SF-36 Physical Component Score Research Articles

#366 Humanistic burden of immunoglobulin A nephropathy and focal segmental glomerulosclerosis on patients and care-partners (HONUS): results for Europe

Abstract Background and Aims Rare proteinuric kidney diseases are associated with clinical and economic burden, however, the humanistic burden associated with these conditions has not been directly evaluated. HONUS is a multi-national, cross-sectional survey study designed to evaluate the humanistic burden associated with two rare kidney diseases, immunoglobulin A nephropathy (IgAN) and focal segmental glomerulosclerosis (FSGS), from the patient and caregiver perspectives in the United States and Europe. The current analysis presents preliminary results from adults with IgAN or FSGS and their care-partners in Spain, Germany, France, or the United Kingdom. Method The study recruited adult patients (≥ 18 years old) with IgAN or FSGS and their adult care-partners. Participants completed an online survey including demographic and clinical characteristics, health-related quality of life (HRQoL) (i.e., Kidney Disease Quality of Life Instrument [KDQoL] including 12-Item Short Form Survey [SF-12]), anxiety and depression (i.e., Generalized Anxiety Disorder Assessment [GAD-7], Patient Health Questionnaire [PHQ-9]), and disease impact on work productivity (i.e., Work Productivity and Activity Impairment [WPAI]). Descriptive analyses were conducted. Results From Europe, 26 IgAN patients, 9 FSGS patients, and their care-partners (4 IgAN and 2 FSGS patients did not report having a care-partner) participated in the survey before November 2023. Mean age for IgAN and FSGS patients was 42.2 years and 51.4 years, with 61.5% and 44.4% being female, respectively. Over two-fifths of both IgAN (42.3%) and FSGS patients (44.4%) were in chronic kidney disease (CKD) stage 3, 4 or 5 (on dialysis), while over one-quarter had received a kidney transplant (IgAN: 26.9%; FSGS: 44.4%). Most patients had worse CKD status since diagnosis (IgAN: 61.5%; FSGS: 77.8%). Hypertension and anemia were the most common comorbidities in both groups. In terms of HRQoL, mean (SD) SF-12 physical and mental component scores (PCS, MCS) indicated IgAN patients were more impacted on the mental component (PCS: 47.3 [11.6], MCS: 43.2 [10.9]), with FSGS patients more impacted on the physical component (PCS: 40.6 [6.0], MCS: 43.8 [9.1]), reflecting generally worse HRQoL (lower scores) than previously published European general population scores (50.0 for PCS and MCS). Moderate to severe anxiety was reported by 30.8% of IgAN and 33.3% of FSGS patients, and moderate to severe depression by 30.8% of IgAN and 44.4% of FSGS patients. Among employed IgAN patients (69.2%), mean percent absenteeism (work time missed) was reported at 13.3%, presenteeism (impairment while working) at 17.7%, and overall work productivity loss at 17.7% due to IgAN-related reasons (only 3 FSGS patients [33.3%] were employed; outcomes not shown). Mean percent activity impairment was reported at 27.3% in IgAN and 43.3% in FSGS patients. Most paired care-partners were partners of patients (IgAN: 81.8%, FSGS: 71.4%), with a mean age of 46.6 years (IgAN) and 50.1 years (FSGS). Mean (SD) SF-12 PCS and MCS of IgAN care-partners were 53.3 (8.8) and 47.9 (10.8), respectively (FSGS: 47.0 [10.6], 49.4 [9.5]). Mild to moderate anxiety was reported in 31.8% (IgAN) and 42.9% (FSGS) of care-partners, with one IgAN care-partner (4.6%) reporting severe anxiety. Mild to moderate depression was reported in 27.3% (IgAN) and 71.4% (FSGS) of care-partners, with two IgAN care-partners (9.1%) reporting moderately severe or severe depression. Among employed IgAN care-partners (86.4%), mean percent absenteeism was reported at 14.2%, presenteeism at 9.3%, and overall work productivity loss at 9.9% due to IgAN-related reasons. Mean percent activity impairment was reported at 18.6% in IgAN and 17.1% in FSGS care-partners. Conclusion Patients with IgAN and FSGS in Europe experience impaired HRQoL compared to previously published European population estimates, as well as anxiety, depression, and impaired productivity. Care-partners of these patients also experience impaired HRQoL, in terms of mental components, and reduced overall productivity.

Safety and efficacy of umbilical cord tissue-derived mesenchymal stem cells in the treatment of patients with aging frailty: a phase I/II randomized, double-blind, placebo-controlled study

BackgroundMesenchymal stem cells (MSCs) hold a great promise for cell-based therapy in the field of regenerative medicine. In this study, we aimed to evaluate the safety and efficacy of intravenous infusion of human umbilical cord-derived MSCs (HUC-MSCs) in patients with aging frailty.MethodsIn this randomized, double-blind, placebo-controlled trial, participants diagnosed with aging frailty were randomly assigned to receive intravenous administrations of HUC-MSCs or placebo. All of serious adverse events and AEs were monitored to evaluate the safety of treatment during the 6-month follow-up. The primary efficacy endpoint was alteration of physical component scores (PCS) of SF-36 qualities of life at 6 months. The secondary outcomes including physical performance tests and pro-inflammatory cytokines, were also observed and compared at each follow-up visits. All evaluations were performed at 1 week, 1, 2, 3 and 6 months following the first intravenous infusion of HUC-MSCs.ResultsIn the MSCs group, significant improvements in PCS of SF-36 were observed from first post-treatment visit and sustained throughout the follow-up period, with greater changes compared to the placebo group (p = 0.042). EQ-VAS scores of MSCs group improved significantly at 2 month (p = 0.023) and continued until the end of the 6-month visit (p = 0.002) in comparison to the placebo group. The timed up and go (TUG) physical performance test revealed significant group difference and showed continual enhancements over 6 months (p < 0.05). MSC transplantation improved the function of 4-m walking test (4MWT) compared with the placebo group with a decrease of 2.05 s at 6 months of follow-up (p = 0.21). The measurement of grip strength revealed group difference with MSCs group demonstrating better performance, particularly at 6 months (p = 0.002). Inflammatory cytokines (TNF-α, IL-17) exhibited declines in MSCs group at 6 months compared to the placebo group (p = 0.034 and 0.033, respectively). There was no difference of incidence of AEs between the two groups.ConclusionIntravenous transplantation of HUC-MSCs is a safe and effective therapeutic approach on aging frailty. The positive outcomes observed in improving quality of life, physical performance, and reducing chronic inflammation, suggest that HUC-MSC therapy may be a promising potential treatment option for aging frailty.Trial Registration: Clinicaltrial.gov; NCT04314011; https://clinicaltrials.gov/ct2/show/NCT04314011.

E047 Utilization of complementary and alternative medicine in patients with rheumatological conditions at Kenyatta National Hospital

Abstract Background/Aims The utilization of complementary and alternative medicine (CAM) has recently increased, especially among patients with rheumatic diseases, particularly those experiencing pain and mobility limitations. CAM use poses potential risks, such as drug interactions and adherence to conventional medicine. This study aimed to determine the prevalence of CAM use and evaluate the reasons and determinants among patients with rheumatological diseases who attended the rheumatology outpatient clinic at Kenyatta National Hospital (KNH). Methods In this cross-sectional descriptive study, patients with a confirmed diagnosis of rheumatic disease were enrolled at the rheumatology outpatient clinic of KNH. Consecutive sampling technique was employed to select patients. Data collection utilized two validated tools: the self-administered NAFKAM International - CAM-Questionnaire (I-CAM-Q) for assessing the prevalence and primary reasons for CAM utilization and the SF-36 questionnaire to assess the quality of life. CAM use was determined as a percentage of all study participants. Health-related quality of life (HRQOL) was assessed using the SF-36 questionnaire, and the results were compiled to established norms. Mean scores and standard deviations were calculated for each SF-36 subscale, and patients’ overall health-related quality of life was categorized as either good or poor based on a 50% cut-off score. To determine the association between CAM use and socio-demographic factors, as well as HRQOL, the Chi-square test was used, with statistical significance set at a p-value of &amp;lt; 0.05. Results A total of 151 patients, predominantly female (137), with a median age of 45 (IQR 31.5-56.5) years, participated in the study. The prevalence of CAM use was 55.6%. Among the CAM modalities, vitamins/minerals (94%), prayer for health (80.9%), and herbs/herbal products (69%) were the most frequently used. The primary motivation for CAM utilization was to enhance overall well-being, followed by its adoption as an alternative or co-medication due to the high cost of conventional medicine. Dissatisfaction with conventional medicine emerged as the least common reason. CAM use was not influenced by age, gender, marital status, or level of education but was associated with parameters indicating poorer health, as evidenced by the mental component score and physical component score of SF-36. Conclusion The study highlights a significant prevalence of CAM use among patients with rheumatic diseases, indicating a noteworthy trend in healthcare choices. This suggests that many individuals with severe conditions may seek CAM as an adjunctive approach to managing their health. Alternatively, CAM usage might interfere with adherence to conventional medication, potentially exacerbating disease severity. Healthcare professionals should be vigilant in recognizing CAM usage and assessing whether it is preferred over conventional medicine, facilitating informed decisions and patient-centered care. This research contributes valuable insights into the complex landscape of healthcare choices among patients with rheumatological diseases, shedding light on the importance of holistic healthcare management. Disclosure K.M. Juma: None. G.O. Oyoo: None. E.G. Kalman: None.

P084 Gout symptom improvements and patient outcomes with SEL-212 in gout refractory to conventional therapy: DISSOLVE I &amp; II phase 3, double-blind, placebo-controlled clinical trials

Abstract Background/Aims Serum uric acid (sUA) ≥6.8 mg/dL often leads to severe clinical manifestations in gout refractory to conventional therapy. Uricase-based therapies can be effective, although immunogenicity can reduce efficacy and increase infusion-reaction risk. SEL-212 is a novel, once-monthly treatment of sequential infusions of tolerogenic nanoparticles containing sirolimus (SEL-110) followed by a pegylated uricase (pegadricase, SEL-037). DISSOLVE I and II (DI and DII) evaluated the safety/efficacy of SEL-212 in adults with refractory gout. Here we report secondary and key safety endpoints. Methods DI (US) and DII (Global) were placebo-controlled, double-blind, randomised clinical trials that evaluated a combination of SEL-110 at either 0.15 mg/kg [high-dose] or 0.1 mg/kg [low-dose] with SEL-037 (0.2 mg/kg) in refractory gout. Six-month outcomes included sUA reduction, number of tender/swollen joints, tophus burden, health-related quality-of-life, and safety. Results 112 participants (96% male, 66% aged ≥50 years, DI) and 153 participants (97% male, 72% ≥50 years, DII) were dosed (Table 1). Mean age was 55 years, 57% (DI) and 67% (DII) had tophi at baseline, and time since gout diagnosis was 12.9 (DI) and 11.0 years (DII). Both SEL-212 doses significantly reduced mean sUA levels versus placebo over 6-months (5.2-5.6 mg/dL in DI and 3.3-4.5 mg/dL in DII, p &amp;lt; 0.001 for all), with marked reductions during the first-treatment period. In DII, tender joints declined significantly between baseline and six-months in the high- and low-dose groups (8.1 joints, p = 0.003; 9.2 joints, p &amp;lt; 0.001, respectively) versus placebo (4.4 joints). Swollen joints were also significantly reduced with high-dose SEL-212 (5.7 joints, p = 0.013) versus placebo (3.4 joints). There were significant improvements in SF-36 physical component score with both SEL-212 doses versus placebo (p ≤ 0.01), while HAQ-DI was significantly improved with low-dose versus placebo (p = 0.018) in DII. In DI non-statistically significant trends favouring SEL-212 across all secondary endpoints were observed. This may reflect the smaller sample size and milder baseline symptom burden of patients in DI versus DII. Infusion reactions occurred in 3.4% of the combined high-dose groups. Conclusion SEL-212 was well tolerated and provided significant reductions in mean sUA over time, improvement in SF-36 physical and HAQ-DI measures and reduced the number of tender/swollen joints. Disclosure H.S.B. Baraf: Corporate appointments; Sobi, Selecta BioScience, Grünenthal, Fresenius Kabi and Olatec. Member of speakers’ bureau; Horizon Pharmaceuticals. Grants/research support; Horizon Pharmaceuticals, Swedish Orphan Biovitrum. P. Khanna: Consultancies; Horizon, Sobi. Other; Investigator: Selecta Bioscience, Dyve Biosciences. A. Patel: Member of speakers’ bureau; Lexicon Pharmaceuticals. J. Sobierska: Other; Employee of Sobi. J. Christie: Shareholder/stock ownership; GSK Pharma. Other; Employee of Sobi. W. DeHaan: Shareholder/stock ownership; Selecta Biosciences. Other; Employee of Selecta Biosciences. R. Azeem: Shareholder/stock ownership; Selecta Biosciences. Other; Employee of Selecta Biosciences;. P.G. Traber: Shareholder/stock ownership; Selecta Biosciences. Other; Employee of Selecta Biosciences. A. Kivitz: Consultancies; AXDEV Group, Amgen, Pfizer, Janssen, Boehringer Ingelheim, AbbVie, Flexion, Gilead, Grünenthal, Orion, Regeneron, Sun Pharma Advance Research, and ECOR1. Shareholder/stock ownership; Pfizer, GSK, Gilead, Novartis, and Amgen. Member of speakers’ bureau; Merck &amp; Co, Eli Lilly, Novartis, Pfizer, Flexion, AbbVie, Amgen, Genentech, Regeneron, UCB, Horizon, and GSK.

Impact of Self-Reported Loss of Balance and Gait Disturbance on Outcomes following Adult Spinal Deformity Surgery.

Background: The objective of this study was to evaluate if imbalance influences complication rates, radiological outcomes, and patient-reported outcomes (PROMs) following adult spinal deformity (ASD) surgery. Methods: ASD patients with baseline and 2-year radiographic and PROMs were included. Patients were grouped according to whether they answered yes or no to a recent history of pre-operative loss of balance. The groups were propensity-matched by age, pelvic incidence-lumbar lordosis (PI-LL), and surgical invasiveness score. Results: In total, 212 patients were examined (106 in each group). Patients with gait imbalance had worse baseline PROM measures, including Oswestry disability index (45.2 vs. 36.6), SF-36 mental component score (44 vs. 51.8), and SF-36 physical component score (p < 0.001 for all). After 2 years, patients with gait imbalance had less pelvic tilt correction (-1.2 vs. -3.6°, p = 0.039) for a comparable PI-LL correction (-11.9 vs. -15.1°, p = 0.144). Gait imbalance patients had higher rates of radiographic proximal junctional kyphosis (PJK) (26.4% vs. 14.2%) and implant-related complications (47.2% vs. 34.0%). After controlling for age, baseline sagittal parameters, PI-LL correction, and comorbidities, patients with imbalance had 2.2-times-increased odds of PJK after 2 years. Conclusions: Patients with a self-reported loss of balance/unsteady gait have significantly worse PROMs and higher risk of PJK.

Proximal Hamstring Repair With All-Suture Anchors and an Accelerated Rehabilitation and Bracing Protocol Demonstrates Good Outcomes at 1-Year Follow-Up

To evaluate patient-reported outcomes and risk for rerupture after surgical treatment of proximal hamstring tendon ruptures using all-suture anchors and a unique postoperative bracing strategy. A retrospective review of a prospectively collected database was conducted of patients undergoing proximal hamstring repair or reconstruction from 2020 to 2022 at a tertiary, academic institution. Patients were included if they reached minimum 1-year follow-up and completed postoperative patient-reported outcomes. The surgical protocol for proximal hamstring repairs included all-suture anchors placed either in an open or endoscopic fashion in the ischial tuberosity. After surgery, all patients underwent an accelerated rehabilitation protocol, including 6 weeks touchdown weight-bearing in a hinged knee brace locked in extension for ambulation, allowing passive knee flexion to 90° while seated. Descriptive statistics were used to analyze the data. Twenty-one patients were included (mean age 50.4 ± 9.5 years, body mass index 24.4 ± 3.5, 66.7% female). Lower Extremity Functional Scale score achieved postoperatively was 74.2 ± 7.5 (out of 80). Patients had minimal pain (mean visual analog scale pain score of 0.9 ± 1.2). 61.9% of patients were able to return to the same level of activity after based on Tegner score by 1 year. Postoperative Single Assessment Numeric Evaluation activity of daily living was 94.3 ± 8.3, and Single Assessment Numeric Evaluation Sports was 82.3 ± 19.0. Mean Short Form Survey (SF-12) postoperative scores were 51.6 ± 6.8 for SF-12 Physical Component Score and 53.9 ± 9.7 for Mental Component Score. 95.2% (20 of 21) patients were satisfied with their outcome. There were no reruptures, infections, or reoperations. One patient of 21 (4.8%) incurred a postoperative deep venous thrombosis, which was treated with therapeutic anticoagulation for 3 months. All-suture anchors for proximal hamstring repair with a unique accelerated postoperative rehabilitation and bracing protocol result in good outcomes and patient satisfaction with minimal risk of complications. Level IV, case series, therapeutic.

Improvement in Pain, Quality of Life, and Urinary Dysfunction following Correction of Lumbar Lordosis and Reduction in Lumbar Spondylolistheses Using Chiropractic BioPhysics® Structural Spinal Rehabilitation: A Case Series with >1-Year Long-Term Follow-Up Exams.

(1) Background: Lumbar spondylolisthesis affects ~20% of the US population and causes spine-related pain and disability. (2) Methods: This series reports on three patients (two females and one male) aged 68-71 years showing improvements in back pain, quality of life (QOL), and urinary dysfunction following correction of lumbar spondylolistheses using CBP® spinal rehabilitation. Pre-treatment radiographs showed lumbar hyperlordosis (-49.6°, ideal is -40°) and anterolisthesis (14.5 mm, ideal is 0 mm). Pre-treatment patient-reported outcome measures (PROMs) included a numeric rating scale (NRS) for back pain (7.3/10, ideal is 0), urinary urgency (8/10, ideal is 0), and SF-36 physical (PCS) and mental component score (MCS) (29.8 and 46.6, ideal is 46.8 and 52.8). Patients underwent 2-3 CBP® sessions per week to correct lumbar hyperlordosis and lumbar anterolistheses. (3) Results: Post-treatment radiographs showed improvements in lumbar curvature (-42.8°) and anterolisthesis (4.2 mm). Post-treatment PROMs showed improvements in NRS for back pain (1/10), urinary urgency (2.3/10), and SF-36 PCS and MCS (50.2 and 57.7). Long-term follow-up radiographs and PROMs showed maintained improvements. (4) Conclusions: This series documents the first-recorded long-term corrections of lumbar spondylolisthesis and concomitant improvements in back pain, urinary urgency, and QOL using CBP®. This series provides evidence for CBP® as a non-surgical approach to lumbar spinal rehabilitation and the possible impacts of spinal alignment on pain, urinary dysfunction, and QOL.

Importance of modifiable non-radiographic functional parameters for adult spinal deformity

We clarified non-radiographic physical parameters associated with the severity of adult spinal deformity (ASD) using community-dwelling adult volunteers. They were subjected to upright entire spine radiographs for standard radiographic parameters and the number of sagittal modifiers of SRS-Schwab ASD classification (Schwab-SM). Clinical evaluations included isometric muscle strength of trunk extensor (TEX), trunk flexor (TFL), quadriceps femoris (QF), gluteus maximus, and iliopsoas; range of motion (ROM) of hip, knee, ankle, and active back extension (BET); SF36 physical component score (PCS), VAS for back and knee pain, and the degree of ambulatory kyphosis (dTIA). Each muscle strength was calibrated by body weight (BW) and expressed as BW ratio. According to our previous study, dTIA ≥ 7.6° was defined as pathological and dTIA ≤ 3.5° as normal. A final total of 409 female volunteers were included, and their demographics were; age 67.0 ± 5.5 years, Schwab-SM 2.1 ± 1.8, TEX 0.90 ± 0.33BW, TFL 0.48 ± 0.15BW, QF 0.45 ± 0.19BW, PCS 33.5 ± 6.5. Subjects were classified as clinical ASD group (cASD, n = 10) with PCS ≤ 27(mean-1SD) and pathological dTIA, robust group (n = 19) with PCS ≥ 40 (mean + 1SD) and normal dTIA, and the rest (non-cASD, n = 338). Statistical analyses showed significant differences in TEX, TFL, QF, knee extension (KEX), and BET between robust and cASD, and the mean values of robust group (TEX ≥ 1.1BW, TFL ≥ 0.5BW, QF ≥ 0.5BW, KEX ≥ 0° and BET ≥ 14 cm) were used as ‘ASD-MJ’ index. Subjects with fully achieving ASD-MJ goals showed significantly better radiographic and clinical outcomes than those with unmet goals. In conclusion, upon prescribing conservative or physical therapies for ASD patients, modifiable clinical goals should be clarified, and ASD-MJ could be a benchmark.

Functional outcome and analysis of factors affecting health-related quality of life of surgically managed pelvic ring fractures: a cross-sectional study.

Pelvic ring fractures, particularly those involving the posterior pelvis, pose significant challenges due to their inherent instability. The posterior pelvic ring is critical in providing structural support and stability to the pelvis. This study evaluates the functional outcomes and health-related Quality of life (HRQOL) of patients who underwent internal fixation for unstable pelvic fractures. Various factors influencing the outcomes are also investigated. A single-center cross-sectional study was conducted on patients with unstable sacral fractures treated with posterior tension band plate or sacroiliac plating with or without symphyseal plating between 2016 and 2020. Patient demographics, injury mechanisms, associated injuries, surgical details, complications, and return-to-work data were collected. HRQOL was assessed using specific pelvic fracture instruments and general HRQOL questionnaires. Logistic regression analysis was performed to identify factors associated with lower SF-12 and SF-36 scores RESULTS: The study included 54 patients, predominantly males (55.6%), aged 18-70 years, with high-energy trauma mechanisms, such as road traffic accidents and occupational injuries. The majority of patients had lateral compression pelvic fractures. Overall, the functional outcomes were favorable, with excellent or good outcomes observed in 86.1% of cases. Patients with associated injuries, such as abdominal, chest, or head injuries, were likelier to have lower SF-12 physical component scores. Sexual satisfaction scores remained stable for most patients post-surgery. This study highlights the favorable functional outcomes and HRQOL for patients undergoing fixation for unstable pelvic ring fractures. Younger, working-age males were the most commonly affected demographic. Associated injuries significantly affected physical HRQOL scores. Despite high-energy trauma, patients generally reported satisfactory sexual function post-surgery.

Atrial fibrillation symptom reduction and improved quality of life following the hybrid convergent procedure: a CONVERGE trial subanalysis.

CONVERGE was a prospective, multicenter, randomized controlled trial that evaluated the safety of Hybrid Atrial Fibrillation Convergent (HC) and compared its effectiveness to endocardial catheter ablation (CA) for the treatment of persistent atrial fibrillation (PersAF) and longstanding PersAF (LSPAF). In 2020, we reported that CONVERGE met its primary safety and effectiveness endpoints. The primary objective of the present study is to report CONVERGE trial results for quality of life (QOL) and Class I/III anti-arrhythmic drug (AAD) utilization following HC. Eligible patients had drug-refractory symptomatic PersAF or LSPAF and a left atrium diameter ≤6.0 cm. Enrolled patients were randomized 2:1 to receive HC or CA. Atrial Fibrillation Severity Scale (AFSS) and the 36-Item Short Form Health Survey (SF-36) were assessed at baseline and 12 months; statistical comparison was performed using paired t-tests. AAD utilization at baseline through 12 and 18 months post-procedure was evaluated; statistical comparison was performed using McNemar's tests. A total of 153 patients were treated with either HC (n=102) or CA (n=51). Of the 102 HC patients, 38 had LSPAF. AFSS and SF-36 Mental and Physical Component scores were significantly improved at 12 months versus baseline with HC overall and for the subset of LSPAF patients treated with either HC or CA. The proportion of HC patients (n=102) who used Class I /III AADs at 12 and 18 months was significantly less (33.3% and 36.3%, respectively) than baseline (84.3%; P<0.001). In LSPAF patients who underwent HC (n=38), AADs use was 29.0% through 18 months follow-up versus 71.1% at baseline (P<0.001). HC reduced AF symptoms, significantly improved QOL, and reduced AAD use in patients with PersAF and LSPAF. NCT01984346.

Effectiveness and safety of mexiletine versus placebo in patients with myotonia: a systematic review and meta-analysis.

The rare nature of dystrophic and non-dystrophic myotonia has limited the available evidence on the efficacy of mexiletine as a potential treatment. To address this gap, we conducted a systematic review and meta-analysis to evaluate the effectiveness and safety of mexiletine for both dystrophic and non-dystrophic myotonic patients. The search was conducted on various electronic databases up to March 2023, for randomized clinical trials (RCTs) comparing mexiletine versus placebo in myotonic patients. A risk of bias assessment was carried out, and relevant data was extracted manually into an online sheet. RevMan software (version 5.4) was employed for analysis. A total of five studies, comprising 186 patients, were included in the meta-analysis. Our findings showed that mexiletine was significantly more effective than placebo in improving stiffness score (SMD = - 1.19, 95% CI [- 1.53, - 0.85]), as well as in reducing hand grip myotonia (MD = - 1.36s, 95% CI [- 1.83, - 0.89]). Mexiletine also significantly improved SF-36 Physical and Mental Component Score in patients with non-dystrophic myotonia only. Regarding safety, mexiletine did not significantly alter ECG parameters but was associated with greater gastrointestinal symptoms (GIT) compared to placebo (RR 3.7, 95% CI [1.79, 7.64]). Other adverse events showed no significant differences. The results support that mexiletine is effective and safe in myotonic patients; however, it is associated with a higher risk of GIT symptoms. Due to the scarcity of published RCTs and the prevalence of GIT symptoms, we recommend further well-designed RCTs testing various drug combinations to reduce GIT symptoms.

Evaluation of differences in quality of life in patients with chronic graft-versus-host disease

Objective: To evaluate the status of, differences in, and factors influencing quality of life (QoL) in patients with chronic graft-versus-host disease (GVHD). Methods: From September 2021 to February 2023, a cross-sectional study of 140 patients with chronic GVHD was conducted at our center. Symptom burden was assessed by the Lee Symptomatology Scale (LSS), and QoL was assessed by the Medical Outcome Study 36-Item Short-Form Health Survey (SF-36) (version 1) and five-level EuroQoL five-dimensional questionnaire (EQ-5D-5L). Results: Data from 140 respondents, including 32 (22.9%) with mild chronic GVHD, 87 (62.1%) with moderate chronic GVHD, and 21 (15.0%) with severe chronic GVHD, were analyzed. Of the respondents, 61.4% were male, and the median transplantation age was 34 (15-68) years. The primary diagnoses were acute myeloid leukemia (50.0%), acute lymphoblastic leukemia (20.0%), and myelodysplastic syndrome (15.0%). The common chronic GVHD-affected organs included the skin in 74 patients (52.9%), the eyes in 57 patients (40.7%), and the liver in 50 patients (35.7%). Among the whole cohort, the eye (20.48±23.75), psychological (16.13±17.00), and oral (13.66±20.55) scores were highest in the LSS group. The physiological function (36.07±11.13), social function (36.10±10.68), and role-emotional functioning (38.36±11.88) scores were lowest in the SF-36 group. The EQ-5D index was 0.764. The total LSS scores for mild, moderate, and severe chronic GVHD were 6.51±6.15, 10.07±5.61, and 20.90±10.09, respectively. The SF-36 physical component scores (PCSs) were 43.12±6.38, 40.73±7.14, and 36.97±6.97, respectively, and the mental component scores (MCSs) were 43.00±8.47, 38.90±9.52, and 28.96±9.63, respectively. The EQ-5D values were 0.810±0.124, 0.762±0.179, and 0.702±0.198, respectively. The multivariate analysis showed that the overall symptom burden (β=-0.517), oral symptom burden (β=-0.456), National Institute of Health (NIH) criteria for the eyes (β=-0.376), and nutrition-related symptom burden (β=-0.211) were significantly negatively correlated with the PCS. The NIH score (β=-0.260) was negatively correlated with the MCS score. Oral symptom burden (β=-0.400), joint/fascia NIH criteria (β=-0.332), number of involved systems (β=-0.253), overall NIH criteria (β=-0.205), and number of immunosuppressants taken (β=-0.171) were significantly negatively correlated with the EQ-5D score (all P<0.05). Medium to strong correlations were found between the EQ-5D score and the SF-36 score (|r|=0.384-0.571, P<0.001). Conclusions: The QoL of patients with chronic GVHD is impaired, and the more severe the disease, the poorer the QoL. Overall symptom burden, severity of eyes, and oral symptom burden were the most important factors affecting QoL.

Incorporating patient-reported outcome data into a predictive calculator for allogeneic hematopoietic cell transplantation recipients.

The Center for International Blood and Marrow Transplant Research (CIBMTR) provides a 1-year overall survival calculator to estimate outcomes for individual patients before they undergo allogeneic hematopoietic cell transplantation (HCT) to inform risk. The calculator considers pre-HCT clinical and demographic characteristics, but not patient-reported outcomes (PROs). Because pre-HCT PRO scores have been associated with post-HCT outcomes, the authors hypothesized that adding PRO scores to the calculator would enhance its predictive power. Clinical data were obtained from the CIBMTR and the Blood and Marrow Transplant Clinical Trials Network. The PRO measures used were the 36-Item Short Form Survey (SF-36) and the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation. One thousand thirty-three adult patients were included. When adjusted for clinical characteristics, the SF-36 physical component score was significantly predictive of 1-year survival (hazard ratio [HR], 0.88; 95% confidence interval [CI], 0.81-0.95; p=.0015), whereas the mental component score was not (HR, 1.02; 95% CI, 0.95-1.10; p=0.6396). The baseline single general health question on the SF-36 was also significantly associated with mortality (HR, 1.91 for those reporting fair/poor health vs. good, very good, or excellent health; 95% CI, 1.33-2.76; p=.0005). The addition of PRO scores to the calculator did not result in a significant change in the model's predictive ability. Self-reported pre-HCT scores were strongly predictive of self-reported health status (odds ratio, 3.35; 95% CI, 1.66-6.75; p=.0007) and quality of life (odds ratio, 3.24; 95% CI, 1.93-5.41; p<.0001) after HCT. The authors confirmed the significant, independent association of pre-HCT PRO scores with overall survival, although adding PRO scores to the survival calculator did not improve its performance. They also demonstrated that a single general health question was as accurate as the full measure for predicting survival, an important finding that may reduce respondent burden and promote its inclusion in routine clinical practice. Validation of these findings should be performed.

Dysfunction of circulating endothelial progenitor cells in major depressive disorder.

Despite mounting evidence demonstrates circulating endothelial progenitor cells (cEPCs) quantitative changes in depression, no study has investigated cEPC functions in major depressive disorder (MDD). We investigated the role of cEPC adhesive and apoptotic functions in MDD. We recruited 68 patients with MDD and 56 healthy controls (HCs). The depression symptoms, anxiety, psychosomatic symptoms, subjective cognitive dysfunction, quality of life, and functional disability were evaluated using the Hamilton Depression Rating Scale and Montgomery-Åsberg Depression Rating Scale, Hamilton Anxiety Rating Scale, Depression and Somatic Symptoms Scale (DSSS), Perceived Deficits Questionnaire-Depression, 12-Item Short Form Health Survey (SF-12), and Sheehan Disability Scale (SDS), respectively. Working memory and executive function were assessed using a 2-back task and Wisconsin Card Sorting Test (WCST). Inflammatory marker (soluble interleukin-6 receptor, C-reactive protein, and tumor necrosis factor-α receptor-1), cEPC adhesive, and apoptotic levels were measured using in vitro assays. The MDD patients showed significantly lower cEPC adhesive levels than the HCs, and this difference in adhesive function remained statistically significant even after adjusting for inflammatory marker levels. The cEPC adhesion levels were in inverse correlations with commission and omission errors in 2-back task, the percent perseverative response and percent perseverative errors in WCST, and the DSSS and SDS scores, but in positive correlations with SF-12 physical and mental component scores. cEPC apoptotic levels did not differ significantly between the groups. The findings indicate that cEPC adhesive function is diminished in MDD and impacts various aspects of cognitive and psychosocial functions associated with the disorder.

Does Preoperative Back Pain Impact Patient-reported Outcomes in Patients Undergoing Minimally Invasive Transforaminal Lumbar Interbody Fusion for Isthmic Spondylolisthesis?

Retrospective review. To determine postoperative clinical outcomes in patients undergoing minimally invasive (MIS) transforaminal lumbar interbody fusion (TLIF) for isthmic spondylolisthesis (IS). Few studies have examined the postoperative clinical trajectory in patients undergoing MIS-TLIF specifically for IS. Patients were separated into two cohorts based on the previously defined Visual Analog Scale (VAS) back pain (BP) for severe pain: VAS-BP <7 and VAS-BP ≥7. Patient-reported outcome measures (PROMs) of Patient-Reported Outcomes Measurement Information System-physical function (PF), 12-item Short Form (SF-12) Physical/Mental Component Score, Patient Health Questionnaire-9, VAS-BP, VAS leg pain, and Oswestry Disability Index were collected preoperatively and up to 2-year postoperatively. Minimum clinically important difference (MCID) was calculated through previously defined thresholds. A total of 160 patients were recorded, with 58 patients in the VAS-BP <7 cohort. The VAS-BP <7 cohort demonstrated significant improvement in all PROMs at one or more postoperative time points. The VAS-BP ≥7 demonstrated significant improvement at 3 or more postoperative time points in all PROMs except for SF-12 Mental Component Score. The VAS-BP <7 cohort reported significantly superior preoperative and postoperative PROMs in all domains, except for SF-12 Physical Component Score. The VAS-BP ≥7 cohort had higher MCID achievement rates at one or more time points in multiple PROMs. Patients undergoing MIS-TLIF for IS demonstrated significant postoperative improvement in PF, mental function, pain, and disability outcomes independent of preoperative severity of BP. Patients with lower preoperative BP demonstrated superior outcomes in PF, mental function, pain, and disability. However, patients with greater preoperative BP achieved higher rates of MCID in mental function, BP, and disability outcomes. Patients with greater severity of preoperative BP undergoing MIS-TLIF for IS may experience greater rates of clinically relevant improvement in mental function, BP, and disability outcomes.

Assessment of quality of life, psychosocial, and epidemiological aspects in patients diagnosed with tuberculous uveitis.

To assess the quality of life in patients diagnosed as having tuberculous uveitis and its association with sociodemographic, clinical, and psychosocial aspects. By conducting standardized interviews, clinical and demographic data were collected using a measure developed in this study. This measure was applied in addition to other measures, namely SF-12, Hospital Anxiety and Depression Scale, and NEI-VFQ-39, which were used to assess health-related quality of life, anxiety and depression symptoms, and visual functioning. The study included 34 patients [mean age: 46.5 ± 15.1 years, female patients: 21 (61.8%)]. The mean of the VFQ-39 score was 74.5 ± 16.6 and that of SF-12 physical and mental component scores were 45.8 ± 10.1 and 51.6 ± 7.5, respectively, for the health-related quality of life. Anxiety symptoms were the most prevalent compared with depression symptoms and were found in 35.3% of the participants. Tuberculous uveitis affects several scales of quality of life, thereby affecting a population economically active with a social, psychological, and economic burden.

Restricted life-space mobility impacts physical but not mental quality of life in older cancer survivors.

Older cancer survivors often value quality of life (QOL) over survival. Life-space mobility (LSM), defined as the individual's spatial geographic mobility range, is an important QOL indicator in older adults with chronic illnesses; however, this relationship is unexplored in older cancer survivors. We examined the longitudinal associations and causal relationships between LSM and QOL in 153 older cancer survivors (≥65 years) from the University of Alabama at Birmingham (UAB) Study of Aging. LSM was assessed using the UAB Life-Space Assessment-Composite score (LSA-C), and QOL was assessed by the SF-12 Mental Component Score (MCS12) and Physical Component Score (PCS12) at 0 (study entry), 6, 18, 36, 54, and 72 months. We examined the causal relationship between LSM and QOL using a cross-lagged panel model (CLPM). The cohort (n = 153) was 76 years old on average and predominantly White (58%), female (58%), and married (55%). Longitudinal analyses found LSM decreased over time (p < 0.0001), and this decrease was associated with decreased QOL (PCS12, p < 0.0001, MCS12, p < 0.0001). In the CLPM causal analysis, lower LSM resulted in worse PCS12 (p < 0.001), but not worse MSC12. Restricted LSM resulted in worse physical QOL over 72 months in a sample of 153 older cancer survivors. Developing and evaluating interventions to preserve greater LSM could be a promising approach to improving QOL.

Quality of Life in the Danish Fontan Population is Unchanged Over the Past Decade—A Nationwide Longitudinal Study

AbstractKnowledge about health-related quality of life (HRQoL) over time in Fontan patients is sparse. We aimed to describe HRQoL over a ten-year period in a population-based Fontan cohort. Further, we compared HRQoL in Fontan patients with the general population. In 2011, Danish Fontan patients were invited to participate in a nationwide study assessing HRQoL. Depending on age, 152 participants filled out either the Pediatric Quality of Life Inventory or the 36-Item Short Form Health Survey. After a decade, patients from the initial study were invited to participate in a follow-up study. All were given the same questionnaire as in the first study, plus the 12-Item Short Form Health Survey (SF-12) as part of the Danish National Health Survey. HRQoL over time was described, and SF-12 scores were compared with the general population. A total of 109 Fontan patients completed the questionnaires in both studies. The mean patient age was 14.9 ± 6.6 years and 25.6 ± 6.5 years respectively. Despite an increase in complications, HRQoL did not decrease during the study period. Physical HRQoL scores were lower than mental HRQoL scores at both time points. The SF-12 physical component score was significantly lower in Fontan patients than in the general population (median score 52 vs. 56, p &lt; 0.001), while the SF-12 mental component score was comparable (median score 51 vs. 50, p = 0.019). HRQoL remained stable over a ten-year period in a contemporary Danish Fontan cohort. Still, the physical HRQoL remained significantly lower than that of the general population.

Patient Acceptable Symptom State Thresholds for the Knee Society Score, Oxford Knee Score, and 36-Item Short Form Survey Ten Years Following Unicompartmental Knee Arthroplasty

BackgroundThe patient acceptable symptom state (PASS) refers to a cutoff value on any patient-reported outcome measures (PROMs) scale, beyond which patients consider themselves as having achieved an acceptable outcome. This study aimed to identify PASS thresholds for knee-specific and generic PROMs at 10 years post-unicompartmental knee arthroplasty (UKA). MethodsThere were 269 patients who underwent UKA for medial osteoarthritis from 2004 to 2007 at a single institution and were surveyed preoperatively and 10 years postoperatively using the Knee Society Function Score (KSFS), Knee Society Knee Score (KSKS), Oxford Knee Score (OKS), 36-Item Short Form Survey (SF-36) Mental Component Score (MCS), and SF-36 Physical Component Score (PCS). Treatment outcomes and expectations were assessed using an anchor question, and PASS attainment was determined using the Youden index on a receiver operating characteristic (ROC) curve. Also, a similar study that identified 2-year long-term PROM PASS thresholds for UKA was referenced and compared. ResultsOverall, 91.1% reported acceptable outcomes. The area under the curve for ROCs of KSKS, OKS, and PCS were 0.80, 0.75, and 0.71, respectively. The area under the curve for ROCs of KSFS and MCS were both 0.64. The PASS thresholds were 67.5 for KSFS, 70.5 for KSKS, 39.5 for OKS, 44.6 for PCS, and 43.8 for MCS. Patients who achieved a PASS were at least 3 times more likely to have satisfactory outcomes. ConclusionsTo our knowledge, this is the first study that identified 10-year long-term PROM PASS thresholds for UKA. Accounting for our finding that a decade-long follow-up yielded lower PASS thresholds, time-specific UKA PROM PASS thresholds should be considered. Level of evidenceIII.

Clinical characteristics of flares in patients with rheumatoid arthritis after total hip and total knee arthroplasty.

Clinical characteristics of flares in patients with rheumatoid arthritis after total hip and total knee arthroplasty.