ABSTRACT
The frailty index (FI) is based on the principle that the more deficits an individual has, the greater their risk of adverse outcomes. It is expressed as a ratio of the number of deficits present to the total number of deficits considered. We developed an MDS-specific FI using a prospective MDS registry and assessed its ability to add prognostic power to conventional prognostic scores in MDS. The 42 deficits included in this FI included measurements of physical performance, comorbidities, laboratory values, instrumental activities of daily living, quality of life and performance status. Of 644 patients, 440 were eligible for FI calculation. The median FI score was 0.25 (range 0.05-0.67), correlated with age and IPSS/IPSS-R risk scores and discriminated overall survival. With a follow-up of 20 months, survival was 27 months (95% CI 24-30.4). By multivariate analysis, age >70, FI, transfusion dependence, and IPSS were significant covariates associated with OS. The incremental discrimination improvement of the frailty index was 37%. We derived a prognostic score with five risk groups and distinct survivals ranging from 7.4 months to not yet reached. If externally validated, the MDS-FI could be used as a tool to refine the risk stratification of current clinical prognostication models.
Subject(s)
Frailty/mortality , Frailty/pathology , Myelodysplastic Syndromes/mortality , Myelodysplastic Syndromes/pathology , Quality of Life , Registries/statistics & numerical data , Risk Assessment/methods , Activities of Daily Living , Aged , Aged, 80 and over , Comorbidity , Female , Follow-Up Studies , Humans , Male , Middle Aged , Prognosis , Prospective Studies , Risk Factors , Survival RateABSTRACT
Central nervous system and skeletal muscles secrete a group of polypeptide hormones called neurotrophins (NTs). More recent studies show that NTs and their receptors are also expressed in the lung, suggesting a role for NTs in lung development. To examine the role of NTs during normal and diseased lung organogenesis, we employed wild-type and amyogenic mouse embryos (designated as Myf5-/-:MyoD-/-). Amyogenic embryos completely lacked skeletal muscles and were not viable after birth due to the respiratory failure secondary to lung hypoplasia. To examine the importance of lung-secreted NTs during normal and hypoplastic lung organogenesis, immunohistochemistry was employed. Distribution of NTs and their receptors was indistinguishable between normal and hypoplastic lungs. To further examine the importance of non-lung-secreted NTs (e.g., from the skeletal muscle and CNS) in lung organogenesis, in utero injections of two NTs were performed. The exogenously introduced NTs (i.e., non-lung-secreted) did not appear to improve development of the lung in amyogenic embryos. Moreover, immunohistochemistry showed significantly reduced number of airway smooth muscle cells (ASMCs) in hypoplastic lungs of amyogenic embryos, suggesting that the number of ASMCs is primarily regulated by the fetal breathing-like movements (i.e., mechanical factors).
Subject(s)
Embryo, Mammalian/cytology , Lung/pathology , Nerve Growth Factors/metabolism , Animals , Gene Expression Regulation , Immunohistochemistry , In Situ Nick-End Labeling , Lung/embryology , Mice , Mice, Transgenic , Movement , MyoD Protein/metabolism , Myocytes, Smooth Muscle/cytology , Myogenic Regulatory Factor 5/metabolism , RespirationABSTRACT
Multiple sclerosis (MS), a chronic disease of the central nervous system, is characterized by a variable and unpredictable course. The most common pattern of the disease is the relapsing-remitting form in which clearly defined relapses (also called exacerbations) are followed by complete or incomplete recovery. Interferon beta-1b (Betaseron), a drug that affects the natural course of the disease, was developed for the treatment of relapsing-remitting MS. Multiple Sclerosis Pathways (MSP), a disease management program, was developed to provide comprehensive and personal support to MS patients taking interferon beta-1b and to serve as an information resource for all people with MS, their families, and healthcare professionals. The MSP program includes personal patient assistance, reimbursement services, a 24-hour nurse hotline, training program, educational resources, and injection supplies. The nurse hotline counselor (NHC) utilizes the nursing process in a unique telephone nursing practice in this program. The positive impact of education and support on adherence to therapy has been validated by training and nurse hotline data.
Subject(s)
Adjuvants, Immunologic/therapeutic use , Counseling/organization & administration , Disease Management , Drug Information Services/organization & administration , Hotlines/organization & administration , Interferon-beta/therapeutic use , Multiple Sclerosis/drug therapy , Multiple Sclerosis/nursing , Patient Education as Topic/organization & administration , Comprehensive Health Care , Disease Progression , Drug Industry , Humans , Interferon beta-1a , Interferon beta-1b , Job Description , Multiple Sclerosis/physiopathology , Multiple Sclerosis/psychology , Patient Compliance/psychology , Patient Compliance/statistics & numerical data , Personal Health Services/organization & administration , Program Evaluation , Reimbursement Mechanisms , Remission, Spontaneous , Social SupportABSTRACT
A teaching program (Betaseron Instruction) for patients beginning interferon beta-1b therapy was developed at St. John's Mercy Medical Center in St. Louis, Missouri according to Bandura's theory of self-efficacy. Then, a study was conducted to determine attitudes of patients with multiple sclerosis (MS) about interferon beta-1b (Betaseron) therapy and evaluate the effectiveness of the teaching program. In that program, teaching skills mastery and the use of modeling and persuasion were used in curriculum development. A survey questionnaire in which information about the patients' evaluation of interferon beta-1b therapy effects was mailed to 60 patients who had participated in the teaching program between December, 1993 and October 1994. Thirty surveys were completed and returned. The mean age of participants was 42 years; 22% were men and 78% were women. The mean duration of therapy for the study participants was 10.5 weeks. Qualitative content analysis and tabulation of the one scaled question from the 30 completed surveys were performed to obtain outcome measurements of cognitive goals and determine the level of self-management of therapy. Responses regarding feelings about therapy, the benefits of the teaching program, confidence in performing skills that resulted from the use of interferon beta-1b therapy, adverse effects produced by the medication and quality of life issues were evaluated. The effectiveness of the Betaseron Instruction program and the importance of access to support systems from peers and healthcare professionals were validated by patient responses. The role of the nurse in patient teaching and the provision of support were indicated to be essential to a positive outcome in all of the patient surveys submitted.
Subject(s)
Adjuvants, Immunologic/administration & dosage , Interferon-beta/administration & dosage , Multiple Sclerosis/therapy , Patient Education as Topic , Adaptation, Psychological , Adjuvants, Immunologic/adverse effects , Adult , Caregivers/education , Caregivers/psychology , Female , Humans , Injections, Subcutaneous , Interferon beta-1a , Interferon beta-1b , Interferon-beta/adverse effects , Male , Middle Aged , Multiple Sclerosis/psychology , Quality of Life , Self Administration , Sick Role , Treatment OutcomeABSTRACT
In adult acute lymphocytic leukemia (ALL) cure is rare. The purpose of this study was to try to improve remission duration and survival by administration of two courses of high-dose chemotherapy, each followed by autologous bone marrow rescue, in first remission. Chemotherapy consisted of cyclophosphamide, BCNU and VP-16-213. Rescue bone marrow was fractionated over a discontinuous albumin gradient to minimize contamination with leukemic cells. Fourteen patients entered the study. Median total remission duration was 14 months. Three patients relapsed after one course of treatment. Five patients relapsed after the second course. Four patients died after the second course and two patients remain alive and well in unmaintained remission, with a total remission duration of 42+ and 47+ months. It is concluded that this regimen is toxic but, with careful selection of patients, may lead to long-term unmaintained remissions.
