A pilot study of US HTC physical therapists' concordance of PT MASAC recommendations and educational needs.

INTRODUCTION: Physical therapists (PTs) are essential providers within the Hemophilia Treatment Centers (HTCs) team caring for persons with inherited blood disorders (PWBD). OBJECTIVE: Little is known regarding PTs understanding and concordance of MASAC PT Recommendations (MASAC#238), educational resources used to maintain competency and support for a mentorship programme. METHODS: PTs at federally funded HTCs were eligible to participate in a descriptive non-validated study exploring: (i) demographics, (ii) educational background, (iii) experience in evaluation and treatment of PWBD, (iv) practice patterns indicative of concordance with MASAC#238 and (v) opinion regarding PT mentorship. RESULTS: Respondents experience caring for PWBD ranged 1-36 years, treating both adults and children. Although most acknowledged awareness of MASAC#238, dropout (14/44, 31.8%) was noted; 28/30 (93.3%) who continued were aware of the recommendations. Level of concordance with MASAC#238 varied (range 64.3%-96.2%) regarding: signs/symptoms, treatment of muscle/joint bleeding and pre/post synovectomy and knee replacement treatment. Many PTs identified patients as individual and unique, thus not all recommendations may apply. PTs utilised available educational programmes. No relationships were noted regarding years of practice, education and years caring for PWBD. All respondents favoured a mentorship programme citing benefits, but also outlined barriers. CONCLUSIONS: Provision of necessary financial support for optimal function of a full-time PT within the HTC can enhance standards of care for PWBD. Supporting educational opportunities may enhance concordance with current MASAC PT Recommendations. Respondents valued development of a structured, hands-on mentorship programme. MASAC#23 has recently been updated in May 2023 to MASAC#275.

Cross-cultural assessment of the influence of the COVID-19 pandemic on the perceived mental health and medical experiences of persons with inherited bleeding disorders and their parents/guardians.

BACKGROUND: Objectives were to 1) assess COVID-19-associated medical and psychological challenges facing persons with inherited bleeding disorders (PIBD) and their parents/guardians (PG) in Germany, the US, and the UK; 2) describe similarities and differences among these countries; 3) identify needs and opportunities for intervention by patient advocacy organizations (PAGs). RESEARCH DESIGN & METHODS: A cross-sectional, international survey was conducted in three countries using validated psychometric instruments and investigator-developed items. RESULTS: Five hundred and four surveys were included. Significant differences between countries were found including experiences with medical care, specific thoughts, and concerns about COVID-19, anxiety, and other mental health measures, as well as resources used to cope with stress. Age, education, income, race, IBD diagnosis, PIBD vs. BD group, and gender had moderating effects on resources used. Communication with friends/relatives and use of PAG and HTCs as resources for information/coping decreased in all countries during the pandemic. CONCLUSIONS: There were similarities and differences between respondents across the country in the perceived impact of the pandemic, mental health scores, and strategies used to cope with stress.  Recommendations: strategies to increase PAG access for PIBD and their PG during pandemics and natural disasters, ongoing assessment and adaptation to provide supportive resources to specific patient subgroups.

Persons with inherited bleeding disorders (PIBD) and their parents faced many challenges during the COVID-19 pandemic. An online survey was conducted within three countries: Germany, the United States, and the United Kingdom to explore these challenges and address how patient advocacy organizations can better meet their needs. Areas explored included experiences with medical care, concerns, and thoughts expressed during the pandemic, and coping resources used before and during the pandemic. In addition, mental health issues were explored addressing anxiety, COVID-related fears, depression, and resilience. Differences were found regarding experiences with medical care, specific thoughts, and concerns about COVID-19, anxiety, and other mental health measures, as well as resources used to cope with stress. These results provide an opportunity for advocacy organizations for PIBD to develop appropriate assessment, adaptation, and education resources to help patients during pandemics and/or natural disasters in the future.

Bridging the gap: Survey highlights challenges and solutions in outreach and identification of people with inherited bleeding disorders.

INTRODUCTION: Inherited bleeding disorders (IBD) are genetic conditions that affect blood clotting, leading to complications such as prolonged or spontaneous bleeding into muscles or joints. Early identification and treatment are crucial to prevent complications and improve outcomes. However, effective patient outreach and identification programs for IBD face significant challenges globally. AIM: This study aimed to identify successful patient outreach initiatives for IBD, barriers encountered during implementation, and approaches used to overcome them. METHODS: The World Federation of Haemophilia (WFH) conducted a survey of its national member organizations and other patient associations, totalling 153 organizations, to identify common strategies, barriers to their implementation, and solutions for outreach and the identification of people with IBD. The survey consisted of both closed-ended and open-ended questions, and the data were analysed using descriptive statistics and thematic analysis. RESULTS: Common challenges included resource and sustainability-related aspects such as financial constraints, limited lab equipment for diagnosis, and inadequate government commitment. Significant barriers also encompassed physical/geographical challenges like difficulty accessing remote areas, and inadequate logistical support and transportation. Seven themes emerged to enhance patient outreach: resource mobilization; awareness-raising and advocacy; knowledge and capacity building; collaboration and partnership; decentralization of services; improved logistical support and infrastructure; utilization of technology and innovation; and financial aid and incentives. CONCLUSION: Multistakeholder collaboration, coupled with secured government commitment, is crucial for improving global outreach, diagnosis rates, and access to care for individuals with IBD. Customized outreach programs should consider regional contexts, financial constraints, and prioritize innovation.

The National Hemophilia Foundation State of the Science Research Summit initiative: executive summary.

INTRODUCTION: The National Hemophilia Foundation State of the Science Research Summit initiative sought to unify research efforts in the US inherited bleeding disorders (BDs) community around key topics of importance to people living with inherited BDs, the lived experience experts. AREAS COVERED: This community-led and -informed project focused on six broad areas - hemophilia A or B; von Willebrand Disease (VWD), platelet dysfunctions and other mucocutaneous inherited BDs; ultra-rare inherited BDs; the unique challenges of people with the potential to menstruate with inherited BDs; diversity, equity and inclusion, health services research, and implementation science; and facilitating research in the inherited BD community through designing an optimizied research infrastructure, enabling resources and funding, and furthering workforce capabilities required to execute the research priorities. EXPERT OPINION: The work summarized here, and in the accompanying supplement manuscripts , has implications not only for the US population but for people globally who have inherited BDs. The information is equally relevant to people living with hemophilia, VWD, the spectrum of inherited platelet disorders, ultra-rare factor deficiencies, and all other inherited BDs as it is to the health care providers and researchers focused on the care and treatment of inherited BDs in the US and globally.

Lived experience experts: a name created by us for us.

Plain Language SummaryPeople affected by a medical disorder, usually called patients, develop a very special expertise by living with it every day. They know, better than anyone else, how it affects their lives, what they go through to get a diagnosis and treatment, how treatments affect them, how symptoms or side effects impact their daily life, and what it is like to interact with the health care system. The people who share their lives, usually close family members like parents, partners, or siblings, develop similar knowledge. When it comes to research, patients are usually seen only as subjects. In the recent National Hemophilia Foundation State of the Science Research Summit and the subsequent National Research Blueprint project, people with inherited bleeding disorders and their family members were invited to participate in creating an agenda of the most important research that needs to be done, and in designing the approach to do the research. As full members of State of the Science Working Groups, and in leadership roles in the National Research Blueprint, they realized they needed a title that recognizes and clearly communicates their unique expertise, so that the people they work with understand what they bring to the table. They chose the term lived experience expert (LEE). Especially in rare disorders, LEEs have unique, valuable expertise to contribute to all stages of research (e.g. planning and designing, participating and recruiting participants, communicating its importance and results). Including LEEs in leadership roles will make research stronger.

Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.

INTRODUCTION: Decades of inherited bleeding disorders (BD) research transformed severe haemophilia from a childhood killer to a disorder managed across a full lifespan for many in economically developed countries. Health equity, a life unimpaired by disease complications, however, remains unimaginable for most people with an inherited BD (PWIBD). AIM: The National Hemophilia Foundation (NHF) and American Thrombosis and Hemostasis Network (ATHN) undertook the development of a community-driven United States (US) National Blueprint for Inherited Bleeding Disorders Research to transform the experience of all PWIBD and those who care for them. METHODS: Extensive community consultations were conducted to identify the issues most important to PWIBD and those who love and care for them. Expert multidisciplinary teams distilled these key areas of need into prioritised research questions, and identified the resources and infrastructure required to pursue them. A summit was held to gather feedback and inform the detailed blueprint. RESULTS: Community-prioritised research areas fell into three broad categories: issues common across inherited BDs, those specific to individual disorders, and issues of infrastructure and capacity. NHF State of the Science Research Summit discussions of the research questions derived from the community priorities by six working groups provided important input for the drafting of the research blueprint for the coming decades. CONCLUSION: The inherited BD community came together to develop the US National Blueprint for Inherited Bleeding Disorders Research dedicated to transforming the lives of all PWIBD including innovating solutions for the rarest disorders and under-represented populations.

Big picture initiatives in bleeding disorders.

INTRODUCTION: The inherited bleeding disorders (IBD) community has witnessed significant therapeutic advances recently, yet important gaps persist, particularly for those with rare disorders and historically underserved populations. AIMS: -To create a national research blueprint agenda, led by the National Hemophilia Foundation (NHF), enhancing patient-centric principles, accelerate research progress and address important gaps in care. -To review critical gaps that remain to be addressed in women with IBDs, who face specific bleeding challenges. METHODS: The NHF research blueprint research agenda was defined by input from across the community, including caregivers and patients who are considered subject matter experts of their IBD, research leaders, allied health professionals and specialists, and representatives of the biopharmaceutical industry. In addition, two medical experts in the field of IBDs performed a comprehensive review to address the knowledge gaps in women with IBDs. RESULTS: Two foundational principles of the NHF blueprint are: (1) it must deliver on key issues that significantly impact the lives of those affected by IBDs, and (2) the priorities defined are relevant and actionable aimed to achieve health equity among all those affected by IBDs. A multidisciplinary approach is necessary for an optimal management of puberty, transition to adulthood and pregnancy. Even if strict guidelines are followed, recent studies show that women with IBDs are still facing a high burden. CONCLUSION: NHF blueprint will be issued in 2022. A specific research agenda is needed in women with IBDs to further improve their management and quality of life.

Physical therapy within US HTCs: A multicentre survey of utilization, practice patterns and pain management approaches.

INTRODUCTION: Practice patterns and utilization of physical therapists (PTs) affiliated with Hemophilia Treatment Centers (HTCs) in the United States (US) are not well known. AIMS: Describe utilization, role responsibilities and practice patterns of US HTC PTs. Identify practice patterns specifically focusing on assessment and treatment of pain. Recognize gaps in utilization and role responsibilities of PTs as part of the multidisciplinary team and suggest guidelines for PT involvement within the HTC. METHODS: Respondents were a subset of a convenience sample of healthcare providers who responded to a non-validated survey developed by a multi-disciplinary panel of haemophilia experts. RESULTS: A 33.0% response-rate (n = 59) representing all regions of US HTCs was achieved. Those working ≥10 hours per week were more likely to provide nutrition education (P = .026) and surgical options education (P < .001). Those who billed insurance for their services during comprehensive visits were more likely to provide education regarding surgical options (P = .046). The majority of PTs (95.0%) evaluated pain regardless of time spent in clinic and felt comfortable treating pain. Fifty-eight percent used a formal pain measurement tool and more likely to use a formal pain measurement tool if billing insurance (P = .004). Top five non-pharmacologic treatments recommended for pain management included splints/braces (84.8%), aquatic therapy (74.6%), orthotics (71.2%), surgical options (47.5%) and yoga (32.2%). CONCLUSIONS: This study demonstrated PT utilization across HTC centres varies widely. Gaps in care may be addressed through salary support, funded education, greater regional/national collaboration of PTs specializing in bleeding disorders and advocacy for insurance coverage for appropriate services.

Understanding the Pain Management Landscape Within the US Bleeding Disorder Community: A Multi-Center Survey.

OBJECTIVES: Pain is a known complication in persons with hemophilia (PWH) as a result of muscle and joint bleeding. Little is known regarding national Hemophilia Treatment Center (HTC) practice patterns related to pain management. The aim of this study was to: 1) Describe pain management practice patterns of HTC providers, 2) Identify gaps and areas of alignment with the CDC pain guidelines, and 3) Address educational opportunities for pain management. This survey is the first extensive description of multidisciplinary practice patterns of pain management for PWH. METHODS: This descriptive study involved physicians, nurse practitioners, nurses, physical therapists, and social workers from federally funded Hemophilia Treatment Centers (HTC) eligible to complete an online survey exploring pain management practice patterns within the CDC pain guidelines. RESULTS: Results of this survey shed light on areas of strength and cohesiveness between HTC providers, including the following: dedication to effective pain management, utilization of non-pharmacological pain options, trial of non-opioid medications first before opioids, maintaining follow-up with patients after opioid prescription initiation, recognizing and utilizing clinically important findings before prescribing opioids, and counseling their patients regarding potential risk factors. CONCLUSIONS: There remain opportunities to incorporate into clinical practice consistent use of tools such as formal screening questionnaires, opioid use agreements, written measurable goals, ongoing prescription monitoring, and written plans for discontinuation of opioid therapy. These results provide opportunities for improvement in education of HTC team members thus optimizing pain management in persons with bleeding disorders.

Cardiac defense reactivity and cognitive flexibility in high- and low-resilience women.

Resilience is a protective health variable that contributes to successful adaptation to stressful experiences. However, in spite of its relevance, few studies have examined the psychophysiological and neuropsychological mechanisms involved in resilience. The present study analyzes, in a sample of 54 young women, the relationships between high- and low-resilience, measured with the Spanish versions of Connor-Davidson Resilience Scale questionnaire and the Resilience Scale, and two indices of psychophysiological and neuropsychological adaptability, the cardiac defense response (CDR) and cognitive flexibility. The CDR is a specific reaction to an unexpected intense noise characterized by two acceleration-deceleration heart rate components. Cognitive flexibility, defined as the ability to adapt our behavior to changing environmental demands, is measured in this study with the CAMBIOS neuropsychological test. The results showed that the more resilient people, in addition to having better scores on mental health questionnaires, had a larger initial acceleration-deceleration of the CDR-indicative of greater vagal control, obtained better scores in cognitive flexibility, and evaluated the intense noise as less unpleasant than the less resilient people. No group differences were found in the second acceleration-deceleration of the CDR-indicative of sympathetic cardiac control, in the skin conductance response, or in subjective intensity of the noise. The present findings broaden the understanding of how resilient people change their adaptable responses to address environmental demands.

Associations between Home Foreclosure and Health Outcomes in a Spanish City.

The financial crisis has caused an exponential increase of home foreclosures in Spain. Recent studies have shown the effects that foreclosures have on mental and physical health. This study explores these effects on a sample of adults in the city of Granada (Spain), in terms of socio-demographic, socio-economic and process characteristics. A cross-sectional survey was administered to obtain information on self-perceived changes in several indicators of physical and mental health, consumption of medications, health-related behaviors and use of health services. A total of 205 persons, going through a foreclosure process, participated in the study. 85.7% of the sample reported an increase of episodes of anxiety, depression, and stress; 82.6% sleep disturbances; 42.8% worsening of previous chronic conditions, and 40.8% an increase in consumption of medication. Women, married persons and persons already in the legal stage of the foreclosure process reported higher probability of worsening health according to several indicators, in comparison with men, not married, and individuals in the initial stages of the foreclosure process. The results of this study reveal a general deterioration of health associated with the foreclosure process. These results may help to identify factors to prevent poor health among populations going through a foreclosure process.

The International Prognostic Index for Patients with Chronic Lymphocytic Leukemia Has the Higher Value in Predicting Overall Outcome Compared with the Barcelona-Brno Biomarkers Only Prognostic Model and the MD Anderson Cancer Center Prognostic Index.

In recent years, new prognostic indexes (PIs) for chronic lymphocytic leukemia (CLL), which include clinical, biological, and genetic variables, have been validated, highlighting the MD Anderson Cancer Center prognostic index (MDACC PI), the CLL-international prognostic index (CLL-IPI), and the Barcelona-Brno biomarkers only prognostic model. The aim of this study is to compare the utility of these PIs in a cohort of Spanish patients. A retrospective analysis of 696 unselected CLL patients newly diagnosed and previously untreated from different Spanish institutions was performed. The MDACC PI, the CLL-IPI, and the biomarkers only PI were applied to these patients, and a comparison of the three PIs was performed. With a median follow-up time of 46 months, 394 patients were alive and 187 had received treatment. The median overall survival (OS) was 173 months and the median time to first therapy (TTFT) was 32 months. Significant differences were obtained in OS and TTFT for all subgroups when applying these PIs, with the CLL-IPI being the one with the higher c-index (0.676 for OS and 0.757 for TTFT). The three PIs were able to discriminate patients in different prognostic subgroups. In our cohort, the CLL-IPI showed higher power in predicting TTFT and OS.

Appetitive drives for ultra-processed food products and the ability of text warnings to counteract consumption predispositions.

OBJECTIVE: The present study aimed to (i) assess the appetitive drives evoked by the visual cues of ultra-processed food and drink products and (ii) investigate whether text warnings reduce appetitive drives and consumers' reported intentions to eat or drink ultra-processed products. DESIGN: In Study I, a well-established psychometric tool was applied to estimate the appetitive drives associated with ultra-processed products using sixty-four image representations. Sixteen product types with four exemplars of a given product were included. Pictures from the International Affective Picture System (IAPS) served as controls. The two exemplars of each product type rated as more appetitive were selected for investigation in the second study. Study II assessed the impact of textual warnings on the appetitive drive towards these thirty-two exemplars. Each participant was exposed to two picture exemplars of the same product type preceded by a text warning or a control text. After viewing each displayed picture, the participants reported their emotional reactions and their intention to consume the product. SETTING: Controlled classroom experiments SUBJECTS: Undergraduate students (Study I: n 215, 135 women; Study II: n 98, 52 women). RESULTS: In Study I, the pictures of ultra-processed products prompted an appetitive motivation associated with the products' nutritional content. In Study II, text warnings were effective in reducing the intention to consume and the appetitive drive evoked by ultra-processed products. CONCLUSIONS: This research provides initial evidence favouring the use of text warnings as a public policy tool to curb the powerful influence of highly appetitive ultra-processed food cues.

Home infusion teaching practices at federally funded hemophilia treatment centers in the United States of America.

BACKGROUND: Home management of hemophilia is standard of care in many countries. This study examined current nursing practices in teaching home infusion (HI) at hemophilia treatment centers (HTC) in the USA. OBJECTIVES: The aims were to identify and compare tools and resources used, areas of unmet needs, and to discuss implications for nurses in practice. METHODS: An anonymous electronic survey was distributed to 574 HTC nurses; 156 responses were analyzed. RESULTS: The data demonstrated that nurses, more specifically nurse coordinators, were most responsible for teaching HI. However, many nurses lack the knowledge and confidence to do so: 23.0% responded feeling somewhat or not very confident with teaching. Of those 36.4% were staff nurses, 11.9% nurse coordinators, and 41.7% advanced practice registered nurses. The majority of nurses have worked more than 5 years as a nurse, with a mean length of time of 23.7 years (SD=11.12, range 3-47) and a mean of 12.9 years (SD=10.29, range 1-42) in a HTC. Thirty-eight and a half percent of nurses have worked less than 5 years in a HTC. Most nurses appeared to follow the Infusion Nurses Society standards when performing venipunctures. Many centers reported using a formal tool or curriculum to teach HI. Nonetheless, these curricula are not uniform and their use is inconsistent between centers and regions. CONCLUSION: There are currently no national guidelines or standards to assist nurses in this task. The data confirmed the need to develop guidelines and a standardized curriculum to teach HI.

Emerging therapies for hemophilia: a new era of care and the role of the interdisciplinary team.

The introduction of new hemophilia management therapies, targeting extended half-lives through bioengineering, ushers in an era of potential promise and increasing complexity, more so for those with hemophilia B than hemophilia A. Questions arise for patients, caregivers, and hemophilia treatment center (HTC) staff about how to assess and incorporate novel therapies and how to determine whether new therapies offer a distinct advantage over established treatment routines. Nurses and other interdisciplinary HTC staff are well positioned to assess, educate, and support patients and families in navigating this rapidly changing landscape. To support these challenging efforts, this review offers a perspective on issues affecting therapeutic transitions and provides tools to foster ongoing adherence.

In vitro activity of daptomycin and comparator agents against Staphylococcus aureus isolates from intravenous drug users with right endocarditis.

There is an increasing need for alternative agents in endocarditis, especially with the increasing incidence of vancomycin-intermediate Staphylococcus aureus (VISA). We evaluated the in vitro activity of daptomycin and several comparator agents against 33 non-duplicate clinical Staphylococcus aureus isolates from intravenous drug users with right endocarditis. Wider microdilution panels were used for all the comparator agents and daptomycin. Daptomycin was also tested using E-test strips. E-test strips were used to confirm the vancomycin MICs. Methicillin-resistant Staphylococcus aureus (MRSA isolates with vancomycin MICs ≥ 2 g/mL were screened using the E-test GRD. In all, 30 isolates were methicillin-susceptible (MSSA) and 3 MRSA. The three MRSA isolates exhibited a false vancomycin MIC >2 g/mL determined by Wider microdilution panels. They were screened using the E-test GRD and they were GRD negative. Their final MIC was 2 g/mL. Three MSSA and three MRSA isolates had a vancomycin MIC of 2 g/mL. Four MSSA isolates had a vancomycin MIC of 1.5 g/mL, daptomycin MIC90 0.25 g/mL, linezolid MIC90 2 g/mL. As regards daptomycin, wider microdilution panels and E-test strips yielded the same results. Our findings suggest that daptomycin and linezolid are a viable alternative for treating right endocarditis and bacteraemia caused by MSSA, MRSA and hVISA.

Two scales to evaluate cocaine craving.

The cocaine craving questionnaire-brief (CCQ-Brief English) asks cocaine users to report their level of craving now. We constructed two brief scales of cocaine craving in a sample of 107 Spanish-speaking natives in treatment for cocaine abuse or dependence: the CCQ-Brief(Spanish) and the pictographic assessment of desire (PAD), which relies less on language. Principal component analyses yielded a one-component solution for the CCQ-Brief, explaining between 62% and 68% of the sample variability. Cronbach's alpha ranged from .92 to .94. The CCQ-Brief and the PAD were strongly correlated; effect sizes ranged from .42 to .68 in separate trials. Laboratory cue-exposure results showed that in both scales craving was higher upon presentation of cocaine-related rather than neutral pictorial stimuli. The CCQ-Brief and PAD are potentially valuable scales for evaluating cocaine craving.

Prevalence of autism spectrum disorders in relatives of patients with selective immunoglobulin A deficiency.

BACKGROUND: An association of selective IgA (immunoglobulin A) deficiency in individuals with autism has been previously described. The objective of this study was to examine the incidence of autism spectrum disorders (ASD) in children and siblings of selective IgA deficient patients. OBJECTIVE: to assess the likelihood of parents with the most common type of primary immunodeficiency (selective IgA deficiency) having children with ASD and to investigate the occurrence of ASD in siblings of the immunodeficient patients. METHODS: A study was conducted in 31 selective IgA deficient patients and 62 age and gender-matched controls. Children and siblings of IgA deficient patients and controls were screened for an ASD (autism spectrum disorder) using a standard questionnaire. RESULTS: Only one patient in the IgA deficient group had classical autism. Three children in that group (10.3%) had an ASD compared to only one in the control group (1.6%) and this difference was statistically significant. In terms of siblings, there was a higher occurrence of an ASD in the IgA deficient group than in the control group, but the difference was not statistically significant. A high incidence of allergies (71%) was documented in IgA deficient patients. All individuals with allergies had food sensitivities. There was a predominance of the male gender in cases identified with an ASD in all groups. CONCLUSIONS: A lower prevalence of ASD was observed in the IgA deficient group, as compared to other reports. The study suggests that screening for an ASD seems appropriate for children of IgA deficient patients.