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Allografting

Stem cell transplantation in patients with severe congenital neutropenia with evidence of leukemic transformation

Bone Marrow Transplantation volume 35, pages 473–477 (2005)Cite this article

Summary:

Severe congenital neutropenia (SCN) is a hematologic condition characterized by arrested maturation of myelopoiesis at the promyelocyte stage of development. With appropriate treatment using recombinant human granulocyte-colony-stimulating factor (r-HuG-CSF), SCN patients are now surviving longer, but are at increased risk of developing myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML). Hematopoietic stem cell transplantation (HSCT) is the only curative option for these patients, but transplantation outcomes after malignant transformation are not well established. We report results for six patients with SCN who underwent HSCT for MDS or AML between 1997 and 2001 at two transplant centers. Two patients transplanted for MDS survived. Both of these patients were transplanted without being given induction chemotherapy. Four patients, who all received induction chemotherapy for AML prior to HSCT, died. Administering induction chemotherapy prior to HSCT resulted in significant morbidity. Rapid transplantation should be the goal for the SCN patient once the diagnosis of MDS/AML is established. SCN patients should be monitored carefully for progression to MDS in order to be treated with HSCT as soon as they have progressed and before developing AML. For SCN patients who progress to AML, HSCT should still be considered, even though the risks appear to be greater.

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Authors and Affiliations

  1. Division of Pediatric Hematology-Oncology, University of Michigan, Ann Arbor, MI, USA

    S W Choi, L A Boxer, R J Hutchinson, G A Yanik, K R Cooke, J L M Ferrara & J E Levine

  2. Division of Hematology/Bone Marrow Transplantation, Primary Children's Hospital, Salt Lake City, UT, USA

    M A Pulsipher

  3. Department of Pathology, University of Michigan, Ann Arbor, MI, USA

    D Roulston

Authors
  1. S W Choi
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  2. L A Boxer
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  3. M A Pulsipher
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  4. D Roulston
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  5. R J Hutchinson
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  6. G A Yanik
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  7. K R Cooke
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  8. J L M Ferrara
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  9. J E Levine
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Correspondence to J E Levine.

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Choi, S., Boxer, L., Pulsipher, M. et al. Stem cell transplantation in patients with severe congenital neutropenia with evidence of leukemic transformation. Bone Marrow Transplant 35, 473–477 (2005). https://doi.org/10.1038/sj.bmt.1704813

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  • DOI: https://doi.org/10.1038/sj.bmt.1704813

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