title: Growth and endocrine function following bone marrow transplantation for thalassemia major.
abstract: Thalassemia major (TM) patients frequently suffer from growth delay and endocrine dysfunction. Thirty-two TM patients who had survived more than 2 years after bone marrow transplantation (BMT) were recruited for growth and endocrine study. Patients were followed up annually for growth, and the height was expressed as height standard deviation score (HtSDS). The HtSDS at baseline was -1.51 and was more reduced in patients older than 7 years (-1.99) as compared with those younger patients (-0.79) (p =.027). The HtSDS gradually improved after BMT and increased by 0.59 (CI 0.16-1.01) at 5 years after BMT. Forty percent of patients were below 2 SD at time of BMT but this decreased to 15% at the latest assessment. The hormonal profiles of gonadotrophins, sex hormones, and thyroid function were assayed regularly after BMT. With a median follow-up of 67 months, ovarian failure was universal among the 10 girls evaluable for puberty and all required hormonal replacement. Eight of 10 boys had spontaneous puberty but 3 of them had gonadal impairment. One patient developed diabetes mellitus and one had growth hormone deficiency after BMT. In conclusion, improvement of growth after BMT in TM was common. Gonadal failure is universal in girls, and boys were less affected.
authors.affiliation: Department of Paediatrics, Prince of Wales Hospital, The Chinese University of Hong Kong, Shatin, China. ckli@cuhk.edu.hk
authors.forename: Chi Kong
authors.initials: CK
authors.lastname: Li
authors.forename: Ki Wai
authors.initials: KW
authors.lastname: Chik
authors.forename: Gary Wing Kin
authors.initials: GW
authors.lastname: Wong
authors.forename: Pik Shun
authors.initials: PS
authors.lastname: Cheng
authors.forename: Vincent
authors.initials: V
authors.lastname: Lee
authors.forename: Matthew Ming Kong
authors.initials: MM
authors.lastname: Shing
mesh_terms: D000293:Adolescent; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D002657:Child Development / Q000502:physiology*; D004703:Endocrine System / Q000502:physiology* / Q000503:physiopathology; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000209:etiology / Q000503:physiopathology; D006130:Growth Disorders / Q000175:diagnosis / Q000209:etiology* / Q000503:physiopathology; D006801:Humans; D008297:Male; D011446:Prospective Studies; D011628:Puberty, Delayed / Q000175:diagnosis / Q000209:etiology; D017086:beta-Thalassemia / Q000150:complications* / Q000503:physiopathology / Q000628:therapy