Mieke Aldenhoven

Publications (4)19.34 Total impact

• Article: Outcomes of transplantation using various hematopoietic cell sources in children with Hurler's syndrome after myeloablative conditioning.

  Jaap Jan Boelens, Mieke Aldenhoven, Duncan Purtill, Annalisa Ruggeri, Todd Defor, Robert Wynn, Ed Wraith, Marina Cavazzana-Calvo, Attilio Rovelli, Alain Fischer, Jakub Tolar, Vinod K Prasad, Maria Escolar, Eliane Gluckman, Anne O'Meara, Paul J Orchard, Paul Veys, Mary Eapen, Joanne Kurtzberg, Vanderson Rocha
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  ABSTRACT: Key points In Hurler Syndrome patients early referral for HCT, with the best available HLA-matched donor offers the best "event free survival".In Hurler syndrome patients HCT with a well matched unrelated cord blood unit is particularly attractive as the unit is readily available.
  Blood 03/2013; · 9.90 Impact Factor
• Article: Dried blood spot analysis: an easy and reliable tool to monitor the biochemical effect of hematopoietic stem cell transplantation in hurler syndrome patients.

  Mieke Aldenhoven, Tom J de Koning, Frans W Verheijen, Berthil H Prinsen, Frits A Wijburg, Ans T van der Ploeg, Monique G M de Sain-van der Velden, Jaapjan Boelens
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  ABSTRACT: Hurler syndrome (HS), the most severe phenotype in the spectrum of mucopolysaccharidosis type I, is caused by a deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). At present, hematopoietic stem cell transplantation (HSCT) is the only treatment able to prevent disease progression in the central nervous system, and therefore considered the treatment of choice in HS patients. Because IDUA enzyme activities after HSCT have been suggested to influence the prognosis of HS patients, monitoring these activities after HSCT remains highly important. The use of dried blood spots (DBS) for enzyme analysis can be a useful alternative to the conventional leukocyte assay. Importantly, this method allows for convenient worldwide shipment, and can therefore be applied to monitor patients from larger areas of the world, or during large-scale international studies. Furthermore, this method requires only a minimal amount of blood. From 13 HS patients receiving HSCT, 36 paired whole blood and DBS samples were analyzed to assess leukocyte and DBS IDUA activities, respectively. To correct for potential interfering factors, simultaneous assay of the alpha-Galactosidase-A (AGA) activity was performed in the DBS samples and an IDUA/AGA ratio was calculated. A strong linear correlation was demonstrated between the DBS IDUA/AGA ratio and the leukocyte IDUA activity (r(2) = .875, P < .001). This correlation was applicable to all enzyme activities, including the activities measured early after HSCT as well as heterozygous activities because of mixed chimerism or the use of a carrier donor. These results demonstrate that the DBS method is reliable to monitor the biochemical effect of HSCT in HS patients.
  Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 05/2010; 16(5):701-4. · 3.15 Impact Factor
• Source

  Available from: Anne O'Meara

  Article: Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome.

  Jaap Jan Boelens, Vanderson Rocha, Mieke Aldenhoven, Robert Wynn, Anne O'Meara, Gerard Michel, Irina Ionescu, Suhag Parikh, Vinod K Prasad, Paul Szabolcs, Maria Escolar, Eliane Gluckman, Marina Cavazzana-Calvo, Joanne Kurtzberg
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  ABSTRACT: Allogeneic stem cell transplantation (SCT) is considered effective in preventing disease progression in patients with Hurler syndrome (HS). Unrelated umbilical cord blood (UCB) grafts are suggested as an alternative to bone marrow (BM) or peripheral blood stem cells (PBSC). We studied 93 HS patients receiving an UCB graft to analyze risk factors for outcomes. The median time from diagnosis to transplant was 4.6 months, median follow-up was 29 months, and median number of nucleated CB cells infused was 7.6 x 10(7)/kg. Most of the patients received 1 or 2 HLA disparate grafts, and the most frequently used conditioning regimen was cyclophosphamide + busulfan (Bu/Cy). All patients received anti-T cell antibody. At post transplant day +60, the cumulative incidence of neutrophil engraftment was 85%. A younger age at transplant and a higher CD34(+) dose at infusion were favorably associated with engraftment. With the exception of 2 patients, all engrafted patients achieved full and sustained donor chimerism. The 3-year event-free survival (EFS) and 3-year overall survival (OS) rates were 70% and 77%, respectively. In a multivariate analyses, use of Bu/Cy and a shorter interval from diagnosis to transplant were predictors for improved EFS rate (82% for patients transplanted within 4.6 months after diagnosis compared to 57% for the rest). Improved outcomes from early transplantation and immediate availability of CB unit lead us to conclude that CB transplantation is a beneficial option, which should be considered expediently for children with HS.
  Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 06/2009; 15(5):618-25. · 3.15 Impact Factor
• Source

  Available from: umcutrecht.nl

  Article: The clinical outcome of Hurler syndrome after stem cell transplantation.

  Mieke Aldenhoven, Jaap Jan Boelens, Tom J de Koning
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  ABSTRACT: Hurler syndrome (HS) is a severe inborn error of metabolism causing progressive multi-system morbidity and death in early childhood. At present, stem cell transplantation (SCT) is the only available treatment that can prevent central nervous system disease progression in HS patients. Although SCT has been shown to be effective for several important clinical outcome parameters, the reported clinical outcome after successful SCT is variable among HS patients and there are still some major limitations. This review will focus on the clinical outcome of HS patients after successful SCT, with particular emphasis on the long-term outcome and complications. In addition, factors that are suggested to contribute to the variable outcome are outlined, as well as the limitations of SCT in HS patients.
  Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 06/2008; 14(5):485-98. · 3.15 Impact Factor