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Plasma-derived von Willebrand factor-containing factor VIII concentrates (pd-VWF/FVIII-C) are the mainstay of treatment in von Willebrand disease (VWD). Real-world data on efficacy and safety of these pd-VWF/FVIII-C are required. To retrospectively evaluate the efficacy and safety of pd-VWF/FVIII-C (Fanhdi® and Alphanate®, Grifols) in clinical practice in Italy. A multicentric, observational, retrospective study at 10 Italian centers was conducted. Eligible patients diagnosed with inherited VWD (ISTH criteria) were treated with either Fanhdi® or Alphanate® for bleeding episodes, prevention of surgical bleeding and secondary long-term prophylaxis (SLTP) according to clinical practice with medical records collected from January 2007 to December 2019. Efficacy/safety of pd-VWF/FVIII-C was assessed according to FDA-agreed objective criteria following regulatory procedures. Fifty-seven patients (M/F: 21/36) were enrolled in the study with the following VWD types: VWD1 (n = 29, 52%), VWD2A (n = 10, 18%), VWD2B (n = 7, 12%), VWD2M (n = 2, 4%), VWD2N (n = 1, 2%), VWD2 unclassified (n = 1, 2%), and VWD3 (n = 7, 12%). These pd-VWF/FVIII-C were used to manage 58 bleeding episodes (n = 24 patients), 100 surgeries (n = 47 patients), and 7 SLTP (n = 6 patients). Global clinical efficacy with these pd-VWF/FVIII-C was reported to be excellent/good in 85% of bleeding episodes, 98% of surgeries, and 100% of SLTP. As far as safety, no adverse-drug-related episodes, immunogenic or thrombotic events were reported. This study confirmed that Fanhdi® and Alphanate® were effective and safe in the management of bleeding episodes, the prevention of bleeding during surgeries and for SLTP in Italian patients with inherited VWD. [ABSTRACT FROM AUTHOR]

Pain is an unpleasant experience that we will all experience in some form over the course of our lives, with chronic pain affecting a significant proportion of the global population. Given these circumstances, this study investigates whether pain is a legitimated phenomenon and considers the processes involved in the creation of such a status. This is an exploratory investigation based on semi-structured interviews with people suffering from chronic pain as a consequence of physical, psychological, emotional, or social circumstances. Our principal objective is to explore the fundamental elements of legitimacy and the processes that bring it into being—i.e., to understand how it is socially constructed. The main finding, however, is that many sufferers of chronic physical, psychiatric/psychological, emotional and social pain perceive that their pain is not considered legitimate when no clear cause can be identified, when the pain prevents them from developing the norms imposed by social roles or when it inhibits them to make a productive contribution to the society in which they live. This is generally due to the disruptive aspect of pain and its impact on the social structure, specifically on productivity, which nowadays constitutes a key element in the legitimation of any social phenomenon. [ABSTRACT FROM AUTHOR]

Background: Post-traumatic growth is a positive psychological change that may aid recovery in individuals experiencing trauma. Owing to the lack of research in the area of parental care for premature infants, we decided to explore the levels and factors influencing post-traumatic growth among parents of premature infants in neonatal intensive care units. We believe that these findings will help reassess existing care practices so that healthcare providers can promptly identify negative emotions and take necessary measures to help develop the potential to enhance post-traumatic growth. Methods: A cross-sectional survey was conducted using convenience sampling between February and September 2022. Data were analysed using independent sample t-tests and one-way analysis of variance (ANOVA). Bivariate correlations were analysed using the Pearson's or Spearman's method, and related factors were analysed using multiple linear regression. We followed the SRQR checklist throughout the study period. Results: A total of 217 patients were effectively treated, with a recovery rate of 98.64%. Univariate analysis showed that the length of hospital stay, presence of only one child, parents' age, marital status, education level, working status, and per capita monthly familial income were influencing factors. Bivariate analysis showed that post-traumatic growth was moderately and positively correlated with perceived social support, rumination, and family resilience. Multiple linear regression showed that purposeful contemplation, family resilience, education, family support, age, and marital status entered into the regression equation and together accounted for 47.4% of the total variation. Conclusions: It is necessary to pay attention to post-traumatic growth and familial stability in these families, provide aid in building a good support system, and encourage parents to mobilise their family and favourable factors to increase post-traumatic growth levels. [ABSTRACT FROM AUTHOR]

Background: Recombinant von Willebrand factor (rVWF, vonicog alfa, Vonvendi/Veyvondi, Takeda Pharmaceuticals USA, Lexington, MA) and several plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrates are available for treating bleeding episodes in patients with von Willebrand disease (VWD). Purpose: To develop population pharmacokinetic (PK)/pharmacodynamic (PD) models that describe VWF:ristocetin cofactor (VWF:RCo) activity and its relationship with FVIII activity (FVIII:C) over time following intravenous administration of either rVWF or a pdVWF/FVIII concentrate (VWF:RCo/FVIII:C 2.4:1) in patients with VWD; to use the final PK/PD models for an in silico comparison of rVWF and pdVWF/FVIII. Methods: The population PK model for rVWF was based on data from four clinical studies in which rVWF was administered to adult patients with VWD type 1, 2 or 3 (phase 1: NCT00816660; phase 3: NCT01410227 and NCT02283268) or severe hemophilia A (phase 1: EudraCT 2011– 004314-42). The PK and PK/PD models for pdVWF/FVIII were based on data from the phase 1 study (NCT00816660) in patients with type 3 VWD who received either rVWF plus recombinant FVIII (rFVIII, octocog alfa, ADVATE®, Takeda Pharmaceuticals USA, Lexington, MA, USA) or pdVWF/FVIII. Results: There was a marked difference in clearance following rVWF administration compared with pdVWF/FVIII in type 3 VWD, leading to a ~1.75 longer mean residence time (ie, persistence of VWF:RCo activity in the body) and half-life for rVWF versus pdVWF/FVIII. Simulations showed that following repeated administration of rVWF (50 IU/kg), a FVIII:C activity of > 40 IU/dL can be maintained for the full 72 h dosing interval. Conclusion: The slower elimination of VWF:RCo following rVWF administration results in a prolonged effect on FVIII turnover compared with pdVWF/FVIII administration. [ABSTRACT FROM AUTHOR]

Objective: To evaluate the efficacy and safety of a plasma-derived factor VIII concentrate containing von Willebrand Factor (pdVWF/FVIII) in standard clinical practice in von Willebrand Disease (VWD) patients. Methods: A retrospective, multicentric, observational study of VWD patients treated with Fanhdi®, a pdVWF/FVIII concentrate, from January 2011 to December 2017 was conducted at 14 centers in Spain. Efficacy and safety were evaluated for acute bleeding episodes, for prevention of bleeding in surgeries, and for secondary long-term prophylaxis. Results: Seventy-two eligible patients, type 1, 2, 3 VWD (25%/38.9%/36.1%) were treated for spontaneous and traumatic bleeding (140 episodes, n = 41 patients), to prevent surgical bleeding (69 episodes, n = 43 patients); and for secondary long-term prophylaxis (18 programs, n = 13 patients). Replacement therapy with pdVWF/FVIII showed an excellent to good clinical efficacy in 96.7% of the bleeding episodes, 100% during surgical procedures and 100% during prophylaxis. No adverse events (AEs), nor serious AEs related to the product were observed. Conclusions: Fanhdi® was effective, safe and well tolerated in the management of bleeding episodes, the prevention of bleeding during surgeries, and for secondary long-term prophylaxis in VWD patients. [ABSTRACT FROM AUTHOR]

Background: Multi-sensory behavioral interventions for preterm infants have the potential to accelerate feeding, growth, and optimize developmental trajectories and increase parents' interactive engagement with their infants. However, few neonatal intensive care units (NICUs) provide evidence-based standardized early behavioral interventions as routine care. Lack of implementation is a major gap between research and clinical practice. H-HOPE, is a standardized behavioral intervention with an infant- directed component (Massage+) and a parent-directed component (four participatory guidance sessions that focus on preterm infants' behaviors and appropriate responses). H-HOPE has well documented efficacy. The purpose of this implementation study is to establish H-HOPE as the standard of care in 5 NICUs.Methods: The study employs a Type 3 Hybrid design to simultaneously examine the implementation process and effectiveness in five NICUs. To stagger implementation across the clinical sites, we use an incomplete stepped wedge design. The five participating NICUs were purposively selected to represent different acuity levels, number of beds, locations and populations served. Our implementation strategy integrates our experience conducting H-HOPE and a well-established implementation model, the Consolidated Framework for Implementation Research (CFIR). The CFIR identifies influences (facilitators and barriers) that affect successful implementation within five domains: intervention characteristics, outer setting (the hospital and external events and stakeholders), inner setting (NICU), implementers' individual characteristics, and the implementation process. NICUs will use the CFIR process, which includes three phases: Planning and Engaging, Executing, and Reflecting and Evaluating. Because sustaining is a critical goal of implementation, we modify the CFIR implementation process by adding a final phase of Sustaining.Discussion: This study builds on the CFIR, adding Sustaining H-HOPE to observe what happens when sites begin to maintain implementation without outside support, and extends its use to the NICU acute care setting. Our mixed methods analysis systematically identifies key facilitators and barriers of implementation success and effectiveness across the five domains of the CFIR. Long term benefits have not yet been studied but may include substantial health and developmental outcomes for infants, more optimal parent-child relationships, reduced stress and costs for families, and substantial indirect societal benefits including reduced health care and special education costs.Trial Registration: ClinicalTrials.gov registration number NCT04555590 , Registered on 8/19/2020. [ABSTRACT FROM AUTHOR]

Background: Regional variation in cost of neonatal intensive care for extremely preterm infant is not documented. We sought to evaluate regional variation that may lead to benchmarking and cost saving.Methods: An analysis of a Canadian national costing data from the payor perspective. We included all liveborn 23-28-week preterm infants in 2011-2015. We calculated variation in costs between provinces using non-parametric tests and a generalized linear model to evaluate cost variation after adjustment for gestational age, survival, and length of stay.Results: We analysed 6932 infant records. The median total cost for all infants was $66,668 (Inter-Quartile Range (IQR): $4920-$125,551). Medians for the regions varied more than two-fold and ranged from $48,144 in Ontario to $122,526 in Saskatchewan. Median cost for infants who survived the first 3 days of life was $91,000 (IQR: $56,500-$188,757). Median daily cost for all infants was $1940 (IQR: $1518-$2619). Regional variation was significant after adjusting for survival more than 3 days, length of stay, gestational age, and year (pseudo-R2 = 0.9, p < 0.01). Applying the model on the second lowest-cost region to the rest of the regions resulted in a total savings of $71,768,361(95%CI: $65,527,634-$81,129,451) over the 5-year period ($14,353,672 annually), or over 11% savings for the total program cost of $643,837,303 over the study period.Conclusion: Costs of neonatal intensive care are high. There is large regional variation that persists after adjustment for length of stay and survival. Our results can be used for benchmarking and as a target for focused cost optimization, savings, and investment in healthcare. [ABSTRACT FROM AUTHOR]

Objectives. We examined how different types of health information-seeking behaviors (HISBs) — no use, illness information only, wellness information only, and illness and wellness information combined — are associated with health risk factors and health indicators to determine possible motives for health information seeking. Methods. A sample of 559 Seattle-Tacoma area adults completed an Internet-based survey in summer 2006. The survey assessed types of HISB, physical and mental health indicators, health risks, and several covariates. Covariate-adjusted linear and logistic regression models were computed. Results. Almost half (49.4%) of the sample reported HISBs. Most HISBs (40.6%) involved seeking a combination of illness and wellness information, but both illness-only (28.6%) and wellness-only (30.8%) HISBs were also widespread. Wellness-only information seekers reported the most positive health assessments and the lowest occurrence of health risk factors. An opposite pattern emerged for illness-only information seekers. Conclusions. Our findings reveal a unique pattern of linkages between the type of health information sought (wellness, illness, and so on) and health self-assessment among adult Internet users in western Washington State. These associations suggest that distinct health motives may underlie HISB, a phenomenon frequently overlooked in previous research. [ABSTRACT FROM AUTHOR]

The purpose of this study was to assess the usefulness of a health information styles segmentation strategy in understanding audience subgroups. We examined the health information styles of men participating in a prostate cancer screening informed decision-making (IBM) intervention and assessed intervention effects on men with distinct health information styles. We classified participants into three health information style groups based on their independence in health decision making (independent versus doctor dependent) and engagement in health enhancement (active versus passive): independent active (IA), doctor-dependent active (DDA) and passive. We developed profiles of men in these groups: IAs tended to be white and more highly educated and to have greater baseline prostate cancer knowledge; DDAs were older, less healthy and more likely to have visited a doctor in the past year and passives tended to be younger, not to have had a recent prostate-specific antigen test and to have lower self-efficacy related to communication with doctors and less positive interactions with doctors. All groups significantly increased their prostate cancer knowledge after the intervention, but passives exhibited the greatest increase in knowledge at a 6-month follow-up. The health information styles segmentation strategy used in this study offers a viable framework for segmenting audiences. [ABSTRACT FROM AUTHOR]

Comments on the critical need for a comprehensive and integrated model to help care providers, patients, and consumers. Importance of better understanding of the health information, communication, and information seeking of patients and consumers; Capability of recognizing the signs and symptoms of disease.

Immune thrombocytopenic purpura as a presentation of childhood tuberculosis is a rare event and occasional reports are described in the pediatric literature. We describe a 8-yr-old girl with mediastinal tubercular lymphadenitis-induced immune thrombocytopenia, who was successfully treated with anti-tubercular drugs. We also review the published reports. Tuberculosis should be considered a cause of immune thrombocytopenia in areas where tuberculosis is highly endemic. [ABSTRACT FROM AUTHOR]

Trichosporon asahii (formerly Trichosporon beigelii)isan emerging fungal pathogen seen particularly in immunolog-ically compromised patients. There are now 100 reported cases of hematogenously disseminated infections with this life-threatening yeast, and no effective antifungal therapy is available. The present case is unusual because the patient did not have neutropenia or evidence of a malignancy. Extensive evaluation for the cause of thrombocytopenia, in-cluding bone marrow and peripheral smear examination, was unrewarding. In particular, the diagnosis of thrombotic throm-bocytopenic purpura could not be established. Renal failure of uncertain cause required hemodialysis that involved use of a vascular catheter in the groin. Total parenteral nutrition was initiated on hospital day 16. Toxic epidermal necrolysis developed. [ABSTRACT FROM AUTHOR]

Background: Gaining a better understanding of the probability, timing and prediction of rehospitalisation amongst preterm babies could help improve outcomes. There is limited research addressing these topics amongst extremely and very preterm babies. In this context, unplanned rehospitalisations constitute an important, potentially modifiable adverse event. We aimed to establish the probability, time-distribution and predictability of unplanned rehospitalisation within 30 days of discharge in a population of French preterm babies.Methods: This study used data from EPIPAGE 2, a population-based prospective study of French preterm babies. Only those babies discharged home alive and whose parents responded to the one-year survey were eligible for inclusion in our study. For Kaplan-Meier analysis, the outcome was unplanned rehospitalisation censored at 30 days. For predictive modelling, the outcome was binary, recording unplanned rehospitalisation within 30 days of discharge. Predictors included routine clinical variables selected based on expert opinion.Results: Of 3841 eligible babies, 350 (9.1, 95% CI 8.2-10.1) experienced an unplanned rehospitalisation within 30 days. The probability of rehospitalisation progressed at a consistent rate over the 30 days. There were significant differences in rehospitalisation probability by gestational age. The cross-validated performance of a ten predictor model demonstrated low discrimination and calibration. The area under the receiver operating characteristic curve was 0.62 (95% CI 0.59-0.65).Conclusions: Unplanned rehospitalisation within 30 days of discharge was infrequent and the probability of rehospitalisation progressed at a consistent rate. Lower gestational age increased the probability of rehospitalisation. Predictive models comprised of clinically important variables had limited predictive ability. [ABSTRACT FROM AUTHOR]