Assessing what matters most to patients with or at risk for Alzheimer's and care partners: a qualitative study evaluating symptoms, impacts, and outcomes.

BACKGROUND: The What Matters Most (WMM) study was initiated to evaluate symptoms, AD-related impacts, treatment-related needs, preferences, and priorities among individuals with or at risk for Alzheimer's disease (AD) and their care partners. The objective of this qualitative study phase was to identify a comprehensive set of concepts of interest that are meaningful to individuals across the AD continuum. METHODS: Interviews were conducted with 60 clinically referred individuals and care partners across 5 AD stages (n = 12 each): group 1 (non-clinically impaired individuals with AD pathology), group 2 (individuals with mild cognitive impairment and AD pathology), group 3 (individuals with mild AD), group 4 (individuals with moderate AD and their care partners), and group 5 (care partners of individuals with severe AD). Interviews were conducted by experienced interviewers, audio-recorded, and transcribed. Dominant trends were identified in each interview and compared across subsequent interviews to generate themes or patterns in descriptions of AD symptoms, impacts, and desired treatment outcomes. RESULTS: All participants endorsed current issues related to memory; nearly all participants (n = 55; 92%) across the five groups endorsed symptoms related to communication and language. Groups 1-3 reported an impact on mood/emotions (n = 23; 64%) and a decrease in social activities or outgoingness (n = 17; 47%). Current and future concerns reported by the overall sample included memory (n = 48; 80%), dependence (n = 40; 67%), and "other" concerns (n = 33; 55.0%) (e.g., uncertainty about the future, burdening others). The most desired AD treatment outcomes were improvement or restoration of memory (n = 40; 67%) and stopping AD progression (n = 35; 58.3%). Group-level differences were observed in the symptoms, impacts, and desired treatment outcomes among patients and care partners across the AD continuum. CONCLUSIONS: Cognitive functioning issues-particularly in memory and communication-are present even in preclinical and early-stage AD, including among those without a formal AD diagnosis. While the impacts of AD vary across the disease-severity spectrum, improved memory and disease modification were treatment outcomes considered most important to participants across all 5 AD stages. Neuropsychological assessments traditionally used in AD clinical trials may not evaluate the often-subtle concepts that are important to patients and care partners. Results from this study will inform the second phase of the WMM project-a quantitative study to elicit the relative importance of these concepts of interest to people at risk for and living with AD and their care partners.

The Role of Noninvasive Endpoints in Predicting Long-Term Outcomes in Pulmonary Arterial Hypertension.

BACKGROUND: Until recently, many clinical trials in patients with pulmonary arterial hypertension (PAH) evaluated exercise capacity with 6-minute walk distance (6MWD) as the primary endpoint. Common secondary endpoints include PAH functional class (FC), which assesses symptoms, and either brain natriuretic peptide (BNP) or the inactive N-terminal cleavage product of its prohormone (NT-proBNP), which assesses cardiac function. OBJECTIVE: Examine the relationships among 6MWD, FC, and BNP/NT-proBNP measured at baseline or follow-up with long-term outcomes in PAH studies. METHODS: Relevant literature from January 1990 to April 2018 were obtained by searching PubMed, Embase, and Cochrane. Articles in English reporting on associations between 6MWD, FC, or BNP/NT-proBNP and outcomes in PAH were identified. Each endpoint was evaluated individually. Prespecified inclusion and exclusion criteria were applied at level 1 (titles/abstracts) and level 2 (full-text review). RESULTS: The database search yielded 836 unique records; 65 full-text articles were reviewed. Twenty-five studies were eligible for inclusion. Findings supported the importance of measuring PAH noninvasive endpoints in predicting long-term outcomes. Patients with shorter or decreased 6MWD, poor (III/IV) or declining FC (e.g., from II to III), or elevated or increasing BNP/NT-proBNP had a higher risk of death and costly events (e.g., hospitalization, lung transplant). FC also predicted health care resource utilization and costs. Collectively, these endpoints establish risk groups that predict likelihood of complications from PAH or death. CONCLUSION: Assessment of 6MWD, FC, and BNP/NT-proBNP provides low-cost, efficient, and noninvasive means of predicting long-term health and economic outcomes in patients with PAH.

Defining cancer survivor and cancer survivorship: the who, what, and when

Over the past three decades various definitions of cancer survivor and cancer survivorship have been proposed. These definitions frequently describe (1) who is considered a cancer survivor and who is experiencing cancer survivorship, (2) what it means to a be survivor and experience survivorship, and (3) when someone is considered a survivor and when survivorship begins. Descriptive epidemiology indicates that certain individuals diagnosed and treated for cancer describe the presence of specific unmet needs following diagnosis and treatment for cancer. Evidence also points to an increased likelihood of cancer recurrence or a new cancer in many cancer patients. These observations should be reflected in the case definitions of cancer survivor and cancer survivorship. This paper reviews the literature for evidence to validate existing definitions of cancer survivor and cancer survivorship and proposes updated definitions for each. Based on this evidence, a cancer survivor is defined as an individual diagnosed with cancer. The period following primary treatment, which is characterized by transitions in care and a need to address the late effects of cancer and its treatment, may be a particularly critical time for the cancer survivor. The definition does not preclude the major involvement of family, friends, caregivers, and providers, but does place the focus on the individual who received the diagnosis of cancer. Children and young adult cancer survivors should be housed in subgroups under the 'cancer survivor' umbrella. In addition, cancer survivorship is defined as the period of time beginning at cancer diagnosis where attention by the survivor and his or her care team is directed at addressing needs related to health, symptoms, function, lifestyle, and well-being; these needs may change through the care trajectory. Coordination of care between oncologist specialists involved in the early phases of cancer diagnosis and treatment and primary care physicians involved in long-term follow-up is critical. As new knowledge is acquired regarding cancer survival and the cancer experience, modifications to these proposed definitions will be imperative

Durante las últimas tres décadas se han propuesto diversas definiciones del superviviente de cáncer y la supervivencia del cáncer. Estas definiciones describen con frecuencia (1) quién se considera un superviviente de cáncer y quién experimenta la supervivencia del cáncer, (2) lo que significa ser superviviente y la experiencia de sobrevivir, y (3) cuándo se considera que alguien es un superviviente y cuándo comienza a ser un superviviente. La epidemiología descriptiva indica que ciertos individuos diagnosticados y tratados de cáncer describen la presencia de necesidades específicas insatisfechas tras el diagnóstico y tratamiento para el cáncer. Los datos disponibles. apuntan a un aumento de la probabilidad de recurrencia del cáncer o un nuevo cáncer en muchos pacientes con cáncer. Estas observaciones deben reflejarse en las definiciones de caso del superviviente de cáncer y la supervivencia del cáncer. Este artículo revisa los datos relativos a la validez de las definiciones existentes de superviviente de cáncer y supervivencia del cáncer y propone definiciones actualizadas para cada uno. Con base en estos hallazgos, se define como superviviente de cáncer a un individuo diagnosticado con cáncer. El periodo que sigue al tratamiento primario, que se caracteriza por las transiciones en el cuidado y la necesidad de abordar los efectos tardíos del cáncer y su tratamiento, puede ser un momento especialmente crítico para el superviviente de cáncer. La definición no excluye la importante participación de la familia, amigos, cuidadores y proveedores, pero pone el foco en la persona que recibió el diagnóstico de cáncer. Los niños y los jóvenes adultos supervivientes de cáncer deben ser ubicados en subgrupos bajo el paraguas de 'superviviente de cáncer'. Además, definimos la supervivencia del cáncer como el período de tiempo que comienza en el diagnóstico del cáncer en el que la atención del equipo de cuidados para el superviviente se centra en las necesidades relativas a la salud, la funcionalidad, los síntomas, el estilo de vida y bienestar; estas necesidades pueden cambiar a través de la trayectoria del cuidado. Es fundamental la coordinación de la atención entre los especialistas oncólogos, que participan en las primeras fases de diagnóstico y tratamiento del cáncer, y los médicos de atención primaria involucrados en el seguimiento a largo plazo de la enfermedad. Las modificaciones a las definiciones propuestas se harán necesarias a medida que se adquieran nuevos conocimientos en relación a la supervivencia del cáncer y la experiencia del cáncer

Neurocognitive deficits following primary brain tumor treatment: systematic review of a decade of comparative studies.

There has been an increase in the prevalence of adults diagnosed with and treated for primary brain tumors. Cognitive deficits are a common long-term effect in brain tumor survivors. The objective of this paper is to examine whether these deficits are specific to those diagnosed with and treated for a primary brain tumor. A systematic review of the medical literature from 2002 to 2012 was conducted to investigate neurocognitive deficits in brain tumor survivors (post-primary treatment) compared to healthy controls. Four studies were identified that met all inclusion criteria. Gliomas were the most common form of tumor included. Neuropsychological evaluation identified cognitive deficits in brain tumor survivors on tests of working memory, cognitive control and flexibility, cognitive processing speed, visual searching, planning and foresight, and general attention. While age, education, and gender can influence cognitive function, the present review indicates that deficits exist beyond those accounted for by these factors. Many primary brain tumor survivors are involved in roles (e.g., employee, parent, spouse/partner, student) that require optimal performance of these cognitive skills. Future research should evaluate brain tumor survivors on functional challenges resulting from these cognitive sequelae and develop effective ways to mitigate them.