TREOSULFAN-BASED CONDITIONING REGIMEN IN SIBLING AND ALTERNATIVE DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CHILDREN WITH SICKLE CELL DISEASE

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
  • Elisabetta Calore
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Manuela Tumino
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Marta Pillon
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Maria Vittoria Gazzola
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Roberta Destro
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Raffaella Colombatti
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Piero Marson
    Department of Transfusion Medicine, Azienda Ospedaliera Padova,
  • Tiziana Tison
    Department of Transfusion Medicine, Azienda Ospedaliera Padova,
  • Anna Colpo
    Department of Transfusion Medicine, Azienda Ospedaliera Padova,
  • Chiara Mainardi
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Maria Gabelli
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Maria Paola Boaro
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Sara Rossin
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Aurora Strano
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Nadia Quaglia
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Federica Menzato
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Giuseppe Basso
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Laura Sainati
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,
  • Chiara Messina
    Pediatric Hematology-Oncology Unit, Department of Women’s and Children’s Health, University of Padova, Italy,

Abstract

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.

Keywords

Anemia, Stem Cell transplantation

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