Abstract
Our objective was to investigate the nutritional requirements in ALS and to determine the feasibility of early intervention with NIPPV. Subjects were enrolled into one of two arms. In the nutrition arm, total daily energy expenditure (TDEE) was determined longitudinally over 48 weeks using the Doubly Labeled Water method. In the NIPPV arm, NIPPV was offered at 80% vs. 50% FVC. Additional measurements were obtained in both arms to ultimately formulate equations to predict TDEE and to estimate sample size for a phase III study of early NIPPV. Eighty subjects were enrolled in the nutrition arm and 73 in the NIPPV arm. Baseline characteristics of the participants are described. TDEE was available for 80 subjects in 249 independent determinations during disease progression. Other variables were measured simultaneously for future modeling of ALS-specific equations to predict TDEE. In the NIPPV arm, rates of progression to the criteria for NIPPV intervention (80% or 50% predicted FVC) were computed. Additional factors were obtained longitudinally to develop indices of early ventilatory insufficiency. The results of this study will allow us to model equations to predict the energy requirements in ALS and to plan a study of early intervention with NIPPV.
Acknowledgements
Study funded by the National Institute of Neurological Disorders and Stroke (NINDS) grant (RO1 NS045087); the General Clinical Research Centers at Columbia (RR00645), Vermont (RR00109), Utah (RR00064), Penn State (RR10732, CO6-RR016499) and Kentucky (RR02602); the ALS Hope Foundation, and the Cynthia Shaw Crispen Endowment. Lan Chi T. Luu provided helpful discussions during the planning phase of the study. The cooperation of the patients and caregivers is gratefully acknowledged. The authors thank Robin A. Conwit and Janice Cordell of NINDS, and the members of the DSMB, for their assistance during the course of the study.
The ALS Nutrition/NIPPV Study Group
Nutrition Sites
University of Kentucky Coordination Center
Edward Kasarskis, Richard Kryscio, Marta S. Mendiondo, Stephen Wells, Christie Shrestha, Margaret Healey, Megan Thompson, Lan Chi T. Luu, Carmen Saylor, Kathryn Vanderpool, Irina Kasarskis, Maria Malguizo, Jason King, Jody Clasey.
Columbia University
Hiroshi Mitsumoto, Jackie Montes, Daniel Bell
Pennsylvania State University
Zachary Simmons, Helen Stephens, Ally Brothers, Susan Deiling, Michelle Heckenluber
University of Utah
Mark B. Bromberg, Summer Davis
University of Vermont
Rup Tandan, Chris Potter, Dwight Matthews, Shannon Lenox, Jesse Gardner
NIPPV Sites
Beth Israel Medical Center; Albert Einstein College of Medicine
Stephen N Scelsa, Theresa Imperato
University of Colorado
Yvonne D. Rollins, Hans Neville, Burleen Hewitt, John Cumming
Drexel University
Terry D. Heiman-Patterson, Roseanne Sattazahn, Michael Sherman, Sara Feldman, Melonie Mitchell
Henry Ford
Daniel S. Newman, Helen Foley
University of Miami
Ashok Verma, Julie Steele, Donald A. Koggan
Upstate Medical University, Syracuse
Jeremy M. Shefner, Mary Lou Watson
University of Texas Health Science Center at San Antonio
Carlayne Jackson, Pamela Kittrell
Data and Safety Monitoring Board
R. L. Sufit, L. Gutmann, P. Huang, N. Lechtzin
NIH Program Management
R. A. Conwit, J. Cordell
Disclosure of interest: The authors report no conflicts of interest and alone are responsible for the content and writing of this paper.