Costs of UK community care for individuals with recessive dystrophic epidermolysis bullosa: Findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study.

Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare inherited skin fragility disorder requiring multidisciplinary management. Information regarding costs of current standard treatment is scant. Objectives: As part of a longitudinal natural history study, we explored the community care costs of UK patients with different forms of RDEB. Methods: Fifty-nine individuals with RDEB provided detailed information on multiple facets of RDEB including disease severity scores (iscorEB, BEBS) and patient reported outcomes (quality of life evaluation in epidermolysis bullosa, iscorEB patient questionnaire). Costs data included time spent doing dressings, frequency of dressing changes, details of materials used, and paid and unpaid care. Results: Overall costs of dressing materials and associated care were high in RDEB. Median annual costs across all subtypes for those using dressings (n = 51) were over £26 000. For severe RDEB (RDEB-S), median costs were almost £90 000 per annum, with a median of 18 h per week spent on dressing changes. Half of working-age adults with RDEB were unemployed and 39% of carers were unable to take on full-time or part-time paid employment, adding to indirect costs and the financial burden from RDEB on families and society. Conclusions: The findings demonstrate the high costs of care of RDEB, particularly for RDEB-S. The current expense supports the drive to develop new therapies which accelerate wound healing and diminish total wound burden, thereby reducing costs of dressings and care. While costly to bring to market, these might ultimately reduce the overall cost of treatment and also the impact on individuals living with this rare disease. The data also highlight the need for adequate reimbursement for EB care which can place significant financial strain on families.

Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study.

BACKGROUND: Itch is common and distressing in epidermolysis bullosa (EB) but has not previously been studied in depth in different recessive dystrophic EB (RDEB) subtypes. OBJECTIVES: As part of a prospective register study of the natural history of RDEB we explored features of itch, medications used, and correlation with disease severity and quality of life. METHODS: Fifty individuals with RDEB aged 8 years and above completed the Leuven Itch Scale (LIS) (total 243 reviews over a 7-year period). Data included itch frequency, severity, duration, distress, circumstances, consequences, itch surface area and medications for itch. The iscorEB disease severity score and the validated EB quality of life tool, QOLEB, were compared to LIS domains and analysed by RDEB subtype. RESULTS: Itch was frequent, present in the preceding month in 93% of reviews. Itch severity and distress were significantly greater in severe (RDEB-S) and pruriginosa (RDEB-Pru) subtypes compared to intermediate RDEB (RDEB-I). Itch medications were reported in just over half of reviews including emollients, topical corticosteroids and antihistamines; the proportion of participants not using medication despite frequent pruritus suggests limited efficacy. In inversa RDEB (RDEB-Inv) and RDEB-I, LIS domains correlated with iscorEB and QOLEB. In contrast to previous studies, correlations were lacking in RDEB-S suggesting that global disease burden relatively reduces the contribution of itch. CONCLUSIONS: This comprehensive study of RDEB-associated itch highlights differences between RDEB subtypes, suggests an unmet need for effective treatments and could serve as control data for future clinical trials incorporating itch as an endpoint.

Clinical effectiveness of decongestive treatments on excess arm volume and patient-centered outcomes in women with early breast cancer-related arm lymphedema: a systematic review.

OBJECTIVE: To identify the effect of decongestive lymphedema treatment on excess arm volume or patient-centered outcomes in women presenting within either 12 months or a mean nine months of developing arm lymphedema following breast cancer treatment. INTRODUCTION: Lymphedema is a common consequence of breast cancer treatment requiring life-long treatment to reduce symptoms and prevent complications. Currently, evidence to inform the optimal decongestive lymphedema treatment package is lacking. INCLUSION CRITERIA: The review included studies on women who received lymphedema treatment within either 12 months or a mean of nine months of developing unilateral breast cancer-related arm lymphedema. The intervention was any decongestive lymphedema treatment delivered with the purpose of reducing arm lymphedema, compared to another form of lymphedema treatment (whether self or practitioner-administered), placebo or no treatment. The clinical outcome was excess arm volume; patient-centered outcomes were health-related quality of life, arm heaviness, arm function, patient-perceived benefit and satisfaction with treatment. Experimental study designs were eligible, including randomized and non-randomized controlled trials, quasi-experimental, prospective and retrospective before and after studies were considered. METHODS: A three-step search strategy was utilized to find published and unpublished studies. The search identified studies published from the inception of each database to July 6, 2016. Reference lists were scanned to identify further eligible studies. Studies were critically appraised using appropriate standardized critical appraisal instruments from the Joanna Briggs Institute. Details describing each study and treatment results regarding outcomes of interest were extracted from papers included in the review using appropriate standardized data extraction tools from the Joanna Briggs Institute. Due to heterogeneity in included studies, results for similar outcome measures were not pooled in statistical meta-analysis. A narrative and tabular format was used to synthesize results from identified and included studies. RESULTS: Seven studies reporting results for outcomes of interest were critically appraised and included in the review: five randomized controlled trials and two descriptive (uncontrolled) studies. Reported outcomes included excess arm volume (five studies), health-related quality of life (three studies), arm heaviness (one study), arm function (two studies) and patient-perceived benefit (two studies). There was some evidence that decongestive treatments were effective for women presenting within either 12 months or a mean of nine months of developing breast cancer-related arm lymphedema, but the wide range of data prevented comparison of treatment findings which limited our ability to answer the review questions. CONCLUSIONS: Weak evidence (grade B) for the impact of decongestive lymphedema treatment on women with early lymphedema (i.e. less than 12 months duration of BCRL symptoms) did not allow any conclusions to be drawn about the most effective treatment to be offered when these women first present for treatment. Findings provided no justification to support change to current practice.Future primary research needs to focus on the most effective treatment for women when they first present with lymphedema symptoms, e.g. treatment provided within 12 months of developing symptoms. Studies should be adequately powered and recruit women exclusively with less than 12 months duration of breast cancer-related lymphedema symptoms, provide longer follow-up to monitor treatment effect over time, with comparable treatment protocols, outcome measures and reporting methods.

Exploring patient perception of success and benefit in self-management of breast cancer-related arm lymphoedema.

PURPOSE: The aim of this study was to identify factors influencing patient perception of success and benefit with self-management of breast cancer-related lymphoedema (BCRL) and explore how patients decide whether their swollen limb has improved or deteriorated. METHODS: This qualitative study used a Grounded Theory approach. Twenty-one women in the BCRL self-management phase participated in one in-depth interview exploring their experience and perspective on self-managing their BCRL. FINDINGS: Seven enablers and blocks to self-management were identified: routine, recognising benefit of self-management and consequences of non-treatment, owning treatment, knowledge and understanding, problem-solving, time required for treatment and aesthetics of hosiery. Women determined treatment outcome by monitoring size, appearance, texture and internal sensations within the affected arm. CONCLUSIONS: Women who participated in this study showed varying degrees of acceptance and adjustment to life with lymphoedema. This appears to directly impact their ability to self-manage lymphoedema. Lymphoedema practitioners and oncology nurses have a valuable role providing knowledge and support to patients transitioning to independent self-care. A better understanding of factors facilitating patients to become experts in their condition may improve longer term outcomes and reduce cost pressures on lymphoedema services.

Treatment and outcomes of head and neck oedema referrals to a hospital-based lymphoedema service.

A retrospective review of all head and neck cancer-related lymphoedema (HNCRL) referrals from 1 October 2009 to 30 September 2013 was undertaken. HNCRL referrals significantly increased over a 4-year period. Lymphoedema clinic records were manually examined for the presentation of HNCRL, treatments provided, and outcomes. Some 207 HNCRL referrals were received, increasing by 251% from 2009 to 2013. A total of 110 men and 50 women were assessed and treated over the 4 years; 67% were discharged, 26% continued treatment/monitoring by clinic; 8% are deceased. The average time to discharge reduced from 16 to 5 months. From the results, it seems that self-management with exercise and counter-pressure/compression is very effective and the requirement for manual lymph drainage is reduced. There is increased patient engagement with effective self-care. Provision of appropriate education and information facilitated simple, effective self-management. This reduced treatment time, shortened time to discharge and released valuable practitioner time to treat patients with complex oedema.

Randomised controlled trial to determine the benefit of daily home-based exercise in addition to self-care in the management of breast cancer-related lymphoedema: a feasibility study.

Exercise is considered to be a key aspect of lymphoedema treatment, although there is little evidence for the therapeutic effect of exercise in managing breast cancer-related lymphoedema (BCRL). This small randomised controlled trial (RCT) was designed to determine the feasibility, prior to undertaking a larger RCT, of researching a daily home-based exercise programme to treat stable BCRL. An experimental design compared the exercise intervention combined with standard lymphoedema self-care to self-care alone over a 6-month period. Twenty-three women with stable unilateral BCRL of ≥10 % excess limb volume (ELV) were randomly allocated to a daily home-based exercise programme and self-care (n = 11) or self-care measures alone (n = 12). The primary objective was to determine difference in limb volume reduction for the two groups. Secondary objectives were to monitor change in other areas that impact BCRL: quality of life, arm function and range of shoulder movement. All 23 women completed the trial, providing full data for each time point. The intervention group showed a clinically and statistically significant improvement in relative ELV at week 26 (95 % confidence interval (CI) -26.57 to -5.12), whereas the control group improvement crossed the line of no effect (95 %CI -17.71 to 1.1). This study demonstrated the feasibility of conducting a RCT of exercise as a therapeutic intervention in the management of BCRL. Although the sample was small, the results support the findings of other exercise studies which have shown trends towards improvement.

Assessment of health-related quality of life in patients with lymphedema of the lower limb.

The purpose of this study was to examine the use of a number of tools in the evaluation of health-related quality of life in patients with lower limb lymphedema, and to determine the consequences of cancer history and concurrent leg ulceration. Patients in one health trust having lower limb lymphedema were identified and interviewed at entry and after 24 weeks. The short form-36 (SF-36), modified Barthel scale, McGill short form pain questionnaire, and Euroqol were administered at both time points. Of the 164 (median age=76.9 years, 70.7% women) patients who comprised the study population, 15.2% had a history of cancer and 30.4% had coexisting current leg ulceration. Internal consistencies were high for all scales (Cronbach's alpha >0.80). There were high ceiling effects for a number of SF-36 scores, and high floor effects in these and the McGill short form pain questionnaire, scales. Despite these limitations, there was strong evidence that treatment led to significant improvements in six of eight scores of the SF-36, three of three scores of the McGill short form pain questionnaire and the modified Barthel scale (all p<0.05). The improvement in physical functioning was significantly greater for patients who entered the study with a leg ulcer (mean different=9.1, 95% confidence interval 2.1-16.1, p=0.011). Patients treated with compression bandaging had significantly greater improvements for physical functioning (10.2) than those treated with compression hosiery (-1.5) or no treatment (-2.0), p=0.001. Of the tools assessed, the SF-36, appears to be the most appropriate for use in this patient group.

Treating breast cancer-related lymphoedema at the London Haven: clinical audit results.

Lymphoedema is recognised as a significant management problem for both patients and clinicians. Around 25% of those undergoing axillary intervention as treatment for breast cancer will go on to develop some degree of lymphoedema, which will impact their everyday life and wellbeing [Moffatt et al., 2003. Quarterly Journal of Medicine 96, 731-738; Mortimer et al., 1996. Quarterly Journal of Medicine 89, 377-380; Tobin et al., 1993. Cancer 72, 3248-3252]. This paper outlines an audit of the presenting characteristics of all 263 patients seen in The London Haven Lymphoedema Service (TLHLS) between 10th February 2000 and 4th June 2003 for treatment of breast cancer-related lymphoedema and reports outcomes of treatment: Results highlight the key issues around current practice at the London Haven and the need for further research in promising areas in lymphoedema management.