Contributions
Abstract: S131
Type: Oral Presentation
Presentation during EHA22: On Friday, June 23, 2017 from 12:15 - 12:30
Location: Room N105
Background
In clinical practice, allogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment offering a definitive cure for patients with beta-thalassemia. Its outcome has improved over the last 3 decades with currently a disease free survival rate of 90% when transplant is performed in childhood from an HLA-identical sibling. Few data are available on long-term toxicity and frequency of chronic complications after transplant.
Aims
The purpose of this study was to evaluate the long-term health status after a successful allogeneic HSCT for beta-thalassemia major in a national cohort of patients.
Methods
This French retrospective study included patients who successfully received allogeneic HSCT between 1985 -2012 and were alive at least 2 years after HSCT. Study was based on data collected in the national registry of patients with beta-thalassemia and conducted in collaboration with the French society of HSCT (SFGM-Tc). Late effect data were recorded by physicians through reference or transplant center visits. Collected data included medical examination results, long-term treatments administered and laboratory tests (serum ferritin, Hb, liver enzymes, creatinine level and thyroid evaluation). Linear-mixed model was used to analyze data evolution over time (for height and weight SDS, SF, Hb values).
Results
A total of 134 patients had received allogeneic HSCT for beta-thalassemia in France from 1985 to 2012. 107/134 patients experienced successful HSCT (6 after a second transplant) and were alive 2 years after transplantation. Six were not analyzed (back to their country or lost of follow-up) and two died of chronic graft-versus-host disease. 99 patients were analyzed for long-term effects. Median age at HSCT was 5.9 years (8 month-26 years). The source was bone marrow in 85% of cases and a matched sibling donor was used in 90% of cases. Conditioning mostly consisted (85%) of busulfan and cyclophosphamide (oral busulfan in 52%). Median age at the last visit was 19 years. Chronic complications, similar to those observed in patients treated with transfusion and chelation therapy occurred after transplant in 12% of patients: 7 hypothyroidism, 2 heart failure, 5 diabetes. 2 patients had chronic respiratory failure related to transplant. The height SDS improved after HSCT if performed at a young age. Weight SDS values increased with time, especially in females. Although gonadal dysfunction was observed in 60% of women aged at least 13 years at the last evaluation, 12/27 aged more than 20 years experimented at least one successful pregnancy. 93 patients had stopped their immunosuppressive treatment two years after HSCT. 37 were treated with iron chelation therapy and/or phlebotomies. At least half of patients are receiving a long-term hormonal treatment or antibiotic prophylaxis at the last visit. Decrease in serum ferritin values after transplant was significantly influenced by age at transplant and pre-transplant serum ferritin value. The median hemoglobin value was 12.5 g/dL (86-165) at a mean age of 18 years and Hb values were significantly influenced by age, the sex of the donor and the presence of donor thalassemia trait.
Conclusion
A comprehensive and regular long-term follow-up should be established for all patients receiving allogeneic HSCT for beta-thalassemia major. In this national cohort, endocrinological complications were frequent after transplant. Fertility can be partly preserved, but this result has to be reevaluated with the more recent use of intravenous busulfan.
Session topic: 26. Thalassemias
Keyword(s): Long-term follow-up, Allo-SCT
Abstract: S131
Type: Oral Presentation
Presentation during EHA22: On Friday, June 23, 2017 from 12:15 - 12:30
Location: Room N105
Background
In clinical practice, allogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment offering a definitive cure for patients with beta-thalassemia. Its outcome has improved over the last 3 decades with currently a disease free survival rate of 90% when transplant is performed in childhood from an HLA-identical sibling. Few data are available on long-term toxicity and frequency of chronic complications after transplant.
Aims
The purpose of this study was to evaluate the long-term health status after a successful allogeneic HSCT for beta-thalassemia major in a national cohort of patients.
Methods
This French retrospective study included patients who successfully received allogeneic HSCT between 1985 -2012 and were alive at least 2 years after HSCT. Study was based on data collected in the national registry of patients with beta-thalassemia and conducted in collaboration with the French society of HSCT (SFGM-Tc). Late effect data were recorded by physicians through reference or transplant center visits. Collected data included medical examination results, long-term treatments administered and laboratory tests (serum ferritin, Hb, liver enzymes, creatinine level and thyroid evaluation). Linear-mixed model was used to analyze data evolution over time (for height and weight SDS, SF, Hb values).
Results
A total of 134 patients had received allogeneic HSCT for beta-thalassemia in France from 1985 to 2012. 107/134 patients experienced successful HSCT (6 after a second transplant) and were alive 2 years after transplantation. Six were not analyzed (back to their country or lost of follow-up) and two died of chronic graft-versus-host disease. 99 patients were analyzed for long-term effects. Median age at HSCT was 5.9 years (8 month-26 years). The source was bone marrow in 85% of cases and a matched sibling donor was used in 90% of cases. Conditioning mostly consisted (85%) of busulfan and cyclophosphamide (oral busulfan in 52%). Median age at the last visit was 19 years. Chronic complications, similar to those observed in patients treated with transfusion and chelation therapy occurred after transplant in 12% of patients: 7 hypothyroidism, 2 heart failure, 5 diabetes. 2 patients had chronic respiratory failure related to transplant. The height SDS improved after HSCT if performed at a young age. Weight SDS values increased with time, especially in females. Although gonadal dysfunction was observed in 60% of women aged at least 13 years at the last evaluation, 12/27 aged more than 20 years experimented at least one successful pregnancy. 93 patients had stopped their immunosuppressive treatment two years after HSCT. 37 were treated with iron chelation therapy and/or phlebotomies. At least half of patients are receiving a long-term hormonal treatment or antibiotic prophylaxis at the last visit. Decrease in serum ferritin values after transplant was significantly influenced by age at transplant and pre-transplant serum ferritin value. The median hemoglobin value was 12.5 g/dL (86-165) at a mean age of 18 years and Hb values were significantly influenced by age, the sex of the donor and the presence of donor thalassemia trait.
Conclusion
A comprehensive and regular long-term follow-up should be established for all patients receiving allogeneic HSCT for beta-thalassemia major. In this national cohort, endocrinological complications were frequent after transplant. Fertility can be partly preserved, but this result has to be reevaluated with the more recent use of intravenous busulfan.
Session topic: 26. Thalassemias
Keyword(s): Long-term follow-up, Allo-SCT