Yazar "Locatelli, Franco" seçeneğine göre listele

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    Age Is a Crucial Determinant of GFRS with Incidence of Severe Chronic GVHD Reducing over Time in Haemopoietic Cell Transplantation for Transfusion Dependent Thalassaemia: Real World Data from 2010-2021. an Analysis of the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Working Party
    (ELSEVIER, 2024) Baronciani, Donatella; de la Fuente, Josu; Galimard, Jacques-Emanuel; Dalissier, Arnaud; Mousavi, Ashrafsadat; Yeşilipek, Akif; Hashem, Hasan; Öztürk, Gülyüz; Locatelli, Franco; Alahmari, Ali; Gaziev, Javid; Antmen, Ali Bülent; Küpesiz, O. Alphan; Fışgın, Tunç; La Nasa, Giorgio; Lankester, Arjan C.; Goussetis, Eugene; Bremathas, Sandrine
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    Busulfan–fudarabine- or treosulfan–fudarabine-based myeloablative conditioning for children with thalassemia major
    (Annals of Hematology, 2021) Lüftinger, Roswitha; Zubarovskaya, Natalia; Galimard, Jacques‑Emmanuel; Cseh, Annamaria; Salzer, Elisabeth; Locatelli, Franco; Algeri, Mattia; Yeşilipek, Akif; Fuente, Josu de la; Isgro, Antonella; Alseraihy, Amal; Angelucci, Emanuele; Smiers, Frans J.; Nasa, Giorgia La La; Zecca, Marco; Fışgın, Tunç; Ünal, Emel; Kleinschmidt, Katharina; Peters, Christina; Lankester, Arjan; Çorbacıoğlu, Selim
    Signifcant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients’ life expectancy. However, transfusion-associated iron overload remains a signifcant barrier to long-term survival with good quality of life. Today, allogeneic hematopoietic stem cell transplantation (HSCT) is the current curative standard of care. Alongside selection of the best available donor, an optimized conditioning regimen is crucial to maximize outcomes for patients with TDT undergoing HSCT. The aim of this retrospective analysis was to investigate the role of busulfan– fudarabine-based and treosulfan–fudarabine-based conditioning in TDT patients undergoing HSCT. We included 772 patients registered in the European Society for Blood and Marrow Transplantation (EBMT) database who underwent frst HSCT between 2010 and 2018. Four hundred ten patients received busulfan–fudarabine-based conditioning (median age 8.6 years) and 362 patients received treosulfan–fudarabine-based conditioning (median age 5.7 years). Patient outcomes were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confdence interval: 89.3–95.1%) after busulfan–fudarabine-based conditioning and 94.7% (95% confdence interval: 91.7–96.6%) after treosulfan–fudarabine-based conditioning. There was a very low incidence of second HSCT overall. The main causes of death were infections, graft-versus-host disease, and rejection. In conclusion, use of busulfan or treosulfan as the backbone of myeloablative conditioning for patients with TDT undergoing HSCT resulted in comparably high cure rates. Long-term follow-up studies are warranted to address the important issues of organ toxicities and gonadal function.
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    Outcomes of HLA-mismatched HSCT with TCR?ß/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity
    (2024) Lum, Su Han; Albert, Michael H.; Gilbert, Patrick; Sirait, Tiarlan; Algeri, Mattia; Muratori, Rafaella; Fournier, Benjamin; Laberko, Alexandra; Karakükcü, Musa; Ünal, Ekrem; Ayas, Mouhab F.; Yadav, Satya Prakash; Fışgın, Tunç; Elfeky, Reem; Fernandes, Juliana Folloni; Faraci, Maura; Cole, Theresa; Schulz, Ansgar S.; Meisel, Roland; Zecca, Marco; Ifversen, Marienne; Biffi, Alessandra; Diana, Jean-Sebastien; Vallee, Tanja C.; Giardino, Stefano; Ersoy, Gizem Zengin; Moshous, Despina; Gennery, Andrew R.; Balashov, Dmitry; Bonfim Carmem M. S.; Locatelli, Franco; Lankester, Arjan C.; Neven, Benedicte; Slatter, Mary A.
    HLA-mismatched transplants with either in vitro depletion of CD3+TCRαβ/CD19 (TCRαβ) cells or in vivo T-cell depletion using post-transplant cyclophosphamide (PTCY) have been increasingly used for patients with inborn errors of immunity (IEI). We performed a retrospective multicenter study via the EBMT registry on 306 children with IEI undergoing first transplant between 2010-2019 from an HLA-mismatched donor using TCRαβ (n=167) or PTCY (n=139). Median age at HSCT was 1.2 years (range, 0.03-19.6 years). The 3-year overall survival (OS) was 78% (95% confidence interval (CI), 71-84%) after TCRαβ and 66% (57-74%) after PTCY (p=0.013). Pre-HSCT morbidity score (hazard ratio (HR) 2.27, 1.07-4.80, p=0.032) and non-Busulfan/Treosulfan conditioning (HR 3.12, 1.98-4.92, p<0.001) were the only independent predictors of unfavorable OS. The 3-year event-free survival (EFS) was 58% (50-66%) after TCRαβ and 57% (48-66%) after PTCY (p=0.804). Cumulative incidence of severe acute GvHD was higher after PTCY (15%, 9-21%) than TCRαβ (6%, 2-9%, p=0.007), with no difference in chronic GvHD (PTCY, 11%, 6-17%; TCRαβ, 7%, 3-11%, p=0.173). The 3-year GvHD-free EFS was 53% (44-61%) after TCRαβ and 41% (32-50%) after PTCY (p=0.080). PTCY had significantly higher rates of veno-occlusive disease (14.4% versus TCRαβ 4.9%, p=0.009), acute kidney injury (12.7% versus 4.6%, p=0.032) and pulmonary complications (38.2% versus 24.1%, p=0.017). Adenoviraemia (18.3% versus PTCY 8.0%, p=0.015), primary graft failure (10%, versus 5%, p=0.048), and second HSCT (17.4% versus 7.9%, p=0.023) were significantly higher in TCRαβ. In conclusion, this study demonstrates that both approaches are suitable options in IEI patients, although characterized by different advantages and outcomes.