Contributions
Abstract: PB2475
Type: Publication Only
Background
Allogeneic hematopoietic stem cell transplantation (HSCT) is the sole available curative therapy for patients with thalassemia major. With the progress human leukocyte antigen (HLA) antigen typing technology and supportive care, the outcome of thalassemia major have greatly improved in recent years, even in high risk patients. However, the problem of finding a suitable donor was still a major limit to cure these patients. In the past decades, haploidentical donor was more and more used in hematologic malignancies HSCT. Here, we explored the outcome of haploidentical donor (HD) HSCT to thalassemia major children based on a FBCA conditioning regimen.
Aims
To anlysis the outcomes of haploidentical hematopoietic stem cell transplantation for thalassemia major based on a FBCA conditioning regimen.
Methods
Eghit patients with thalassemia major (age 3-14 year old, meidan 5.5 year old) underwent haploidentical hematopoietic stem cell transplantation.The conditioning regimen was consists of fludarabin(Flu), busulfan(Bu), cyclophosphamide(Cy) and antithymocyte globulin(ATG). All donors were HLA mismatched family members including fathers、mothers and sister. ABO blood type between donor and receipt were incompatible in three patients.GVHD prophylaxis included cyclosporine (CSA), and short course of methotrexate (MTX). Chimerism studies were performed with blood nucleated cells whth STR-PCR.
Results
The median of nucleated cell and CD34+ cell dose in the infused product were 9.7×108/kg (range, 6.9-26.7×108/kg) and 10.1×106/kg (range, 8.2-27.2×106/Kg), respectively. The median time to achieve absolute neutrophil recovery was in 10 days (range,10 to 15 days), and platelet recovery was in 13 days (range,10 to 102 days).Four patients (50%) have experienced grade I-II acute GVHD; Two patients suffered from grade III-IV (25%) acute GVHD and one of which turned into local chronic GVHD (skin).Chimerism studies were performed for all patients after transplantation. The data showing all patients have achieved full donor chimerism(100%) at post-transplantation +30 day and mixed chimerism status was not observed in all eight patients with the mdeian one year follow up.
So far, all of the patients have a stable engraftment and were transfusion independent in daily life.
Conclusion
The acute and chronic GVHD of haploidentical hematopoietic stem cell transplantation for thalassemia major based on this FBCA conditioning regimen is accpitable and the outcome prompt us to study further.
Session topic: 23. Stem cell transplantation - Clinical
Keyword(s): Haploidentical stem cell transplantation, Outcome, Thalassemia
Abstract: PB2475
Type: Publication Only
Background
Allogeneic hematopoietic stem cell transplantation (HSCT) is the sole available curative therapy for patients with thalassemia major. With the progress human leukocyte antigen (HLA) antigen typing technology and supportive care, the outcome of thalassemia major have greatly improved in recent years, even in high risk patients. However, the problem of finding a suitable donor was still a major limit to cure these patients. In the past decades, haploidentical donor was more and more used in hematologic malignancies HSCT. Here, we explored the outcome of haploidentical donor (HD) HSCT to thalassemia major children based on a FBCA conditioning regimen.
Aims
To anlysis the outcomes of haploidentical hematopoietic stem cell transplantation for thalassemia major based on a FBCA conditioning regimen.
Methods
Eghit patients with thalassemia major (age 3-14 year old, meidan 5.5 year old) underwent haploidentical hematopoietic stem cell transplantation.The conditioning regimen was consists of fludarabin(Flu), busulfan(Bu), cyclophosphamide(Cy) and antithymocyte globulin(ATG). All donors were HLA mismatched family members including fathers、mothers and sister. ABO blood type between donor and receipt were incompatible in three patients.GVHD prophylaxis included cyclosporine (CSA), and short course of methotrexate (MTX). Chimerism studies were performed with blood nucleated cells whth STR-PCR.
Results
The median of nucleated cell and CD34+ cell dose in the infused product were 9.7×108/kg (range, 6.9-26.7×108/kg) and 10.1×106/kg (range, 8.2-27.2×106/Kg), respectively. The median time to achieve absolute neutrophil recovery was in 10 days (range,10 to 15 days), and platelet recovery was in 13 days (range,10 to 102 days).Four patients (50%) have experienced grade I-II acute GVHD; Two patients suffered from grade III-IV (25%) acute GVHD and one of which turned into local chronic GVHD (skin).Chimerism studies were performed for all patients after transplantation. The data showing all patients have achieved full donor chimerism(100%) at post-transplantation +30 day and mixed chimerism status was not observed in all eight patients with the mdeian one year follow up.
So far, all of the patients have a stable engraftment and were transfusion independent in daily life.
Conclusion
The acute and chronic GVHD of haploidentical hematopoietic stem cell transplantation for thalassemia major based on this FBCA conditioning regimen is accpitable and the outcome prompt us to study further.
Session topic: 23. Stem cell transplantation - Clinical
Keyword(s): Haploidentical stem cell transplantation, Outcome, Thalassemia