Forging the Future: 2018 Alopecia Areata Research Summit Summary Report.

Alopecia areata (AA) is a common autoimmune skin disease resulting in the loss of hair on the scalp and elsewhere on the body that affects over 146 million people worldwide at some point in their lives. Founded in 1981, the National AA Foundation (NAAF) is a nonprofit organization that supports research to find a cure or acceptable treatment for AA, supports those with the disease, and educates the public about AA. NAAF conducts research summits every two years to review progress and create new directions in its funded and promoted research. This report from the seventh AA Research Summit, Forging the Future, held December 4-5, 2018 in New York City provides highlights of the research presented and future research priorities identified during targeted discussion sessions.

Building and Crossing the Translational Bridge: 2016 Alopecia Areata Research Summit Highlights.

Alopecia areata (AA) is a common autoimmune skin disease that results in the loss of hair on the scalp and elsewhere on the body and affects over 146 million people worldwide at some point in their lives. Founded in 1981, the National Alopecia Areata Foundation is a nonprofit organization that supports research to find a cure or acceptable treatment for AA, supports those with the disease, and educates the public about AA. The National Alopecia Areata Foundation conducts research summits every 2 years to review progress and create new directions in its funded and promoted research. The Foundation brings together scientists from all disciplines to get a broad and varied perspective. These AA research summits are part of the Foundation's main strategic initiative, the AA Treatment Development Program, to enhance the understanding of AA and accelerate progress toward a viable treatment.

From Targets to Treatments: Bridging Autoimmune Research to Advance Understanding of Alopecia Areata.

Alopecia areata is a common autoimmune skin disease resulting in the loss of hair on the scalp and elsewhere on the body that affects over 146 million people worldwide at some point in their lives. Founded in 1981, the National Alopecia Areata Foundation (NAAF) is a nonprofit organization that supports research to find a cure or acceptable treatment for alopecia areata, supports those with the disease, and educates the public about alopecia areata. NAAF conducts research summits every 2 years that are central to achieving the goals of a major strategic initiative, the Alopecia Areata Treatment Development Program, which are: to accelerate progress toward a safe, effective, affordable treatment or a cure for alopecia areata. These summits have played a key role in transforming the understanding of alopecia areata from largely inflammatory and dermatological perspectives to a focus on the genetic and immunological factors that are now recognized as driving and active determinants of the disease process.

Cumulative Life Course Impairment of Alopecia Areata.

Alopecia areata (AA), an unpredictable, nonscarring hair loss, is commonly perceived as a cosmetic, rather than medical, concern. However, substantial evidence exists describing the negative impact on quality of life, as the disease affects patients personally, socially, financially, and physically. Over time, the cumulative disability may perpetuate poor confidence, social disconnection, negative coping strategies, and failure to achieve a full life potential. Here, we describe the cumulative life course impairment (CLCI) of AA by examining the complex interaction of (1) stigmatization, (2) physical and psychiatric comorbidities, and (3) coping strategies. The model aggregates existing cross-sectional data, which have previously captured disease burden only as snapshots in time. Thus, by examining cumulative effects, the CLCI model serves as a proxy for longitudinal data to better describe life course epidemiology of the disease.

Long-term outcomes in patients with treated childhood hydrocephalus.

OBJECT: The goal in this study was to determine the long-term effects of childhood hydrocephalus. METHODS: A patient-reported survey completed by 1953 participants was used to collect data in a subgroup of 1459 individuals who had been treated for hydrocephalus in childhood. Data on shunt complications, including total shunt revisions and infections, were examined in those whose hydrocephalus had been diagnosed at least 10 years earlier (718 patients). Social and functional outcomes were examined in patients who were 20 years of age or older at the time of survey completion (403 individuals). Specific questions addressed the presence of depression, the patient's marital status, independent living arrangements, and the educational level attained. Shunt complications were common; 54% of patients had four or more shunt revisions, and 9% had three or more shunt infections. Depression requiring treatment occurred in 45% of participants. Other measures of social functioning all reflected a major impact of childhood hydrocephalus. In general, a worse outcome was found in patients whose hydrocephalus was diagnosed before 18 months of age. CONCLUSIONS: The lifelong morbidity associated with shunt placement to treat childhood hydrocephalus is substantial, and it includes shunt-related complications and comorbidities that adversely affect social functioning.

Priorities for hydrocephalus research: report from a National Institutes of Health-sponsored workshop.

OBJECT: Treatment for hydrocephalus has not advanced appreciably since the advent of cerebrospinal fluid (CSF) shunts more than 50 years ago. Many questions remain that clinical and basic research could address, which in turn could improve therapeutic options. To clarify the main issues facing hydrocephalus research and to identify critical advances necessary to improve outcomes for patients with hydrocephalus, the National Institutes of Health (NIH) sponsored a workshop titled "Hydrocephalus: Myths, New Facts, and Clear Directions." The purpose of this paper is to report on the recommendations that resulted from that workshop. METHODS: The workshop convened from September 29 to October 1, 2005, in Bethesda, Maryland. Among the 150 attendees was an international group of participants, including experts in pediatric and adult hydrocephalus as well as scientists working in related fields, neurosurgeons, laboratory-based neuroscientists, neurologists, patient advocates, individuals with hydrocephalus, parents, and NIH program and intramural staff. Plenary and breakout sessions covered injury and recovery mechanisms, modeling, biomechanics, diagnosis, current treatment and outcomes, complications, quality of life, future treatments, medical devices, development of research networks and information sharing, and education and career development. RESULTS: The conclusions were as follows: 1) current methods of diagnosis, treatment, and outcomes monitoring need improvement; 2) frequent complications, poor rate of shunt survival, and poor quality of life for patients lead to unsatisfactory outcomes; 3) investigators and caregivers need additional methods to monitor neurocognitive function and control of CSF variables such as pressure, flow, or pulsatility; 4) research warrants novel interdisciplinary approaches; 5) understanding of the pathophysiological and recovery mechanisms of neuronal function in hydrocephalus is poor, warranting further investigation; and 6) both basic and clinical aspects warrant expanded and innovative training programs. CONCLUSIONS: The research priorities of this workshop provide critical guidance for future research in hydrocephalus, which should result in advances in knowledge, and ultimately in the treatment for this important disorder and improved outcomes in patients of all ages.

What we don't (but should) know about hydrocephalus.

In an effort to identify critical gaps in the prevailing knowledge of hydrocephalus, the authors formulated 10 key questions. 1) How do we define hydrocephalus? 2) How is cerebrosinal fluid (CSF) absorbed normally and what are the causes of CSF malabsorption in hydrocephalus? 3) Why do the ventricles dilate in communicating hydrocephalus? 4) What happens to the structure and function of the brain when it is compressed and stretched by the expanding ventricles? 5) What is the role of cerebrovenous pressure in hydrocephalus? 6) What causes normal-pressure hydrocephalus? 7) What causes low-pressure hydrocephalus? 8) What is the pathophysiology of slit ventricle syndrome? 9) What is the pathophysiological basis for neurological impairment in hydrocephalus, and to what extent is it reversible? 10) How is the brain of a child with hydrocephalus different from that of a young or elderly adult? Rigorous answers to these questions should lead to more effective and reliable treatments for this disorder.

Headaches in patients with shunts.

Headache is one of the most common afflictions suffered by humans. Headache in patients with a shunt triggers a series of events that includes utilization of expensive technologies and often potentially dangerous surgical intervention. The purpose of this study was to determine the incidence of headaches in patients with shunts and, hopefully, the relationship of those headache disorders to the treatment of hydrocephalus. The Hydrocephalus Association maintains a self-reporting database recorded from individuals treated for hydrocephalus and their families. This database was mined to determine the incidence of severe headaches requiring treatment and interfering with normal life in patients who have been treated for hydrocephalus. There were 1,242 responders between the ages of 19 months and 45 years of age. Of these, 1,233 answered the question, "Do you or your family member suffer from (does your child complain of) frequent or chronic headaches?" This subset forms the basis of this study. Three groups were defined by age: children (19 months-12 years), adolescents (13 years-19 years), and young adults (20 years-45 years). Most respondents were initially treated during infancy (before 18 months of age); 84% of children and 69% of both adolescents and young adults were treated very early in life. Severe headaches became a more frequent problem as the age of the population treated for hydrocephalus increased. In terms of frequency and severity of headaches, direct comparisons with epidemiologic studies of normal populations are difficult because of the limitations of data available in the database. However, it is likely that this population has a higher incidence of severe headaches than normal populations. The cost of management of headaches in this population is very high, and the patients are at risk throughout life. Early treatment decisions have a significant effect on later quality of life. Strategies that lead to normalization of cerebrospinal fluid dynamics and life without shunt dependency are justified if they can be shown to improve later quality of life.