Title :

Late effects after hematopoietic stem cell transplantation for beta-thalassemia major: the French national experience.

Author(s) :

Rahal, Ilhem [Auteur]
Galambrun, Claire [Auteur]
Bertrand, Yves [Auteur]
Garnier, Nathalie [Auteur]
Paillard, Catherine [Auteur]
Frange, Pierre [Auteur]
Pondarre, Corinne [Auteur]
Dalle, Jean-Hugues [Auteur]
Peffault De Latour, Regis [Auteur]
Michallet, Mauricette [Auteur]
Steschenko, Dominique [Auteur]
Moshous, Despina [Auteur]
Lutz, Patrick [Auteur]
Stephan, Jean-Louis [Auteur]
Rohrlich, Pierre-Simon [Auteur]
Yakoub-Agha, Ibrahim [Auteur]
Lille Inflammation Research International Center - U 995 [LIRIC]
Bernaudin, Francoise [Auteur]
Piguet, Christophe [Auteur]
Aladjidi, Nathalie [Auteur]
Badens, Catherine [Auteur]
Berger, Claire [Auteur]
Socie, Gerard [Auteur]
Dumesnil, Cecile [Auteur]
Castex Marie, Pierre [Auteur]
Poiree, Marilyne [Auteur]
Lambilliotte, Anne [Auteur]
Thomas, Caroline [Auteur]
Simon, Pauline [Auteur]
Auquier, Pascal [Auteur]
Aix Marseille Université [AMU]
Michel, Gerard [Auteur]
Loundou, Anderson [Auteur]
Aix Marseille Université [AMU]
Agouti, Imane [Auteur]
Thuret, Isabelle [Auteur]

Journal title :

Haematologica

Abbreviated title :

Haematologica

Volume number :

103

Pages :

1143-1149

Publication date :

2018-07-01

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

In this retrospective study, we evaluate long-term complications in nearly all beta-thalassemia-major patients who successfully received allogeneic hematopoietic stem cell transplantation in France. Ninety-nine patients ...
Show more >In this retrospective study, we evaluate long-term complications in nearly all beta-thalassemia-major patients who successfully received allogeneic hematopoietic stem cell transplantation in France. Ninety-nine patients were analyzed with a median age of 5.9 years at transplantation. The median duration of clinical follow up was 12 years. All conditioning regimens were myeloablative, most were based on busulfan combined with cyclophosphamide, and more than 90% of patients underwent a transplant from a matched sibling donor. After transplantation, 11% of patients developed thyroid dysfunction, 5% diabetes, and 2% heart failure. Hypogonadism was present in 56% of females and 14% of males. Female patients who went on to normal puberty after transplant were significantly younger at transplantation than those who experienced delayed puberty (median age 2.5 vs 8.7 years). Fertility was preserved in 9 of 27 females aged 20 years or older and 2 other patients became pregnant following oocyte donation. In addition to patient's age and higher serum ferritin levels at transplantation, time elapsed since transplant was significantly associated with decreased height growth in multivariate analysis. Weight growth increased after transplantation particularly in females, 36% of adults being overweight at last evaluation. A comprehensive long-term monitoring, especially of endocrine late effects, is required after hematopoietic stem cell transplantation for thalassemia.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Inserm
Université de Lille
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Research team(s) :

Immunity, inflammation and fibrsis in auto and allo-reactivity

Submission date :

2019-03-01T14:08:16Z
2024-01-23T12:01:32Z