Sustained and full fetal hemoglobin production after failure of bone marrow transplant in a patient homozygous for beta 0-thalassemia: a clinical remission despite genetic disease and transplant rejection.
An adult patient affected by beta(0)-thalassemia major underwent allogeneic bone marrow transplant (BMT) from a matched related donor. Forty days after transplant, allogeneic engraftment failure and autologous beta(0)-thalassemic bone marrow recovery were documented. Red blood cell transfusions were required until 118 days post-transplant. Thereafter, the haemoglobin (Hb) levels stabilized over 11.8 gr/dl throughout the ongoing 34-month follow-up, abolishing the need for transfusion support. The Hb electrophoresis showed 100% Hb Fetal (HbF). This unexplained case suggests full HbF production may occur in an adult patient with beta(0)-thalassemia major.
Paciaroni
Katia
K
Gallucci
Cristiano
C
De Angelis
Gioia
G
Alfieri
Cecilia
C
Roveda
Andrea
A
Lucarelli
Guido
G
D000293:Adolescent; D016026:Bone Marrow Transplantation; D005319:Fetal Hemoglobin / Q000096:biosynthesis*; D006084:Graft Rejection / Q000097:blood*; D006801:Humans; D017086:beta-Thalassemia / Q000097:blood* / Q000235:genetics / Q000601:surgery
D005319:Fetal Hemoglobin