In this review, we present the following four main approaches for improving long-term disease control: 2. Modulating myelotoxicity 2.1. Busulfan dose The intensity of the conditioning regimen, especially the alkylating agent busulfan, has been challenged in several studies with conflicting results (...
Cumulative incidences of complete remission and disease-related mortality were compared using Gray-test. Results From April 2007 to January 2013, 163 pts were included: 34 (21%) pts had no donor; 115 (71%) pts had an HLA-matched donor (34% sibling and 37% unrelated) and 14 (9%) pts...
H. Myelodysplastic syndromes: revisiting the role of the bone marrow microenvironment in disease pathogenesis. Int J Hematol 95, 17–25 (2012). Article PubMed Google Scholar Geyh, S. et al. Insufficient stromal support in MDS results from molecular and functional deficits of mesenchymal stromal...
dInformation on whether patients had IPSS Low- or Intermediate-1-risk disease was available for 243 lenalidomide-treated and 116 untreated patients. The remaining patients had either Low- or Intermediate-1-risk disease, but exact IPSS scores were not clearly definable due to missing data. eData ...
Ideally, evidence supporting the beneficial effects of iron chelation in MDS should rest on proof of improved life expectancy and in exceptional cases, reversal of myocardial disease. However, such evidence is presently unavailable. The selection of MDS patients who may benefit from ICT involves a ...
The heterogeneity of the disease is seen as the culprit for the limited success. Perhaps we were focusing in the wrong direction, making treatment success nearly impossible. In the absolute knowledge that life is never endless, we should shift our focus towards HRQoL as the most important ...
The risk of disease related mortality without the transplant should be balanced with the NRM. Higher risk patients have short life expectancy with chemotherapy, and will generally benefit from the HSCT. Risk for NRM should be carefully eva-luated, and relies on functional age assessment related ...
Mice transgenic for the first exon of the human Huntington's disease (HD) gene carrying an expanded CAG repeat expansion (R6/2 line) develop a progressive neurologic phenotype with symptoms resembling those seen in HD. The overt symptoms of R6/2 mice worsen with age, resulting in a rapid ...
Assessment of prognosis is of paramount importance in heterogeneous MDS. Insights in pathophysiological mechanisms and development of investigational tools has brought forward a high number of features with a possible use as prognostic factors in this disease. After theoretical considerations on prognosis an...
Currently the only strategy with the potential of curing the disease is hematopoietic cell transplantation (HCT). In view of the patient's young age and the karyotype, HCT from an HLA-identical related or unrelated donor should be considered as recommended by 70% of respondents. We prefer relat...