Long-term Patient-reported Outcomes Research Articles

Female non-obstetric urogenital fistula repair: long-term patient-reported outcomes and a scoping literature review.

To investigate long-term and patient-reported outcomes, including sexual function, in women undergoing urogenital fistula (UGF) repair, addressing the lack of such data in Western countries, where fistulas often result from iatrogenic causes. We conducted a retrospective analysis at a tertiary referral centre (2010-2023), classifying fistulas based on World Health Organisation criteria and evaluating surgical approaches, aetiology, and characteristics. Both objective (fistula closure, reintervention rates) and subjective outcomes (validated questionnaires) were assessed. A scoping review of patient-reported outcome measures in UGF repair was also performed. The study included 50 patients: 17 (34%) underwent transvaginal and 33 (66%) transabdominal surgery. History of hysterectomy was present in 36 patients (72%). The median (interquartile range [IQR]) operating time was 130 (88-148) min. Fistula closure was achieved in 94% of cases at a median (IQR) follow-up of 50 (16-91) months and reached 100% after three redo fistula repairs. Seven patients (14%) underwent reinterventions for stress urinary incontinence after transvaginal repair (autologous fascial slings). Patient-reported outcomes showed median (IQR) scores on the International Consultation on Incontinence Questionnaire Female Lower Urinary Tract Symptoms Modules (ICIQ-FLUTS) of 5 (3-7) for filling symptoms, 1 (0-2) for voiding symptoms and 4.5 (1-9) for incontinence symptoms. The median (IQR) score on the ICIQ Female Sexual Matters Associated with Lower Urinary Tract Symptoms Module (ICIQ-FLUTSsex) was 3 (1-5). The median (IQR) ICIQ Satisfaction (ICIQ-S) outcome score and overall satisfaction with surgery item score was 22 (18.5-23.5) and 10 (8.5-10), respectively. Higher scores indicate higher symptom burden and treatment satisfaction, respectively. Our scoping review included 1784 women, revealing mixed aetiology and methodological and aetiological heterogeneity, thus complicating cross-study comparisons. Urogenital fistula repair at a specialised centre leads to excellent outcomes and high satisfaction. Patients with urethrovaginal fistulas are at increased risk of stress urinary incontinence, possibly due to the original trauma site of the fistula.

Patient reported outcomes and patient experiences of immune checkpoint modulators for advanced or recurrent melanoma: a mixed methods study.

Little is known about late and long-term patient-reported outcomes (PROs) of immune checkpoint modulators (ICMs) outside clinical trials. We conducted a cross-sectional, mixed-methods study to describe long-term PROs among advanced melanoma patients who began standard of care treatment with ICMs at least 1year previously. All participants completed the Functional Assessment of Cancer Therapy-Immune Checkpoint Modulator (FACT-ICM), assessing 46 immune-related side effects on a 5-point Likert scale, and a subset completed individual interviews. Descriptive statistics were computed for quantitative data and applied thematic analysis was used to examine qualitative data. Participants (N = 80) had a mean age of 67years, and the majority were male (66%), non-Hispanic White (96%), and college graduates (61%). Single-agent nivolumab was the most common first (47%) and current/recent ICM (64%). On the FACT-ICM, 98% of participants reported at least one side effect, and 78% reported moderate or severe side effects. The most common moderate or severe side effects were aching joints (43%) and fatigue (38%). In interviews (n = 20), we identified five themes regarding patients' longer-term experiences after ICMs: lasting fatigue or decline in functioning, minimal side effects, manageable thyroid and pituitary dysfunction, skin conditions can be difficult to manage, and treating the cancer is worth the side effects. Nearly all patients reported side effects of ICMs at least 1year after starting treatment. Our findings suggest that ICM side effect screening and management-especially for aching joints and fatigue-are indicated during long-term care of people living with advanced melanoma.

Abstract PO2-22-12: Patient-reported outcomes 3 and 18 months after prepectoral implant-based breast reconstruction: Results from the UK Pre-BRA multicentre prospective cohort study

Abstract Background: Prepectoral implant-based reconstruction (PPBR) with and without mesh is becoming the standard of care for prosthetic reconstruction in the UK. In addition to being easier and quicker to perform, the procedure is perceived to be associated with better patient-centred outcomes including decreased post-operative pain, avoidance of animation deformity and improved cosmesis. Evidence to support these benefits, however, is lacking. The UK PreBRA prospective multicentre cohort study (ISRCTN11898000) aimed to assess patient-reported outcomes (PROs) before and 3 and 18 months following PPBR to provide much needed evidence to support the effectiveness of the technique. Methods: Women aged 18 or over undergoing mastectomy for breast cancer or risk-reduction who opted to have PPBR between July 2019 and December 2020 were eligible to participate in the study. Demographic, operative, oncology and three-month complication data were collected. Participants were asked to complete the validated BREAST-Q questionnaire at before surgery and at 3 and 18 months following the procedure. Questionnaires were scored according to the developers’ instructions and scores compared over time. Exploratory analysis of covariance, adjusting for baseline scores was performed to explore factors impacting PROs following PPBR. Results: 347 women underwent PPBR at 40 centres across the UK. Of these, 334 (96.3%) completed PROMs at baseline with 237 (68.3%) completing at least two of the three main BREAST-Q scales at both 3 and 18 months. The median age of the cohort was 49 (range 23-74). Most women (n=291, 87.1%) had mastectomy for malignancy with a quarter (n=82, 24.6%) undergoing bilateral surgery. Approximately a fifth (n=68, 20.4%) had a body mass index >30 and 12% (n=40) were active smokers at the time of surgery. At 3 months, 68 (20.4%) had experienced a complication requiring readmission and/or re-operation and 27 (8.1%) experienced an implant loss. In the cohort overall, women undergoing PPBR reported slight increases in their ‘Satisfaction with Breasts’ scores from baseline to 3 months after surgery but by 18 months, their scores had fallen below baseline values (Table 1). ‘Physical well-being’ scores decreased from baseline to 3 months but then remained stable. No changes were seen in ‘Psychosocial well-being’ scores across the three timepoints. At 18 months, after adjusting for baseline BREAST Q scores, women having surgery for risk-reduction reported scores that were 10-16 points lower than those having surgery for malignancy across all four main domains for the BREAST-Q. This exceeded the minimum clinically meaningful difference and was highly statistically significant. Women with high mastectomy weights ( >600g) reported lower scores for ‘Physical’, ‘Psychosocial’ and ‘Sexual’ well-being but not ‘Satisfaction with Breasts’. Smoking adversely impacted ‘Psychosocial well-being’ and ‘Satisfaction with Breasts’ and women with BMI >30 reported lower ‘Physical well-being’ scores at 18 months. Having a major complication requiring readmission/re-operation did not affect BREAST-Q scores, but when considered separately, implant loss dramatically impacted all BREAST-Q domains Conclusions: Women having PPBR report short-term improvements in ‘Satisfaction with Breasts’ scores but these are not maintained over time and decrease to below baseline 18 months after surgery. ‘Physical well-being’ also decreased after PPBR despite no disruption to the pectoralis muscle. Women having surgery for risk-reduction reported the worse PROs. Further work is needed to establish the long-term patient-reported outcomes of PPBR and factors impacting these. Citation Format: Leigh Johnson, Kate Harvey, Parisa Sinai, Nicola Mills, Paul White, Chris Holcombe, Shelley Potter. Patient-reported outcomes 3 and 18 months after prepectoral implant-based breast reconstruction: Results from the UK Pre-BRA multicentre prospective cohort study [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO2-22-12.

Long-term functional success and patient-reported outcomes after female one-stage buccal mucosal graft urethroplasty

Introduction and objectivesFemale urethral strictures are a rare condition that significantly impacts patients’ quality of life. Patient-reported outcomes are crucial, yet data regarding sexual function and treatment satisfaction are scarce. We aimed to provide insights from a reconstructive referral center. Patients and methodsWe conducted a retrospective analysis of women treated with ventral onlay one-stage buccal mucosa graft urethroplasty for urethral strictures between 2009–2023. We assessed objective (retreatment-free survival, ΔQmax) and subjective outcomes (validated patient-reported outcomes). ResultsOf 12 women, 83% and 17% had iatrogenic and idiopathic strictures, respectively. Median number of prior interventions was 6. Strictures were located meatal and mid-urethral in 25% and 75%, respectively, 22% had the bladder neck involved. Median graft length was 2 cm. At median follow-up of 66 months, 33% of patients underwent stricture retreatment, but only one case occurred within the first 2 years postoperatively. The median improvement in maximum flow rate (ΔQmax) was 10 ml/s. Median International Consultation on Incontinence Questionnaire Female Lower Urinary Tract Symptoms Modules (ICIQ-FLUTS) scores were 8 for filling symptoms, 6 for voiding symptoms, and 3 for incontinence symptoms. Median ICIQ-FLUTSsex score was 4. Higher scores indicate a higher symptom burden. Median ICIQ-Satisfaction outcome and satisfaction scores were 18 and 7, respectively, reflecting high treatment satisfaction. ConclusionsBuccal mucosal graft urethroplasty by ventral onlay for female urethral strictures yields effective, durable, and positively received outcomes. However, larger studies across multiple institutions are necessary to further assess its efficacy, especially regarding patient-reported experiences and sexual function.

Quality of life in 583 head and neck cancer survivors assessed with the FACE-Q head and neck cancer module

ObjectivesLong-term health-related quality of life (HRQOL) is frequently affected in head and neck cancer (HNC) survivors. Due to the shortage of studies investigating long-term patient-reported outcomes, we investigated long-term HRQOL using the novel FACE-Q HNC Module. MethodsA retrospective cross-sectional single-center study was performed, including all patients who underwent curative treatment for HNC between 2006 and 2013. All eligible patients (n = 863) were invited to participate of whom 540 completed the questionnaires. Additionally, a prospective longitudinal cohort of 43 HNC patients treated between 2020 and 2022 was included who preoperatively filled in the FACE-Q, and at three, six, and 12 months postoperatively. HRQOL domain scores were analyzed to visualize group characteristics by tumor site and type of surgical resection. ResultsBoth surgical and non-surgical HNC treatments predominantly affected long-term functional HRQOL domains (eating, salivation, speech, and swallowing), eating distress, and speaking distress, with distinct profiles depending on tumor site and type of treatment. In contrast, few long-term intergroup differences between HNC patients were observed for appearance, smiling, drooling distress, and smiling distress. Longitudinal data showed significant deterioration across all functional HRQOL domains in the short-term following treatment. Patients predominantly reported long-lasting negative treatment effects at 12 months follow-up for functional domains, whereas psychological domains showed near-complete recovery at group level. ConclusionsAt long-term, various function-related and psychosocial HRQOL domains still are affected in head and neck cancer survivors. The results may serve to better inform patients with regard to specific treatments and highlight HRQOL domains which may potentially be optimized.

Electronic Patient-Reported Outcome-Based Symptom Management Versus Usual Care After Lung Cancer Surgery: Long-Term Results of a Multicenter, Randomized, Controlled Trial.

Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.We previously reported superior symptom control of electronic patient-reported outcome (ePRO)-based symptom management after lung cancer surgery for up to 1 month postdischarge. Here, we present the long-term results (1-12 months) of this multicenter, randomized trial, where patients were assigned 1:1 to receive postoperative ePRO-based symptom management or usual care daily postsurgery, twice weekly postdischarge until 1 month, and at 3, 6, 9, and 12 months postdischarge. Long-term patient-reported outcomes were assessed with MD Anderson Symptom Inventory-Lung Cancer module. Per-protocol analyses were performed with 55 patients in the ePRO group and 57 in the usual care group. At 12 months postdischarge, the ePRO group reported significantly fewer symptom threshold events (any of the five target symptom scored ≥4; median [IQR], 0 [0-0] v 0 [0-1]; P = .040) than the usual care group. From 1 to 12 months postdischarge, the ePRO group consistently reported significantly lower composite scores for physical interference (estimate, -0.86 [95% CI, -1.32 to -0.39]) and affective interference (estimate, -0.70 [95% CI, -1.14 to -0.26]). Early intensive ePRO-based symptom management after lung cancer surgery reduced symptom burden and improved functional status for up to 1 year postdischarge, supporting its integration into standard care.

Outcomes and Quality of Life After Resorbable Synthetic Ventral Hernia Repair in Contaminated Fields.

The ideal mesh type for hernia repair in a contaminated field remains controversial. Data regarding outcomes beyond 1 year and the impact on quality of life (QoL) are unknown. This study aims to evaluate the long-term surgical outcomes and patient-reported outcomes (PROs) after contaminated repair with poly-4-hydroxybutyrate (P4HB) mesh. Patients undergoing a contaminated hernia repair with P4HB between 2015 and 2020 were identified. Surgical site occurrences and hernia recurrences were recorded. Long-term PROs as defined by the Abdominal Hernia-Q and Hernia-Related Quality-of-Life Survey were assessed. Fifty-five patients were included with a mean age of 54.5 years, a body mass index of 34 kg/m2, and a defect size of 356.9 ± 227.7 cm2. Median follow-up time was 49 months with a reoperation rate of 14.5% and a hernia recurrence rate of 7.3%. Of the 55 patients, 37 completed preoperative and postoperative PRO questionnaires. When comparing preoperative with postoperative Abdominal Hernia-Q, there was significant improvement in overall PROs (2.1 vs 3.5, P < 0.001). This improvement was maintained with no significant changes between postoperative scores over the course of 5 years. Patients with complications saw the same improvement in postoperative PRO scores as those without complications. Our study found a low hernia recurrence and acceptable long-term reoperation rates in patients undergoing hernia repair with P4HB mesh in a contaminated setting. We demonstrate a sustained significant improvement in QoL scores after hernia repair. These data yield insight into the long-term outcomes and QoL improvement, providing physicians further knowledge to better counsel their patients.

Long-term patient-reported outcomes among patients undergoing revascularization vs medical therapy for intermittent claudication

ObjectiveSociety for Vascular Surgery guidelines recommend revascularization for patients with intermittent claudication (IC) if it can improve patient function and quality of life. However, it is still unclear if patients with IC achieve a significant functional benefit from surgery compared with medical management alone. This study examines the relationship between IC treatment modality (operative vs nonoperative optimal medical management) and patient-reported outcomes for physical function (PROMIS-PF) and satisfaction in social roles and activities (PROMIS-SA). MethodsWe identified patients with IC who presented for index evaluation in a vascular surgery clinic at an academic medical center between 2016 and 2021. Patients were stratified based on whether they underwent a revascularization procedure during follow-up vs continued nonoperative management with medication and recommended exercise therapy. We used linear mixed-effect models to assess the relationship between treatment modality and PROMIS-PF, PROMIS-SA, and ankle-brachial index (ABI) over time, clustering among repeat patient observations. Models were adjusted for age, sex, diabetes, Charlson Comorbidity Index, Clinical Frailty Score, tobacco use, and index ABI. ResultsA total of 225 patients with IC were identified, of which 40% (n = 89) underwent revascularization procedures (42% bypass; 58% peripheral vascular intervention) and 60% (n = 136) continued nonoperative management. Patients were followed up to 6.9 years, with an average follow-up of 5.2 ± 1.6 years. Patients who underwent revascularization were more likely to be clinically frail (P = .03), have a lower index ABI (0.55 ± 0.24 vs 0.72 ± 0.28; P < .001), and lower baseline PROMIS-PF score (36.72 ± 8.2 vs 40.40 ± 6.73; P = .01). There were no differences in patient demographics or medications between treatment groups. Examining patient-reported outcome trends over time; there were no significant differences in PROMIS-PF between groups, trends over time, or group differences over time after adjusting for covariates (P = .07, P = .13, and P =.08, respectively). However, all patients with IC significantly increased their PROMIS-SA over time (adjusted P = .019), with patients managed nonoperatively more likely to have an improvement in PROMIS-SA over time than those who underwent revascularization (adjusted P = .045). ConclusionsPatient-reported outcomes associated with functional status and satisfaction in activities are similar for patients with IC for up to 7 years, irrespective of whether they undergo treatment with revascularization or continue nonoperative management. These findings support conservative long-term management for patients with IC.

172 Does the Number of Social Risk Factors Affect Long-term Patient-Reported Outcomes and Satisfaction in Those With Cervical Myelopathy? A QOD Study

INTRODUCTION: It is not clear whether there is an additive effect of social risk factors in affecting patients with cervical spondylotic myelopathy (CSM) from achieving both a minimum clinically important difference (MCID) in outcomes and satisfaction after surgery. METHODS: This was a retrospective study of the prospective Quality Outcomes Database Cervical Spondylotic Myelopathy (QOD CSM) cohort. Social risk factors included race, education, employment, and insurance. Patients were considered to have improved from surgery if 1) they reported a score of 1 or 2 on the NASS index and 2) met the MCID in patient-reported outcomes (VAS neck and arm pain, NDI, EQ5D, or mJOA score) at 24-month follow-up. RESULTS: Of the 1,141 patients included in the study, 205 (18.0%) had zero, 347 (30.4%) had one, 334 (29.3%) had two, and 255 (22.3%) had three social risk factors. The 24-month follow-up rate was 87.4% for patient-reported outcomes. After adjusting for all significant covariates, patients with one or more social risk factors were less likely to improve from surgery in all measured outcomes including VAS neck and arm pain, NDI, EQ-5D, and mJOA (all p &lt; 0.05) compared to those without any social risk factors. Patients with two or three social risk factors were more likely to fare worse in all outcomes compared to those with only one social risk factor (all p &lt; 0.05). CONCLUSIONS: Compared to those without any social risk factors, patients who had at least one social risk factor were less likely to achieve MCID and feel satisfied after surgery. The effect of social risk factors is additive in that those with a higher number of risk factors are less likely to improve compared to those with only one social risk factor.

Poor postoperative pain control is associated with poor long-term patient-reported outcomes after elective spine surgery: an observational cohort study

Background ContextA significant proportion of patients experience poorly controlled surgical pain and fail to achieve satisfactory clinical improvement after spine surgery. However, a direct association between these variables has not been previously demonstrated. PurposeTo investigate the association between poor postoperative pain control and patient-reported outcomes after spine surgery. Study DesignAmbispective cohort study. Patient SampleConsecutive adult patients (≥ 18-years old) undergoing inpatient elective cervical or thoracolumbar spine surgery. Outcome MeasurePoor surgical outcome was defined as failure to achieve a minimal clinically important difference (MCID) of 30% improvement on the Oswestry Disability Index or Neck Disability Index at follow-up (3-months, 1-year, and 2-years). MethodsPoor pain control was defined as a mean numeric rating scale score of >4 during the first 24-hours after surgery. Multivariable mixed-effects regression was used to investigate the relationship between poor pain control and changes in surgical outcomes while adjusting for known confounders. Secondarily, the Calgary Postoperative Pain After Spine Surgery (CAPPS) score was investigated for its ability to predict poor surgical outcome. ResultsOf 1305 patients, 47.8%, 37.3%, and 39.8% failed to achieve the MCID at 3-months, 1-year, and 2-years, respectively. The incidence of poor pain control was 56.9%. Multivariable analyses showed poor pain control after spine surgery was independently associated with failure to achieve the MCID (OR 2.35 [95%CI=1.59-3.46], p<0.001) after adjusting for age (p=0.18), female sex (p=0.57), any nicotine products (p=0.041), ASA physical status >2 (p<0.001), ≥3 motion segment surgery (p=0.008), revision surgery (p=0.001), follow-up time (p<0.001), and thoracolumbar surgery compared to cervical surgery (p=0.004). The CAPPS score was also found to be independently predictive of poor surgical outcome. ConclusionPoor pain control in the first 24-hours after elective spine surgery was an independent risk factor for poor surgical outcome. Perioperative treatment strategies to improve postoperative pain control may lead to improved patient-reported surgical outcomes.

Clinical Pharmacist Counselling Improves Long-term Medication Safety and Patient-reported Outcomes in Anti-TNF-treated Patients With Inflammatory Bowel Diseases: The Prospective, Randomized AdPhaNCED Trial.

Antitumor necrosis factor (anti-TNF) antibody treatment has led to marked improvements in the management of patients with inflammatory bowel diseases (IBDs). Nevertheless, anti-TNF therapy is associated with potential adverse drug reactions (ADRs). Our prospective, randomized trial investigated the effect of intensified clinical pharmacist counselling in a multidisciplinary team on medication safety in anti-TNF-treated IBD patients. Patients with IBD with ongoing anti-TNF treatment were enrolled in our tertiary center AdPhaNCED trial and randomized to either receive conventional standard of care (control group) or additional clinical pharmacist counselling (intervention group) over 12 months. The primary end point consisted of the number and severity of ADRs associated with anti-TNF therapy. Secondary end points included patient satisfaction with medication information and medication safety. One hundred twenty-seven IBD patients were included in this study. Anti-TNF-related ADRs were significantly lower in the intervention compared with the control group (0.20 vs 0.32 [mean] ADR/patient/month, P = .006) after 12 months. The risk of more severe ADRs (Common Terminology Criteria for Adverse Events [CTCAE] grade ≥2) was significantly higher in the control compared with the intervention group (hazard ratio, 0.34; P = .001). The probability of ADR resolution (hazard ratio, 2.02; P < .001) and patient satisfaction with medication information (14.82 vs 11.60; P < .001) were significantly higher in the intervention group compared with the control group. Our study results demonstrate that intensified pharmacist counselling significantly reduces the occurrence and severity of therapy-related ADRs and improves patient satisfaction. Clinical pharmacists should therefore be part of a holistic approach to IBD care delivered by a multidisciplinary team.

Abstract MP35: Long-Term Trajectories of Self-Rated Health Are Similar Following Incident Diabetic Foot Disease and Acute Myocardial Infarction Among Adults With Diabetes in the Atherosclerosis Risk in Communities (ARIC) Study

Introduction: Incident diabetic foot disease is associated with significant morbidity and mortality. However, data are lacking on long-term patient-reported outcomes. We aimed to evaluate trajectories of self-rated health among adults with diabetes before and after incident foot disease compared to incident acute myocardial infarction (MI), another major diabetes complication. Methods: We conducted a longitudinal analysis of participants in the ARIC Study with diabetes and incident diabetic foot disease (N=749 hospitalizations, N=73 outpatient encounters) or incident MI (N=703). Diagnosis and procedure codes from active hospitalization surveillance and Medicare claims were used to identify events; only MI events were adjudicated. Self-rated health was collected during annual telephone calls and rated from 1 (poor) to 4 (excellent); a score of 0 was assigned to deaths. We used linear regression to examine changes in self-rated health from 1 year pre-event (baseline). Relative effects of index event type over time were estimated by repeated-measures ANOVA. Results: Self-rated health was similar between groups at baseline ( Figure ). Participants with MI had sharper declines from baseline at 1 year post-event than those with diabetic foot disease (-0.93 vs -0.50 points, P&lt;0.01), largely due to higher mortality (29.4% after MI vs 15.9% after diabetic foot disease). Self-rated health stabilized in the outpatient diabetic foot group after 2 years post-event, whereas it continued to decline over 10 years following both MI and inpatient diabetic foot disease ( Figure ). Conclusions: Our findings suggest outpatient diabetic foot disease is primarily associated with a short-term drop in self-rated health, but inpatient diabetic foot disease is associated with long-term declines in self-rated health similar to those experienced after MI. These results reinforce the importance of early diagnosis and treatment of foot disease and of including patient-reported outcomes to measure sequelae of diabetes complications.

Timing of surgery in acute deep partial-thickness burns: A study protocol.

For deep partial-thickness burns no consensus on the optimal treatment has been reached due to conflicting study outcomes with low quality evidence. Treatment options in high- and middle-income countries include conservative treatment with delayed excision and grafting if needed; and early excision and grafting. The majority of timing of surgery studies focus on survival rather than on quality of life. This study protocol describes a study that aims to compare long-term scar quality, clinical outcomes, and patient-reported outcomes between the treatment options. A multicentre prospective study will be conducted in the three Dutch burn centres (Rotterdam, Beverwijk, and Groningen). All adult patients with acute deep-partial thickness burns, based on healing potential with Laser Doppler Imaging, are eligible for inclusion. During a nine-month baseline period, standard practice will be monitored. This includes conservative treatment with dressings and topical agents, and excision and grafting of residual defects if needed 14-21 days post-burn. The subsequent nine months, early surgery is advocated, involving excision and grafting in the first week to ten days post-burn. The primary outcome compared between the two groups is long-term scar quality assessed by the Patient and Observer Scar Assessment Scale 3.0 twelve months after discharge. Secondary outcomes include clinical outcomes and patient-reported outcomes like quality of life and return to work. The aim of the study is to assess long-term scar quality in deep partial-thickness burns after conservative treatment with delayed excision and grafting if needed, compared to early excision and grafting. Adding to the ongoing debate on the optimal treatment of these burns. The broad range of studied outcomes will be used for the development of a decision aid for deep partial-thickness burns, to fully inform patients at the point of consent to surgery and support optimal person-centred care.

Six-Year, Real-World Use of Prophylaxis with Recombinant Factor IX-Albumin Fusion Protein (rIX-FP) in Persons with Hemophilia B: A Single-Center Retrospective-Prospective Study.

Background: Extended half-life (EHL) factor IX (FIX) concentrates allow for prophylaxis with prolonged dosing intervals and high bleeding protection in persons with hemophilia B. Long-term real-world studies are lacking. Methods: In a retrospective-prospective study, the six-year use of prophylaxis with the EHL recombinant FIX-albumin fusion protein (rIX-FP) was analyzed, comparing outcomes with previous standard half-life (SHL) FIX in patients already on prophylaxis. Results: Prophylaxis with rIX-FP was prescribed in 15 patients (10 severe, 5 moderate; follow-up: 57 ± 17 months). Based on a pharmacokinetic assessment and clinical needs, the first regimen was 47 ± 7 IU/Kg every 9 ± 2 days. All but one patient remained on rIX-FP prophylaxis, adjusting infusion frequency and/or dose; the last prescribed frequency was ≥10 days in 10/13 patients, being reduced in seven and increased in four vs. the first regimen. The weekly FIX dose was unchanged; FIX trough levels were >5% in all patients. The annual infusion number and FIX IU/Kg significantly decreased (~60%) in eight patients previously on SHL FIX prophylaxis, with similar concentrate costs. Very low bleeding rates (most traumatic bleeds and the last quartile of the infusion interval), improved orthopedic and pain scores, unchanged HEAD-US scores and problem joints, and high treatment adherence (>90%) and satisfaction were registered. Conclusions: Personalized, carefully adjusted rIX-FP regimens contribute to the diffusion and optimization of prophylaxis in persons with severe and moderate hemophilia B, with long-term favorable bleeding, joint, and patient-reported outcomes.

Association of 5-Year Hip Arthroscopy Outcomes with Hip Morphology and Cartilage Status at Time of Surgery: A National Registry Study With HAGOS Outcomes in 281 Patients With Femoroacetabular Impingement Syndrome.

The effects of specific bony hip morphologies, cam and dysplasia, and cartilage damage on mid- and long-term (≥5 years) patient-reported outcomes (PROs) are understudied. To investigate if changes in PROs from preoperatively to 5 years after hip arthroscopy are associated with preoperative bony hip morphology and cartilage status in patients with femoroacetabular impingement syndrome. Cohort study; Level of evidence, 3. Patients were identified in the Danish Hip Arthroscopy Registry. Hip and groin function was assessed from preoperatively to 5 years postoperatively with the Copenhagen Hip and Groin Outcome Score (HAGOS) Activities of Daily Living (ADL) and Sports and Recreation (Sport) subscales. Morphology was defined using the anterior alpha angle (AA) and lateral center-edge angle (LCEA) as follows: mild to moderate cam (55°≤ AA < 78°), severe cam (AA ≥ 78°), pincer (LCEA > 39°), and borderline dysplasia (20°≤ LCEA < 25°). Joint space width (JSW) was defined as slightly reduced (3.1 mm ≤ JSW ≤ 4 mm) or severely reduced (2.1 mm ≤ JSW ≤ 3 mm). Acetabular cartilage status was defined by modified Beck grades 0 to 4 and femoral head cartilage status by International Cartilage Regeneration & Joint Preservation Society grades 0 to 4. Acetabular and femoral cartilage injury areas were categorized as <1, 1 to 2, or >2 cm2. Multiple regression analyses assessed adjusted associations between hip morphology and cartilage injuries with improvement in HAGOS-ADL and HAGOS-Sport. The study included 281 patients (age, 35 ± 10 years; 52.3% female). No cam and mild-to-moderate cam were associated with greater improvement in HAGOS-ADL (16 points [P = .002] and 7 points [P = .038], respectively) compared with severe cam. Normal JSW was associated with greater improvement in HAGOS-ADL (21 points; P = .026) compared with severely reduced JSW. Femoral head cartilage injury area <1 cm2 was associated with greater improvements in HAGOS-ADL (17 points; P = .03) and HAGOS-Sport (21 points; P = .035) compared with femoral cartilage injury area >2 cm2. Patients having no-to-moderate cam morphology, normal JSW, or femoral head cartilage injury area <1 cm2 had greater improvement in PROs 5 years after hip arthroscopy compared with patients having severe cam morphology, severely reduced JSW, or femoral cartilage injury area >2 cm2.

Round ligament management during minimally invasive groin hernia repair in women: a systematic review and meta-analysis.

Female sex has been associated with worse outcomes after groin hernia repair (GHR), including a higher rate of chronic pain and recurrence. Most of the studies in GHR are performed in males, and the recommendations for females extrapolate from these studies, even though females have anatomy intricacies. The round ligament of the uterus (RLU) is associated with pelvic stabilization and plays a role in sensory function. Transection of the RLU during GHR is controversial as it can allow easier mesh placement but can favor genitourinary complications and chronic pain. As no previous meta-analysis compared preserving versus transecting the RLU during minimally invasive (MIS) GHR, we aim to perform a systematic review and meta-analysis evaluating surgical outcomes comparing the approaches. Cochrane Central, Embase, and PubMed databases were systematically searched for studies comparing transection versus preservation of the RLU in MIS groin hernia surgeries. Outcomes assessed were operative time, bleeding, surgical site events, hospital stay, chronic pain, paresthesia, recurrence rates, and genital prolapse rates. Statistical analysis was performed using RevMan 5.4.1. Heterogeneity was assessed with I2 statistics. A review protocol for this meta-analysis was registered at PROSPERO (CRD 42023467146). 1738 studies were screened. A total of six studies, comprising 1131 women, were included, of whom 652 (57.6%) had preservation of the RLU during MIS groin hernia repair. We found no statistical difference regarding chronic pain, paresthesia, recurrence rates, and postoperative complications. We found a longer operative time for the preservation group (MD 6.84min; 95% CI 3.0-10.68; P = 0.0005; I2 = 74%). Transecting the RLU reduces the operative time during MIS GHR with no difference regarding postoperative complication rates. Although transection appears safe, further prospective randomized studies with long-term follow-up and patient-reported outcomes are necessary to define the optimal management of RLU during MIS GHR.

Patient-Reported Outcomes and Mortality in Cutaneous Chronic Graft-vs-Host Disease

Chronic graft-vs-host disease (GVHD) is associated with impaired quality of life and symptom burden. The independent association of skin involvement with patient-reported outcomes (PROs) and their utility as a clinical prognostic marker remain unknown. Identification of patients with cutaneous chronic GVHD and impaired PROs could assist in initial risk stratification and treatment selection. To compare the association of sclerotic and epidermal-type chronic GVHD with longitudinal PROs and to evaluate whether PROs can identify patients with cutaneous chronic GVHD at high risk for death. This multicenter prospective cohort study involved patients from the Chronic GVHD Consortium of 9 US medical centers, enrolled between August 2007 and April 2012, and followed up until December 2020. Participants included adults 18 years and older with a diagnosis of chronic GVHD requiring systemic immunosuppression and with skin involvement during the study period. Patient-reported symptom burden was assessed using the Lee Symptom Scale (LSS) skin subscale with higher scores indicating worse outcomes. Quality of life was measured using the Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT) instrument with lower scores indicating worse outcomes. Nonrelapse mortality, overall survival, and their association with PROs at diagnosis were also assessed. Among 436 patients with cutaneous chronic GVHD (median [IQR] age at transplant, 51 [41.5-56.6] years; 261 [59.9%] male), 229 patients had epidermal-type chronic GVHD (52.5%), followed by 131 with sclerotic chronic GVHD (30.0%), and 76 with combination disease (17.4%). After adjusting for confounders, patients with sclerotic chronic GVHD had mean FACT-BMT scores 6.1 points worse than those with epidermal disease (95% CI, 11.7-0.4; P = .04). Patients with combination disease had mean LSS skin subscale scores 9.0 points worse than those with epidermal disease (95% CI, 4.2-13.8; P < .001). Clinically meaningful differences were defined as at least 7 points lower for FACT-BMT and 11 points higher for LSS skin subscale. At diagnosis, clinically meaningful worsening in FACT-BMT score was associated with an adjusted odds of nonrelapse mortality increased by 9.1% (95% CI, 2.0%-16.7%; P = .01). Similarly, for clinically meaningful worsening in LSS skin subscale score, adjusted odds of nonrelapse mortality increased by 16.4% (95% CI, 5.4%-28.5%; P = .003). These associations held true after adjusting for clinical severity by the National Institutes of Health Skin Score. The results of this cohort study demonstrated that skin chronic GVHD was independently associated with long-term PRO impairment, with sclerotic and combination disease carrying the highest morbidity. The degree of impairment at skin chronic GVHD diagnosis was a prognostic marker for mortality. Therefore, PROs could be useful for risk stratification and treatment selection in clinical practice and clinical trials.

Evaluation of the long-term patient-reported outcomes after hip arthroplasty or joint preserving with Sugioka femoral osteotomy in patients with femoral head osteonecrosis

PurposeThis study retrospectively evaluated long-term clinical outcomes and patient-reported outcome measures (PROMs) in patients with osteonecrosis of the femoral head (ONFH) who underwent transtrochanteric rotational osteotomy (TRO), curved varus osteotomy (CVO), and total hip arthroplasty (THA).MethodsWe retrospectively reviewed the 109 hips in 96 patients (46 men, 50 women) who underwent CVO, TRO, or THA for ONFH treatment. The mean follow-up period for the TRO, CVO, and THA groups was 14.8, 11.5, and 13.3 years, respectively.ResultsThe THA conversion rate of the TRO patients was significantly higher than that of the patients with CVO, and the final clinical scores in the patients with TRO did not improve compared with preoperative scores. Postoperative PROMs showed that the total and pain scores of the patients with THA were significantly higher than those of patients with TRO and CVO, while the PROM score did not change between patients with TRO and CVO. The analysis further showed that the preoperative type C2, stage 3A, or postoperative type C1 and C2 were significant predictors of decreased final PROM scores.ConclusionThis study found that CVO and THA are clinically effective treatments for ONFH, with significant improvements compared with preoperative scores. However, THA was associated with significantly higher PROMs and pain scores than those of CVO and TRO in long-term follow-up. Furthermore, our results suggest that postoperative PROMs depend mainly on the preoperative level of collapse and postoperative transposed intact ratio of the articular surface of the femoral head.

The Insertion of a Subacromial Balloon Spacer Can Provide Symptom Relief and Functional Improvement at a Minimum 5-Year Follow-Up in Patients With Massive Irreparable Rotator Cuff Tears

To examine long-term patient-reported outcomes and range of motion in patients with massive irreparable rotator cuff tears (MIRCTs) who underwent subacromial balloon spacer implantation. A retrospective review of all patients who underwent subacromial balloon placement procedure for MIRCTs at our institution was conducted. Patients with adequate preoperative and postoperative data, with at least 5 years of follow-up, were included in our study. Outcome measures were range of motion (forward elevation), American Shoulder and Elbow Surgeon (ASES) score, and visual analog scale (VAS) score. Independent t test was conducted to check for statistically significant differences between preoperative and postoperative outcome scores, with P < .05 deemed significant. Ten patients were identified: 4were lost to follow-up beyond 2 years and were excluded. One was converted to an arthroplasty at the 1-year mark and was then lost to follow-up (conversion rate: 16.6%). Five patients had at least 5 years of follow-up after the balloon procedure and were involved in our case series analysis. Mean age was 63.1 years, and mean follow-up was 5.8 years (range, 5-7 years). Preoperatively, mean forward elevation was 110 degrees, mean ASES score was 40.68, and mean VAS score was 6.2. On follow-up, mean forward elevation was 163 degrees (P= .007), mean ASES score was 90.97 (P= .001), and mean VAS score was 0.9 (P= .004). All patients showed significant improvements in all outcome measures, and none had any significant complications. In this study, we found that the use of a subacromial balloon spacer can lead to good outcomes at a minimum 5-year follow-up in patients with MIRCTs. Level IV, therapeutic case series.

Avelumab first-line maintenance (1LM) for advanced urothelial carcinoma (aUC): Long-term patient-reported outcomes (PROs) in the phase 3 JAVELIN Bladder 100 trial.

Avelumab first-line maintenance (1LM) for advanced urothelial carcinoma (aUC): Long-term patient-reported outcomes (PROs) in the phase 3 JAVELIN Bladder 100 trial.