Outcome of transplantation with unrelated donor bone marrow in children with severe thalassaemia

Abstract

Summary:We conducted a study of unrelated donor bone marrow transplantation (BMT) in 11 children with severe thalassaemia. The conditioning regimen consisted of busulphan, cyclophosphamide and antilymphocyte globulin. All received T-cell nondepleted bone marrow. The median marrow-nucleated cell dose was 4.9 Â 10 8 /kg (range; 3.5À8.0 Â 10 8 /kg). Median time of granulocyte recovery was 16 days (range; 13-21 days), and of platelet recovery was 39 days (range; 14-196). Grade 2-4 acute graft-versus-host disease (GVHD) developed in six patients (54%), and grade 3-4 in one patient (9%). Three (27%) of 11 evaluable patients had chronic GVHD (limited stage). All 11 patients are alive without thalassaemia after a median follow-up time of 397 days (range; 171-814 days). This study lends support to consideration of unrelated donor BMT as an acceptable therapy to cure severe thalassaemia especially in patients who are young and do not yet show irreversible severe complications of iron overload.

Donor/recipient mixed chimerism does not predict graft failure in children with -thalassemia given an allogeneic cord blood transplant from an HLA-identical sibling

“…[4][5][6] In the last two decades, cord blood (CB) has progressively become an extensively used alternative source of hematopoietic stem cells for transplanting patients with either malignant or non-malignant hematologic diseases. 7,9 As compared to bone marrow transplantation (BMT), cord blood transplantation (CBT) offers significant advantages, including easy and safe cell collection, low risk of transmitting viral infections, immediate availability of hematopoietic stem cells, elimination of risk and discomfort to donors, as well as a reduced incidence and severity of both acute and chronic graft-versus-host disease (GvHD), this last advantage translating into the possibility of using unrelated donors HLA disparate with respect to the recipient.…”

Multiorgan engraftment and differentiation of human cord blood CD34 + Lin − cells in goats assessed by gene expression profiling

“…H ematopoietic stem cell (HSC) transplantation can compensate for tissue damage elicited by a wide variety of disorders, including malignant and͞or inherited diseases (1)(2)(3)(4)(5). However, broader clinical application is still limited because of a number of biological and technical problems.…”

Unrelated donor bone marrow transplantation for β-thalassemia major: an experience from China

“…7 These results highlight the possibility of using unrelated BMT to reconstitute allogeneic bone marrow and cure thalassemia. In the present study, we investigated the effect of unrelated BMT in nine Chinese children with b-thalassemia major and found that eight patients had full engraftment, while one developed recurrence of thalassemia that continued to depend on treatment with blood transfusions and iron chelation.…”

“…Although the following trials to test the efficacy of unrelated BMT were generally disappointing and neglected, 4-6 two recent reports have brought thalassemic patients new hope of using unrelated donors for BMT. 2,7 In La Nasa's investigation, among 32 patients transplanted with unrelated BMT, 22 had full engraftment and the need for blood transfusion was eliminated.…”