title: Identification and management of poor response to growth-promoting therapy in children with short stature.
abstract: Growth hormone (GH) is widely prescribed for children with short stature across a range of growth disorders. Recombinant human (rh) insulin-like growth factor-1 (rhIGF-1) therapy is approved for severe primary IGF-I deficiency - a state of severe GH resistance. Evidence is increasing for an unacceptably high rate of poor or unsatisfactory response to growth-promoting therapy (i.e. not leading to significant catch up growth) in terms of change in height standard deviation score (SDS) and height velocity (HV) in many approved indications. Consequently, there is a need to define poor response and to prevent or correct it by optimizing treatment regimens within accepted guidelines. Recognition of a poor response is an indication for action by the treating physician, either to modify the therapy or to review the primary diagnosis leading either to discontinuation or change of therapy. This review discusses the optimal investigation of the child who is a candidate for GH or IGF-1 therapy so that a diagnosis-based choice of therapy and dosage can be made. The relevant parameters in the evaluation of growth response are described together with the definitions of poor response. Prevention of poor response is addressed by discussion of strategy for first-year management with GH and IGF-1. Adherence to therapy is reviewed as is the recommended action following the identification of the poorly responding patient. The awareness, recognition and management of poor response to growth-promoting therapy will lead to better patient care, greater cost-effectiveness and increased opportunities for clinical benefit.
authors.affiliation: Division of Pediatrics, Department of Clinical and Experimental Medicine, Faculty of Health Sciences, Linköping University, Linköping, Sweden. peter.bang@liu.se
authors.forename: Peter
authors.initials: P
authors.lastname: Bang
authors.forename: S Faisal
authors.initials: SF
authors.lastname: Ahmed
authors.forename: Jesús
authors.initials: J
authors.lastname: Argente
authors.forename: Philippe
authors.initials: P
authors.lastname: Backeljauw
authors.forename: Markus
authors.initials: M
authors.lastname: Bettendorf
authors.forename: Gianni
authors.initials: G
authors.lastname: Bona
authors.forename: Régis
authors.initials: R
authors.lastname: Coutant
authors.forename: Ron G
authors.initials: RG
authors.lastname: Rosenfeld
authors.forename: Marie-José
authors.initials: MJ
authors.lastname: Walenkamp
authors.forename: Martin O
authors.initials: MO
authors.lastname: Savage
mesh_terms: D001827:Body Height / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D006130:Growth Disorders / Q000188:drug therapy* / Q000378:metabolism; D013006:Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D007334:Insulin-Like Growth Factor I; D013006:Growth Hormone