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

Abstract

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). These categories are prognostic for treatment outcome after marrow transplantation. Class 3 patients have more transplant-related mortality than other patients. This study examines transplantation outcome for class 3 patients. Records were reviewed of 215 patients in class 3 who received transplants in Pesaro from HLA-identical related donors between May 1, 1984 and May 1, 1994. The influence of pretransplant, peritransplant, and posttransplant variables on survival, relapse, and transplant-related mortality was examined by product-limit and proportional-hazards multivariate analysis. Age and conditioning regimen were influential on survival, and regimens with less than 200 mg/kg cyclosporine (CY) were associated with 5-year survival probabilities of .74 and .63 patients younger than 17 years and older patients, respectively. Transfusion history and regimen were influential on rejection with 5 year probabilities of .53 and .24 in patients who received less than or greater than 100 red blood cell transfusions before transplantation and regimens containing less than 200 mg/kg CY. Results of transplantation for patients with advanced thalassemia treatment have improved with the introduction of conditioning regimens with less CY. This has been associated with an increase in rejection (particularly in patients who have received < 100 red blood cell transfusions before transplant). Efforts at reducing the rejection rate by modifying the conditioning regimen should be concentrated on younger patients who have received a small number of transfusions. Patients with thalassemia who have HLA-identical family members should be transplanted before they are in class 3.

Treosulfan‐based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major

“…Remarkably, the outcome of our patients transplanted from UDs was at least as good as that of patients previously reported and prepared with a busulfan-based regimen (La Nasa et al, 2005). Although the number of patients investigated was small, our data also suggest that this regimen might abolish the difference usually observed among thalassaemia patients of different risk classes (Lucarelli et al, 1996). While the outcome of thalassaemia patients given reducedintensity preparative regimens has been reported to be dismal (Iannone et al, 2003), we demonstrated that, although this preparation was associated with limited extra-medullary toxicity, the majority of patients benefited from sustained donor engraftment.…”

Unrelated Bone Marrow Transplantation for β‐Thalassemia Patients: The Experience of the Italian Bone Marrow Transplant Group

“…In class 3 patients, the risk of mortality is high even when the donor is an HLA-identical sibling. 10,25,26 In this cohort of 38 class 3 patients, a higher incidence of deaths was observed in the group of patients conditioned with the protocol, including TT (9/22 = 41%) compared to patients conditioned with BU-CY alone (4/16 = 25%). Although this difference was not significant, a conditioning regimen with three drugs (BU-TT-CY) is likely to be too toxic for this category of patients.…”

Treatment of Hepatitis C Virus Infection in Thalassemia

“…BMT provides a curative therapy for patients with thalassemia. 29,30 With improvements in conditioning regimen, modulation of the conditioning regimen based upon pretransplantation risk factors, and expansion of the sources of hematopoietic cells, the availability and success of transplantation have improved over the past two decades. Despite these improvements in outcome, the presence of active HCV with liver inflammation (elevated transaminases) remains a contraindication to transplantation.…”