Browsing by Author "Bordon, Victoria"

Filter results by typing the first few letters
Now showing 1 - 2 of 2
  • No Thumbnail Available
    ItemMetadata only
    Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.
    (Blood Adv, 2018-01-09) van den Broek, Brigitte TA; Page, Kristin; Paviglianiti, Annalisa; Hol, Janna; Allewelt, Heather; Volt, Fernanda; Michel, Gerard; Diaz, Miguel Angel; Bordon, Victoria; O'Brien, Tracey; Shaw, Peter J; Kenzey, Chantal; Al-Seraihy, Amal; van Hasselt, Peter M; Gennery, Andrew R; Gluckman, Eliane; Rocha, Vanderson; Ruggeri, Annalisa; Kurtzberg, Joanne; Boelens, Jaap Jan
    Leukodystrophies (LD) are devastating inherited disorders leading to rapid neurological deterioration and premature death. Hematopoietic stem cell transplantation (HSCT) can halt disease progression for selected LD. Cord blood is a common donor source for transplantation of these patients because it is rapidly available and can be used without full HLA matching. However, precise recommendations allowing care providers to identify patients who benefit from HSCT are lacking. In this study, we define risk factors and describe the early and late outcomes of 169 patients with globoid cell leukodystrophy, X-linked adrenoleukodystrophy, and metachromatic leukodystrophy undergoing cord blood transplantation (CBT) at an European Society for Blood and Marrow Transplantation center or at Duke University Medical Center from 1996 to 2013. Factors associated with higher overall survival (OS) included presymptomatic status (77% vs 49%;P= .006), well-matched (≤1 HLA mismatch) CB units (71% vs 54%;P= .009), and performance status (PS) of >80 vs <60 or 60 to 80 (69% vs 32% and 55%, respectively;P= .003). For patients with PS≤60 (n = 20) or 60 to 80 (n = 24) pre-CBT, only 4 (9%) showed improvement. Of the survivors with PS >80 pre-CBT, 50% remained stable, 20% declined to 60 to 80, and 30% to <60. Overall, an encouraging OS was found for LD patients after CBT, especially for those who are presymptomatic before CBT and received adequately dosed grafts. Early identification and fast referral to a specialized center may lead to earlier treatment and, subsequently, to improved outcomes.
  • Thumbnail Image
    ItemOpen Access
    Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.
    (Blood, 2015-03) Aldenhoven, Mieke; Wynn, Robert F; Orchard, Paul J; O'Meara, Anne; Veys, Paul; Fischer, Alain; Valayannopoulos, Vassili; Neven, Benedicte; Rovelli, Attilio; Prasad, Vinod K; Tolar, Jakub; Allewelt, Heather; Jones, Simon A; Parini, Rossella; Renard, Marleen; Bordon, Victoria; Wulffraat, Nico M; de Koning, Tom J; Shapiro, Elsa G; Kurtzberg, Joanne; Boelens, Jaap Jan
    Mucopolysaccharidosis type I-Hurler syndrome (MPS-IH) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Although hematopoietic cell transplantation (HCT) has been performed in these patients for more than 30 years, large studies on the long-term outcome of patients with MPS-IH after HCT are lacking. The goal of this international study was to identify predictors of the long-term outcome of patients with MPS-IH after successful HCT. Two hundred seventeen patients with MPS-IH successfully engrafted with a median follow-up age of 9.2 years were included in this retrospective analysis. Primary endpoints were neurodevelopmental outcomes and growth. Secondary endpoints included neurologic, orthopedic, cardiac, respiratory, ophthalmologic, audiologic, and endocrinologic outcomes. Considerable residual disease burden was observed in the majority of the transplanted patients with MPS-IH, with high variability between patients. Preservation of cognitive function at HCT and a younger age at transplantation were major predictors for superior cognitive development posttransplant. A normal α-l-iduronidase enzyme level obtained post-HCT was another highly significant predictor for superior long-term outcome in most organ systems. The long-term prognosis of patients with MPS-IH receiving HCT can be improved by reducing the age at HCT through earlier diagnosis, as well as using exclusively noncarrier donors and achieving complete donor chimerism.