Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future

M Bhatia, MC Walters - Bone marrow transplantation, 2008 - nature.com
Abstract β-Thalassemia major and sickle cell disease (SCD) are among the most common
hereditary disorders worldwide. The supportive treatment of β-thalassemia major requires …

Hematopoietic stem cell transplantation in thalassemia

M Algeri, M Lodi, F Locatelli - Hematology/Oncology Clinics, 2023 - hemonc.theclinics.com
In the last half-century, the life expectancy of thalassemia major (TM) patients has
dramatically improved and, with the proper administration of supportive care, consisting of …

Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling

F Locatelli, N Kabbara, A Ruggeri… - Blood, The Journal …, 2013 - ashpublications.org
We analyzed the outcomes of 485 patients with thalassemia major (TM) or sickle cell
disease (SCD) receiving HLA-identical sibling cord blood transplantation (CBT, n= 96) or …

Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease

F Locatelli, V Rocha, W Reed… - Blood, The Journal …, 2003 - ashpublications.org
Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted
treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with …

Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report

MC Walters, R Storb, M Patience… - Blood, The Journal …, 2000 - ashpublications.org
Fifty children who had symptomatic sickle cell disease received matched sibling marrow
allografts between September 1991 and March 1999, with Kaplan-Meier probabilities of …

Target antigens determine graft-versus-host disease phenotype

DH Kaplan, BE Anderson, JM McNiff, D Jain… - The Journal of …, 2004 - journals.aai.org
Chronic graft-vs-host disease (cGVHD) is an increasingly frequent complication of
allogeneic stem cell transplantation. Phenotypically, cGVHD differs from patient to patient; in …

Marrow transplantation for patients with thalassemia: results in class 3 patients

G Lucarelli, RA Clift, M Galimberti, P Polchi… - 1996 - ashpublications.org
Thalassemia patients can be categorized as class 1 (minimal liver damage and iron
overload), class 3 (extensive liver damage from iron overload), and class 2 (intermediate) …

Long-term survival of ex-thalassemic patients with persistent mixed chimerism after bone marrow transplantation

M Andreani, S Nesci, G Lucarelli, P Tonucci… - Bone marrow …, 2000 - nature.com
Twenty-six transplanted thalassemic patients out of 295 analyzed, showed the presence of
persistent mixed chimerism, over a period of time varying between 2 and 11 years after BMT …

Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene

C May, S Rivella, A Chadburn… - Blood, The Journal of …, 2002 - ashpublications.org
The β-thalassemias are caused by more than 200 mutations that reduce or abolish β-globin
production. The severity of the resulting anemia can lead to lifelong transfusion dependency …

HLA-matched sibling bone marrow transplantation for β-thalassemia major

M Sabloff, M Chandy, Z Wang… - Blood, The Journal …, 2011 - ashpublications.org
We describe outcomes after human leukocyte antigen-matched sibling bone marrow
transplantation (BMT) for 179 patients with β-thalassemia major. The median age at …