Subcutaneous IGF-1 is not beneficial in 2-year ALS trial

被引:194
作者
Sorenson, E. J. [1 ]
Windbank, A. J. [1 ]
Mandrekar, J. N. [1 ]
Bamlet, W. R. [1 ]
Appel, S. H. [2 ]
Armon, C. [3 ]
Barkhaus, P. E. [4 ]
Bosch, P. [5 ]
Boylan, K. [6 ]
David, W. S. [7 ]
Feldman, E. [8 ]
Glass, J. [9 ]
Gutmann, L. [10 ]
Katz, J. [11 ]
King, W. [12 ]
Luciano, C. A. [13 ]
McCluskey, L. F. [14 ]
Nash, S. [12 ]
Newman, D. S. [15 ]
Pascuzzi, R. M. [16 ]
Pioro, E. [17 ]
Sams, L. J. [18 ]
Scelsa, S. [19 ]
Simpson, E. P. [2 ]
Subramony, S. H. [20 ]
Tiryaki, E. [7 ]
Thornton, C. A. [21 ]
机构
[1] Mayo Clin, Dept Neurol, Rochester, MN 55905 USA
[2] Methodist Hosp, Houston, TX 77030 USA
[3] Baystate Med Ctr, Springfield, MA USA
[4] Med Coll Wisconsin, Milwaukee, WI 53226 USA
[5] Mayo Clin Arizona, Scottsdale, AZ USA
[6] Mayo Clin Jacksonville, Jacksonville, FL 32224 USA
[7] Hennepin Cty Med Ctr, Minneapolis, MN 55415 USA
[8] Univ Michigan, Med Ctr, Ann Arbor, MI USA
[9] Emory Univ, Med Ctr, Atlanta, GA 30322 USA
[10] W Virginia Univ, Med Ctr, Morgantown, WV 26506 USA
[11] Forbes Norris MDA ALS Res Ctr, San Francisco, CA USA
[12] Ohio State Univ, Med Ctr, Columbus, OH 43210 USA
[13] Univ Puerto Rico, San Juan, PR 00936 USA
[14] Penn Neurol Inst, Philadelphia, PA USA
[15] Henry Ford Med Ctr, Detroit, MI USA
[16] Indiana Univ, Med Ctr, Indianapolis, IN USA
[17] Cleveland Clin, Cleveland, OH USA
[18] Univ Cincinnati, Cincinnati, OH 45221 USA
[19] Beth Israel Deaconess Med Ctr, New York, NY 10003 USA
[20] Univ Mississippi, Med Ctr, Jackson, MS 39216 USA
[21] Univ Rochester, Med Ctr, Rochester, NY 14627 USA
关键词
D O I
10.1212/01.wnl.0000335970.78664.36
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Background: Previous human clinical trials of insulin-like growth factor type I (IGF-1) in amyotrophic lateral sclerosis (ALS) have been inconsistent. This phase III, randomized, double-blind, placebo-controlled study was undertaken to address whether IGF-1 benefited patients with ALS. Methods: A total of 330 patients from 20 medical centers were randomized to receive 0.05 mg/kg body weight of human recombinant IGF-1 given subcutaneously twice daily or placebo for 2 years. The primary outcome measure was change in their manual muscle testing score. Secondary outcome measures included tracheostomy-free survival and rate of change in the revised ALS functional rating scale. Intention to treat analysis was used. Results: There was no difference between treatment groups in the primary or secondary outcome measures after the 2-year treatment period. Conclusions: Insulin-like growth factor type I does not provide benefit for patients with amyotrophic lateral sclerosis. Neurology (R) 2008; 71: 1770-1775
引用
收藏
页码:1770 / 1775
页数:6
相关论文
共 20 条
[1]  
BECK AT, 1962, ARCH GEN PSYCHIAT, V4, P561
[2]   A placebo-controlled trial of insulin-like growth factor-I in amyotrophic lateral sclerosis [J].
Borasio, GD ;
Robberecht, W ;
Leigh, PN ;
Emile, J ;
Guiloff, RJ ;
Jerusalem, F ;
Silani, V ;
Vos, PE ;
Wokke, JHJ ;
Dobbins, T .
NEUROLOGY, 1998, 51 (02) :583-586
[4]   NERVE SPROUTING IN INNERVATED ADULT SKELETAL-MUSCLE INDUCED BY EXPOSURE TO ELEVATED LEVELS OF INSULIN-LIKE GROWTH-FACTORS [J].
CARONI, P ;
GRANDES, P .
JOURNAL OF CELL BIOLOGY, 1990, 110 (04) :1307-1317
[5]   The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function [J].
Cedarbaum, JM ;
Stambler, N ;
Malta, E ;
Fuller, C ;
Hilt, D ;
Thurmond, B ;
Nakanishi, A .
JOURNAL OF THE NEUROLOGICAL SCIENCES, 1999, 169 (1-2) :13-21
[6]   Preclinical testing of neuroprotective neurotrophic factors in a model of chronic motor neuron degeneration [J].
Corse, AM ;
Bilak, MM ;
Bilak, SR ;
Lehar, M ;
Rothstein, JD ;
Kuncl, RW .
NEUROBIOLOGY OF DISEASE, 1999, 6 (05) :335-346
[7]   A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis [J].
Cudkowicz, ME ;
Shefner, JM ;
Schoenfeld, DA ;
Brown, RH ;
Johnson, H ;
Qureshi, M ;
Jacobs, M ;
Rothstein, JD ;
Appel, SH ;
Pascuzzi, RM ;
Heiman-Patterson, TD ;
Donofrio, PD ;
David, WS ;
Russell, JA ;
Tandan, R ;
Pioro, EP ;
Felice, KJ ;
Rosenfeld, J ;
Mandler, RN ;
Sachs, GM ;
Bradley, WG ;
Raynor, EM ;
Baquis, GD ;
Belsh, JM ;
Novella, S ;
Goldstein, J ;
Hulihan, J .
NEUROLOGY, 2003, 61 (04) :456-464
[8]   Muscle expression of a local Igf-1 isoform protects motor neurons in an ALS mouse model [J].
Dobrowolny, G ;
Giacinti, C ;
Pelosi, L ;
Nicoletti, C ;
Winn, N ;
Barberi, L ;
Molinaro, M ;
Rosenthal, N ;
Musarò, A .
JOURNAL OF CELL BIOLOGY, 2005, 168 (02) :193-199
[9]   Venous thrombosis in an ALS population over four years [J].
Elman, LB ;
Siderowf, A ;
Houseman, G ;
Kelley, M ;
Mccluskey, LF .
AMYOTROPHIC LATERAL SCLEROSIS, 2005, 6 (04) :246-249
[10]   Synergy of insulin-like growth factor-1 and exercise in amyotrophic lateral sclerosis [J].
Kaspar, BK ;
Frost, LM ;
Christian, L ;
Umapathi, P ;
Gage, FH .
ANNALS OF NEUROLOGY, 2005, 57 (05) :649-655