The ALS Nutrition/NIPPV Study: Design, feasibility, and initial results

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.

Key words::

• Nutrition
• energy expenditure
• Doubly Labeled Water
• NIPPV
• respiratory scales

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.