Quality of life and informal care burden associated with duchenne muscular dystrophy in Portugal: the COIDUCH study.

BACKGROUND: To describe the reduced health-related quality of life (HRQoL) of duchenne muscular dystrophy (DMD) patients and their caregiver burden and to present its relationship with disease progression. METHODS: This cross-sectional study assessed patient HRQoL with the 3-level version of the EuroQol-5D (EQ-5D-3L) and caregiver burden with the Work Productivity and Activity Impairment: General Health questionnaire. DMD patients and their caregivers were identified through Portuguese Neuromuscular Association (APN). RESULTS: A total of 46 DMD main caregivers, of eight ambulant and 38 non-ambulant patients, completed the questionnaires. Over half (58.7%) of all non-ambulant patients were on ventilation support, either full-time (15.2%) or non full-time (43.5%). Non-ambulant patients had a lower mean utility scores than ambulant patients (- 0.05 versus 0.51, p value < 0.001). Caregivers of non-ambulant patients reported a significant mean daily activity impairment as compared to caregivers of ambulant patients (68% versus 23%, p value < 0.001). Among non-ambulant patients, both utility scores and caregiver impairment appeared to deteriorate according to a higher need for ventilation support, however, these results were not statistically significant. CONCLUSIONS: These results emphasise the significant negative impact that DMD progression has on the patient HRQoL, as well as caregivers' ability to conduct their daily activities. Therapeutic options that stop or slow the disease progression could have a beneficial impact for both patients and caregivers.

Headache related to personal protective equipment in healthcare workers during COVID-19 pandemic in Mexico: baseline and 6-month follow-up.

BACKGROUND AND AIM: Headaches related to the use of personal protective equipment (PPE) could affect performance at work in healthcare personnel. Our aim was to describe the prevalence and risk factors for headaches related to PPE, in the personnel of a specialized coronavirus disease 2019 (COVID-19) tertiary hospital. METHODS: In this cross-sectional survey study, we invited healthcare workers from COVID-19 referral center in Mexico (May 22-June 19, 2020) to answer a standardized structure questionnaire on characteristics of new-onset PPE-related headache or exacerbation of primary headache disorder. Participants were invited regardless of whether they had a current headache to avoid selection bias. This is the primary analysis of these data. RESULTS: Two hundred and sixty-eight subjects were analyzed, 181/268 (67.5%) women, 177/268 (66%) nurses, mean age 28 years. The prevalence of PPE-related headache was 210/268 (78.4%). Independent risk factors were occupation other than physician (OR 1.59, 95% CI 1.20-2.10), age > 30 years (OR 2.54, 95% CI 1.25-5.14), and female sex (OR 3.58, 95% CI 1.86-6.87). In the 6-month follow-up, 13.1% of subjects evolve to chronic headache, with stress as predictive risk factor. CONCLUSION: The frequency of PPE-associated headache is high, and a subgroup could evolve to chronic headache. More studies are necessary to improve the knowledge about this condition.

Adherence of mental therapy for mental disorder patients to drug health treatment [corrected]. / Adesão do portador de transtorno mental à terapêutica medicamentosa no tratamento em saúde menta.

OBJECTIVE: To assess the adherence of mental disorder patients to drug therapy for mental health treatment and the association between demographic, socioeconomic, clinical and pharmacotherapeutic variables to treatment adherence. METHOD: A cross-sectional study conducted with mental disorder patients in two Psychosocial Care Centers in Curitiba/Paraná in 2014. Data from structured interviews and medical records were submitted to descriptive and bivariate analysis. RESULTS: 300 patients with mental disorders participated in the study. 51% of participants adhered to the drug therapy, the highest adherence was among males with no family history of mental disorder, diagnosed with schizophrenia, with disease duration of less than 1 year, who did not forget to take the medicine not even once in the previous month and who relied on family participation. Adherence was lower among the interviewees with individual income lower than one minimum wage, perception of regular and poor health, diagnosis of depression associated with another disorder, treatment time in the service over 2 years and with a history of attempted suicide. CONCLUSION: Low adherence to the drug therapy was observed. The variables associated with adherence were gender, individual income, family history of mental disorder, perception about their health, diagnosis of mental disorder, duration of illness and treatment, suicide attempt, failing to take the medication at least once in the previous month and family participation.

Social and economic value of Portuguese community pharmacies in health care.

BACKGROUND: Community pharmacies are major contributors to health care systems across the world. Several studies have been conducted to evaluate community pharmacies services in health care. The purpose of this study was to estimate the social and economic benefits of current and potential future community pharmacies services provided by pharmacists in health care in Portugal. METHODS: The social and economic value of community pharmacies services was estimated through a decision-model. Model inputs included effectiveness data, quality of life (QoL) and health resource consumption, obtained though literature review and adapted to Portuguese reality by an expert panel. The estimated economic value was the result of non-remunerated pharmaceutical services plus health resource consumption potentially avoided. Social and economic value of community pharmacies services derives from the comparison of two scenarios: "with service" versus "without service". RESULTS: It is estimated that current community pharmacies services in Portugal provide a gain in QoL of 8.3% and an economic value of 879.6 million euros (M€), including 342.1 M€ in non-remunerated pharmaceutical services and 448.1 M€ in avoided expense with health resource consumption. Potential future community pharmacies services may provide an additional increase of 6.9% in QoL and be associated with an economic value of 144.8 M€: 120.3 M€ in non-remunerated services and 24.5 M€ in potential savings with health resource consumption. CONCLUSIONS: Community pharmacies services provide considerable benefit in QoL and economic value. An increase range of services including a greater integration in primary and secondary care, among other transversal services, may add further social and economic value to the society.

[Cholelithiasis during pregnancy and postpartum: prevalence, presentation and consequences in a Referral Hospital in Baja California Sur]. / Colelitiasis en el embarazo y posparto. Prevalencia, presentación y consecuencias en un hospital de referencia en Baja California Sur.

INTRODUCTION: Pregnancy and the postpartum period are risk factors for developing biliary sludge, gallstones, and any of their complications. OBJECTIVE: To determine the prevalence, presentation, and consequences of cholestasis during pregnancy and postpartum in a referral hospital of Baja California Sur. MATERIAL AND METHODS: This was a retrospective, observational study that enrolled pregnant or postpartum patients diagnosed with gallstones with any presentation. RESULTS: 137 patients were included, with 22 ± 4 years of age; 33 were pregnant and 104 in the postpartum period. Only 14% of the group had a history of cholelithiasis, and overweight/obesity was observed in 66.7 and 66.3% of pregnant and postpartum patients, respectively (p = 0.94). Of pregnant patients, 33.3% presented with acute cholecystitis, a condition observed in 16.3% of the postpartum patients (p = 0.04). Pancreatitis and choledocholithiasis were slightly more common in pregnant women (21.23% vs. 19.2%; p = 0.56). There was no maternal mortality and one case of spontaneous abortion was exclusively observed. CONCLUSIONS: It is a priority to diagnose and monitor cholelithiasis in pregnant women because the acute cases observed occurred more frequently, but choledocholithiasis and pancreatitis occurred similarly in both groups.

Prevalence of dry eye syndrome in residents of surgical specialties.

BACKGROUND: The aim of this study was to determine the prevalence and severity of dry eye syndrome in a group of Mexican residents of different surgical specialties. METHODS: A cross-sectional descriptive study where the residents were studied using the Ocular Surface Disease Index, together with diagnostic tests for dry eye syndrome, such as tear breakup time, Oxford Schema, Schirmer's test I, and meibomian gland dysfunction testing. Statistical analyses were performed by Pearson's chi-squared test for categorical variables and student's t-test for quantitative variables. Any P value < 0.05 was considered statistically significant. RESULTS: One hundred and twenty-three residents were included (246 eyes); 90 (73 %) were male and 33 (27 %) were female. The mean age was 27.8 ± 2.1 years. A higher number of residents with dry eye syndrome was found in the cardiothoracic surgery (75 %) and otorhinolaryngology (71 %) specialties; 70 % of them reported ocular symptoms, with teardrop quality involvement in >50 % of them. CONCLUSIONS: We found a prevalence of 56 % for mild-to-moderate/severe stages of the condition. Their presence in the operating room predisposes surgical residents to dry eye syndrome because of environmental conditions.

Hyaluronic acid covers in burn treatment: a systematic review.

OBJECTIVE: To evaluate the effectiveness of hyaluronic acid in the healing of partial thickness burns. METHOD: Systematic review of randomized controlled trials on the use of hyaluronic acid for the topical treatment of skin burns, based on recommendations of the Cochrane Handbook for Systematic Reviews of Interventions. RESULTS: Two randomized controlled trials that analyzed 143 patients with partial thickness burns and/or deep partial thickness burns were selected. They compared the application of hyaluronic acid 0.2% associated to silver sulfadiazine 1% 5g/cm2 versus silver sulfadiazine 1% 5g/cm2 alone for the outcome of complete healing. CONCLUSION: This review emphasizes the need for new well-designed randomized controlled trials to establish the therapeutic relevance of hyaluronic acid with respect to the healing of burns of partial thickness or deep partial thickness. OBJETIVO: Avaliar a efetividade do ácido hialurônico na cicatrização de queimaduras de espessura parcial. MÉTODO: Revisão sistemática de ensaios clínicos randomizados sobre a utilização de ácido hialurônico no tratamento tópico de queimaduras de pele, baseada nas recomendações do Cochrane Handbook for Systematic Reviews of Interventions. RESULTADOS: Foram recuperados dois ensaios clínicos randomizados que analisaram 143 pacientes portadores de queimaduras de espessura parcial e/ou espessura parcial profunda, comparando a aplicação de Ácido Hialurônico 0,2% associado à Sulfadiazina de Prata 1% 5g/cm2, versus Sulfadiazina de Prata 1% 5g/cm2 isolada, para o desfecho cicatrização completa. CONCLUSÃO: Esta revisão enfatiza a necessidade de novos ensaios clínicos randomizados bem delineados para estabelecimento da relevância terapêutica do ácido hialurônico no que tange à cicatrização de queimaduras de espessura parcial ou espessura parcial profunda.

Rectal indomethacin versus placebo to reduce the incidence of pancreatitis after endoscopic retrograde cholangiopancreatography: results of a controlled clinical trial.

BACKGROUND: Acute pancreatitis is the most common major complication after endoscopic retrograde cholangiopancreatography (ERCP). Many drugs have been evaluated for prophylaxis, including nonsteroidal anti-inflammatory drugs (NSAIDs), which are potent inhibitors of phospholipase A2 and play a role in the pathogenesis of acute pancreatitis. Rectal NSAIDs have been shown in prospective studies to decrease the incidence of this complication, but the indication is not generalized in clinical practice. The aim of this study was to evaluate the efficacy of rectal administration of indomethacin in reducing the incidence of post-ERCP pancreatitis in high-risk patients. METHODS: This was a controlled clinical trial where patients with an elevated risk of developing post-ERCP pancreatitis were assigned to receive 100 mg of rectal indomethacin or a 2.6 g suppository of glycerin immediately after ERCP, without placement of a pancreatic stent. The patients were determined to be at high risk based on validated patient- and procedure-related risk factors. Post-ERCP pancreatitis was defined as the presence of new upper abdominal pain, hyperamylasemia/hyperlipasemia (at least three times the upper limit) 2 hours after the procedure and hospitalization at least 48 hours because of the complication. Pancreatitis severity was defined according to Cotton's criteria. RESULTS: One hundred sixty-six patients were included; 82 in the study group and 84 in the placebo group. Patients had at least one major and/or two minor risk factors for developing post-ERCP pancreatitis. The incidence of the complication was 4.87% (4/82) in the study group and 20.23% (17/84) in the placebo group; this difference was significant (P = 0.01). According to Cotton's criteria, 17 patients (80.9%) developed mild pancreatitis and 4 (19.1%) had moderate pancreatitis; 3 of these 4 patients belonged to the placebo group (P = 0.60). Based on these results, an absolute risk reduction of 0.15 (15%), a relative risk reduction of 0.75 (75%) and a number needed to treat of 6.5 patients were calculated to prevent an episode of post-ERCP pancreatitis. There was no mortality. CONCLUSIONS: Rectal indomethacin reduced the incidence of post-ERCP pancreatitis among patients at high risk of developing this complication. TRIAL REGISTRATION: National Clinical Trials NCT02110810. Date April 7, 2014.

Efficacy of metronidazole versus placebo in pain control after hemorrhoidectomy. Results of a controlled clinical trial.

INTRODUCTION: Hemorrhoidal disease occurs in 50% of people aged > 40 years and is the most common reason for anorectal surgery. Pain is the main complication. Multiple topical and systemic drugs have been investigated for pain control, but there is no ideal treatment. Metronidazole has been shown to decrease postoperative pain but is not used widely. OBJECTIVE: To evaluate the effect of oral metronidazole versus placebo and to assess postoperative pain following hemorrhoidectomy. MATERIAL AND METHODS: Controlled clinical trial in adult patients who underwent elective hemorrhoidectomy for grade III/IV hemorrhoids. Patients were assigned to receive metronidazole (500 mg q8 h orally; study group, SG) or placebo (control group, CG) for 7 days after surgery. Pain was assessed using a visual analog scale after surgery. Analgesic administration (time and use of analgesics) and resumption of daily life activities were also assessed. RESULTS: Forty-four patients were included, 22 in each group. Postoperative pain differed significantly between the SG and CG at 6 h (3.86 ± 0.56, 6.64 ± 1.49), 12 h (5.59 ± 1.33, 8.82 ± 0.79), 24 h (6.86 ± 1.49, 9.73 ± 0.45), day 4 (5.32 ± 2.10, 9.50 ± 0.59), day 7 (3.14 ± 1.03, 7.36 ± 1.39), and day 14 (2.14 ± 0.46, 5.45 ± 1.29). The first analgesia dose was required at 21.27 ± 5.47 h in the CG and 7.09 ± 2.36 h in the SG (p < 0.05), the time of analgesic use was 6.86 ± 1.61 days in the CG and 13.09 ± 2.48 days in the SG (p < 0.05), and resumption of daily activities occurred at 7.59 ± 1.56 days in the CG and 14.73 ± 3.76 days in the SG (p < 0.05). CONCLUSION: Oral administration of metronidazole is effective in pain management after hemorrhoidectomy.

Time-dependent endpoints as predictors of overall survival in multiple myeloma.

BACKGROUND: Supporting health care sector decisions using time-dependent endpoints (TDEs) such as time to progression (TTP), progression-free survival (PFS), and event-free survival (EFS) remains controversial. This study estimated the quantitative relationship between median TDE and median overall survival (OS) in multiple myeloma (MM) patients. METHODS: Studies (excluding allogeneic transplantation) published from 1970 to 2011 were systematically searched (PubMed). The nonparametric Spearman's rank correlation coefficient measured the association between median TDE and OS. The quantitative relationship between TDEs and OS was estimated with a two-step approach to a simultaneous Tobit model. RESULTS: We identified 153 studies: 230 treatment arms, 22,696 patients and mean study duration of 3.8 years. Mean of median TDEs was 22.5 months and median OS was 39.1 months. Correlation coefficients of median TTP, PFS, and EFS with median OS were 0.51 (P = 0.003), 0.75 (P < 0.0001), and 0.84 (P < 0.0001), respectively. We estimate a 2.5 month (95% confidence interval, 1.7-3.2) increase in median OS for each additional month reported for median TDEs. There was no evidence that this relationship differed by type of surrogate. CONCLUSION: TDEs predict OS in MM patients; this relationship may be valuable in clinical trial design, drug comparisons, and economic evaluation.

Epidemiological Modeling of the Impact of Public Health Policies on Hepatitis C: Protocol for a Gamification Tool Targeting Microelimination.

BACKGROUND: Hepatitis C is a disease with a strong social component, as its main transmission route is via blood, making it associated with lifestyle. Therefore, it is suitable to be worked on from the perspective of public health policy, which still has a lot of room to explore and improve, contrary to diagnoses and treatments, which are already very refined and effective. OBJECTIVE: An interactive gamified policy tool, designated as Let's End HepC (LEHC), was created to understand the impact of policies related to hepatitis C on the disease's epidemiology on a yearly basis until 2030. METHODS: To this end, an innovative epidemiological model was developed, integrating Markov chains to model the natural history of the disease and adaptive conjoint analysis to reflect the degree of application of each of the 24 public health policies included in the model. This double imputation model makes it possible to assess a set of indicators such as liver transplant, incidence, and deaths year by year until 2030 in different risk groups. Populations at a higher risk were integrated into the model to understand the specific epidemiological dynamics within the total population of each country and within segments that comprise people who have received blood products, prisoners, people who inject drugs, people infected through vertical transmission, and the remaining population. RESULTS: The model has already been applied to a group of countries, and studies in 5 of these countries have already been concluded, showing results very close to those obtained through other forms of evaluation. CONCLUSIONS: The LEHC model allows the simulation of different degrees of implementation of each policy and thus the verification of its epidemiological impact on each studied population. The gamification feature allows assessing the adequate fulfillment of the World Health Organization goals for the elimination of hepatitis C by 2030. LEHC supports health decision makers and people who practice patient advocacy in making decisions based on science, and because LEHC is democratically shared, it ends up contributing to the increase of citizenship in health. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): RR1-10.2196/38521.

Cost-effectiveness Analysis of Ocrelizumab for the Treatment of Relapsing and Primary Progressive Multiple Sclerosis in Portugal.

OBJECTIVES: Ocrelizumab demonstrated significant clinical benefit for the treatment of relapsing (RMS) and primary progressive (PPMS) multiple sclerosis (MS), an incurable disease characterized by disability progression. This study evaluated the clinical and economic impact of ocrelizumab relative to current clinical practice, including other disease-modifying therapies (DMT), available in Portugal. METHODS: Markov models for MS were adapted to estimate the impact of ocrelizumab across three patient populations: treatment-naïve RMS, previously treated RMS, and PPMS. Health states were defined according to the Expanded Disability Status Scale. For RMS, the model further captured the occurrence of relapses and progression to secondary progressive multiple sclerosis (SPMS). A lifetime time-horizon and Portuguese societal perspective were adopted. RESULTS: For RMS patients, ocrelizumab was estimated to maximize the expected time (years) without progression to SPMS (10.50) relative to natalizumab (10.10), dimethyl fumarate (8.64), teriflunomide (8.39), fingolimod (8.38), interferon ß-1a (8.33) and glatiramer acetate (8.18). As the most effective option, with quality-adjusted life year (QALY) gains between 0.3 and 1.2, ocrelizumab was found to be cost-saving relative to natalizumab and fingolimod, and presented incremental cost-effectiveness ratios (ICER) below €16,720/QALY relative to the remaining DMT. For PPMS patients, the ICER of ocrelizumab versus best supportive care was estimated at €78,858/QALY. CONCLUSIONS: Ocrelizumab provides important health benefits for RMS and PPMS patients, comparing favourably with other widely used therapies. In RMS, ocrelizumab was revealed to be either cost-saving or have costs-per-QALY likely below commonly accepted cost-effectiveness thresholds. In PPMS, ocrelizumab fills a clear clinical gap in the current clinical practice. Overall, ocrelizumab is expected to provide good value for money in addressing the need of MS patients.

Hypertrophic cardiomyopathy in an adult patient with Noonan syndrome with multiple lentigines.

Noonan syndrome with multiple lentigines (NSML) is a rare RASopathy caused by pathogenic variants (PV) predominantly in PTPN11 gene. We report a 54-year-old male with apical hypertrophic cardiomyopathy, who was diagnosed with NSML due to his short stature, multiple lentigines, winged neck, pectus excavatum, and a heterozygous PV in PTPN11 c.836A > ¡G.

Review and Assessment of Policy Options for Improving Access to Combination Therapies in Oncology in Europe.

OBJECTIVES: Combinations of on-patent therapies (CTs) are increasingly common in oncology. They cause challenges for funding and affordability, and hence patient access, especially when constituent therapies are owned by different manufacturers. The aim of our study was to develop policy proposals for the assessment, pricing, and funding of CTs and identify which might be relevant in different European countries. METHODS: Following a review of available literature, seven hypothetical policy proposals were developed and subsequently assessed through 19 semi-structured interviews with health policy, pricing, technology assessment and legal experts in seven European countries to identify those most likely to gain traction. RESULTS: Experts saw a need for agreed approaches within a country to manage affordability and funding challenges for CTs. Changes to health technology assessment (HTA) and funding models were considered unlikely, but other policy proposals were seen as mostly useful, with country-specific adaptations. Bilateral discussions between manufacturers and payers were deemed important, and less challenging and protracted than arbitrated dialogue between manufacturers. Usage-specific pricing, possibly using weighted average prices, was considered a prerequisite for the financial management of CTs. CONCLUSIONS: There is a growing need to ensure that CTs are affordable to health systems. It would appear that there is no one set of policies that is appropriate for all countries in Europe, so countries wishing to ensure that patients have (or continue to have) access to CTs of value to them must explore and implement the policies that are best suited to their general approach to funding health care and to the assessment and reimbursement of medicines.

Hope and depression in Brazilian head and neck cancer patients during the COVID-19 pandemic.

Introduction: Head and neck cancer is characterised as traumatic, when compared to other types of cancer, due to the physical, physiological and social impact it has on the patient. Objective: To evaluate hope and severe depression in patients with head and neck cancer during the COVID-19 pandemic. Method: Quantitative, observational, and analytical; conducted in the outpatient department of the head and neck department of an oncological hospital in the city of Curitiba, Paraná, Brazil, with 60 patients with head and neck cancer being treated with chemotherapy and radiotherapy. Data collection took place between May and September 2020, with the application of three questionnaires: socio-demographic and clinical; Patient Health Questionnaire-9 (PHQ-9), to evaluate severe depression; and the Herth hope scale. Data analysis was through descriptive statistics and non-parametric Mann- Whitney, Kruskal-Wallis and Spearman correlation tests. Results: The age of participants ranged from 34 to 85 years, with 61.7% being male. The diagnosis of cancer occurred in the oral cavity (53.3%) and larynx (33.3%); 28.3% (n = 1 7) presented with a diagnosis of depression; 8.6% (n = 5) are in treatment with psychology; and 10.3% (n = 6) are in treatment with psychotropic drugs. The analysis of the association of the PHQ-9 score with sex showed a mean score of 7.7 ± 6.2, with a higher level of depression in women. The mean hope score was 41.3 ± 3.1; however, during the pandemic, 35% (n = 21) reported feelings of anguish, anxiety and fear, with the latter being predominant and in association with the PHQ-9 score showing a mean of 8.2 ± 6.2 (p = 0.123). The association of hope with the number of children was statistically significant (p = 0.034) and in the education variable with the PHQ-9 score (p = 0.019). Conclusion: The use of tools that assess both levels of hope and depression in patients undergoing chemotherapy and radiotherapy provides health professionals with support for the implementation of targeted actions to cope with the disease.

The Healthcare Failure Mode and Effect Analysis as a tool to evaluate care protocols.

OBJECTIVES: to identify, classify, and analyze modes of failure in the medication process. METHODS: evaluative research that used the Healthcare Failure Mode and Effect Analysis (HFMEA) in a service of bone marrow transplant from June to September 2018, with the participation of 35 health workers. RESULTS: 207 modes of failure were identified and classified as mistakes in verification (14%), scheduling (25.6%), administration (29%), dilution (16.4%), prescription (2.4%), and identification (12.6%). The analysis of risk showed a moderate (51.7%) and high (30.9%) need of intervention, leading to the creation of an internal quality assurance group and of continued education activities. CONCLUSIONS: the Healthcare Failure Mode and Effect Analysis showed itself to be a tool to actively identify, classify, and analyze failures in the process of medication, contributing for the proposal of actions aimed at patient safety.

Cost of Illness in Patients with Duchenne Muscular Dystrophy in Portugal: The COIDUCH Study.

OBJECTIVE: The aim of this study was to estimate the cost of illness (COI) of Duchenne muscular dystrophy (DMD) and its relation to disease progression, using age as a proxy, and according to the ambulatory status of patients. METHODS: We conducted a cross-sectional study of patients diagnosed with DMD identified through the Portuguese Neuromuscular Patients Association (APN). Data regarding patient and caregiver demographics, patient health status, resource utilization and cost, and informal care were collected using a custom semistructured questionnaire. Labor productivity and absenteeism losses were captured using the Work Productivity and Activity Impairment questionnaire. Costs were valued using a societal perspective. RESULTS: A total of 46 patient-caregiver pairs were included, of which eight of the patients were ambulant and 38 were nonambulant. Age had a decreasing effect on COI, independent of the patient's disease stage. Annualized lifetime costs were at their highest in nonambulant patients around the mean age of loss of ambulation (10 years of age). The mean per patient stage-specific costs (year 2019 values) of DMD were estimated at €48,991 in the nonambulant stage and €19,993 in the ambulant stage. Direct nonmedical costs were the main cost drivers, followed by indirect costs. CONCLUSIONS: Our results indicate a close relation between overall disease costs and disease progression. DMD is associated with a substantial economic burden, which appears to be larger around the time ambulation is lost (10 years of age). The availability of new therapeutic options that delay disease progression, especially loss of ambulation, may prove to be highly beneficial for not only patients with DMD but also their families and society.

Assessment of quality of life three years from hematopoietic stem cell transplant.

OBJECTIVE: To assess the domains of quality of life related to hematologic cancer patient health in the first three years from autologous and allogeneic hematopoietic stem cell transplantation. METHOD: A prospective cohort from September 2013 to February 2019 at a reference service in Latin America with 55 patients. The instruments Quality of Life Questionnaire Core C30 and Functional Assessment Cancer Therapy - Bone Marrow Transplantation were used. For data analysis, Generalized Linear Mixed Model was used. RESULTS: The domains global and overall quality of life presented the lowest scores in the pancytopenia phase: 59.3 and 91.4 in autologous, 55.3 and 90.3 in allogeneic. The mixed method analysis has shown that there was a significant change in scores between the phases throughout the treatment (p< 0.05). CONCLUSION: Health-related quality of life presented significant changes in the domains between the phases throughout time. Understanding these results enables nursing interventions directed at the domains which were damaged during treatment.

Guided imagery relaxation in quality of life of patients undergoing hematopoietic stem cell transplantation: a quasi-experiment.

OBJECTIVE: The aim of this study was to evaluate the effects of the relaxation technique with guided imagery by means of virtual reality on health-related quality of life in patients undergoing hematopoietic stem cell transplantation. METHODS: A quasi-experiment conducted in a Bone Marrow Transplantation Service of a public hospital in southern Brazil. From October 2019 to October 2020, forty-two adult participants who underwent transplantation were included, 35 in the intervention group and seven in the control group. A guided imagery intervention, with audio guiding the relaxation associated with nature images in 360º, was performed during the hospitalization period. Data were collected on the first day of hospitalization, on the transplantation day, during the neutropenia stage, and at pre-hospital discharge. The Functional Assessment of Cancer Therapy-Bone Marrow Transplantation (FACT-BMT), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) and Functional Assessment of Cancer Therapy-Neutropenia (FACT-N) were used to assess health-related quality of life, fatigue and neutropenia. Data were analyzed using the Generalized Linear Mixed Model for the evolution of the health-related quality of life assessments over time, considering the groups and stages. Pearson's correlation coefficient was adopted for the correlation analyses. RESULTS: Allogeneic transplantation was predominant: 28 (80%) in the intervention group and 5 (71.43%) in the control group. There were improvements in the health-related quality of life scores, although not significant. A significant difference was found among the stages (p <0.050) and a significant positive correlation (p <0.000) among the variables on general quality of life, additional concerns, fatigue and neutropenia in all stages. CONCLUSION: Patients undergoing hematopoietic stem cell transplantation suffer changes in their quality of life. Interventions based on integrative practices emerge as an option to minimize them. 
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Quality of life and changes in the social dimension of hematopoietic stem cell transplants recipients.

OBJECTIVES: to assess/correlate health-related quality of life with the social dimension of hematopoietic, autologous and allogeneic stem cell transplant patients in the three years post-transplant. METHODS: longitudinal, observational study with 55 patients, in a reference hospital in Latin America, from September 2013 to February 2019, using the Quality of Life Questionnaire-Core and Functional Assessment Cancer Therapy Bone Marrow Transplantation. RESULTS: A total of 71% underwent allogeneic transplantation. The social dimension had low averages since the baseline stage (55, 21) and low scores (56) for quality of life in pancytopenia. There was a significant positive correlation between social dimension, quality of life in pancytopenia (p<0.01) and follow-up after hospital discharge (p<0.00). There is a significant difference (p<0.00) throughout the stages, however, not in terms of the type of transplant (p>0.36/0.86). CONCLUSIONS: patients with better assessments in the social dimension have a better quality of life. Interventions focusing on the multidimensionality of the quality of life construct are necessary.