurate in prediction of patient subjective improvement in symptoms following OBTX-A (AUC: 0.64), again outperforming other algorithms and human experts (AUC 0.41 – 0.62). ConclusionsThe neural network outperformed human experts and other machine learning approaches in prediction of objective and patient-reported OBTX-A outcomes for overactive bladder in a challenging independent validation cohort. Clinical implementation could improve counseling and treatment selection.

Great expectations: A multisite, randomized controlled open-label trial of a novel patient expectations communication tool.

In emergency medicine (EM), discerning patient expectations to inform patient-centered care poses unique challenges. We devised a novel questionnaire to facilitate clinicians' understanding of patients' expectations for their visit. We conducted a multisite, randomized controlled trial. A brief questionnaire soliciting patient expectations was developed through feedback from clinicians and patients. At the beginning of their visit, the intervention group patients completed the questionnaire and provided it to their treating clinician. The control group patients received standard care. Participants in both groups completed a survey at time of disposition assessing five satisfaction domains. The primary ordinal logistic regression analysis modeled the extent to which the intervention led to patient-reported improvement in clinician understanding of expectations with adjustment for demographic factors and site. Among the 308 participants, 141 intervention and 123 control exit surveys were collected. In the primary analysis, the intervention group had 2.1 times greater odds of strongly agreeing that their clinician understood their expectations (odds ratio: 2.1, 95% confidence interval: 1.2, 3.7, p-value: 0.01), roughly equivalent to a net number needed to treat of 11 for one more improved satisfaction rating. Although the secondary outcome results were not significantly different between groups, all results tended toward the intervention group having more favorable answers. While more patients reported that their expectations were addressed when the novel tool was used, no significant difference was found for conventional satisfaction measures. Future studies may examine whether a tool to elicit patient expectations can lead to other improved outcomes.

Outcomes of a step-up approach to the treatment of neurogenic cough

IntroductionNeurogenic cough (NC) is thought to be related to sensory neuropathy in the hypopharynx and larynx. Defined as a cough persisting longer than 8 weeks refractory to standard therapy, it is a diagnosis of exclusion when other common etiologies (asthma, gastroesophageal reflux disease (GERD), medication side effects) are ruled out. It affects roughly 11 % of Americans and can negatively impact quality of life. MethodsFollowing institutional review board approval, we evaluated the medical records of adult patients seen at the University of Arizona's tertiary laryngology center from 2018 to 2023. Patients were included if their cough persisted for >8 weeks, and they either did not respond to prior proton pump inhibitor and asthma therapy or had GERD and asthma ruled out. These patients underwent a progressive escalation of therapy, which included neuromodulators with or without cough suppression therapy, superior laryngeal nerve (SLN) block, and laryngeal botulinum toxin injections. The primary outcome was patient-reported improvement in cough symptoms rated on a 1–5 scale: 1 = no response, 2 = mild improvement, 3 = moderate improvement, 4 = significant improvement, 5 = complete resolution. ResultsA total of 56 patients were included. Mean (SD) age was 64.6 (14.8) years, and 66 % were female. Overall, 42 patients (75.0 %) responded to treatment. Among responders, 7 (16.7 %) experienced mild improvement, 14 (33.3 %) experienced moderate improvement, 17 (40.5 %) experienced significant improvement, and 4 (9.5 %) experienced complete resolution of their cough.33 patients (58.9 %) were managed exclusively with neuromodulators ± cough suppression therapy; 27 responded, with an average response rating of 3.0 (SD = 1.2). 11 patients (19.6 %) failed medical therapy and underwent SLN block without subsequent botox treatment; 7 responded, with an average response rating of 2.5 (SD = 1.4). 9 patients (16.1 %) failed all previous therapies and underwent laryngeal botulinum toxin injections; 6 responded with an average response rating of 2.4 (SD = 1.3). The remaining 3 patients underwent cough suppression therapy alone, with 2 responding and an average response rating of 3.3 (SD = 1.7). ConclusionsNeurogenic cough can be effectively treated with a stepwise multimodal approach, including neuromodulators, cough suppression therapy, SLN block, and laryngeal botulinum toxin injections.

Identifying SCS Trial Responders Immediately After Postoperative Programming with ECAP Dose-Controlled Closed-Loop Therapy.

Drawbacks of fixed-output spinal cord stimulation (SCS) screening trials may lead to compromised trial outcomes and poor predictability of long-term success. Evoked compound action potential (ECAP) dose-controlled closed-loop (CL) SCS allows objective confirmation of therapeutic neural activation and pulse-to-pulse stimulation adjustment. We report on the immediate patient-reported and neurophysiologic treatment response post-physiologic CL-SCS and feasibility of early SCS trial responder prediction. Patient-reported pain relief, functional improvement, and willingness to proceed to permanent implant were compared between the day of the trial procedure (Day 0) and end of trial (EOT) for 132 participants in the ECAP Study undergoing a trial stimulation period. ECAP-based neurophysiologic measurements from Day 0 and EOT were compared between responder groups. A high positive predictive value (PPV) was achieved with 98.4% (60/61) of patients successful on the Day 0 evaluation also responding at EOT. The false-positive rate (FPR) was 5.6% (1/18). ECAP-based neurophysiologic measures were not different between patients who passed all Day 0 success criteria ("Day 0 successes") and those who did not ("needed longer to evaluate the therapy"). However, at EOT, responders had higher therapeutic usage and dose levels compared to non-responders. The high PPV and low FPR of the Day 0 evaluation provide confidence in predicting trial outcomes as early as the day of the procedure. Day 0 trials may be beneficial for reducing patient burden and complication rates associated with extended trials. ECAP dose-controlled CL-SCS therapy may provide objective data and rapid-onset pain relief to improve prognostic ability of SCS trials in predicting outcomes. The ECAP Study is registered with ClinicalTrials.gov (NCT04319887).

Linking Patient-Reported Outcome Measure Scores to Adverse Event Data to Gain Insight into Overestimation of Postoperative Patient-Reported Outcome Measure Improvement After Total Hip Arthroplasty and Total Knee Arthroplasty Due to Selective Nonresponse

BackgroundThe purpose of the study was to gain insight into how clinically relevant improvement in patient-reported outcome measure scores after total hip arthroplasty (THA) and total knee arthroplasty (TKA) may be underestimated or overestimated, we compared patient-reported outcome measure respondents and nonrespondents on their adverse event rates and assessed whether adverse event occurrence was associated with clinically relevant patient-reported outcome measure improvement from those without adverse events. MethodsAll primary THAs and TKAs performed in 19 Dutch hospitals between January 2017 and December 2019 were included. The hip disability and osteoarthritis outcome score-physical function short form (HOOS-PS) and knee injury and osteoarthritis outcome score-physical function short form (KOOS-PS) were used to assess the physical function after THA and TKA, respectively. Adverse events included 1-year revision, 30-day readmission, 30-day complications, and long (ie, >75th percentile) length of stay. A clinically relevant improvement was defined as at least a 10-point decrease in HOOS-PS and 9 points in KOOS-PS scores. Associations between adverse events and clinically relevant HOOS-PS and KOOS-PS improvement were assessed using binary logistic regression models adjusted for patient characteristics and clustering of patients within hospitals. ResultsThere were 20,338 THA and 18,082 TKA procedures included. Adverse events occurred more frequently in HOOS-PS and KOOS-PS nonrespondents than in respondents. The THA patients experiencing revision, complications, or long length of stay were less likely to experience clinically relevant HOOS-PS improvements (odds ratios of 0.11 [0.06 to 0.20], 0.44 [0.30 to 0.63], and 0.66 [0.50 to 0.88], respectively). The TKA patients experiencing revision or long length of stay were less likely to experience clinically relevant KOOS-PS improvements (odds ratios of 0.26 [0.12 to 0.55] and 0.63 [0.50 to 0.80], respectively). ConclusionsClinically relevant HOOS-PS and KOOS-PS improvements are likely overestimated, as nonrespondents had higher adverse event rates which were associated with lower likelihood to achieve clinically relevant HOOS-PS and KOOS-PS improvements.

Associations between surgeons' preoperative expectations of lumbar surgery and patient-reported 2-year outcomes.

Surgeons' preoperative expectations of lumbar surgery may be associated with patient-reported postoperative outcomes. Preoperatively spine surgeons completed a validated Expectations Survey for each patient estimating amount of improvement expected (range 0-100). Preoperative variables were clinical characteristics, spine-specific disability (ODI), and general health (RAND-12). Two years postoperatively patients again completed these measures and global assessments of satisfaction. Surgeons' expectations were compared to preoperative variables and to clinically important pre- to postoperative changes (MCID) in ODI, RAND-12, and pain and to satisfaction using hierarchical models. Mean expectations survey score for 402 patients was a 57 (IQR 44-68) reflecting moderate expectations. Lower scores were associated with preoperative older age, abnormal gait, sensation loss, vacuum phenomena, foraminal stenosis, prior surgery, and current surgery to more vertebrae (all p ≤ .05). Lower scores were associated postoperatively with not attaining MCID for the ODI (p = .02), RAND-12 (p = .01), and leg pain (p = .01). There were no associations between surgeons' scores and satisfaction (p = .06-.27). 55 patients (14%) reported unfavorable global outcomes and were more likely to have had fracture/infection/repeat surgery (OR 3.2, CI 1.6-6.7, p = .002). Surgeons' preoperative expectations were associated with patient-reported postoperative improvement in symptoms and function, but not with satisfaction. These findings are consistent with clinical practice in that surgeons expect some but not complete improvement from surgery and do not anticipate that any particular patient will have markedly unfavorable satisfaction ratings. In addition to preoperative discussions about expectations, patients and surgeons should acknowledge different types of outcomes and address them jointly in postoperative discussions.

Incentives and individualized coaching improve completion rates of supervised exercise therapy for claudication

ObjectiveSupervised exercise therapy (SET) provides clinical benefit for patients suffering from intermittent claudication and has been widely recommended as first-line therapy before endovascular or surgical intervention. However, published rates of SET program completion range from 5% to 55%, with historic completion of 54% at our own institution. As such, we sought to identify if targeted patient-supportive interventions improve SET completion rates while still maintaining efficacious SET programming. MethodsPatients who were diagnosed with intermittent claudication, as defined by ankle-brachial index (ABI) <0.9 without rest pain, were offered enrollment in a prospective quality improvement protocol for our 12-week SET for peripheral artery disease program. Program completion was defined as ≥24 of 36 offered sessions over 12 weeks. A three-pronged approach was utilized to improve completion during the study, including financial incentives up to $180, scheduled coaching with our advanced practitioner staff, and informational materials on the importance of SET programming and lifestyle modification. Patient-reported improvements in walking symptoms were tracked via regularly administered questionnaires. Functional measures of SET programming including total walking duration and distance, metabolic equivalent of task, and ABIs; vascular intervention within 12-months after enrollment was also collected and compared using univariate paired analysis. ResultsIn total, seventy-three patients were enrolled in SET for peripheral artery disease programming over the study period. Utilizing our three-pronged coaching approach, 56 patients completed SET programming, increasing our SET completion rate to 76.7% over a 2-year study period. Compared with pre-SET baseline, patients who completed SET noted less pain, aching, cramps in calves when walking (P = .004), and less difficulty walking 1 block (P = .038). Additionally, patients significantly increased their metabolic equivalent of task (3.1 vs 2.6; P < .001), total walking duration (30 mins vs 13.5 mins; P < .001), and total walking distance (0.7 vs 0.3 miles; P < .001) from their pre-SET baseline. There were no changes in participant ABIs from enrollment to completion in participants. Patients who completed SET programming also delayed vascular intervention compared with those who did not complete SET or declined participation (213.5 vs 122.5 days from enrollment; P = .041). ConclusionsTargeted incentives, including cost-coverage vouchers and personalized coaching with instructional materials, successfully improved patient completion of a prescribed SET program. Patients who completed SET programming reported subjective improvement in walking symptoms and objective walking benefits. In addition, these patients had delayed time to vascular intervention, supporting current vascular guidelines advocating for effective SET therapy prior to offering vascular intervention for intermittent claudication.

Onabotulinum toxin injections for shoulder and chest wall muscle pain in breast cancer survivors: retrospective study – preliminary report

ObjectivesThe primary objective of this retrospective review is to describe patient-reported improvement in muscular pain after initial treatment with onabotulinum toxin. A secondary objective was to determine other physiatry (physical...

Mid-term outcomes using a ‘kinematic retaining’ total knee replacement – A multicentre prospective study at five years follow-up

BackgroundAlthough total knee replacement (TKR) surgery has succeeded in improving pain and deformity, a proportion of patients remain incompletely satisfied with their outcome. This prospective study aims to assess the survivorship, clinical, and radiological outcomes using a novel ‘kinematic retaining’ (KR) implant. Methods156 patients underwent TKR surgery for primary osteoarthritis using the Physica KR implant at three European Centres. Patients were followed up for five years using both radiographic and clinical evaluations. ResultsWithin 6 months post-operatively, 79.4% and 85.9% had good-excellent clinical and functional KSS values, this was maintained to 76.9% and 79.5% at five years. Mean Knee Society Score (KSS) improvement at 5 years was 32.8 (from 23 to 40) and 37.4 (from 30 to 50) (p < 0.01). All Knee Injury and Osteoarthritis Outcome Score (KOOS) sub-scores showed statistically significant improvement from before surgery at a mean of 34.7 (SD ± 16.1) to a mean of 86.6 (SD ± 16.1) at five years. The mean Oxford Knee Score (OKS) was 43.7 (±5.6), with over 80% of the patients having a good-excellent outcome at five years. OKS improved significantly by six weeks after surgery (p < 0.01) and remained constant throughout the 5-year follow-up. Visual Analogue Score (VAS) Satisfaction scores improved significantly after the post-operative time point of six weeks. From 1 year to 5 years, the average VAS was over 85 mm. The Forgotten Joint Score (FJS) increased from 64.5 at 1 year to 79.2 at 5 years after surgery (p < 0.01). No progressive adverse radiographic features were noted. Two patients were revised during the study period: one for infection and the other for aseptic loosening. ConclusionsThis novel ‘kinematic retaining’ knee prosthesis has shown exceptional clinical and patient-reported improvements, with a remarkable 99.4% survivorship (95.5–99.9) at five years.

An Interdisciplinary Protocol for Ventriculoperitoneal Shunt Patient Selection in Normal Pressure Hydrocephalus

BackgroundNormal pressure hydrocephalus (NPH) can be treated with ventriculoperitoneal shunt (VPS) placement, but no broadly implemented indication for VPS exists. MethodsOur protocol consists of physical therapy and occupational therapy practitioners administering validated tests of gait, balance, and cognition before and after lumbar drain (LD) placement. Specific tests include: Timed “Up & Go”, Tinetti Gait and Balance Assessment, Berg Balance Scale, Mini Mental Status Exam, Trail Making Test Part B, and the Rey Auditory and Visual Learning Test. Minimal clinically important difference values for each test were determined from literature review. A retrospective review of patients treated under this protocol was performed. The primary outcomes were candidacy for VPS based on the protocol and patient-reported symptomatic improvement after VPS placement. Results48/75 (64%) patients received VPS. 43/48 (89.6%) of those shunted reported improved symptoms at 6 week follow up. However, 10/22 (45.5%) reported worsening symptoms at 1 year follow up. The mean Tinetti score significantly increased after LD in patients who improved with VPS compared to the no shunt group (4.27 vs -0.48, p<0.001). 6/33 (18%) patients with post-op imaging had a subdural fluid collection identified and 3/49 (6%) had other complications, including 1 seizure, 1 intracerebral hemorrhage, and 1 stroke. ConclusionsStandardized assessment of gait, balance, and cognition before and after temporary CSF diversion identifies patients with NPH likely to benefit from VPS placement with a low complication rate. One year after VPS, approximately half of patients had symptoms recur.

Surgical Intervention is Associated with Improvement in Health-Related Quality of Life Outcomes in Patients with Symptomatic Sacral Tarlov Cysts: Results from a Prospective Longitudinal Cohort Study

The treatment of symptomatic Tarlov cysts remains a controversial topic within neurosurgery. We describe our experience with patients who underwent surgical intervention for sacral Tarlov cysts at a single institution. General and disease-specific outcome measures were used to assess health-related quality of life. Patients who underwent surgical treatment for one or more sacral Tarlov cysts between 2018 and 2021 were included. The Tarlov Cyst Quality of Life (TCQoL), a validated disease-specific measure, was the primary outcome of the study. Secondary outcomes included general outcome measures: 36-Item Short Form Survey, the Oswestry Disability Index, and Visual Analog Scale. Patients were followed at 3, 6, and 12 months postoperative. Repeated measures analyses were used to assess change from preoperative to 12 months postoperative. Data were obtained from 144 patients who underwent surgery for sacral Tarlov cysts, average age 52.3 ± 11.3 years, 90.3% female. Patients reported significant mean improvement on the TCQoL over time (preoperative 3.2±0.1; 3-months postoperative 2.1±0.1; 6-months 1.9±0.1; 12-months 1.9±0.1; P< 0.001). Patient age and duration of symptoms were not associated with outcome. 82.3% of patients reported improvement on TCQoL. There was not a significant difference in the proportion of patients reporting improvement on TCQoL by cyst size (small 90.9% vs. large 77.9%; P = 0.066). Our longitudinal series demonstrated patient-reported improvement following surgery for symptomatic sacral Tarlov cysts using a validated disease-specific health-related quality of life scale through 12 months following surgery. Patient age and preoperative duration of symptoms were not correlated with outcome.