Antonio Marzollo1, Elisabetta Calore1, Manuela Tumino1, Marta Pillon1, Maria Vittoria Gazzola1, Roberta Destro1, Raffaella Colombatti1, Piero Marson2, Tiziana Tison2, Anna Colpo2, Chiara Mainardi1, Maria Gabelli1, Maria Paola Boaro1, Sara Rossin1, Aurora Strano1, Nadia Quaglia1, Federica Menzato1, Giuseppe Basso1, Laura Sainati1 and Chiara Messina1
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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). 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.
|Table 1. Patient characteristics and outcomes. Abbreviations: ATG = antithymoglobulin; FLU = fludarabine in mg/m2; RITUX = rituximab; TREO = Treosulfan in g/m2; TT = Thiotepa in mg/kg.|
|Table 2. Brain imaging before and after HSCT for patients with cerebral vasculopathy. WHM: white matter hyperintensity; n/a: not available|
|Table 3. Transcranial Doppler Ultrasonography for patients with cerebral vasculopathy. Results were categorized as normal, conditional or abnormal according to the STOP trail criteria.40 n/a: not available.|