Science Advance & A Studio: Prognosis and epidemiology of amyotrophic lateral sclerosis

SummaryNeurology Clinical Practice

Amyotrophic lateral sclerosis (ALS) is a disease with highly variable clinical features and prognosis. Atlanta Clinical & Translational Science Institute (ACTSI) investigators analyzed the prognostic indicators of age, sex, bulbar or spinal onset, body mass index (BMI), and forced vital capacity (FVC) for 728 deceased patients from the Emory ALS Clinic, an ACTSI Clinical Research Network (CRN) Site. The median overall survival was 29.8 months from symptom onset, 15.8 months from diagnosis, and 14.3 months from the initial clinic visit. While univariate analyses revealed that each of the identified clinical features was strongly associated with patient survival, in multivariable analyses only age, BMI, and FVC measured at the first clinic visit were independent prognostic indicators; bulbar onset and sex were not significantly associated with survival prognosis after adjustment for the other clinical features.

The ACTSI supported this work through a Studio Consultation by the institute’s CRN, Biomedical Informatics Program (BIP), and Biostatistics, Epidemiology, & Research Design (BERD) program. “The ACTSI-supported work for this publication involved assessing risk factors for prognosis among ALS patients treated at the Emory ALS Clinic. However, perhaps the most exciting part of the story is the ongoing connections that were fostered,” said Robert Lyles, PhD, ACTSI BERD program director and professor of Biostatistics and Bioinformatics, Rollins School of Public Health, Emory University. The manuscript by Kim Traxinger, MD is co-authored by Crystal Kelly, MPH, Brent Johnson, PhD, Lyles, and Jonathan Glass, MD. 

The Studio Consultation spurred an ongoing and productive relationship between BERD, Glass, the Emory ALS Clinic, and Emory’s Department of Biostatistics and Bioinformatics. From the relationships formed through the Studio, Glass is now on a dissertation committee with Lyles for a doctoral student co-advised by Johnson in the Department of Biostatistics and Bioinformatics. The student is developing complex statistical methods motivated by further questions related to Glass’ ALS patient registry. “I’ve used ACTSI resources since its inception in 2007. The ACTSI biostats and biomedical informatics programs and Clinical Research Sites have supported several of our ALS projects leading to a number of publications. The Studio Consultation allowed us access and expertise from these three ACTSI programs during one meeting. The consultation was time effective and was the key factor in establishing a lasting relationship between the Emory ALS Clinic and Emory’s Department of Biostatistics and Bioinformatics,” said Glass.

Studio Consultations aid in the successful design and implementation of clinical and translational science research proposals. The Studio Consultation concept involves a clinical investigator(s) presenting their proposals to representatives of the BERD, BIP, and CRN who in-turn give the investigator their expert feedback. By pulling together experts from these key programs, ACTSI hopes to improve a proposal's design, implementation, and analysis to maximize competitiveness. In short, a Studio Consultation is a pre-review by a panel of experts designed to improve an investigator's chances of success. A Studio will assist researchers in biostatistics, bioinformatics, database development, project generation planning, research design, and/or protocol planning and implementation. Consultations are available to Emory University, Morehouse School of Medicine, or Georgia Institute of Technology faculty. To arrange for a Studio Consultation, please submit a request.