TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE
Background and objectives Lack of suitable donors and regimen related toxicity are major barriers for hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) when employing the most frequently used Busulfan-based conditioning regimen. The aim of the study is the as...
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PAGEPress Publications
2017-02-01
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| Series: | Mediterranean Journal of Hematology and Infectious Diseases |
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| Online Access: | http://www.mjhid.org/index.php/mjhid/article/view/2783 |
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| author | Antonio Marzollo Elisabetta Calore Manuela Tumino Marta Pillon Maria Vittoria Gazzola Roberta Destro Raffaella Colombatti Piero Marson Tiziana Tison Anna Colpo Chiara Mainardi Maria Gabelli Maria Paola Boaro Sara Rossin Aurora Strano Nadia Quaglia Federica Menzato Giuseppe Basso Laura Sainati Chiara Messina |
| author_facet | Antonio Marzollo Elisabetta Calore Manuela Tumino Marta Pillon Maria Vittoria Gazzola Roberta Destro Raffaella Colombatti Piero Marson Tiziana Tison Anna Colpo Chiara Mainardi Maria Gabelli Maria Paola Boaro Sara Rossin Aurora Strano Nadia Quaglia Federica Menzato Giuseppe Basso Laura Sainati Chiara Messina |
| author_sort | Antonio Marzollo |
| collection | DOAJ |
| description | Background and objectives Lack of suitable donors and regimen related toxicity are major barriers for hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) when employing the most frequently used Busulfan-based conditioning regimen. The aim of the study is the assessment of efficacy and toxicity of Treosulfan-based conditioning regimen for SCD also when alternative donors such as mismatched unrelated donor and haploidentical donor are employed.
Methods We report our single-center experience: 11 patients with sickle cell disease received HSCT with a Treosulfan/Thiotepa/Fludarabine/Anti-thymoglobulin conditioning regimen between 2010 and 2015. The donor was a matched sibling donor (n= 7), a haploidentical parent (n= 2), a matched unrelated donor (n= 1) or a mismatched unrelated donor (n=1). The haploidentical and mismatched unrelated donor grafts were manipulated by removing TCRαβ and CD19 positive cells.
Results All patients survived the procedure and achieved stable engraftment. Stable mixed chimerism but no SCD manifestation was observed in 5/11 patients. Grade III-IV regimen related toxicity was limited to mucositis and no grade III-IV graft-versus-host disease (GvHD) was observed. Organ function evaluation showed no long term pulmonary, cardiac or renal toxicity; cerebral vasculopathy improved in 3/5 evaluable patients. Gonadal failure was observed in 1/4 evaluable patients.
Conclusion Our data suggest that Treosulfan retains the myeloablative potential of Busulfan while reducing the toxicity also when haploidentical or unrelated donors are employed. |
| format | Article |
| id | doaj-art-8c3bd97e90b0408eb3b6b8beb942d596 |
| institution | DOAJ |
| issn | 2035-3006 |
| language | English |
| publishDate | 2017-02-01 |
| publisher | PAGEPress Publications |
| record_format | Article |
| series | Mediterranean Journal of Hematology and Infectious Diseases |
| spelling | doaj-art-8c3bd97e90b0408eb3b6b8beb942d5962025-08-20T02:50:59ZengPAGEPress PublicationsMediterranean Journal of Hematology and Infectious Diseases2035-30062017-02-0191e2017014e201701410.4084/mjhid.2017.0141666TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASEAntonio Marzollo0Elisabetta Calore1Manuela Tumino2Marta Pillon3Maria Vittoria Gazzola4Roberta Destro5Raffaella Colombatti6Piero Marson7Tiziana Tison8Anna Colpo9Chiara Mainardi10Maria Gabelli11Maria Paola Boaro12Sara Rossin13Aurora Strano14Nadia Quaglia15Federica Menzato16Giuseppe Basso17Laura Sainati18Chiara Messina19Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Via Giustiniani 3, Padova 35128, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyDepartment of Transfusion Medicine, Azienda Ospedaliera PadovaDepartment of Transfusion Medicine, Azienda Ospedaliera PadovaDepartment of Transfusion Medicine, Azienda Ospedaliera PadovaPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyPediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, ItalyBackground and objectives Lack of suitable donors and regimen related toxicity are major barriers for hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) when employing the most frequently used Busulfan-based conditioning regimen. The aim of the study is the assessment of efficacy and toxicity of Treosulfan-based conditioning regimen for SCD also when alternative donors such as mismatched unrelated donor and haploidentical donor are employed. Methods We report our single-center experience: 11 patients with sickle cell disease received HSCT with a Treosulfan/Thiotepa/Fludarabine/Anti-thymoglobulin conditioning regimen between 2010 and 2015. The donor was a matched sibling donor (n= 7), a haploidentical parent (n= 2), a matched unrelated donor (n= 1) or a mismatched unrelated donor (n=1). The haploidentical and mismatched unrelated donor grafts were manipulated by removing TCRαβ and CD19 positive cells. Results All patients survived the procedure and achieved stable engraftment. Stable mixed chimerism but no SCD manifestation was observed in 5/11 patients. Grade III-IV regimen related toxicity was limited to mucositis and no grade III-IV graft-versus-host disease (GvHD) was observed. Organ function evaluation showed no long term pulmonary, cardiac or renal toxicity; cerebral vasculopathy improved in 3/5 evaluable patients. Gonadal failure was observed in 1/4 evaluable patients. Conclusion Our data suggest that Treosulfan retains the myeloablative potential of Busulfan while reducing the toxicity also when haploidentical or unrelated donors are employed.http://www.mjhid.org/index.php/mjhid/article/view/2783Anemia, Stem Cell transplantation |
| spellingShingle | Antonio Marzollo Elisabetta Calore Manuela Tumino Marta Pillon Maria Vittoria Gazzola Roberta Destro Raffaella Colombatti Piero Marson Tiziana Tison Anna Colpo Chiara Mainardi Maria Gabelli Maria Paola Boaro Sara Rossin Aurora Strano Nadia Quaglia Federica Menzato Giuseppe Basso Laura Sainati Chiara Messina TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE Mediterranean Journal of Hematology and Infectious Diseases Anemia, Stem Cell transplantation |
| title | TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE |
| title_full | TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE |
| title_fullStr | TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE |
| title_full_unstemmed | TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE |
| title_short | TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE |
| title_sort | treosulfan based conditioning regimen in sibling and alternative donor hematopoietic stem cell transplantation for children with sickle cell disease |
| topic | Anemia, Stem Cell transplantation |
| url | http://www.mjhid.org/index.php/mjhid/article/view/2783 |
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