Hydroxyurea and sickle cell anemia: effect on quality of life

BACKGROUND: The Multicenter Study of Hydroxyurea (HU) in Sickle Cell Anemia (MSH) previously showed that daily oral HU reduces painful sickle cell (SS) crises by 50% in patients with moderate to severe disease. The morbidity associated with this disease is known to have serious negative impact on the overall quality of life(QOL) of affected individuals. METHODS: The data in this report were collected from the 299 patients enrolled in the MSH. Health quality of llife (HQOL) measures were assessed in the MSH as a secondary endpoint to determine if the clinical benefit of HU could translate into a measurable benefit perceptible to the patients. HQOL was assessed with the Profile of Mood States, the Health Status Short Form 36 (SF-36), including 4-week pain recall, and the Ladder of Life, self-administered twice 2-weeks apart pre-treatment and every 6 months during the two-year, randomized, double-blind, treatment phase. The effects of factors including randomized treatment, age, gender, pre-treatment crises frequency, Hb-F level mean, daily pain from 4-week pre-treatment diaries, and 2-year Hb-F response level (low or high) were investigated. RESULTS: Over two years of treatment, the benefit of HU treatment on QOL, other than pain scales, was limited to those patients taking HU who maintained a high HbF response, compared to those with low HbF response or on placebo. These restricted benefits occurred in social function, pain recall and general health perception. Stratification according to average daily pain prior to treatment showed that responders to HU whose average daily pain score was 5–9 (substantial pain) achieved significant reduction in the tension scale compared to the placebo group and to non-responders. HU had no apparent effect on other QOL measures. CONCLUSION: Treatment of SS with HU improves some aspects of QOL in adult patients who already suffer from moderate-to-severe SS

Quality of life at the end of life

<p>Abstract</p> <p>Background</p> <p>Little is known about self-perceived quality of life (QOL) near the end of life, because such information is difficult to collect and to interpret. Here, we describe QOL in the weeks near death and determine correlates of QOL over time, with emphasis on accounting for death and missing data.</p> <p>Methods</p> <p>Data on QOL were collected approximately every week in an ongoing randomized trial involving persons at the end of life. We used these data to describe QOL in the 52 weeks after enrollment in the trial (prospective analysis, N = 115), and also in the 10 weeks just prior to death (retrospective analysis, N = 83). The analysis consisted of graphs and regressions that accounted explicitly for death and imputed missing data.</p> <p>Results</p> <p>QOL was better than expected until the final 3 weeks of life, when a terminal drop was observed. Gender, race, education, cancer, and baseline health status were not significantly related to the number of “weeks of good-quality life” (WQL) during the study period. Persons younger than 60 had significantly higher WQL than older persons in the prospective analysis, but significantly lower WQL in the retrospective analysis. The retrospective results were somewhat sensitive to the imputation model.</p> <p>Conclusion</p> <p>In this exploratory study, QOL was better than expected in persons at the end of life, but special interventions may be needed for persons approaching a premature death, and also for the last 3 weeks of life. Our descriptions of the trajectory of QOL at the end of life may help other investigators to plan and analyze future studies of QOL. Methodology for dealing with death and the high amount of missing data in longitudinal studies at the end of life needs further investigation.</p

An open-label, multicenter study to evaluate the safe and effective use of the single-use autoinjector with an Avonex® prefilled syringe in multiple sclerosis subjects

<p>Abstract</p> <p>Background</p> <p>The ability to self-inject in patients with multiple sclerosis (MS) has been associated with a reduced risk of missed injections and drug discontinuation, and a beneficial effect on patients' independence. However, injection anxiety, needle phobia and disease-related disability are major barriers to a patient's ability to self-administer treatment. Use of an autoinjector may improve patients' ability to self-inject. This study evaluated the safe and effective use of Avonex Pen™ (prefilled pen), a single use autoinjector, for intramuscular delivery of interferon beta-1a (IM IFNβ-1a, Avonex) in MS patients.</p> <p>Methods</p> <p>This was a Phase IIIb, open-label, single-country, multicenter trial in MS patients currently using IM IFNβ-1a prefilled syringes. Patients received weekly 30 mcg IM IFNβ-1a treatment over 4 weeks. On Day 1, patients self-administered IM IFNβ-1a using a prefilled syringe at the clinic. On Day 8, patients received training on the prefilled pen and self-administered IM IFNβ-1a using the device. On Day 15, patients self-administered IM IFNβ-1a at home using the prefilled pen. A final injection occurred at the clinic on Day 22 when patients self-administered IM IFNβ-1a using the prefilled pen while clinic staff observed and completed a detailed questionnaire documenting patients' ability to self-inject with the device. Serum neopterin levels were evaluated pre and post-injection on Days 1 and 8. Adverse events were monitored throughout.</p> <p>Results</p> <p>Seventy-one (96%) patients completed the study. The overall success rate in safely and effectively using the prefilled pen was 89%. No device malfunctions occurred. One unsuccessful administration occurred at Day 22 due to patient error; no patient injury resulted. Patients gave the prefilled pen high ratings (8.7-9.3) on a 10-point scale for ease of use (0 = extremely difficult, 10 = extremely easy). Ninety-four percent of patients preferred the prefilled pen over the prefilled syringe. Induction of serum neopterin levels, serving as a biomarker for type 1 interferon action, was similar to that of the prefilled syringe. The prefilled pen demonstrated a safety profile comparable to the prefilled syringe.</p> <p>Conclusions</p> <p>The prefilled pen is a safe and effective device for administration of IM IFNβ-1a and represents an alternative method for self-injection for MS patients using this therapy.</p> <p>Trial registration</p> <p>This study is registered at clinicaltrials.gov, identifier: <a href="http://www.clinicaltrials.gov/ct2/show/NCT00828204">NCT00828204</a></p

Measuring patient-reported outcomes: moving beyond misplaced common sense to hard science

Interest in the patient's views of his or her illness and treatment has increased dramatically. However, our ability to appropriately measure such issues lags far behind the level of interest and need. Too often such measurement is considered to be a simple and trivial activity that merely requires the application of common sense. However, good quality measurement of patient-reported outcomes is a complex activity requiring considerable expertise and experience. This review considers the most important issues related to such measurement in the context of chronic disease and details how instruments should be developed, validated and adapted for use in additional languages. While there is often consensus on how best to undertake these activities, there is generally little evidence to support such accord. The present article questions these orthodox views and suggests alternative approaches that have been shown to be effective

Impacto na qualidade de vida em pacientes com hanseníase: correlação do Dermatology Life Quality Index com diversas variáveis relacionadas à doença Impact on the quality of life of patients with Hansen’s disease: correlation between Dermatology Life Quality Index and disease status

FUNDAMENTOS: A hanseníase é doença infectocontagiosa crônica causada pelo Micobacterium leprae. Caracteriza-se por acometimento dermatoneurológico, variando em espectro entre dois pólos estáveis. Apesar de curável, ainda representa relevante problema de saúde pública, sendo o Brasil o segundo país mais endêmico do mundo. Sua maior morbidade associa-se aos estados reacionais e ao acometimento neural, podendo causar incapacidades físicas permanentes que comprometem significativamente a qualidade de vida dos pacientes, com auto-estigmatização e vergonha. OBJETIVO: Avaliar o grau de comprometimento da qualidade de vida nos pacientes com hanseníase. MÉTODOS: Estudo observacional com correlação entre variáveis clínicas de gravidade da doença e questionário de avaliação da qualidade de vida: Dermatology Life Quality Index. RESULTADOS: Dos 40 pacientes avaliados, a maioria apresentou comprometimento da qualidade de vida de grave a muito grave segundo score obtido no Dermatology Life Quality Index. As variáveis analisadas individualmente também mostraram correlação de gravidade com o número de pontos obtido. CONCLUSÃO: A hanseníase causa sofrimento que ultrapassa a dor e o mal-estar estritamente vinculados ao prejuízo físico, com grande impacto social e psicológico, justificando tanto avanços para abordagem multidisciplinar ao paciente quanto a necessidade de ações de saúde que visem ao controle da doença.<br>BACKGROUND: Hansen’s disease is a chronic infecto-contagious illness caused by Mycobacterium leprae. It is characterized by dermato-neurological damage, varying in spectrum between two steady poles. Albeit curable, it still represents an important public health problem, Brazil being the second most endemic country in the world. Its higher morbidity is associated with reactions and neural damage, which is able to cause permanent disabilities that significantly compromising the quality of life of the patients, with self-stigmatization and shame. OBJECTIVE: To evaluate the degree of compromise in the quality of life of patients with Hansen’s disease. METHODS: Observational study with correlation between clinical indicators of severity and a questionnaire on quality of life: the Dermatology Life Quality Index (DLQI). RESULTS: Of the 40 evaluated patients, the majority presented an impairment in the quality of life, which ranged from the serious to the very serious score in the Dermatology Life Quality Index. Individual analysis of disease indicators also showed a correlation between severity and the Dermatology Life Quality Index. CONCLUSION: Hansen’s disease causes suffering that exceeds the pain and the malaise strictly related to the physical damage, having a great social and psychological impact. This justifies both improvements to a multidisciplinary health care approach and the need of health actions aiming at disease control