Predicting pituitary iron and endocrine dysfunction.
Patients with thalassemia major (TM) require lifelong transfusion therapy to survive, leading to toxic iron overload in the endocrine glands and heart. The pituitary gland is one of the most vulnerable targets, leading to irreversible hypogonadotropic hypogonadism in approximately half of patients. Improvements in magnetic resonance imaging (MRI) technology and understanding have allowed earlier recognition of preclinical iron deposition in the heart, pancreas, and liver; prior work also supports a similar role for the pituitary. The purpose of this study is threefold, (1) to develop age-specific nomograms for pituitary iron and volume metrics; (2) to determine the prevalence, severity, and age of onset of pituitary iron deposition and volume loss in TM patients, and (3) to determine whether deferasirox monotherapy can modify the trajectory of pituitary iron accumulation and damage over a two-year period. This article outlines relevant background studies and methodological details as well as providing preliminary results from our first two aims.
Wood
John C
JC
Division of Pediatric Cardiology, Children's Hospital Los Angeles, Los Angeles, California, USA.
Noetzl
Leila
L
Hyderi
Aleya
A
Joukar
Mehrdad
M
Coates
Thomas
T
Mittelman
Steven
S
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D004700:Endocrine System Diseases / Q000209:etiology / Q000503:physiopathology*; D006801:Humans; D007501:Iron / Q000378:metabolism*; D019190:Iron Overload / Q000150:complications / Q000209:etiology / Q000503:physiopathology*; D008279:Magnetic Resonance Imaging; D010902:Pituitary Gland / Q000737:chemistry* / Q000473:pathology / Q000502:physiology; D065227:Transfusion Reaction; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications / Q000628:therapy
D007501:Iron