BACKGROUND: We evaluated the impact of gender on disease severity, health-related quality of life (HRQoL), treatment management, and patient-healthcare professional (HCP) interactions from the perspectives of patients with psoriatic arthritis (PsA).
METHODS: Data were collected from a global online patient survey conducted by The Harris Poll (November 2, 2017 to March 12, 2018). Eligible patients were aged ≥ 18 years, with a self-reported diagnosis of PsA for > 1 year, had visited a rheumatologist/dermatologist in the past 12 months, and had reported previously using ≥ 1 conventional synthetic or biologic disease-modifying antirheumatic drug. Data were stratified by gender and analyzed descriptively, inferentially by binomial (chi-square) tests, and by multivariate logistic regression models.
RESULTS: Data from 1286 patients who participated were included: 52% were female, 48% were male. Varying perceptions of disease severity between males and females were indicated by differences in symptoms leading to a diagnosis of PsA, and in symptoms reported despite treatment; more females than males reported joint tenderness, skin patches/plaques, and enthesitis. More females than males reported a major/moderate impact of PsA on their physical activity and emotional/mental well-being. Reasons for switching medication differed between genders, with more females switching because they perceived their medication to not be effective enough related to their joint symptoms. More females than males were very satisfied with their communication with their rheumatologist and were more likely to discuss the impact of PsA on their daily lives, their treatment satisfaction, and treatment goals with their rheumatologist.
CONCLUSIONS: Patients\' perceptions of the impact of PsA on HRQoL, treatment management, and interactions with HCPs varied between males and females. More females than males reported major/moderate physical and emotional impacts of PsA. When treating patients, it is important for HCPs to consider the potential impact of gender on patients\' experience of PsA and its symptoms. Graphical plain language summary available for this article.

Variants in the retinitis pigmentosa GTPase regulator (RPGR) gene are responsible for the majority of X-linked retinitis pigmentosa cases, which not only affects male patients but also some heterozygous females. Vision-related disability and anxiety of patients with RPGR-associated retinal degeneration have never been explored before. This study aimed to evaluate self-reported visual function and vision-related anxiety in a Portuguese cohort of male and female patients with RPGR-associated retinal degeneration using two validated patient-reported outcome measures. Cross-sectional data of thirty-two genetically-tested patients was examined, including scores of the Michigan retinal degeneration questionnaire (MRDQ) and Michigan vision-related anxiety questionnaire. Patients were classified according to retinal phenotypes in males (M), females with male phenotype (FM), and females with radial or focal pattern. Both M and FM revealed higher rod-function and cone-function anxiety scores (p < 0.017). Most MRDQ disability scores were higher in M and FM (p < 0.004). Overall, positive correlations (p < 0.004) were found between every MRDQ domain and both anxiety scores. In RPGR-associated retinal degeneration, males and females with male phenotype show similar levels of increased vision-related anxiety and disability. Every MRDQ visual function domain showed a strong correlation with anxiety scores.

UNASSIGNED: Over the last 3 decades, hematopoietic stem cell transplantation (HSCT) has been successfully used to treat severe and refractory autoimmune diseases (AIDs). A multidisciplinary appraisal of potential benefits and risks by disease and transplant specialists is essential to determine individual suitability for HSCT.
UNASSIGNED: Our aim was to observe that patient-reported outcomes (PROs) and health-related quality of life instruments can capture the unique patient perspective on disease burden and impact of treatment.
UNASSIGNED: Herein, we describe the basis and complexity of end points measuring patient-reported perceptions of efficacy and tolerability used in clinical practice and trials for patients with AIDs undergoing autologous HSCT.
UNASSIGNED: PRO measures and patient-reported experience measures are key tools to evaluate the impact and extent of disease burden for patients affected by AIDs. For formal scientific assessment, it is essential that validated general instruments are used, whereas adaptations have resulted in disease-specific instruments that may help guide tailored interventions. An additional approach relates to qualitative evaluations, from carefully structured qualitative research to informal narratives, as patient stories. The patients\' subjectively reported responses to HSCT may be influenced by their preprocedure expectations and investment in the HSCT journey.
UNASSIGNED: The complexity of AIDs advocates for individualized and multidisciplinary approach to positively affect the patient journey. PROs and health-related quality of life need to be collected using validated instruments in clinical practice and trials to enable robustness of data and to ensure the impact of the intervention is comprehensively assessed, addressing the main questions and needs of the involved stakeholders.

OBJECTIVE: X-linked hypophosphatemic rickets (XLH) is due to loss-of-function mutations in the phosphate-regulating endopeptidase homologue on the X chromosome (PHEX) that lead to increased fibroblast growth factor 23 (FGF23) production. FGF23 excess causes renal phosphate wasting and insufficient 1,25-dihydroxyvitamin D (1,25(OH)2D) synthesis with reduced intestinal phosphate absorption, ultimately resulting in chronic hypophosphatemia. Children with XLH show typical skeletal lesions of rickets, deformities of the lower limbs, stunted growth with disproportionate short stature, bone pain, and physical dysfunctions. Burosumab, a fully human IgG1 monoclonal antibody that binds to FGF23 to inhibit its activity, is more effective to improve the biochemical and clinical signs of XLH than conventional treatment with phosphate supplements and vitamin D active metabolites. Data on adolescents with XLH during the transition period to young adulthood are few. In this prospective case series, we aimed to assess safety and efficacy of burosumab in adolescents with XLH who discontinued long-term conventional therapy.
METHODS: Five Caucasian adolescents (4 males, 1 female; mean age 15.4 ± 1.5 years) with XLH were recruited and switched from conventional treatment to burosumab (0.8 to 1.2 mg/kg, s.c. QW2). Burosumab was continued for 12 to 48 months and, once discontinued, patients were followed-up for 6 to 12 months. In all patients, serum calcium, phosphate, alkaline phosphatase (ALP), parathyroid hormone (PTH), and 1,25(OH)2D levels, and renal tubular reabsorption of phosphate (TmP/GFR) values were assessed at entry and during burosumab. Intact FGF23 plasma levels were measured at entry. Patient-reported outcomes (PROs) were assessed at entry and every 3-6 months to evaluate the impact of low extremity pain, stiffness, and difficulties performing daily activities.
RESULTS: At entry, all patients showed hypophosphatemia, increased intact FGF23 levels, reduced TmP/GFR, insufficient 1,25(OH)2D levels, and in four out of five increased ALP levels. Two patients had radiological signs of rickets. During burosumab, all patients showed a significant increase in serum phosphate and 1,25(OH)2D levels, and in TmP/GFR values (P <0.05 - P<0.0001). Serum ALP levels significantly declined (P <0.05) to normal values. No changes of serum calcium and PTH levels (P = NS) were found during burosumab. PROs significantly improved (P<0.02 - P<0.0001) in all patients. Four patients discontinued burosumab when they turned 18 or 19, whereas one continued the treatment since he was still younger than 18 during the study period. Four patients who suspended burosumab showed a rapid decline in serum phosphate and 1,25(OH)2D levels and in TmP/GFR values; serum ALP levels increased, and PROs progressively worsened with a significant reduction in quality of life. These consequences were not observed in the patient who continued burosumab treatment.
CONCLUSIONS: Our data showed that conventional treatment improved only in part the signs and symptoms of XLH. Burosumab was well tolerated and was effective in improving phosphate metabolism, bone health, and PROs. All the benefits of burosumab were lost after its discontinuation. These results suggested that continuing burosumab is required to achieve and maintain the clinical benefits of the treatment during the transition to young adulthood in patients with XLH.

BACKGROUND: Breast augmentation ranks among the most popular plastic surgery procedures. Yet, reports on post-operative patient-reported quality of life (QoL) and satisfaction remain conflicting.
METHODS: A systematic review was conducted following the PRISMA guidelines. Three databases were searched for eligible studies that reported pre-and/or post-operative Breast-Q™ augmentation scores for patient QoL (psychosocial, sexual, and physical well-being) and/or satisfaction.
RESULTS: A total of 39 studies (53 patient cohorts and 18,322 patients) were included in the quantitative synthesis. The pairwise meta-analysis revealed significant improvements in patient-reported psychosocial (MD: +38.10) and sexual well-being (MD: +40.20) as well as satisfaction with breast (MD: +47.88) (all p < 0.00001). Physical well-being improved slightly after breast augmentation (MD: +6.97; p = 0.42). The single-arm meta-analysis yielded comparable results, with Breast-Q™ scores in psychosocial and sexual well-being as well as satisfaction with breast increasing from 37.2, 31.1, and 26.3 to 75.0, 70.6, and 72.7, respectively (all p < 0.00001). Physical well-being improved by 8.1 (75.8 pre-operatively to 83.9 post-operatively; p = 0.17). Subgroup analyses highlighted higher QoL and satisfaction following breast augmentation for purely esthetic purposes and alloplastic mammaplasty. Although patient-reported physical and sexual well-being increased in the long term, psychosocial well-being was the highest in the short term.
CONCLUSIONS: Patient satisfaction with breast, psychosocial, and sexual well-being increased significantly after breast augmentation. In contrast, patient-reported physical well-being yielded ambivalent results, varying by mammaplasty technique and post-operative follow-up time. Plastic surgeons should be sensitized about our findings to refine eligibility criteria and gain a deeper understanding of the patients\' perceived surgical experience.
UNASSIGNED: CRD42023409605.

BACKGROUND: Total knee arthroplasty (TKA) is performed on approximately 790,000 patients annually in the United States and is projected to increase to 1.5 million by 2050. This study aimed at assessing the use of preoperative cryoneurolysis on patients undergoing TKA by analyzing: 1) pain severity; 2) opioid use; 3) functional status; and 4) sleep disturbance over 6 months following discharge.
METHODS: Patients enrolled in the Innovations in Genicular Outcomes Registry (iGOR) between September 2021 and February 2024 were followed for 6 months. Our analyses included patients undergoing unilateral primary TKA with no pre-operative opioid prescription who either received, or did not receive, cryoneurolysis. Baseline patient demographics were collected before TKA and tabulated. Pain management was assessed via the Brief Pain Inventory-Short Form (BPI-SF) instrument for pain severity. Sleep disturbance was measured using the Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire. Each outcome measure was assessed prior to TKA, weekly, and at monthly follow-up. Data was analyzed by a generalized linear mixed-effect regression model to compare cryoneurolysis versus control patients, with a P < 0.05 as significant.
RESULTS: There were 80 patients who were treated with preoperative cryoneurolysis, while 60 control patients did not have treatment. Patients receiving cryoneurolysis experienced significantly lower pain severity and sleep disturbance over the 6-month follow-up than control patients (P = 0.046). Cryoneurolysis was also associated with a trend toward greater functional improvement that did not reach statistical significance (P = 0.061). Further, patients who underwent cryoneurolysis were 72% less likely than controls to take opioids over six months following discharge (P <0.001).
CONCLUSIONS: Pre-operative cryoneurolysis therapy in opioid-naïve patients undergoing TKA is associated with improved pain, decreased opioid use, and improved sleep disturbance for 6 months postoperatively. Cryoneurolysis, a non-opioid pain relief modality administered pre-operatively, demonstrated substantial benefits in patients who underwent TKA.

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Conventionally, peripheral nerve stimulation (PNS) for treatment of chronic pain has involved a two-stage process: a short-term (e.g., 7 days) trial and, if significant pain relief is achieved, a permanent PNS system is implanted. A percutaneous PNS treatment is now available where a coiled lead may be implanted for up to 60 days with the goal of producing sustained relief. In the present review, published prospective trials using percutaneous PNS treatment were identified and synthesized. The collected evidence indicates that percutaneous PNS treatment for up to 60 days provides durable clinically significant improvements in pain and pain interference. Similar efficacy across diverse targets and etiologies supports the broad applicability for use within the chronic pain population using this nonopioid technology.
What is this review about? This review looks at a drug-free way to treat chronic pain called percutaneous peripheral nerve stimulation (PNS). Percutaneous means it is placed through the skin. PNS applies small amounts of electricity to the nerves to reduce chronic pain. Most PNS systems involve a two-step process. A short trial is first performed to see if a patient has pain relief. A permanent system is then placed if the person had pain relief. Percutaneous PNS treatments are different. They use a thin wire called a lead placed in the body for up to 60 days. The lead is taken out at the end of the treatment period. Studies have shown that this type of PNS treatment can reduce chronic pain even after the treatment is over. No previous article has collected all these studies of percutaneous PNS in one place.What evidence was gathered? This review found evidence from studies on treatment of chronic pain. Pain types included shoulder pain, neuropathic pain and low back pain. It found that percutaneous PNS treatment for up to 60 days can reduce pain and how pain interferes with daily life.How can these data lead to better care for patients? These findings mean that percutaneous PNS treatments could be a useful, non-drug option for many types of chronic pain.

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OBJECTIVE: Symptom assessment based on patient-reported outcome (PRO) can correlate with disease severity, making it a potential tool for threshold alerts of postoperative complications. This study aimed to determine whether shortness of breath (SOB) scores on the day of discharge could predict the development of post-discharge complications in patients who underwent lung cancer surgery.
METHODS: Patients were from a study of a dynamic perioperative rehabilitation cohort of lung cancer patients focusing on patient-reported outcomes. Patients were assessed using the Perioperative Symptom Assessment Scale for Lung surgery (PSA-Lung). Logistic regression model was used to examine the potential association between SOB on the day of discharge and complications within 3 months after discharge. The post-discharge complications were taken as the anchor variable to determine the optimal cutpoint for SOB on the day of discharge.
RESULTS: Complications within 3 months post-discharge occurred in 71 (10.84%) of 655 patients. Logistic regression analysis revealed that being female (OR 1.764, 95% CI 1.006-3.092, P < 0.05) and having two chest tubes (OR 2.026, 95% CI 1.107-3.710, P < 0.05) were significantly associated with post-discharge complications. Additionally, the SOB score on the day of discharge (OR 1.125, 95% CI 1.012-1.250, P < 0.05) was a significant predictor. The optimal SOB cutpoint was 5 (on a scale of 0-10). Patients with an SOB score ≥ 5 at discharge experienced a lower quality of life 1 month later compared to those with SOB score<5 at discharge (73 [50-86] vs. 81 [65-91], P < 0.05).
CONCLUSIONS: SOB on the day of discharge may serve as an early warning sign for the timely detection of 3 month post-discharge complications.