Allogreffe de cellules souches hématopoïétiques; Drépanocytose; Conditionnement; Indications
Abstract :
[fr] La drépanocytose est une pathologie associée à des complications sévères et une mortalité
précoce chez l'adulte. Chez l'enfant, l'allogreffe familiale HLA-identique de cellules souches
hématopoïétiques permet de stopper la progression de la maladie et ainsi de « guérir » plus
de 95 % patients, mais cette thérapeutique reste peu proposée chez les adultes du fait de sa
toxicité potentielle. Cet atelier a eu pour objectif de définir les indications et les modalités
d'allogreffe chez l'enfant et l'adulte atteints de drépanocytose. Les indications d'allogreffe
familiale HLA-identique retenues chez l'enfant et chez l'adulte sont : la vasculopathie cérébrale,
la survenue d'événements vaso-occlusifs sous hydroxycarbamide, l'atteinte rénale ou l'hépatopathie chronique secondaire à la drépanocytose, l'anémie chronique < 7 g/dL malgré
l'hydroxycarbamide, la nécessité de maintenir plus de six mois un programme d'échange érythrocytaire,
les difficultés transfusionnelles majeures par allo-immunisation antiglobules rouges.
Chez l'enfant ayant un donneur familial HLA-identique, le conditionnement myéloablatif à base
debusulfan, cyclophosphamide et sérum anti-lymphocytaire reste la référence compte tenu des
excellents résultats obtenus avec cette approche. Chez les patients de plus de 15 ans, nous
recommandons l'utilisation d'un conditionnement d'intensité réduite tel que celui rapporté par
l'équipe du NIH (association d'alemtuzumab, d'irradiation corporelle totale à 3 Gy) suivie d'un
greffon de cellules souches périphériques puis de sirolimus. En l'absence de donneur familial HLA-
identique, il n'y a pas d'argument formel pour privilégier une allogreffe à partir d'un donneur non
apparenté versus une allogreffe haplo-identique, mais nous recommandons que ces allogreffes
alternatives soient évaluées dans le cadre de protocoles prospectifs.
Disciplines :
Hematology
Author, co-author :
Dhédin, Nathalie
Paillard, Catherine
Dalle, Jean-Hugues
Ouachée, Marie
Buchbinder, Nimrod
Brissot, Eolia
BEGUIN, Yves ; Centre Hospitalier Universitaire de Liège - CHU > Département de médecine interne > Service d'hématologie clinique
Masouridi-Levrat, Stavroula
Yakoub-Agha, Ibrahim
Bernit, Emmanuelle
Pondarre, Corinne
Language :
French
Title :
Allogreffe de cellules souches hématopoïétiques dans la drépanocytose de l'enfant et de l'adulte : indications et modalités
Alternative titles :
[en] Allogeneic hematopoietic stem cell transplant in children and adults with sickle cell disease: Indications and modalities
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