@article{Jan-Henning Klusmann_Dirk Reinhardt_Martin Zimmermann_Bernhard Kremens_Josef Vormoor_Michael Dworzak_Ursula Creutzig_Thomas Klingebiel_2011, place={Pavia, Italy}, title={The role of matched sibling donor allogeneic stem cell transplantation in pediatric high-risk acute myeloid leukemia: results from the AML-BFM 98 study}, volume={97}, url={https://haematologica.org/article/view/6184}, DOI={10.3324/haematol.2011.051714}, abstractNote={<strong>Background</strong> The role of allogeneic stem cell transplantation in post-remission management of children with high-risk acute myeloid leukemia remains controversial. In the multi-center AML-BFM 98 study we prospectively evaluated the impact of allogeneic stem cell transplantation in children with high-risk acute myeloid leukemia in first complete remission.<strong>Design and Methods</strong> HLA-typed patients with high-risk acute myeloid leukemia, who achieved first complete remission (n=247), were included in this analysis. All patients received double induction and consolidation. Based on the availability of a matched-sibling donor, patients were allocated by genetic chance to allogeneic stem cell transplantation (n=61) or chemotherapy-only (i.e. intensification and maintenance therapy; n=186). The main analysis was done on an intention-to-treat basis according to this allocation.<strong>Results</strong> Intention-to-treat analysis did not show a significantly different 5-year disease-free survival (49±6% <em>versus</em> 45±4%, <em>P</em><sub>log rank</sub>=0.44) or overall survival (68±6% <em>versus</em> 57±4%, <em>P</em><sub>log rank</sub>=0.17) between the matched-sibling donor and no-matched-sibling donor groups, whereas late adverse effects occurred more frequently after allogeneic stem cell transplantation (72.5% <em>versus</em> 31.8%, <em>P</em><sub>Fischer</sub><0.01). These results were confirmed by as-treated analysis corrected for the time until transplantation (5-year overall survival: 72±8% <em>versus</em> 60±4%, <em>P</em><sub>Mantel-Byar</sub> 0.21). Subgroup analysis demonstrated improved survival rates for patients with 11q23 aberrations allocated to allogeneic stem cell transplantation (5-year overall survival: 94±6% <em>versus</em> 52±7%, <em>P</em><sub>log-rank</sub>=0.01; n=18 <em>versus</em> 49) in contrast to patients without 11q23 aberrations (5-year overall survival: 58±8% <em>versus</em> 55±5%, <em>P</em><sub>log-rank</sub>=0.66).<strong>Conclusions</strong&gt; Our analyses defined a genetic subgroup of children with high-risk acute myeloid leukemia who benefited from allogeneic stem cell transplantation in the prospective multi-center AML-BFM 98 study. For the remainder of the pediatric high-risk acute myeloid leukemia patients the prognosis was not improved by allogeneic stem cell transplantation, which was, however, associated with a higher rate of late sequelae.}, number={1}, journal={Haematologica}, author={Jan-Henning Klusmann and Dirk Reinhardt and Martin Zimmermann and Bernhard Kremens and Josef Vormoor and Michael Dworzak and Ursula Creutzig and Thomas Klingebiel}, year={2011}, month={Dec.}, pages={21-29} }