Should we treat genetic syndromes?

Citation
Rg. Rosenfeld et Ck. Buckway, Should we treat genetic syndromes?, J PED END M, 13, 2000, pp. 971-981
Citations number
10
Categorie Soggetti
Endocrinology, Nutrition & Metabolism
Journal title
JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM
ISSN journal
0334018X → ACNP
Volume
13
Year of publication
2000
Supplement
2
Pages
971 - 981
Database
ISI
SICI code
0334-018X(200009)13:<971:SWTGS>2.0.ZU;2-B
Abstract
With the greater availability of GH due to the development of recombinant h uman GH, GH therapy has expanded well beyond the original FDA indication fo r GHD, We examine the NCGS database, comparing data for FDA-approved indica tions with data for "other" conditions to see whether such expanded use is warranted, Although statistical analyses are of questionable validity becau se of the small size of some subpopulations and other factors discussed her ein, certain trends emerge from the data captured by NCGS, We conclude that many clinical syndromes characterized by short stature are responsive to G H, at least in the short term, For many such syndromes, responsiveness is o f the same magnitude as that seen in Turner's syndrome and, occasionally, G HD, If responsiveness to GH is the most important criterion for GH therapy, these "other" conditions warrant an open-minded, prospective evaluation.