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Titolo:
Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen to disease type 1b
Autore:
Calderwood, S; Kilpatrick, L; Douglas, SD; Freedman, M; Smith-Whitley, K; Rolland, M; Kurtzberg, J;
Indirizzi:
Good Samaritan Reg Med Ctr, City Hope Samaritan Bone Marrow Transplant Progra, Phoenix, AZ USA Good Samaritan Reg Med Ctr Phoenix AZ USA splant Progra, Phoenix, AZ USA Childrens Hosp Philadelphia, Div Infect Dis & Immunol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia Philadelphia PA USA 19104 lphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia Philadelphia PA USA 19104 lphia, PA 19104 USA Hosp Sick Children, Div Hematol, Toronto, ON M5G 1X8, Canada Hosp Sick Children Toronto ON Canada M5G 1X8 Toronto, ON M5G 1X8, Canada Duke Univ, Med Ctr, Div Pediat, Durham, NC USA Duke Univ Durham NC USADuke Univ, Med Ctr, Div Pediat, Durham, NC USA
Titolo Testata:
BLOOD
fascicolo: 2, volume: 97, anno: 2001,
pagine: 376 - 382
SICI:
0006-4971(20010115)97:2<376:RHGCFT>2.0.ZU;2-F
Fonte:
ISI
Lingua:
ENG
Soggetto:
STORAGE-DISEASE; CSF TREATMENT; IN-VITRO; IB; CELLS; CORRECTS; DEFECTS;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Life Sciences
Citazioni:
27
Recensione:
Indirizzi per estratti:
Indirizzo: Kilpatrick, L City Hope Natl Med Ctr, Dept Pediat, 1500 E Duarte Rd, Duarte, CA 91010 USA City Hope Natl Med Ctr 1500 E Duarte Rd Duarte CA USA 91010
Citazione:
S. Calderwood et al., "Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen to disease type 1b", BLOOD, 97(2), 2001, pp. 376-382

Abstract

The purpose of this study was to evaluate the efficacy and toxicity of recombinant human granulocyte colony-stimulating factor (rhG-CSF) therapy in patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease (GSD) type 1b, Thirteen patients with neutropenia and/or neutrophil dysfunction secondary to GSD type 1b were treated with rhG-CSF, The effects of therapy on neutrophil numbers and in vitro neutrophil function and on bone marrow cellularity and morphology were studied. The clinicalstatus of the patients and the occurrence of adverse events associated with rhG-CSF use were monitored. Use of rhG-CSF therapy was associated with a significant increase in circulating neutrophil numbers (P < .01) and an improvement in neutrophil function as assessed in vitro. In addition, rhG-CSF therapy produced a significant increase in marrow cellularity and an increase in myeloid:erythroid (M:E) ratio, indicating stimulation of granulopoeisis, No adverse effects on marrow function were noted; in particular, no myelodysplasia or marrow exhaustion was seen. Use of rhG-CSF therapy was associated with objective and subjective improvements in infection-related morbidity, The therapy was well tolerated, although ail patients developed splenomegaly, and 5 patients developed mild hypersplenism that did not require any specific treatment. rhG-CSF therapy is efficacious in the management of neutropenia and neutrophil dysfunction associated with GSD type 1b, Patients on this therapy need to be monitored for hypersplenism. Continued follow-up will be necessary to confirm long-term safety; however, no significant short-term toxicity was noted. (C) 2001 by The American Society of Hematology.

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Documento generato il 11/07/20 alle ore 04:21:42