BACKGROUND: Surgical techniques for Terrible Triad injuries developed 20 years ago. Good and excellent short- and medium-term functional results have been reported. No long-term (over 10 years) functional outcomes have previously been reported. This case-series is the longest follow-up of patients treated for acute, isolated terrible triad injuries using a standard treatment protocol.
METHODS: 20 Patients with acute, isolated, surgically managed terrible triad injuries were treated between October 2001 and May 2008. 10 of these patients were seen face-to face for a clinical follow-up and if required a radiological assessment. Mayo Elbow Performance Scores (MEPS) and Disability of the Arm, Shoulder and Hand (DASH) scores, requirement for further surgery and elbow instability were recorded.
RESULTS: The average length of follow-up was 18.8 years. The mean MEPS was 88 and the mean DASH score was 12.3. The average loss of pronation was 8 degrees. The average loss of supination was 13 degrees. The re-operation rate was 40%, only one of these was a functionally limiting operation. A trend towards osteoarthritis was observed but there were no conversions to total elbow replacement.
CONCLUSIONS: This is the longest-term follow-up study of these injuries and demonstrates the functional performance that the majority of patients achieve. The low follow-up rate can be expected with such a long interval between treatment and assessment. A relatively high re-operation rate is largely made up of minor procedures (removal of metalwork and cubital tunnel release) which did not impact the patients\' functional status.
CONCLUSIONS: This study adds to the evidence that the terrible triad of the elbow is surgically treatable to allow a high functional standard not only in the short-term but also in the long-term. As such this is a useful adjunct to have both when informing patients of what can be expected in their long-term recovery from this injury.

暂无摘要。

OBJECTIVE: The short-term efficacy and safety of daridorexant, a dual orexin receptor antagonist, has been demonstrated in Japanese patients with insomnia disorder. The objective of this study was to evaluate, in a non-overlapping patient population to the short-term study, the long-term safety and efficacy of daridorexant in Japanese patients with insomnia disorder.
METHODS: In this Phase 3 open-label study conducted in Japan, 154 patients with insomnia disorder were randomized to daridorexant 50 mg (n = 102) or 25 mg (n = 52) for 52 weeks. The primary objective was to assess the safety and tolerability of daridorexant for up to 1 year. Secondary exploratory objectives were to evaluate the long-term efficacy of daridorexant on subjective sleep parameters (total sleep time, latency to sleep onset and wake after sleep onset) and daytime functioning (Insomnia Daytime Symptoms and Impacts Questionnaire).
RESULTS: The incidence of treatment-emergent adverse events (TEAEs) was 74 % and 58 % in the 50 mg and 25 mg groups respectively. No serious drug-related TEAEs were reported. Both doses improved next-morning sleepiness (Visual Analog Scale) throughout the study. Five adjudicated adverse events of special interest were reported; excessive daytime sleepiness (n = 1, 25 mg; n = 2, 50 mg), sleep paralysis (n = 1, 50 mg) and nightmare (n = 1, 25 mg). Improvements in sleep and daytime functioning were maintained from Week 2 (first assessment) through to Week 52 in both dose groups.
CONCLUSIONS: Up to 52-weeks, daridorexant was well tolerated with sustained improvement in sleep onset, sleep maintenance and daytime functioning, supporting its long-term use in Japanese patients with insomnia disorder.

BACKGROUND: Achieving long-term clinical remission in Crohn\'s disease (CD) with antitumor necrosis factor α (anti-TNF-α) agents remains challenging.
OBJECTIVE: This study aims to establish a prediction model based on patients\' clinical characteristics using a machine-learning approach to predict the long-term efficacy of infliximab (IFX).
METHODS: Three cohorts comprising 746 patients with CD were included from 3 inflammatory bowel disease (IBD) centers between June 2013 and January 2022. Clinical records were collected from baseline, 14-, 30-, and 52-week post-IFX treatment. Three machine-learning approaches were employed to develop predictive models based on 23 baseline predictors. The SHapley Additive exPlanations (SHAP) algorithm was used to dissect underlying predictors, and latent class mixed model (LCMM) was applied for trajectory analysis of the longitudinal change of blood routine tests along with long-term IFX therapy.
RESULTS: The XGBoost model exhibited the best discrimination between long-term responders and nonresponders. In the internal training and testing set, the model achieved an AUC of 0.91 (95% CI, 0.86-0.95) and 0.71 (95% CI, 0.66-0.87), respectively. Moreover, it achieved a moderate predictive performance in the independent external cohort, with an AUC of 0.68 (95% CI, 0.59-0.77). The SHAP algorithm revealed disease-relevant laboratory measurements, notably hemoglobin (HB), white blood cells (WBC), erythrocyte sedimentation rate (ESR), albumin (ALB), and platelets (PLT), alongside age at diagnosis and the Montreal classification, as the most influential predictors. Furthermore, 2 distinct patient clusters based on dynamic laboratory tests were identified for monitoring the long-term remission.
CONCLUSIONS: The established prediction model demonstrated remarkable discriminatory power in distinguishing long-term responders from nonresponders to IFX therapy. The identification of distinct patient clusters further emphasizes the need for tailored therapeutic approaches in CD management.
The study developed a machine-learning model using clinical data to predict long-term efficacy of IFX in Crohn’s disease. The XGBoost model demonstrated strong discriminatory power, revealing influential predictors and distinct patient clusters, emphasizing the importance of tailored therapeutic approaches in CD management.

OBJECTIVE: There is limited research on the proportion of individuals with epilepsy who maintain response to ketogenic diet therapy (KDT) after discontinuing treatment. We aimed to determine the proportion of individuals who did / did not maintain response post KDT and explore factors that may influence the likelihood of maintaining response.
METHODS: Retrospective data were collected from 97 individuals from 9 KDT centres. Individuals had achieved ≥50 % seizure reduction on KDT for at least 12 months, with seizure frequency data available at 3 months+ post diet. Outcome 1 was: recurrence of seizures or increase in seizure frequency post diet; outcome 2: recurrence of seizures, increase in seizure frequency or an additional anti-seizure treatment started post diet.
RESULTS: 61/97 (62.9 %) individuals maintained response at latest follow-up (mean 2.5[2.0] years since stopping KDT). Approximately one third maintained response without further anti-seizure treatments. One quarter of individuals had an increase in frequency or recurrence of seizures within 6 months (95 %CI 4, 12) for outcome 1 and within 3 months (3, 6) for outcome 2. Individuals who did not achieve seizure freedom on diet were significantly more likely to have an increase in seizures or to require additional anti-seizure treatments post diet compared to those who were seizure-free on diet (hazard ratio 4.02, 95 %CI (1.46, 11.16) p < 0.01).
CONCLUSIONS: Our findings should help guide clinical teams with the information they provide patients and their families regarding likelihood of long-term seizure response to KDT. Realistic costings for KDT services may need to be considered.

Pulmonary rehabilitation (PR) has been shown to alleviate dyspnea, increase exercise capacity, and improve quality of life in patients with chronic obstructive pulmonary disease (COPD). However, such PR programs have focused on short-term effects. Thus, this study aimed to report our experience with a COPD patient who underwent PR once a week for one year. An 84-year-old male with stage II COPD, which was classified by the Global Initiative for Obstructive Lung Disease, presented symptoms of dyspnea while walking. The patient underwent PR once a week for one year, which included exercise training, self-management support, instructions on breathing during exertion, and respiratory muscle stretching. Before and after PR, we assessed the patient\'s physical function, dyspnea, and quality of life. For one year, no adverse events were recorded. We observed that the patient\'s physical function, dyspnea, and quality of life improved over time. In particular, his six-minute walking distance (6MWD) reached the minimal clinically important difference at three months and the predictive value of 6MWD for healthy adults at six months. The present case showed that a PR program conducted once a week for one year might be feasible and effective.

OBJECTIVE: Osteochondritis dissecans (OCD) can lead to detrimental effects in the affected joints. Osteochondral autologous transplantation (OAT) allows to restore the articular surface with an autologous osteochondral unit. While short-term results are documented, there is a lack of long-term data. Aim of this study was to analyze the long-term clinical results of single-plug OAT for the treatment of knee OCD.
METHODS: Twenty patients (14 men, 6 women) were treated with single plug-OAT. Mean age was 23.6 ± 9.9 years and BMI was 23.3 ± 3.6 kg/m2. Lesion size was 2.3 ± 1.6 cm2 and defects included 14 medial femoral condyles (MFC) and 6 lateral femoral condyles (LFC). Patients were followed up prospectively at baseline, 24 months, 60 months, and at minimum ten years (12.6 ± 2.0 years) using the IKDC subjective score and through an overall judgment on treatment satisfaction. The activity level was evaluated with the Tegner score and adverse events and failures were also recorded. Factors influencing the clinical outcomes, including age, sex, BMI, lesions size, and lesion location were also investigated.
RESULTS: No severe adverse events and no surgical failures were reported and 85.0% of patients were satisfied at a minimum ten year follow-up. Subjective IKDC showed a significant and stable improvement at all follow-ups, passing from 45.3 ± 16.5 at baseline to 73.7 ± 16.6 at 24 months (p < 0.0005), to 72.9 ± 16.6 at 60 months (p < 0.0005), and to 74.1 ± 20.8 at long-term follow-up (p < 0.0005). Patients with OCD lesions localized on the LFC obtained lower results compared to those with MFC lesions at two years and five years (p = 0.034 and p = 0.023). The highest long-term scores were obtained in patients with lesion size lower than 2 cm2 (89.1 ± 8.8) compared to patients with lesion size between 2 and 4 cm2 (69.2 ± 15.7), and patients with lesion size larger than 4 cm2 (63.8 ± 34.6).
CONCLUSIONS: OAT is a suitable technique to treat knee OCD in young patients and offers a high patient satisfaction and a significant improvement in terms of clinical subjective scores, with results remaining stable over time, although without reaching the pre-injury activity level. No severe adverse events and no surgical failures have been documented confirming OAT as a valid treatment option, although the best long-term results for lesions smaller than 2 cm2 and for MFC lesions should be considered when choosing this procedure to address knee OCD lesions.

BACKGROUND: Helsinki University Hospital has developed a digital care pathway (DCP) for people with multiple sclerosis (MS) to improve the care quality. DCP was designed for especially newly diagnosed patients to support adaptation to a chronic disease.
OBJECTIVE: This study investigated the MS DCP user behavior and its impact on patient education-mediated changes in health care use, patient-perceived impact of MS on psychological and physical functional health, and patient satisfaction.
METHODS: We collected data from the service launch in March 2020 until the end of 2022 (observation period). The number of users, user logins, and their timing and messages sent were collected. The association of the DCP on health care use was studied in a case-control setting in which patients were allowed to freely select whether they wanted to use the service (DCP group n=63) or not (control group n=112). The number of physical and remote appointments either to a doctor, nurse, or other services were considered in addition to emergency department visits and inpatient days. The follow-up time was 1 year (study period). Furthermore, a subgroup of 36 patients was recruited to fill out surveys on net promoter score (NPS) at 3, 6, and 12 months, and their physical and psychological functional health (Multiple Sclerosis Impact Scale) at 0, 3, 6, and 12 months.
RESULTS: During the observation period, a total of 225 patients had the option to use the service, out of whom 79.1% (178/225) logged into the service. On average, a user of the DCP sent 6.8 messages and logged on 7.4 times, with 72.29% (1182/1635) of logins taking place within 1 year of initiating the service. In case-control cohorts, no statistically significant differences between the groups were found for physical doctors\' appointments, remote doctors\' contacts, physical nurse appointments, remote nurse contacts, emergency department visits, or inpatient days. However, the MS DCP was associated with a 2.05 (SD 0.48) visit increase in other services, within 1 year from diagnosis. In the prospective DCP-cohort, no clinically significant change was observed in the physical functional health between the 0 and 12-month marks, but psychological functional health was improved between 3 and 6 months. Patient satisfaction improved from the NPS index of 21 (favorable) at the 3-month mark to the NPS index of 63 (excellent) at the 12-month mark.
CONCLUSIONS: The MS DCP has been used by a majority of the people with MS as a complementary service to regular operations, and we find high satisfaction with the service. Psychological health was enhanced during the use of MS DCP. Our results indicate that DCPs hold great promise for managing chronic conditions such as MS. Future studies should explore the potential of DCPs in different health care settings and patient subgroups.

Fanconi anemia (FA) is a rare and complex inherited genetic disorder characterized by impaired DNA repair mechanisms leading to genomic instability. Individuals with FA have increased susceptibility to congenital anomalies, progressive bone marrow failure, leukemia and malignant tumors, endocrinopathies and other medical issues. In recent decades, steadily improved approaches to hematopoietic cell transplantation (HCT), the only proven curative therapy for the hematologic manifestations of FA, have significantly increased the life expectancy of affected individuals, illuminating the need to understand the long-term consequences and multi-organ ramifications. Utilizing a systematized review approach with narrative synthesis of each primary issue and organ system, we shed light on the challenges and opportunities for optimizing the care and quality of life for individuals with FA and identify knowledge gaps informing future research directions.

UNASSIGNED: At 12 months in the phase 2 TOPAZ study, treatment with apitegromab was associated with both an improved motor function in patients with Type 2 or 3 spinal muscular atrophy (SMA) and with a favorable safety profile. This manuscript reports the extended efficacy and safety in the nonambulatory group of the TOPAZ study at 36 months.
UNASSIGNED: Patients who completed the primary study (NCT03921528) could enroll in an open-label extension, during which patients received apitegromab 20 mg/kg by intravenous infusion every 4 weeks. Patients were assessed periodically via the Hammersmith Functional Motor Scale-Expanded (HFMSE), Revised Upper Limb Module (RULM), World Health Organization (WHO) motor development milestones, Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT) Daily Activities and Mobility domains, and Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue questionnaire.
UNASSIGNED: Of the 58 patients enrolled in TOPAZ, 35 were nonambulatory (mean age 7.3 years). The mean change at 36 months in HFMSE score from baseline was +4.0 (standard deviation [SD]: 7.54), and + 2.4 (3.24) for RULM score (excluding n = 7 after scoliosis surgery). Caregiver-reported outcomes (PEDI-CAT and PROMIS Fatigue) showed improvements from baseline over 36 months. In addition, most patients (28/32) improved or maintained WHO motor milestones achieved at baseline. The most frequently reported treatment-emergent adverse events were pyrexia (48.6%), nasopharyngitis (45.7%), COVID-19 infection (40.0%), vomiting (40.0%), and upper respiratory tract infection (31.4%).
UNASSIGNED: The benefit of apitegromab treatment observed at 12 months was sustained at 36 months with no new safety findings.