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Objectives: To investigate recruitment processes across a range of clinical trials and from the perspective of parents, young people and practitioners to identify strategies to improve recruitment and its conduct across the spectrum of trials of medicines for children. Design: Qualitative interview and observational study. Setting: Eleven paediatric clinical trial centres recruiting to four trials. Participants: Members of 60 families approached to consider entry to one of the participating trials and 31 practitioners. Interventions: None. Main outcome measures: Data were verbatim transcripts of (1) audio-recorded trial recruitment discussions between practitioners and families (n = 41) and (2) semi-structured interviews with parents (n = 62), young people (n = 22) and practitioners (19 doctors and 12 research nurses). Analyses were interpretive, following the general principles of the constant comparative method. Results: Practitioners were concerned to avoid overburdening parents and some indicated that they found approaching families about trials to be aversive. By contrast, even in the most difficult situations, parents did not mind being asked about trials and they did not describe the approach as burdensome. Some parents viewed the trial approach as a positive or exciting opportunity. Parents and young people took little active part in the trial discussions and asked few questions. Despite this, they were satisfied with how they had been approached, and spoke of how they had felt involved, valued, cared for and comfortable to interject during the discussion. However, we identified several parents who had important misunderstandings about the trial. There were few differences between parents who consented and those who declined a trial. Regardless of whether they consented or declined, parents’ trial decisions were influenced by their perceptions of the trial in relation to their child’s safety and well-being, potential benefits to the child and family, potential benefits to others and the practicality of participation. Of these, parents’ paramount consideration was safety. Parents’, young people’s and practitioners’ views of what was important when considering a trial were broadly convergent, although families gave greater importance than practitioners to the trial’s practical requirements. All parties valued the face-to-face trial discussion highly and wanted shorter and less complex written information. Parents did not feel pressured by the trial team to participate, but some described how their personal values made them reluctant to decline, and several parents who did decline described a passing sense of discomfort. Conclusions: The concerns of some practitioners that families would be overburdened were unfounded, as parents did not object to being asked about research. Practitioners may benefit from support that helps them feel personally more at ease in approaching families about trials. Parents and young people often described the trial discussions in strongly positive terms and emphasised the importance of the social and emotional aspects of these encounters. Informed consent training could be enhanced if it similarly emphasised these aspects of recruitment; the misunderstandings we identified indicate how this training could also help practitioners to improve the clarity of their trial discussions with families. Guidelines on informed consent documents should take account of findings that all groups thought that these documents should be shorter and more straightforward. Funding: This research was commissioned by the National Institute for Health Research Health Technology Assessment programme.

With therapeutic trials on the horizon for Charcot-Marie-Tooth type 1A (CMT1A), reliable, valid, and responsive clinical outcome assessments and biomarkers are essential. Accelerate Clinical Trials in CMT (ACT-CMT) is an international study designed to address important gaps in CMT1A clinical trial readiness including the lack of a validated, responsive functional outcome measure for adults, and a lack of validated biomarkers for multicenter application in clinical trials in CMT1A. The primary aims of ACT-CMT include validation of the Charcot-Marie-Tooth Functional Outcome Measure, magnetic resonance imaging of intramuscular fat accumulation as a lower limb motor biomarker, and in-vivo reflectance confocal microscopy of Meissner corpuscle sensory receptor density, a sensory biomarker. Initial studies have indicated that these measures are feasible, reliable and valid. A large prospective, multi-site study is necessary to fully validate and examine the responsiveness of these outcome measures in relation to existing outcomes for use in future clinical trials involving individuals with CMT1A. Two hundred 15 adults with CMT1A are being recruited to participate in this prospective, international, multi-center study. Serial assessments, up to 3 years, are performed and include the CMT-FOM, CMT Exam Score-Rasch, Overall Neuropathy Limitations Scale, CMT-Health Index, as well as nerve conduction studies, and magnetic resonance imaging and Meissner corpuscle biomarkers. Correlations using baseline data will be examined for validity. Longitudinal analyses will document the changes in function, intramuscular fat accumulation, Meissner corpuscle sensory receptor density. Lastly, we will use anchor-based and other statistical methods to determine the minimally clinically important change for these clinical outcome assessments and biomarkers in CMT1A. Reliable, and responsive clinical outcome assessments of function and disease progression biomarkers are urgently needed for application in early and late phase clinical trials in CMT1A. The ACT-CMT study protocol will address this need through the prospective, longitudinal, multicenter examination in unprecedented detail of novel and existing clinical outcome assessments and motor and sensory biomarkers, and enhance international clinical trial infrastructure, training and preparedness for future therapeutic trials in CMT and related neuropathies.

Abstract Objective Experimental therapies under development for Friedreich’s Ataxia (FRDA) require validated biomarkers. In‐vivo reflectance confocal microscopy (RCM) of skin is a noninvasive way to quantify Meissner’s corpuscle (MC) density and has emerged as a sensitive measure of sensory polyneuropathies. We conducted a prospective, cross‐sectional study evaluating RCM of MCs and conventional peripheral nerve measures as candidate peripheral nerve markers in FRDA. Methods Sixteen individuals with FRDA and 16 age‐ and gender‐matched controls underwent RCM of MC density and morphology, skin biopsies for epidermal nerve fiber density (ENFD), nerve conduction studies (NCS), and quantitative sensory testing (QST) including touch, vibration, and cooling thresholds. Results MC densities were measurable in all participants with FRDA, and were lower at digit V (hand), thenar eminence, and arch (foot) compared to controls. By contrast, sensory NCS showed floor effects and were obtainable in only 13% of FRDA participants. QST thresholds for touch, vibration, and cooling were higher at the hand and foot in FRDA than controls. Reductions in ENFDs were present in more severely affected individuals with FRDA (Friedreich’s Ataxia Rating Scale (FARS) >60) compared to matched controls, although skin biopsies were not well tolerated in children. MC densities, ENFDs, and touch and vibration thresholds were associated with clinical disease severity (FARS and modified FARS) and duration since symptom onset. Interpretation MC density, ENFD, and QST thresholds provide structural and physiologic markers of sensory involvement in FRDA. Longitudinal evaluation is needed to determine whether these measures can identify changes associated with disease progression or treatment.

Aim: As heart failure (HF) with preserved ejection fraction (HFpEF) prevalence increases, it remains frequently underdiagnosed and poorly managed. Recent positive pharmacological trials have increased interest in HFpEF but challenges of diagnosis and management remain. The survey aim was to examine consensus between primary and secondary care providers regarding HFpEF diagnosis and management., Methods: As part of a larger programme of work, survey questions were developed in an online format and piloted with healthcare providers (HCPs). The survey link was distributed via professional networks and social media. Analysis included frequencies of responses, comparison by main professional groups and thematic analysis free-text responses. A virtual workshop of HCPs was conducted to discuss and refine survey findings., Results: HCPs (n=66) across the UK participated: 19 general practitioners (GPs), 20 HF specialist nurses (HFSN), 17 cardiologists and 10 others. Consensus was high (92%) that diagnosing the type of HF was very important and most favoured inclusion of HFpEF in Quality Outcome Framework indicators. No clear consensus was reached that ongoing management should be in primary care (47.5% of GPs, 35% of HFSN and 31.3% of cardiologists 'somewhat agreed'). Opinions differed between GPs (52.3)% and specialists (HFSN 80% and cardiologists 81.3%) for practice nurses to be upskilled and assume HFpEF management. No HCPs reported any level of disagreement for HFSN management of HFpEF. Free-text comments highlighted resource barriers to HFpEF diagnosis and management and confirmed the need to develop better HFpEF services., Conclusions: Consensus was reached regarding importance of diagnosing HFpEF, but agreement on methods and responsibilities for diagnosis and management varied. Free-text comments identified HCPs concerns related to overwhelmed primary and secondary care services and lack of sufficient resources to meet existing patient demands. Creation of collaborative care pathways is needed to support the increasing number of older patients with HFpEF., Trial Registration Number: ClinicalTrials. gov (reference number: NCT03617848)., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ Group.)

Aims: To investigate how heart failure with preserved ejection fraction (HFpEF), within the context of limited clinical services, impacts patients' lives., Methods and Results: Secondary thematic analysis informed by the cumulative complexity model (CCM), of interview transcripts from 77 people diagnosed with HFpEF and their carers. Four themes corresponding to the core concepts of workload, capacity, access, and outcome described in the CCM were generated. Theme 1: Shouldering a heavy workload described the many tasks expected of people living with HFpEF. Theme 2: The multiple threats to capacity described how patients and carers strived to engage with this work, but were often faced with multiple threats such as symptoms and mobility limitations. Deficient illness identity (Theme 3) reflects how HFpEF either was not recognized or was perceived as a more benign form of HF and therefore afforded less importance or priority. These themes contributed to a range of negative physical, social, and psychological outcomes and the perception of loss of control described in Theme 4: Spiraling complexity., Conclusions: The constellation of HFpEF, multi-morbidity, and ageing creates many demands that people with HFpEF are expected to manage. Concurrently, the same syndromes threaten their ability to physically enact this work. Patients' recollections of their interactions with health professionals suggest that there is a widespread misunderstanding of HFpEF, which can prohibit access to care that could potentially reduce or prevent deterioration., Competing Interests: Conflicts of interest: J.M. has done consultancy work for BMS/Pfizer and Omron. C.D. has consulted for Astra Zeneca. All other authors declare no conflicts of interest., (© The Author(s) 2022. Published by Oxford University Press on behalf of the European Society of Cardiology.)

Introduction: Caring for a child with obsessive-compulsive disorder (OCD) can be extremely difficult, yet evidence-based support strategies for parents/carers are limited. A detailed understanding of parent support needs is an important first step in intervention development and qualitative research with this focus is currently lacking. In this study, the viewpoints of parents and professionals were used to understand support needs and preferences when caring for a child with OCD. This qualitative descriptive study formed part of a wider UK-based project aimed at developing better support for parents of children with OCD., Method: Individual semi-structured interviews (and an optional one-week journal) with a purposive sample of parents of children and young people (CYP) with OCD, aged 8-18, and focus groups (or individual interviews where preferred) with a purposive sample of professionals supporting CYP with OCD. Data comprised transcripts of audio-recorded interviews and focus groups, and text from journals. Analysis was informed by the Framework approach involving inductive and deductive coding, supported by NVivo 12.0 software. Co-production methods were adopted throughout the research process, including the involvement of a parent co-researcher and charity collaborators., Results: Interviews were undertaken with 20 parents, of which 16 completed a journal. Twenty-five professionals took part in a focus group or interview. Five key themes relating to parent support challenges and support needs/preferences were identified (1) Coping with the impact of OCD; (2) Getting help for my child; (3) Understanding parents' role; (4) Making sense of OCD; (5) Joined-up care., Conclusion: Parents caring for children with OCD have clear caregiver support needs which are currently not being met. Through triangulation of parent and professional accounts, this study has identified parent support challenges (e.g., emotional impact of OCD, visibility of caring role, misunderstanding about OCD) and support needs/ preferences (e.g., headspace/respite, compassion/sensitivity, guidance on accommodation) to lay the vital foundations for the development of effective parent support interventions. There is now an urgent need to develop and test an intervention to support parents in their caregiving role, with the aim of preventing and/or reducing their levels of burden and distress and ultimately, improving their quality of life., (© 2023. The Author(s).)

Aims: Heart failure with preserved ejection fraction (HFpEF) accounts for 50% of all heart failure cases; yet remains poorly understood, diagnosed, and managed, which adds complexity to the carer role. No study to date has investigated the experiences of informal carers of people with HFpEF. The aim of this study was to explore the role and experiences of informal carers of people with HFpEF., Methods and Results: A qualitative study using semi-structured interviews involving carers alone, patients alone, or carer/patient dyads. The interviews were part of a larger programme of research in HFpEF. Participants were recruited from three regions of England. Interviews were recorded, transcribed verbatim, and analysed thematically. Twenty-two interviews were conducted with 38 participants, 17 were informal carers. Three inter-related themes were identified: Theme 1, the complex nature of informal caregiving ('spinning plates'); Theme 2, the barriers to caregiving ('the spinning falters'); and Theme 3, the facilitators of caregiving ('keeping the plates spinning')., Conclusions: Informal carers play an important role in supporting people with HFpEF. The experience of caregiving in HFpEF is similar to that described for Heart Failure with reduced Ejection Fraction, but complicated by challenges of limited information and support specific to HFpEF, and high burden of multi-morbidity. Healthcare providers should assess the needs of informal carers as part of patient care in HFpEF. Carers and patients would benefit from improved information and co-ordinated management of HFpEF and multi-morbidities. Helping carers 'keep the plates spinning' will require innovative approaches and co-ordination across the care continuum., Competing Interests: Conflict of interest: none declared., (© The Author(s) 2022. Published by Oxford University Press on behalf of the European Society of Cardiology.)

Objectives: To explore the perspectives of general practitioners (GPs) on the identification and management of people, including those from ethnic minority groups, with Heart Failure with Preserved Ejection Fraction (HFpEF)., Methods: Qualitative study. Semi-structured, face-to-face or telephone interviews and focus groups were conducted with 35 GPs in England, which were audio-recorded and transcribed verbatim. Framework analysis was used to manage and interpret data., Results: Themes presented reflect four inter-related challenges: GPs' 1) lack of understanding HFpEF, impacting on 2) difficulties in communicating the diagnosis, leading to 3) uncertainty in managing people with HFpEF, further hindered by (4) discontinuity across the primary/secondary interface. All were considered more challenging by GPs when managing people from different cultures and languages., Discussion: HFpEF is not well understood by GPs, leading to diagnostic difficulty, management uncertainty and potential inequity in care offered. People with HFpEF are seen as complex, with multiple long-term conditions and requiring personalised care. Challenges in their management occur across the healthcare system. This study has identified learning needs for GPs around identification and on-going support for people with HFpEF in primary care. It will contribute to the development of more flexible and patient-centred pathways across the primary/secondary care interface.

Background: Severe acute respiratory coronavirus 2 (SARS-CoV-2), also known as coronavirus disease 2019 (COVID-19), resulted in unprecedented societal and healthcare provision change, which has been implemented at pace. Little is known about the indirect impacts of these changes and what the future effects may be., Aim: To explore patients' and clinicians' experiences of managing heart failure (HF) during the COVID-19 pandemic., Design & Setting: Qualitative study in three regions of the UK: Cambridgeshire, Greater Manchester, and the West Midlands., Method: Semi-structured interviews ( n = 30) were conducted with older adults with established HF and healthcare providers from primary and secondary health services involved in their care. Interviews were analysed thematically., Results: Compliance with the government guidance 'Stay at home, protect the NHS, and save lives' during the COVID-19 pandemic, and perceptions relating to risk from COVID-19 and underlying morbidity, drove 'being careful' behaviours and organisational changes. Enacting behavioural change and implementing organisational change resulted in opportunities and challenges for health and healthcare practice., Conclusion: Perception of risk led to significant behavioural and organisational change during the pandemic. Some changes described by both patients and clinicians, such as enhanced relationships and self-monitoring, present as opportunities, and consideration should be given as to how to maintain or develop these. Equally, indirect impacts of COVID-19 and the associated lockdown, such as disengagement and withdrawal, and the fallout from reluctance to access health services, should be acknowledged and interventions to address these challenges are needed., (Copyright © 2021, The Authors.)

Background: About half of all people with heart failure have heart failure with preserved ejection fraction (HFpEF), in which the heart is stiff. This type of heart failure is more common in older people with a history of hypertension, obesity, and diabetes mellitus. Patients with HFpEF are often managed in primary care, sometimes in collaboration with specialists. Knowledge about how best to manage this growing population is limited, and there is a pressing need to improve care for these patients., Aim: To explore clinicians' and patients'/carers' perspectives and experiences about the management of HFpEF to inform the development of an improved model of care., Design and Setting: A multiperspective qualitative study involving primary and secondary care settings across the east of England, Greater Manchester, and the West Midlands., Method: Semi-structured interviews and focus groups were conducted. Transcribed data were analysed using framework analysis and informed by the normalisation process theory (NPT)., Results: In total, 50 patients, nine carers/relatives, and 73 clinicians were recruited. Difficulties with diagnosis, unclear illness perceptions, and management disparity were identified as important factors that may influence management of HFpEF. The NPT construct of coherence reflected what participants expressed about the need to improve the identification, understanding, and awareness of this condition in order to improve care., Conclusion: There is a pressing need to raise the public and clinical profile of HFpEF, develop a clear set of accepted practices concerning its management, and ensure that systems of care are accessible and attuned to the needs of patients with this condition., (© British Journal of General Practice 2020.)

Background: Heart failure with preserved ejection fraction (HFpEF) is less well understood than heart failure with reduced ejection fraction (HFrEF), with greater diagnostic difficulty and management uncertainty., Aim: The primary aim is to develop an optimised programme that is informed by the needs and experiences of people with HFpEF and healthcare providers. This article presents the rationale and protocol for the Optimising Management of Patients with Heart Failure with Preserved Ejection Fraction in Primary Care (OPTIMISE-HFpEF) research programme., Design & Setting: This is a multi-method programme of research conducted in the UK., Method: OPTIMISE-HFpEF is a multi-site programme of research with three distinct work packages (WPs). WP1 is a systematic review of heart failure disease management programmes (HF-DMPs) tested in patients with HFpEF. WP2 has three components (a, b, c) that enable the characteristics, needs, and experiences of people with HFpEF, their carers, and healthcare providers to be understood. Qualitative enquiry (WP2a) with patients and providers will be conducted in three UK sites exploring patient and provider perspectives, with an additional qualitative component (WP2c) in one site to focus on transitions in care and carer perspectives. A longitudinal cohort study (WP2b), recruiting from four UK sites, will allow patients to be characterised and their illness trajectory observed across 1 year of follow-up. Finally, WP3 will synthesise the findings and conduct work to gain consensus on how best to identify and manage this patient group., Results: Results from the four work packages will be synthesised to produce a summary of key learning points and possible solutions (optimised programme) which will be presented to a broad spectrum of stakeholders to gain consensus on a way forward., Conclusion: HFpEF is often described as the greatest unmet need in cardiology. The OPTIMISE-HFpEF programme aims to address this need in primary care, which is arguably the most appropriate setting for managing HFpEF., (Copyright © 2019, The Authors.)

Background: Assent is used to take children's wishes into account when they are invited into clinical trials, but the concept has attracted considerable criticism. We investigated children's accounts of decision-making with the aim of informing practice in supporting children when invited to join a clinical trial., Methods: We audio-recorded qualitative, semi-structured interviews with 22 children aged 8-16 years about being invited to take part in a clinical trial. Most children were interviewed with their parents. Analysis of the transcribed interviews examined the content of participants' accounts thematically, whilst also drawing on principles of discourse analysis, which examines how individuals use talk to achieve certain effects or social practices., Results: It was not possible to separate children's knowledge of the clinical trial, or their decision-making processes from that of their parents, with parents taking a substantial mediating role in producing their children's decisions. Decision-making gradually unfolded across time and events and was interwoven within the family context, rather than happening in one moment or in the clinical setting. Whilst children valued their parents' role, a case study of child-parent disagreement indicated how children can struggle to be heard., Conclusions: Decisions happen within a process of family dynamics, in contrast to ideas of assent that isolate it from this context. Parents have a substantial role in children's decisions, and thus how families come to provide consent. Reflecting this we argue that assent practices need to focus on supporting parents to support their children in learning and deliberating about trials. However, this needs to be accompanied by practitioners being alert to the possibility of divergence in child and parent views and enabling children's perspectives to be heard., (© 2016 The Authors. Child: Care, Health and Development published by John Wiley & Sons Ltd.)

Ensuring parents make an informed decision about their child's participation in a clinical trial is a challenge for practitioners as a parent's comprehension of a trial may differ from that intended by the practitioners responsible for recruitment. We explored what issues parents consider important when making a decision about participation in a paediatric clinical trial and their comprehension of these issues to inform future recruitment practice. This qualitative interview and observational study examined recruitment in four placebo-controlled, double-blind randomised clinical trials of medicines for children. Audio-recorded trial recruitment discussions between practitioners and parents (N = 41) were matched with semi-structured interviews with parents (N = 41). When making a decision about trial entry parents considered clinical benefit, child safety, practicalities of participation, research for the common good, access to medication and randomisation. Within these prioritised issues parents had specific misunderstandings, which had the potential to influence their decisions. While parents had many questions and concerns about trial participation which influenced their decision-making, they rarely voiced these during discussions about the trials with practitioners. Those involved in the recruitment of children to clinical trials need to be aware of parents' priorities and the sorts of misunderstandings that can arise with parents. Providing trial information that is tailored to what parents consider important in making a decision about a clinical trial may improve recruitment practice and ultimately benefit evidence-based paediatric medicine.

Background: Methotrexate (MTX) is a first-line disease-modifying agent and anchor drug for biologic therapy used in rheumatoid arthritis and other inflammatory rheumatic disorders. Adverse effects are a common cause of drug discontinuation and include preventable serious incidents that may result in patient harm or death., Objectives: The objective of this study was to audit adherence by health professionals to national and international guidelines for patient education and risk reduction in patients prescribed MTX for inflammatory rheumatic diseases., Methods: A combination of interviews, case record reviews, and self-administered patient knowledge questionnaires with individual patient feedback was used. The setting was the rheumatology outpatient department of a district general hospital., Results: Fifty-one patients participated in the audit. The mean age was 58.6 (SD, 13.1) years and median duration of disease was 3.7 years (interquartile range, 1.7-7.6 years). Nurse-led patient education was documented at baseline for 94.1% of participants. Despite this, only 11.8% of participants recognized the potentially lethal drug-drug interaction with trimethoprim/Septrin (co-trimoxazole), and less than 60.8% recognized possible major adverse effects related to MTX. Although lifestyle implications relating to alcohol consumption and pregnancy/breast-feeding were recognized by the majority, only 52.9% of males were aware of recommendations in relation to conception. Univariable and multivariable analyses identified male sex, not speaking English as a first language, and a longer duration of therapy as predictors of lower levels of patient knowledge., Conclusions: Despite consistent baseline patient education, end-user knowledge and awareness pertinent to MTX safety are limited. Good-quality written information in the most appropriate language, patient feedback on educational programs, follow-up testing of patient knowledge, and targeted reeducation are recommended to address individual deficiencies in core knowledge.

Wegener's granulomatosis is a systemic necrotizing granulomatous vasculitis of small- to medium-sized vessels typically affecting upper and lower airways, lungs, and kidneys. Cardiac involvement is less common and conducting tissue involvement is extremely rare. Cardiac manifestations are often not clinically apparent, but are associated with increased mortality. We report the case of a 36-year-old female with Wegener's thought to be in remission, presenting in complete atrioventricular (AV) block, with echocardiographic evidence of basal interatrial septum and basal lateral left atrial wall thickening. Despite immunosuppression therapy a permanent pacemaker was required for recurring complete AV block. Although rare, this case emphasizes the need for careful and regular screening for cardiac involvement in this multi-system condition.

Background: Recruiting children to clinical trials is perceived to be challenging. To identify ways to optimise recruitment and its conduct, we compared how parents and practitioners described their experiences of recruitment to clinical trials., Methods and Findings: This qualitative study ran alongside four children's clinical trials in 11 UK research sites. It compared analyses of semi-structured interviews with analyses of audio-recordings of practitioner-family dialogue during trial recruitment discussions. Parents from 59 families were interviewed; 41 had participated in audio-recorded recruitment discussions. 31 practitioners were interviewed. Parents said little in the recruitment discussions contributing a median 16% of the total dialogue and asking a median of one question. Despite this, parents reported a positive experience of the trial approach describing a sense of comfort and safety. Even if they declined or if the discussion took place at a difficult time, parents understood the need to approach them and spoke of the value of research. Some parents viewed participation as an 'exciting' opportunity. By contrast, practitioners often worried that approaching families about research burdened families. Some practitioners implied that recruiting to clinical trials was something which they found aversive. Many were also concerned about the amount of information they had to provide and believed this overwhelmed families. Whilst some practitioners thought the trial information leaflets were of little use to families, parents reported that they used and valued the leaflets. However, both parties agreed that the leaflets were too long and wanted them to be more reader-friendly., Conclusions: Parents were more positive about being approached to enter their child into a clinical trial than practitioners anticipated. The concerns of some practitioners, that parents would be overburdened, were unfounded. Educating practitioners about how families perceive clinical trials and providing them with 'moral' support in approaching families may benefit paediatric research and, ultimately, patients.

Background: Influenza and pneumococcal vaccination are recommended for a number of clinical risk groups including patients treated with major immunosuppressant disease modifying anti-rheumatic drugs. Such immunisation is not only safe but immunogenic in patients with rheumatic diseases. We sought to establish dual vaccination rates and significant influencing factors amongst our hospital rheumatology outpatients., Method: We audited a sample of 101 patients attending hospital rheumatology outpatient clinics on any form of disease modifying treatment by clinical questionnaire and medical record perusal. Further data were collected from the local immunisation coordinating agency and analysed by logistic regression modelling., Results: Although there was a high rate of awareness with regard to immunisation, fewer patients on major immunosuppressants were vaccinated than patients with additional clinical risk factors against influenza (53% vs 93%, p < 0.001) or streptococcus pneumoniae (28% vs 64%, p = 0.001). The presence of additional risk factors was confirmed as significant in determining vaccination status by logistic regression for both influenza (OR 10.89, p < 0.001) and streptococcus pneumoniae (OR 4.55, p = 0.002). The diagnosis of rheumatoid arthritis was also found to be a significant factor for pneumococcal vaccination (OR 5.1, p = 0.002). There was a negative trend suggesting that patients on major immunosuppressants are less likely to be immunised against pneumococcal antigen (OR 0.35, p = 0.067)., Conclusion: Influenza and pneumococcal immunisation is suboptimal amongst patients on current immunosuppressant treatments attending rheumatology outpatient clinics. Raising awareness amongst patients may not be sufficient to improve vaccination rates and alternative strategies such as obligatory pneumococcal vaccination prior to treatment initiation and primary care provider education need to be explored.

Background: Pneumocystis pneumonia (PCP) is an increasing problem amongst patients on immunosuppression with autoimmune inflammatory disorders (AID). The disease presents acutely and its diagnosis requires bronchoalveolar lavage in most cases. Despite treatment with intravenous antibiotics, PCP carries a worse prognosis in AID patients than HIV positive patients. The overall incidence of PCP in patients with AID remains low, although patients with Wegener's granulomatosis are at particular risk., Discussion: In adults with AID, the risk of PCP is related to treatment with systemic steroid, ill-defined individual variation in steroid sensitivity and CD4+ lymphocyte count. Rather than opting for PCP prophylaxis on the basis of disease or treatment with cyclophosphamide, we argue the case for carrying out CD4+ lymphocyte counts on selected patients as a means of identifying individuals who are most likely to benefit from PCP prophylaxis., Summary: Corticosteroids, lymphopenia and a low CD4+ count in particular, have been identified as risk factors for the development of PCP in adults with AID. Trimethoprim-sulfamethoxazole (co-trimoxazole) is an effective prophylactic agent, but indications for its use remain ill-defined. Further prospective trials are required to validate our proposed prevention strategy.