Differences in longitudinal growth patterns of children and adolescents with transfusion-dependent hemoglobin E/β-thalassemia and those achieving successful hematopoietic stem-cell transplantation.
Children
Growth
Hematopoietic stem-cell transplantation
Thalassemia
Transfusion-dependent
Journal
International journal of hematology
ISSN: 1865-3774
Titre abrégé: Int J Hematol
Pays: Japan
ID NLM: 9111627
Informations de publication
Date de publication:
Apr 2022
Apr 2022
Historique:
received:
06
09
2021
accepted:
14
12
2021
revised:
14
12
2021
pubmed:
23
2
2022
medline:
2
4
2022
entrez:
22
2
2022
Statut:
ppublish
Résumé
Short stature is a very common endocrinopathy among children with transfusion-dependent (TD) thalassemia. Hematopoietic stem-cell transplantation (HSCT) is the only effective curative treatment for TD thalassemia. This study aimed to identify and compare the longitudinal growth patterns of children with TD hemoglobin E (Hb E)/β-thalassemia against those of children successfully undergoing HSCT. We reviewed the medical records of 39 patients with TD Hb E/β-thalassemia receiving regular blood transfusions, and 39 post-HSCT patients. Longitudinal weight and height Z-scores at each year of age were recorded for TD patients, and longitudinal weight and height Z-scores at each year before and after HSCT were obtained for post-HSCT patients. The mean weight and height Z-scores of TD patients decreased gradually and were lowest at age 13. However, post-HSCT subjects saw significant improvement in their mean weight and height Z-scores 6 and 3 years after HSCT, respectively, relative to pre-HSCT baseline values. Longitudinal growth patterns differed between patients successfully undergoing HSCT and children and adolescents with TD Hb E/β-thalassemia. HSCT significantly improved height outcomes of children and adolescents with TD Hb E/β-thalassemia.
Sections du résumé
BACKGROUND
BACKGROUND
Short stature is a very common endocrinopathy among children with transfusion-dependent (TD) thalassemia. Hematopoietic stem-cell transplantation (HSCT) is the only effective curative treatment for TD thalassemia. This study aimed to identify and compare the longitudinal growth patterns of children with TD hemoglobin E (Hb E)/β-thalassemia against those of children successfully undergoing HSCT.
MATERIALS AND METHODS
METHODS
We reviewed the medical records of 39 patients with TD Hb E/β-thalassemia receiving regular blood transfusions, and 39 post-HSCT patients. Longitudinal weight and height Z-scores at each year of age were recorded for TD patients, and longitudinal weight and height Z-scores at each year before and after HSCT were obtained for post-HSCT patients.
RESULTS
RESULTS
The mean weight and height Z-scores of TD patients decreased gradually and were lowest at age 13. However, post-HSCT subjects saw significant improvement in their mean weight and height Z-scores 6 and 3 years after HSCT, respectively, relative to pre-HSCT baseline values.
CONCLUSIONS
CONCLUSIONS
Longitudinal growth patterns differed between patients successfully undergoing HSCT and children and adolescents with TD Hb E/β-thalassemia. HSCT significantly improved height outcomes of children and adolescents with TD Hb E/β-thalassemia.
Identifiants
pubmed: 35192188
doi: 10.1007/s12185-021-03279-4
pii: 10.1007/s12185-021-03279-4
doi:
Substances chimiques
Hemoglobin E
9034-61-1
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
575-584Informations de copyright
© 2022. Japanese Society of Hematology.
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