Consensus recommendations on holistic care in hereditary ATTR amyloidosis: an international Delphi survey of patient advocates and multidisciplinary healthcare professionals.

BACKGROUND: Hereditary transthyretin-mediated amyloidosis is a rare, progressive and potentially life-limiting multisystem disease, affecting every aspect of a patient's life. OBJECTIVES: This online international Delphi survey aimed to evolve clinical-patient-led practical guidance, to inspire and encourage a holistic approach to care that is managed in specialist settings by multidisciplinary teams and supported by allied healthcare professionals (HCPs) and patient advocacy groups (PAGs). DESIGN: A 14-member joint patient advocate-HCP primary panel was convened including representation from PAGs and key clinical specialties (neurology, cardiology, internal medicine, physiotherapy, clinical psychology, dietetics and specialist nursing). Guidance evolved on the care provision needed to support seven core goals: early diagnosis and treatment; disease monitoring and organisation of care; maintenance of physical and mental health; family-centred care and caregiver support; patient-doctor dialogue; access to social support and social networking. PARTICIPANTS: From June to October 2022, 252 HCPs and 51 PAG representatives from 27 countries were invited to participate in a Delphi survey. Of the 122 respondents who answered at least one survey question, most were HCPs (100, 82%) from specialist centres; the remainder were PAG representatives (22, 18%). MAIN OUTCOME MEASURE: Both level of agreement and feasibility in practice of each recommendation was tested by two anonymised online Delphi voting rounds. RESULTS: Based on an a priori threshold for consensus of ≥75% agreement, the clinical-patient community endorsed all but one recommendation. However, only 17/49 (35%) recommendations were identified by most HCPs as a core part of routine care; the remainder (32/49 (65%)) were identified as part of core care by <50% of HCPs respondents, or as largely achievable by 30%-45% of HCPs. By comparison, PAGs recorded lower implementation levels. CONCLUSIONS: Further consideration is needed on how to evolve multidisciplinary services (supported by allied HCPs and PAGs) to address the complex needs of those affected by this disease.

Factors affecting antidepressant use by patients requiring home parenteral nutrition.

BACKGROUND: Home parenteral nutrition (HPN) is a life-saving therapy for patients with chronic intestinal failure but can be associated with a degree of psychological distress. The factors associated with the need for antidepressants (ADs) in this cohort have not yet been described. METHODS: The study involved prospective data collection from patients attending an HPN clinic at a national intestinal failure referral center. Patients requiring HPN as a result of active malignancy were excluded. Patients were divided in 2 groups according to AD usage; demographic, anthropometric, socioeconomic characteristics, and intravenous supplementation (IVS) regimens were compared between groups. RESULTS: A total of 184 patients were recruited between July 2018 and April 2019, with an overall prevalence of AD use of 41.7% (70/168 patients). Daily mean IVS volume was significantly higher among patients taking AD ("AD" group; 2125.48 ± 991.8 ml/day, "no-AD" group; 1828.54 ± 847.0 ml/day, P = .039), with the proportion of patients needing high-volume IVS (≥3000 ml/day) being 3 times higher in the AD group (20.0%(14/70 patients) vs 6.1% (6/98 patients), P = .006). The average energy IVS infusion per day was similar between the groups. CONCLUSION: This is the first study to demonstrate that AD use correlates with higher IVS volume rather than energy requirements in HPN patients, suggesting that high IVS volume requirements may be better associated with the patient's disease burden. Early and tailored mental health intervention may be beneficial in those with high IVS volume requirements.

Successful implementation of remote video consultations for patients receiving home parenteral nutrition in a national UK Centre.

RATIONALE: Our intestinal failure unit provides care for patients from a wide geographical area. Patients dependent on home parenteral nutrition (HPN) are routinely reviewed in the clinic at 3-6 monthly intervals. Between March 2008 and 2015, we noted a significant rise in the number of patients under our care, with an associated 51% increase in clinic appointments offered. We evaluated whether telemedicine would provide a strategy to reduce patients' need to travel while maintaining safe clinical standards. METHODS: Implementation began in December 2015 via patient consultation and small tests of change. Clinical data were obtained from a prospectively maintained database. Remote video consultation discussions were carried out via internet video call service (Skype). An anonymous satisfaction questionnaire was offered to patients for completion following consultation. The number of miles saved by obviating the need to attend hospital was calculated for each patient. RESULTS: During the study period, patients receiving HPN rose by 18% to 288. Twenty-five patients used telemedicine for HPN follow-up, three of these for follow-up with the psychologist. By avoiding hospital attendance, this saved a mean travel distance of 56.7 miles with a total of 18 346.6 cumulative miles saved. Sixty-three per cent of patients rated their satisfaction with the system at ≥90%, with a mean satisfaction of 85%. Eight per cent of the telemedicine cohort was admitted with an HPN complication, compared with an admission rate of 24% for the whole HPN cohort. One emergency admission was avoided. CONCLUSION: Telemedicine can obviate the need for clinic attendance in HPN-dependent patients, so reducing the need of individuals with chronic illness to travel while maintaining standards for follow-up.

Needs-based quality of life in adults dependent on home parenteral nutrition.

BACKGROUND & AIMS: Home parenteral nutrition (HPN) provides life sustaining treatment for people with chronic intestinal failure. Individuals may require HPN for months or years and are dependent on regular intravenous infusions, usually 12-14 h overnight between 1 and 7 days each week. This regime can have adverse impact on the life of people dependent on the treatment. The aim of this study was to establish mean values for the Parenteral Nutrition Impact Questionnaire (PNIQ) and to determine the effect of disease, frequency of infusions per week and patient characteristics on quality of life of patients fed HPN. METHOD: The PNIQ was distributed to patients across nine UK HPN clinics. Data were analysed using linear regression, with PNIQ score as the dependent variable and potential confounders as independent variables. Unadjusted and adjusted models are presented. Higher PNIQ scores reflect poorer quality of life. RESULTS: Completed questionnaires were received from 466 people dependent on HPN. Mean PNIQ score was 11.04 (SD 5.79). A higher PNIQ score (effect size 0.52, CI 0.184 to 0.853) was recorded in those dependent on a higher frequency of HPN infusions per week. Respondents with cancer had a similar mean PNIQ score to those with inflammatory bowel disease (mean 10.82, SD 6.00 versus 11.04, SD 5.91). Those with surgical complications reported a poorer QoL (effect size 3.03, CI 0.642 to 5.418) and those with severe gastro-intestinal dysmotility reported a better QoL (effect size -3.03, CI -5.593 to -0.468), compared to other disease states. CONCLUSIONS: This large cohort study of quality of life in chronic intestinal failure demonstrates that HPN impacts individuals differently depending on their underlying disease. Furthermore, since the number of HPN infusions required per week is inversely related to an individual's needs-based quality of life, therapies that reduce PN burden should lead to an improvement in QoL.

Poor Social Support and Unemployment Are Associated With Negative Affect in Home Parenteral Nutrition-Dependent Patients With Chronic Intestinal Failure.

BACKGROUND: Home parenteral nutrition (HPN) provides life-sustaining treatment for people with chronic intestinal failure (CIF). Poor quality of life is reported in association with the burden of HPN and the underlying condition leading to intestinal failure (IF). However, levels of negative affect (NA), incorporating anxiety and depression, have not been reported in CIF. This study examined the occurrence and risk factors for NA in a large CIF population using the validated Hospital Anxiety & Depression Scale (HADS) and Multidimensional Scale of Perceived Social Support (MSPSS) questionnaires. METHODS: A survey pack including HADS and MSPSS were distributed to patients managed by a specialist IF center. Data from these measures were analyzed against factors including underlying disease, length of time receiving HPN, employment status, and demographics. RESULTS: Of 85 patients who completed this study, 56% had clinical levels of anxiety and/or depression. Linear regression analysis confirmed significant correlations between social support and HADS scores, with poorer perceived social support associated with higher levels of NA (-0.26 MSPSS + 33.24; R2 = 0.29; P < .0001). There were also significant associations between unemployment and higher anxiety (P = .004), depression (P = .008), and NA scores (P = .003). By contrast, there were no significant associations between patient age, duration of time receiving parenteral nutrition, pathophysiological mechanisms of CIF, and NA levels. CONCLUSIONS: Clinical anxiety and depression are common, affecting more than half of patients with CIF. Patients with poor social networks and those of working age who have ceased employment are particularly vulnerable and should be prioritized for psychological support because of their elevated risk.

Measurement of Utilities Associated with Parenteral Support Requirement in Patients with Short Bowel Syndrome and Intestinal Failure.

PURPOSE: Short bowel syndrome (SBS) is the main cause of intestinal failure. Intravenous supplementation (parenteral support [PS]) helps patients regain health but can affect patients' health-related quality of life (HRQoL). The value of health states associated with the number of days on PS per week is unknown in the United Kingdom. The objectives of the present study were to develop health state vignettes for SBS and PS, and to estimate health state utilities by using the time trade-off (TTO) technique. METHODS: Vignettes were developed and reviewed through various processes. Eight states described the impact of 0 days (weaned off PS) through to 7 days on PS; each state comprised the conditions, symptoms, treatments, and impacts related to EuroQol-5 dimensions. A sample of the UK general public viewed each state in interviews; they provided ratings using a visual analog scale and utility scores using the TTO. Participants completed background questionnaires. FINDINGS: One hundred participants rated and valued each health state. Visual analog scale and utility scores showed a steady decline for the health states associated with increasing numbers of days on PS. With "full health" equivalent to a utility score of 1, the most burdensome state was "7 days on PS" (mean [SD] utility score, 0.36 [0.35]), whereas weaned off ("0 days on PS") showed the least burden (0.82 [0.22]). IMPLICATIONS: More days of PS are perceived by members of the public to have an increasing negative impact on HRQoL. Therapies aimed at reducing the number of days on PS may be beneficial for improving patients' HRQoL.

The chronic intestinal pseudo-obstruction subtype has prognostic significance in patients with severe gastrointestinal dysmotility related intestinal failure.

BACKGROUND & AIMS: Severe gastrointestinal dysmotility (GID) is a significant cause of chronic intestinal failure (CIF) with unclear benefits of sub-classifying into Chronic Intestinal Pseudo-obstruction (CIPO) and non-CIPO sub-types. We compared outcomes between CIPO and non-CIPO sub-types in a tertiary cohort of patients with CIF resulting from severe GID. METHODS: Adults with primary GID, commenced on home parenteral nutrition (HPN) over a 16-year period at a national referral centre, were included. All patients satisfied GID clinical criteria which mandated evidence of small bowel involvement either objectively (abnormal antroduodenal manometry) or pragmatically (failure to progress on small bowel feeding). Clinical outcomes including HPN dependency and survival were compared between CIPO and non-CIPO sub-types. RESULTS: Patients with primary GID requiring HPN (n = 45, age 38 ± 2, 33 females, 23/45 (51%) CIPO, 22/45 (49%) non-CIPO) were included. Patients with CIPO had more surgical interventions (P = 0.03), higher incidence of bacterial overgrowth (P = 0.006), greater parenteral energy (P = 0.02) and volume requirements (P = 0.05). Overall, during a mean 6 years' follow-up, 36/45 (80%) patients remained HPN dependent. Multivariate analyses confirmed that the non-CIPO sub-type (P = 0.04) and catheter related blood stream infections/1000 days (P = 0.01) were predictive factors for time to discontinuing HPN. Overall 5-year survival on HPN was 85%, with no difference between sub-types (P = 0.83). CONCLUSIONS: The CIPO sub-type is associated with higher HPN dependency and should be recognized as a separate entity in severe GID. In multidisciplinary settings with continuous close monitoring of risks and benefits, our data confirm HPN is a safe, life-preserving therapy in severe GID related CIF.

The active problem solving of patients dependent on home parenteral nutrition: A qualitative analysis.

BACKGROUND & AIMS: People with chronic, type 3, intestinal failure often require long-term home parenteral nutrition (HPN). People receiving HPN have frequent interactions with their healthcare, due to the need for close monitoring and due to recurrent hospital admissions. Individuals' responses to, and interactions with, their health care service provides are poorly described. We conducted a service evaluation to explore people's experiences of HPN-related healthcare interactions in order to identify how service providers can best meet the individualised needs of patients. METHODS: We interviewed ten people receiving HPN. The participants were asked about their healthcare interactions related to HPN. The data were analysed using a qualitative research method known as interpretive phenomenological analysis. This analytical approach is concerned with the meaning that people ascribe to particular events, in this case their HPN-related healthcare interactions. RESULTS: The participants in this study described a range of psychosocial problems related to their HPN healthcare interactions, including reliance, risk and restrictions. Participants' solutions to these problems included adaptation of their routine, self-guardianship and passivity. Sometimes, these solutions generated secondary problems of their own, including concerns with resource use, negative healthcare interactions and conflicts of responsibility. A range of contextual factors influenced how participants interpreted their healthcare interactions and the solutions available to them. These contextual factors included continuity of healthcare professionals, the attitude of staff, and information and knowledge about HPN. CONCLUSIONS: By attending to the individual meaning ascribed to healthcare events, and the contextual factors that surround these events, we have been able to better understand the decisions made by patients dependent on HPN. This suggests that healthcare professionals may also better understand their patients' decision making by attending to the individual meaning that patients ascribe to healthcare events and to contextual factors. We propose a model to describe the process of problem -> resolution -> problem in which participants are actively engaged.