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Marrow transplantation for patients with thalassemia: results in class 3 patients
G Lucarelli1, R A Clift, M Galimberti
1Divisione Ematologica e Centro Trapianto Midollo Osseo di Muraglia, Pesaro, Italy.
Insights
Class 3 thalassemia patients undergoing marrow transplantation have improved survival with reduced cyclosporine (CY) conditioning regimens. However, this leads to increased rejection, especially in younger patients with fewer transfusions. Early transplantation is recommended.
Area of Science:
- Hematology
- Transplantation Immunology
- Pediatric Oncology
Background:
- Thalassemia patients are classified by liver damage and iron overload (Class 1, 2, 3).
- Class 3 patients face higher transplant-related mortality.
- Understanding transplantation outcomes for Class 3 patients is critical.
Purpose of the Study:
- To examine marrow transplantation outcomes in Class 3 thalassemia patients.
- To identify pretransplant, peritransplant, and posttransplant factors influencing survival, relapse, and mortality.
- To evaluate the impact of conditioning regimens on transplant success.
Main Methods:
- Retrospective review of 215 Class 3 patients receiving transplants from HLA-identical related donors (1984-1994).
- Analysis using product-limit and proportional-hazards multivariate methods.
- Assessment of variables including age, conditioning regimen, and transfusion history.
Main Results:
- Age and conditioning regimen significantly impacted survival.
- Reduced cyclosporine (CY) regimens (<200 mg/kg) improved survival (5-year probabilities: 0.74 for <17 years, 0.63 for older).
- Fewer transfusions (<100) and reduced CY regimens were linked to higher rejection rates (5-year probability: 0.53 vs. 0.24).
Conclusions:
- Conditioning regimens with less CY have improved outcomes for advanced thalassemia.
- Increased rejection rates necessitate strategies to mitigate this, focusing on younger patients with fewer transfusions.
- Transplanting thalassemia patients before reaching Class 3 is strongly advised.
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.