Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000–2010

Abstract

Allogeneic hemopoietic stem cell transplantation (HSCT) is the only method currently available to cure transfusion-dependent thalassemia major that has been widely used worldwide. To verify transplantation distribution, demography, activity, policies and outcomes inside the European Group for Blood and Marrow Transplantation (EBMT), we performed a retrospective non-interventional study, extracting data from the EBMT hemoglobinopathy prospective registry database. We included 1493 consecutive patients with thalassemia major transplanted between 1 January 2000 and 31 December 2010. In total, 1359 (91%) transplants were performed on patients o18 years old, 1061 were from a human leukocyte Ag-identical sibling donor. After a median observation time of 2 years, the 2-year overall survival (OS) and event-free survival (EFS; that is, thalassemia-free survival) were 88 ± 1% and 81 ± 1%, respectively. Transplantation from a human leukocyte Ag-identical sibling offered the best results, with OS and EFS of 91 ± 1% and 83 ± 1%, respectively. No significant differences in survival were reported between countries. The threshold age for optimal transplant outcomes was around 14 years, with an OS of 90-96% and an EFS of 83-93% when transplants were performed before this age. Allogeneic HSCT for thalassemia is a curative approach that is employed internationally and produces excellent results.

Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency

“…A negative effect of age on survival is also reported for other forms of hereditary anemia. 11,12 Concurrently, we noticed a striking difference in survival between patients treated in Asian and European centers, which could possibly be explained by the difference in age at which patients were transplanted. In addition, haematologica 2018; 103:e82 CASE REPORTS …”

“…Compared to published survival rates for other forms of hereditary anemias, cohorts that are otherwise comparable in age, time period and transplant hospital, the overall survival rate after HSCT in PKD is relatively low. [11][12][13] The present analysis of all 16 PKD patients known to be transplanted to date showed a three-year overall survival of 65%. Significantly better survival was observed for patients transplanted before the age of ten.…”

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

“…[2][3][4][5] HLAmatched sibling donor transplants account for the majority of transplants performed worldwide for hemoglobinopathy. 6,7 However, only 18% of patients with SCD have an HLA-matched sibling donor in the United States. 8 HLA-matched adult unrelated donors (URDs) have been used to expand the donor pool for nonmalignant hematologic disorders, but their role in SCD transplants is unclear.…”

Haploidentical haematopoietic stem cell transplantation for thalassaemia major based on an FBCA conditioning regimen

“…However, the lack of a matched family donor always limits HSCT for these patients (Baronciani et al , 2016; Chaudhury et al , 2017). Although HLA‐haploidentical donors, which must be family members, have been thought to solve the problem of HSCT for haematological malignancies(Kanakry et al , 2016), the experience for thalassaemia major was still limited.…”

“…Currently, allogeneic haematopoietic stem cell transplantation (HSCT) is the only curative therapy for thalassaemia major. For Pesaro class 1–2 patients, the overall survival (OS) and thalassaemia‐free survival (TFS) have been reported as 80–90% (Hussein et al , 2013; La Nasa et al , 2013; Baronciani et al , 2016). However, in reality a human leucocyte antigen (HLA)‐matched donor can be found for only 20–30% of thalassaemia major patients (Shenoy & Thompson, 2016).…”