Medial malleolar osteotomy in the treatment of osteochondral lesions of the Talus - incidence and impact on functional outcome: a retrospective analysis based on data from the German Cartilage Register (KnorpelRegister DGOU).

BACKGROUND: There has long been discussion regarding the impact of medial malleolar osteotomies (MMO) as an adjunctive treatment for osteochondral lesions of the talus (OCLT). MMO may improve the visibility and accessibility of the talus, but they also pose a risk of periprocedural morbidity. There is a lack of research about the prevalence and consequences of MMO in the surgical treatment of OCLT. METHODS: This study retrospectively evaluated data from the German Cartilage Register (KnorpelRegister DGOU) from its implementation in 2015 to December 2020. The impact of MMO on patient-reported outcome measures (PROMs) was investigated. Wherever possible, subgroups were built and matched using a propensity score which matched a group undergoing OCLT without MMO. Matching included age, sex, weight, localization of the OCLT, the international cartilage repair society (ICRS) grading, surgical procedure and preoperative symptoms using the Foot and Ankle Ability Measure (FAAM) and the Activities of Daily Living Subscale (ADL). RESULTS: The prevalence of MMO in the operative treatment of OCLT was 15.9%. Most of the osteotomies were performed in OCL of the medial talar dome (76.8%) and in more serious lesions with an ICRS grade of III (29.1%) and IV (61.4%). More than half of the osteotomies (55.6%) were performed during revision surgery. A matched pair analysis of n = 44 patients who underwent AMIC® via arthrotomy and MMO vs. arthrotomy alone showed no significant differences in patient-reported outcome measures (PROMs, i.e. FAAM-ADL, and FAOS) at 6,12 and 24 months. CONCLUSIONS: MMO are mostly used in the treatment of severe (≥ ICRS grade 3) OCL of the medial talar dome and in revision surgery. Functional and patient-reported outcome measures are not significantly affected by MMO compared to arthrotomy alone. TRIAL REGISTRATION: The German Cartilage Register (KnorpelRegister DGOU) was initially registered at the German Clinical Trials Register ( https://www.drks.de , register number DRKS00005617, Date of registration 03.01.2014) and was later expanded by the ankle module.

Optimizing erectile function evaluation pattern in postoperative pelvic fracture-urethral injury patients: introducing PFUI pEF PROM.

PURPOSE: To establish a psychometric validated pelvic fracture-urethral injury postoperative erectile function patient reported outcome measure (PFUI pEF PROM). We also aim to explore PFUI patients' potential classification and suitable postoperative erectile function assessment pattern. METHODS: A total of 93 PFUI patients who treated by excision and primary anastomotic (EPA) urethroplasty from January 2020 to August 2022 were enrolled to this study. Patients who had intercourse completed the IIEF-5, those who had sexual stimulation other than intercourse finished PFUI pEF PROM, and those who performed both completed the IIEF-5, and PFUI pEF PROM. Erection Hardness Score (EHS) was completed by all patients. This PROM was performed psychometric validation and used to find PFUI patients potential classification through latent class analysis. Then, we determined the cut-off value though receiver-operating characteristic (ROC) curve and performed univariate analysis subgroups feature. RESULTS: The PFUI pEF PROM demonstrated high reliability and validity with a Cronbach's alpha of 0.928. It correlated significantly with IIEF-5 (r = 0.550, p < 0.001) and EHS (r = 0.909, p < 0.001). The latent class analysis identified three patient subgroups, with 14.5 as the subgroup cut-off value. Urethral stricture length, IIEF-5, and EHS score were identified as influence factors for maximal erection potential. An assessment pattern combining IIEF-5, EHS, and the PFUI pEF PROM covered 92.5% of patients. CONCLUSION: This PROM effectively addresses the current limitation in assessing erectile function in PFUI patients. This study provides a promising tool for stratified assessment, prediction erection recovery, and treatment guidance in the PFUI Erectile dysfunction field.

Patient-reported outcome measures (PROMs) to personalise follow-up care of ovarian cancer: what do patients think? A qualitative interview study.

PURPOSE: The purpose of this study was to explore ovarian cancer patients' preferences regarding follow-up care and, in particular, the use of patient-reported outcome measures (PROMs) as an approach to personalise follow-up care. METHODS: Between May and June 2021, semi-structured interviews were conducted with ovarian cancer patients, who had finished their primary treatment at least 6 months prior and were receiving follow-up care at our centre. Interviews were transcribed verbatim and analysed using an inductive thematic approach. A thematic flow chart was created describing interacting themes. RESULTS: Seventeen patients were interviewed, of which 11 were familiar with PROMs. Two key themes emerged from the data: the need for reassurance and the wish for personalised care. A follow-up scheme using PROMs was identified as a separate theme with the potential to personalise care. Several barriers and facilitators of PROMs were mentioned. CONCLUSIONS: Ovarian cancer patients have a desire for personalised follow-up care and seek reassurance. PROMs may be able to support both of these needs. Future research is needed to determine the most effective, patient-centred way to implement them. IMPLICATIONS FOR CANCER SURVIVORS: By understanding what patients' preferences are regarding follow-up care, more initiatives can be set up to personalise follow-up care, through which patient anxiety and dissatisfaction can be reduced.

Bridging the gap between statistical significance and clinical relevance: A systematic review of minimum clinically important difference (MCID) thresholds of scales reported in movement disorders research.

Background: Minimum clinically important difference (MCID) is the smallest change in an outcome measure that is considered clinically meaningful. Using validated MCID thresholds for outcomes powers trials adequately to detect meaningful treatment effects, aids in their interpretation and guides development of new outcome measures. Objectives: To provide a comprehensive summary of MCID thresholds of various symptom severity scales reported in movement disorder. Methods: We conducted systematic review of the literature and included studies of one or more movement disorders, and reporting MCID scales. Results: 2763 reports were screened. Final review included 32 studies. Risk of bias (RoB) assessment showed most studies were of good quality. Most commonly evaluated scale was Unified Parkinson's Disease Rating Scale (UPDRS) (11 out of 32). Four studies assessing MDS-UPDRS had assessed its different sub-parts, reporting a change of 2.64,3.05,3.25 and 0.9 points to detect clinically meaningful improvement and 2.45,2.51,4.63 and 0.8 points to detect clinically meaningful worsening, for the Part I, II, III and IV, respectively. For Parts II + III, I + II + III and I + II + III + IV, MCID thresholds reported for clinically meaningful improvement were 5.73, 4.9, 6.7 and 7.1 points respectively; while those for clinically meaningful worsening were 4.7, 4.2, 5.2 and 6.3 points, respectively. MCID thresholds reported for other scales included Abnormal Involuntary Movement Scale (AIMS), Toronto Western Spasmodic Torticollis Rating Scale (TWSRS), and Burke-Fahn-Marsden Dystonia Scale (BFMD). Conclusion: This review summarizes all the MCID thresholds currently reported in Movement disorders research and provides a comprehensive resource for future trials, highlighting the need for standardized and validated MCID scales in movement disorder research.

A prospective study evaluating patient reported outcome measures in patients who have undergone chest wall perforator flaps.

AIMS: To evaluate Patient Reported Outcome Measures (PROMs) and surgical outcomes in patients undergoing Chest Wall Perforator Flaps (CWPFs). METHODS: This was an observational single cohort study using an audit approach and a survey instrument. 84 patients who had undergone CWPFs in the last 5 years at the Department of Breast Surgery, City Hospital Birmingham, were identified from a pre-existing database. Surgical outcomes were recorded. Patients were contacted telephonically or in person at the time of follow-up and were asked to fill up a PROMs questionnaire. RESULTS: Out of 84 patients, 58 patients chose to respond. The average age of the patients was 51.3 years (±8.2 years). The average follow-up was 15.4 months (±9.9 months). The most common histological subtype was Infiltrating ductal carcinoma (IDC)-Not otherwise specified 34/58 (58.6%). Majority of the patients had T2 cancers-28/58 (48.3%). 26/58 (44.8%) were node negative. Eight patients (13.7%) had post-operative complications. No patient had total/partial flap loss. Nine patients (15.5%) had margin re-excision. One patient developed distant metastasis while 1 patient developed a second primary. Fifty-one patients (88%) were either satisfied or very satisfied with the post-operative appearance of the breasts. Thirty-six patients (62%) had no/little persistent pain or tenderness post-surgery. Eighty-six per cent (38/44) of the patients undergoing Lateral Intercostal Artery Perforator (LICAP) Flap and 16/18 (89%) of patients undergoing Anterior Intercostal Artery Perforator (AICAP) flap had no/little difficulty in carrying out normal activities at follow up. CONCLUSION: CWPFs are associated with a low complication rate and a high patient satisfaction rate.

Glycemic outcomes and patient satisfaction and self-management improves in transition from standard to virtual multidisciplinary care.

AIMS: With advances in cloud-based technologies, there has been a rise in remote T1D care. We hypothesized that transitioning T1DM care to a virtual, multidisciplinary clinic could improve measures beyond HbA1c. METHODS: To assess the impact of transitioning from standard to virtual T1DM care, we evaluated glycemic measures and patient reported outcomes. RESULTS: Sixty-one adults with T1DM were included, with mean age 40.2 ± 13.5 years and diabetes duration 16.9 ± 9.0 years. Most patients were treated with insulin pumps and CGM. The number of annual diabetes care encounters rose from 2.1 ± 4.2 to 12.8 ± 5.5. Baseline HbA1c was 7.9 ± 1.6 %(63 ± 16.9 mmol/mol), declining to 7.3 ± 1.1 %(56 ± 8.5 mmol/mol) and 7.1 ± 1.0 %(54 ± 7.7 mmol/mol) at 6 and 12 months respectively (p < 0.001 for both). In parallel, TIR improved from 63.1 ± 19.3 % to 69.2 ± 13.8 % (p < 0.001) and 67.5 ± 19.4 % (p = 0.03) at 6 and 12 months respectively, while TBR declined. Scores from validated diabetes treatment and self-management questionnaires rose significantly and these rises were associated with a reduction in HbA1c, the latter score was also associated with increased TIR. There was a trend toward a correlation between encounter frequency and improvement in HbA1c and TIR. CONCLUSIONS: Transitioning from standard to virtual, coordinated, multidisciplinary T1DM care is associated with increased visit frequency, improving glycemic control, treatment satisfaction and self-care behaviors.

Patient-reported outcome measures in patients with familial cerebral cavernous malformations: results from the Treat_CCM trial.

Background: The Phase 1/2 Treat_CCM randomized controlled trial for people with familial cerebral cavernous malformations (FCCMs) confirmed the safety of propranolol and suggested beneficial effects on intracerebral hemorrhage or new focal neurological deficits, but the effects on patient-reported outcome measures have not been reported. Methods: Participants completed self-reported questionnaires at baseline, 1 and 2 years. Depression was assessed with the Beck Depression Inventory-II (BDI-2); Anxiety with the State-Trait Anxiety Inventory X1 and X2 (STAI X-1 and STAI X-2); and Quality of Life with the Short Form 36 (SF-36), split into the physical and mental component scales (PCS and MCS). Differences between treatment groups and the general population were assessed. Change over time by treatment was assessed by means of mixed models. Results: In total, 71 participants (48 propranolol and 23 standard care) were enrolled, of whom 61 (73%) completed questionnaires at baseline and 2-year FU. At baseline, no differences between treatment groups for any of the questionnaires were present. Twenty (31.7%) patients were considered depressed at baseline, while this proportion was lower in the propranolol group after 2 years (28.6% vs. 55.5%, p = 0.047). The STAI X-1 and X-2 scores were stable over time. PCS was lower in FCCM patients as compared with the general Italian population, while the MCS was similar to the general population. No effect of propranolol was found for both PCS and MCS. Conclusion: Depression is common among patients with FCCM. Patients randomized to propranolol had a lower proportion of participants with depression after 2 years.Clinical trial registration: https://clinicaltrials.gov/, identifier (NCT03589014).

Clinic-based perspectives on the integration of patient-reported outcomes (PROs) in a tertiary cancer center.

PURPOSE: This study examines providers' and clinic staff's perspectives on patient-reported outcomes (PROs) implementation at an academic medical center. METHODS: An anonymous and voluntary survey was administered to Henry Ford Cancer providers and clinic staff 18 months after PROs program implementation in September 2020, to obtain their feedback on perceived barriers, impact on workflows, and PROs administration frequency in routine cancer care. RESULTS: A total of 180 providers and 40 clinic staff were invited to complete the survey; 31% and 63% completed the survey, respectively. Approximately 68% of providers reported that electronically integrated PROs scores were either beneficial or somewhat beneficial to their patients, while only 28% of the clinic staff reported that PROs were beneficial or somewhat beneficial to patients. According to the clinic staff, the most common barriers to PROs completion included lack of patients' awareness of the utility of the program with respect to their care, patients' health status at check-in, and PROs being offered too frequently. CONCLUSION: There is favorable acceptance of the PROs program by providers, but clinic staff found it less favorable. Interventions to address barriers and improve program engagement are needed to ensure broad adoption of PROs in oncology practice.

Smartphone-based activity tracking for spine patients: Current technology and future opportunities.

Activity trackers and wearables allow accurate determination of physical activity, basic vital parameters, and tracking of complex medical conditions. This review attempts to provide a roadmap for the development of these applications, outlining the basic tools available, how they can be combined, and what currently exists in the marketplace for spine patients. Various types of sensors currently exist to measure distinct aspects of user movement. These include the accelerometer, gyroscope, magnetometer, barometer, global positioning system (GPS), Bluetooth and Wi-Fi, and microphone. Integration of data from these sensors allows detailed tracking of location and vectors of motion, resulting in accurate mobility assessments. These assessments can have great value for a variety of healthcare specialties, but perhaps none more so than spine surgery. Patient-reported outcomes (PROMs) are subject to bias and are difficult to track frequently - a problem that is ripe for disruption with the continued development of mobility technology. Currently, multiple mobile applications exist as an extension of clinical care. These include Manage My Surgery (MMS), SOVINITY-e-Healthcare Services, eHealth System, Beiwe Smartphone Application, QS Access, 6WT, and the TUG app. These applications utilize sensor data to assess patient activity at baseline and postoperatively. The results are evaluated in conjunction with PROMs. However, these applications have not yet exploited the full potential of available sensors. There is a need to develop smartphone applications that can accurately track the functional status and activity of spine patients, allowing a more quantitative assessment of outcomes, in contrast to legacy PROMs.

Minimal Clinically Important Difference of Scales Reported in Stroke Trials: A Review.

There is a growing awareness of the significance of using minimum clinically important differences (MCIDs) in stroke research. An MCID is the smallest change in an outcome measure that is considered clinically meaningful. This review is the first to provide a comprehensive summary of various scales and patient-reported outcome measures (PROMs) used in stroke research and their MCID values reported in the literature, including a concise overview of the concept of and methods for determining MCIDs in stroke research. Despite the controversies and limitations surrounding the estimation of MCIDs, their importance in modern clinical trials cannot be overstated. Anchor-based and distribution-based methods are recommended for estimating MCIDs, with patient self-evaluation being a crucial component in capturing the patient's perspective on their health. A combination of methods can provide a more comprehensive understanding of the clinical relevance of treatment effects, and incorporating the patient's perspective can enhance the care of stroke patients.

Exploring Patients' Perceptions About Chronic Kidney Disease and Their Treatment: A Qualitative Study.

BACKGROUND: Unhelpful illness perceptions can be changed by means of interventions and can lead to improved outcomes. However, little is known about illness perceptions in patients with chronic kidney disease (CKD) prior to kidney failure, and no tools exist in nephrology care to identify and support patients with unhelpful illness perceptions. Therefore, this study aims to: (1) identify meaningful and modifiable illness perceptions in patients with CKD prior to kidney failure; and (2) explore needs and requirements for identifying and supporting patients with unhelpful illness perceptions in nephrology care from patients' and healthcare professionals' perspectives. METHODS: Individual semi-structured interviews were conducted with purposive heterogeneous samples of Dutch patients with CKD (n = 17) and professionals (n = 10). Transcripts were analysed using a hybrid inductive and deductive approach: identified themes from the thematic analysis were hereafter organized according to Common-Sense Model of Self-Regulation principles. RESULTS: Illness perceptions considered most meaningful are related to the seriousness (illness identity, consequences, emotional response and illness concern) and manageability (illness coherence, personal control and treatment control) of CKD. Over time, patients developed more unhelpful seriousness-related illness perceptions and more helpful manageability-related illness perceptions, caused by: CKD diagnosis, disease progression, healthcare support and approaching kidney replacement therapy. Implementing tools to identify and discuss patients' illness perceptions was considered important, after which support for patients with unhelpful illness perceptions should be offered. Special attention should be paid towards structurally embedding psychosocial educational support for patients and caregivers to deal with CKD-related symptoms, consequences, emotions and concerns about the future. CONCLUSIONS: Several meaningful and modifiable illness perceptions do not change for the better by means of nephrology care. This underlines the need to identify and openly discuss illness perceptions and to support patients with unhelpful illness perceptions. Future studies should investigate whether implementing illness perception-based tools will indeed improve outcomes in CKD.

Patient adherence to patient-reported outcome measure (PROM) completion in clinical care: current understanding and future recommendations.

BACKGROUND: Patient-reported outcome measures (PROMs) are increasingly being used as an assessment and monitoring tool in clinical practice. However, patient adherence to PROMs completions are typically not well documented or explained in published studies and reports. Through a collaboration between the International Society for Quality-of-Life Research (ISOQOL) Patient Engagement and QOL in Clinical Practice Special Interest Groups (SIGs) case studies were collated as a platform to explore how adherence can be evaluated and understood. Case studies were drawn from across a range of clinically and methodologically diverse PROMs activities. RESULTS: The case studies identified that the influences on PROMs adherence vary. Key drivers include PROMs administeration methods within a service and wider system, patient capacity to engage and clinician engagement with PROMs information. It was identified that it is important to  evaluate  PROMs integration and adherence from multiple perspectives. CONCLUSION: PROM completion rates are an important indicator of patient adherence. Future research prioritizing an understanding of PROMs completion rates by patients is needed.

Impact of a wearable-based physical activity and sleep intervention in multimorbidity patients: protocol for a randomized controlled trial.

BACKGROUND: The benefits of physical activity (PA) and adequate sleep are well documented, and their importance strengthens with the increasing prevalence of chronic diseases and multimorbidity (MM). Interventions to promote physical activity and sleep that use commercial activity trackers may be useful non-pharmacological approaches to managing individual health; however, limited evidence exists on their use to improve physical activity in older adult patients with MM. METHODS: This study aims to measure the effects of behavioral change techniques (BCTs) delivered by a wearable device on physical activity and quality of sleep (QS) in older adult patients with MM. We designed an open-label randomized controlled trial with participants recruited through primary care and a specialist outpatient clinic. Participants must be more than 65 years old, have MM, and have access to smartphones. All eligible participants will receive PA promotion content and will be randomly assigned to wear a smartwatch. The primary outcome will be the participants' PA measurement at baseline and at six months using the International Physical Activity Questionnaire - Short Form (IPAQ-SF). Secondary outcomes will include changes in the participants' frailty status, biometric measurements, quality of life, and biopsychosocial assessments. A sample size of 40 participants per arm was calculated to detect group differences, with 50 participants planned to recruit and randomize into each arm. DISCUSSION: This study aims to contribute to a better understanding of PA patterns and the impact of wearable-based PA interventions in patients with MM. In addition, we aim to contribute to more knowledge about the relationship between PA patterns, Patient Reported Outcomes Measures (PROMs), and healthcare resource utilization in patients with MM. To achieve this, the study will leverage a locally developed PROMs registry and assess data from participants' medical records, in order to understand the added impact of wearable data and medical information data on predicting PROMs and unplanned hospital admissions. TRIAL REGISTRATION: NCT05777291.

From simple to even simpler, but not too simple: a head-to-head comparison of the Better-Worse and Drop-Down methods for measuring patient health status.

BACKGROUND: We recently developed a novel, preference-based method (Better-Worse, BW) for measuring health status, expressed as a single metric value. We have since expanded it by developing the Drop-Down (DD) method. This article presents a head-to-head comparison of these two methods. We explored user feasibility, interpretability and statistics of the estimated coefficients, and distribution of the computed health-state values. METHODS: We conducted a cross-sectional online survey among patients with various diseases in the USA. The BW and DD methods were applied in the two arms of the study, albeit in reverse order. In both arms, patients first performed a descriptive task (Task 1) to rate their own health status according to the 12 items (each with 4 levels) in the CS-Base health-outcome instrument. They then performed Task 2, in which they expressed preferences for health states by the two methods. We then estimated coefficients for all levels of each item using logistic regression and used these to compute values for health states. RESULTS: Our total sample comprised 1,972 patients. Completion time was < 2 min for both methods. Both methods were scored as easy to perform. All DD coefficients were highly significant from the reference level (P < 0.001). For BW, however, only the second-level coefficient of "Cognition" was significantly different (P = 0.026). All DD coefficients were more precise with narrower confidence intervals than those of the BW method. CONCLUSIONS: Both the BW and DD are novel methods that are easy to apply. The DD method outperformed the BW method in terms of the precision of produced coefficients. Due to its task, it is free from a specific distorting factor that was observed for the BW method.

What is the association between gender and self-perceived health status when controlling for disease-specific conditions? A retrospective data analysis of pre- and post-operative EQ-5D-5L differences in total hip and knee arthroplasty.

BACKGROUND: The self-perceived health status of patients undergoing total hip and knee arthroplasty (THA and TKA) might differ post-operatively due to gender, age, or comorbidities. Patient-reported outcome measures (PROMs) such as the EQ-5D-5L measure the self-perceived health status. This study investigates whether the index score of the EQ-5D-5L is a valid tool for interpreting gender differences in outcomes for patients undergoing THA and TKA. METHODS: Routine and PROM-data of elective primary THA or TKA patients in two German hospitals between 2016 and 2018 were analyzed. Univariate analysis with Pearson's chi-square was conducted to identify control variables for gender. To quantify the association between gender and the EQ-5D-5L dimensions, a cumulative odds ordinal logistic regression with proportional odds was conducted. RESULTS: Two thousand three hundred sixty-eight​​ THA patients (m = 978; f = 1390) and 1629 TKA patients (m = 715; f = 914) were considered. The regression analysis of the individual EQ-5D-5L dimensions showed that female gender was significantly associated with better self-care (THA and TKA) and better post-operative mobility (THA). In contrast, male gender was significantly associated with less pain/discomfort (TKA) and less anxiety/depression (THA) pre-surgery and 3-months post-surgery. CONCLUSION: Our results confirmed that the self-perceived health status improved after surgery. However, due to the different associations of gender to the individual dimensions of the EQ-5D-5L, the weighted index score clouds the comparability between patients with different gender undergoing THA or TKA. Therefore, we argue to use the individual five dimensions for health status analysis, to reveal relevant additional information.

Patients undergoing total hip and knee arthroplasty (THA and TKA) can fill out standardized questionnaires pre- and post-surgery, such as the EQ-5D-5L, to measure the improvement in the self-perceived health status. The EQ-5D-5L includes mobility, self-care, usual activity, pain/discomfort, and anxiety/depression. We do not know whether male and female patients experience the same improvement in the dimensions or whether significant differences exist. Currently, only index scores of the EQ-5D-5L are used for the comparison of pre- and post-operative health status. However, due to the questionnaire's weighted composition, relevant changes in individual dimensions might be easily missed. Thus, we investigated whether significant differences between gender and the EQ-5D-5L dimensions in patients undergoing TKA and TKA are observable. We found that female patients reported significantly better scores in self-care (THA and TKA) and post-operative mobility (THA). In contrast, male gender was significantly associated with less pain/discomfort (TKA) and less anxiety/depression (THA) pre-surgery and 3-months post-surgery. The EQ-5D-5L's weighted index score, however, does not directly represent these differences. Therefore, we argue to use the individual five dimensions for health status analysis, as relevant additional information on improvement over time would otherwise be missed.

Minimal Clinically Important Difference (MCID) in Patient-Reported Outcome Measures for Neurological Conditions: Review of Concept and Methods.

The concept of the minimal clinically important difference (MCID) emerged from the recognition that statistical significance alone is not enough to determine the clinical relevance of treatment effects in clinical research. In many cases, statistically significant changes in outcomes may not be meaningful to patients or may not result in any tangible improvements in their health. This has led to a growing emphasis on the importance of measuring patient-reported outcome measures (PROMs) in clinical trials and other research studies, in order to capture the patient perspective on treatment effectiveness. MCID is defined as the smallest change in scores that is considered meaningful or important to patients. MCID is particularly important in fields such as neurology, where many of the outcomes of interest are subjective or based on patient-reported symptoms. This review discusses the challenges associated with interpreting outcomes of clinical trials based solely on statistical significance, highlighting the importance of considering clinical relevance and patient perception of change. There are two main approaches to estimating MCID: anchor-based and distribution-based. Anchor-based approaches compare change scores using an external anchor, while distribution-based approaches estimate MCID values based on statistical characteristics of scores within a sample. MCID is dynamic and context-specific, and there is no single 'gold standard' method for estimating it. A range of MCID thresholds should be defined using multiple methods for a disease under targeted intervention, rather than relying on a single absolute value. The use of MCID thresholds can be an important tool for researchers, neurophysicians and patients in evaluating the effectiveness of treatments and interventions, and in making informed decisions about care.

Does the application of autologous injectable Platelet-Rich Fibrin (i-PRF) affect the patient's daily performance during the retraction of upper canines? A single-centre randomized split-mouth controlled trial.

BACKGROUND: Previous studies have assessed different aspects concerning the applications of i-PRF in the oral cavity. However, nothing is known regarding patients' perceptions of the injection of autologous platelet-rich fibrin (i-PRF). OBJECTIVES: To investigate patients' perceptions after injecting platelet-rich fibrin (i-PRF) in the course of retracting upper canines. METHODS: Twenty-one patients, whose treatments required extractions of both upper first premolars, were recruited. Extraction side was randomly allocated to the intervention or control sides. After the alignment phase, i-PRF was injected twice with a one-month interval on the buccal and palatal aspects of the extraction sites (intervention side). Patients' perceptions were evaluated with two questionnaires: the first was used to assess the level of pain, discomfort, swelling, eating and swallowing difficulties as well as jaw movement restriction after 1 h (T1), 2 h (T2), 6 h (T3), 24 h (T4) and 48 h (T5) of the second injection; the second questionnaire was used to assess the acceptance of the i-PRF injection and overall satisfaction with this technique at the end of canine retraction phase. Visual Analogue Scale (VAS) was adopted for this purpose. Wilcoxon Signed Rank Test was used to compare between both sides at all time points while Friedman's Test was the selected test for detecting variables' changes over time. Post-hoc Wilcoxon Matched-Pairs Signed-Rank Tests were applied when any of the results were significant. As to the multiplicity of tests, Bonferroni Correction was implemented. RESULTS: Pain and swelling levels were significantly higher on the experimental compared to the control sides at T1, T2, and T3 (P < 0.05), whereas they declined sharply and went back to almost normal values at T4 (after 24 h). At T5 they were 0. Discomfort and difficulty in mastication on intervention sides were significant only at T1 and T2. Pain, swelling, and chewing difficulties were significant (P < 0.001) during the 4 assessed time points. The increase was insignificant regarding swallowing difficulties and jaw movement limitations at all time intervals. CONCLUSIONS: Injecting autologous (i-PRF) during orthodontic canine retraction is a well-perceived and well-tolerated method due to the limited discomfort which significantly diminishes 24 h afterwards. TRIAL'S REGISTRATION: ClinicalTrials.gov (Identifier Number: NCT03399422. 16/01/2018).

Real-world analysis of ruxolitinib in myelofibrosis: interim results focusing on patients who were naïve to JAK inhibitor therapy treated within the JAKoMo non-interventional, phase IV trial.

Ruxolitinib (RUX) is a Janus kinase 1/2 inhibitor (JAKi) approved in the EU for treating disease­related splenomegaly or symptoms in adults patients with myelofibrosis (MF). This is an interim analysis of JAKoMo, a prospective, non­interventional, phase IV study in MF. Between 2012-2019 (cutoff March 2021), 928 patients (JAKi-naïve and -pretreated) enrolled from 122 German centers. This analysis focuses on JAKi-naïve patients. RUX was administered according to the Summary of Product Characteristics. Compared to the COMFORT-I, -II, and JUMP trials, patients in JAKoMo were older (median 73 years), had poorer Eastern Cooperative Oncology Group (ECOG) performance statuses (16.5% had ECOG ≥ 2), and were more transfusion dependent (48.5%). JAKoMo represents the more challenging patients with MF encountered outside of interventional studies. However, patients with low-risk International Prognostic Scoring System (IPSS) scores or without palpable splenomegaly were also included. Following RUX treatment, 82.5% of patients experienced rapid (≤ 1 month), significant decreases in palpable spleen size, which remained durable for 24 months (60% patients). Symptom assessment scores improved significantly in Month 1 (median -5.2) up to Month 12 (-6.2). Common adverse events (AEs) were anemia (31.2%) and thrombocytopenia (28.6%). At cutoff, 54.3% of patients had terminated the study due to, death, AEs, or deterioration of health. No new safety signals were observed. Interim analysis of the JAKoMo study confirms RUX safety and efficacy in a representative cohort of real-world, elderly, JAKi-naïve patients with MF. Risk scores were used in less than half of the patients to initiate RUX treatment.Trial registration: NCT05044026; September 14, 2021.