Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.
Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.
christina.peters@stanna.at.
Emanuele
E
Angelucci
Susanne
S
Matthes-Martin
Donatella
D
Baronciani
Françoise
F
Bernaudin
Sonia
S
Bonanomi
Maria Domenica
MD
Cappellini
Jean-Hugues
JH
Dalle
Paolo
P
Di Bartolomeo
Cristina Díaz
CD
de Heredia
Roswitha
R
Dickerhoff
Claudio
C
Giardini
Eliane
E
Gluckman
Ayad Achmed
AA
Hussein
Naynesh
N
Kamani
Milen
M
Minkov
Franco
F
Locatelli
Vanderson
V
Rocha
Petr
P
Sedlacek
Frans
F
Smiers
Isabelle
I
Thuret
Isaac
I
Yaniv
Marina
M
Cavazzana
Christina
C
Peters
D000755:Anemia, Sickle Cell / Q000175:diagnosis / Q000628:therapy*; D002648:Child; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D017086:beta-Thalassemia / Q000175:diagnosis / Q000628:therapy*