Efficacy and safety of mecasermin rinfabate.
There has been interest in using recombinant human (rh) insulin-like growth factor (IGF)-I (rhIGF-I) to treat short stature, either alone or in combination with its binding protein (insulin-like growth factor binding protein [IGFBP]-3). IGF-I has been shown to increase growth velocity in children with IGF deficiency, either as a result of growth hormone insensitivity syndrome (GHIS) or IGF gene deletion. However, there have been adverse events, particularly hypoglycaemia, reported with administration of unbound rhIGF-I. In addition, the serum half-life of unbound rhIGF-I is shorter when administered to patients with GHIS, who have low serum concentrations of its binding proteins IGFBP-3 and acid-labile subunit (ALS), than when administered to normal volunteers or to the patient with an IGF-I gene deletion (who had normal levels of IGFBP-3). iPlex (mecasermin rinfabate), an equimolar mixture of IGF-I and its binding protein IGFBP-3, was developed to prolong the half-life and to counteract acute adverse events (particularly hypoglycaemia) associated with administration of IGF-I. Although there are no published data on the efficacy of mecasermin rinfabate in treating growth disorders, it does appear that mecasermin rinfabate has a longer half-life in patients with GHIS than unbound IGF-I, and fewer reports of adverse events (including hypoglycaemia) when administered to patients with diabetes.
Kemp
Stephen F
SF
University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR 72202-3591, USA. KempStephenF@uams.edu
Fowlkes
John L
JL
Thrailkill
Kathryn M
KM
D002056:Burns / Q000188:drug therapy; D002986:Clinical Trials as Topic; D003920:Diabetes Mellitus / Q000188:drug therapy; D004338:Drug Combinations; D006130:Growth Disorders / Q000188:drug therapy*; D006801:Humans; D018972:Insulin-Like Growth Factor Binding Protein 3 / Q000493:pharmacokinetics / Q000627:therapeutic use*; D007334:Insulin-Like Growth Factor I / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D011994:Recombinant Proteins
D004338:Drug Combinations; D018972:Insulin-Like Growth Factor Binding Protein 3; D011993:Recombinant Fusion Proteins; D011994:Recombinant Proteins; C475619:somatoKine; D007334:Insulin-Like Growth Factor I