Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation

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dc.contributor.authorZeidler, C
dc.contributor.authorWelte, K
dc.contributor.authorBarak, Y
dc.contributor.authorBarriga, F
dc.contributor.authorBolyard, AA
dc.contributor.authorBoxer, L
dc.contributor.authorCornu, G
dc.contributor.authorCowan, MJ
dc.contributor.authorDale, DC
dc.contributor.authorFlood, T
dc.contributor.authorFreedman, M
dc.contributor.authorGadner, H
dc.contributor.authorMandel, H
dc.contributor.authorO'Reilly, RJ
dc.contributor.authorRamenghi, U
dc.contributor.authorReiter, A
dc.contributor.authorSkinner, R
dc.contributor.authorVermylen, C
dc.contributor.authorLevine, JE
dc.date.accessioned2025-01-21T01:31:33Z
dc.date.available2025-01-21T01:31:33Z
dc.date.issued2000
dc.description.abstractSevere congenital neutropenia (CN) (Kostmann syndrome) is a hematologic disorder characterized by a maturation arrest of myelopoiesis at the promyelocyte/myelocyte stage of development, This arrest results in severe neutropenia leading to absolute neutrophil counts (ANC) below 0.2 x 10(9)/L associated with severe bacterial infections from early infancy. Data on over 300 patients with CN collected by the Severe Chronic Neutropenia International Registry (SCNIR) beginning in 1994 indicate that more than 90% of these patients respond to recombinant human granulocyte colony stimulating factor (r-HuG-CSF) treatment with an ANC greater than 1.0 x 10(9)/L. For patients who are refractory to r-HUG-CSF treatment and continue to have severe and often life-threatening bacterial infections, hematopoietic stem cell transplantation is the only currently available treatment. We report on a total of 11 patients with CN reported to the SCNIR who underwent transplantation for reasons other than malignant transformation between 1976 and 1998. Of these patients, 8 were nonresponders or showed only partial response to r-HuG-CSF treatment with ongoing infections. Results from these patients suggest that transplantation of stem cells from an HLA-identical sibling is beneficial for patients refractory to r-HuG-CSF. (Blood. 2000;95:1195-1198) (C) 2000 by The American Society of Hematology.
dc.fuente.origenWOS
dc.identifier.issn0006-4971
dc.identifier.urihttps://repositorio.uc.cl/handle/11534/97092
dc.identifier.wosidWOS:000085261600015
dc.issue.numero4
dc.language.isoen
dc.pagina.final1198
dc.pagina.inicio1195
dc.revistaBlood
dc.rightsacceso restringido
dc.subject.ods03 Good Health and Well-being
dc.subject.odspa03 Salud y bienestar
dc.titleStem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation
dc.typeartículo
dc.volumen95
sipa.indexWOS
sipa.trazabilidadWOS;2025-01-12
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