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Article Dans Une Revue Haematologica Année : 2018

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

1 AMU MED - Aix-Marseille Université - Faculté de médecine
2 Service de pédiatrie spécialisée et médecine infantile (neurologie, pneumologie, maladies héréditaires du métabolisme) [Hôpital de la Timone - APHM]
3 IHOPe - Institut d'hématologie et d'oncologie pédiatrique [CHU - HCL]
4 CIC 1405 - Centre d'Investigation Clinique [CHU Clermont-Ferrand]
5 Service d'immuno-hématologie pédiatrique [CHU Necker]
6 CHI Créteil
7 Unité d'Hémato-Immunologie pédiatrique [Hôpital Robert Debré, Paris]
8 AP-HP - Hopital Saint-Louis [AP-HP]
9 Service d'hématologie [Hôpital Edouard Herriot - HCL]
10 CHRU Nancy - Centre Hospitalier Régional Universitaire de Nancy
11 CEREDIH - Centre de Référence Déficits Immunitaires Héréditaires
12 Service de pédiatrie
13 CHU ST-E - Centre Hospitalier Universitaire de Saint-Etienne [CHU Saint-Etienne]
14 Service d'hématologie
15 UAM - Unités d'Activité Médicale [Lille]
16 LIRIC - Lille Inflammation Research International Center - U 995
17 Service de Pédiatrie médicale - Spécialités médicales [CHU Limoges]
18 Service d'Hémato-oncologie Pédiatrique
19 Centre de référence maladie rare Thalassémie
20 Hôpital Saint-Louis
21 CHU Rouen
22 PANTHER - Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices
23 Service d'Allergologie et d'Immunologie [CHU Toulouse]
24 Hopital L'Archet-II
25 Service d'hématologie pédiatrique-oncologie
26 CHU Nantes - Centre Hospitalier Universitaire de Nantes
27 CHU Pointe-à-Pitre/Abymes [Guadeloupe]
28 CEReSS - Centre d'études et de recherche sur les services de santé et la qualité de vie
29 FEMTO-ST - Franche-Comté Électronique Mécanique, Thermique et Optique - Sciences et Technologies (UMR 6174)
30 TIMONE - Hôpital de la Timone [CHU - APHM]
Corinne Pondarré
  • Fonction : Auteur

Résumé

In this retrospective study, we evaluate long-term complications in nearly all β-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.
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Dates et versions

hal-02413103 , version 1 (06-02-2020)

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Paternité - Pas d'utilisation commerciale

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Citer

Ilhem Rahal, Claire Galambrun, Yves Bertrand, Nathalie Garnier, Catherine Paillard, et al.. Late effects after hematopoietic stem cell transplantation for β-thalassemia major: the French national experience. Haematologica, 2018, 103 (7), pp.1143-1149. ⟨10.3324/haematol.2017.183467⟩. ⟨hal-02413103⟩
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