{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,11]],"date-time":"2025-10-11T13:07:48Z","timestamp":1760188068164},"reference-count":27,"publisher":"American Society of Hematology","issue":"2","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2001,1,15]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>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 clinical status 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\u2009&amp;lt;\u2009.01) 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 all 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.<\/jats:p>","DOI":"10.1182\/blood.v97.2.376","type":"journal-article","created":{"date-parts":[[2002,7,27]],"date-time":"2002-07-27T12:47:58Z","timestamp":1027774078000},"page":"376-382","update-policy":"http:\/\/dx.doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":64,"title":["Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and\/or neutrophil dysfunction secondary to glycogen storage disease type 1b"],"prefix":"10.1182","volume":"97","author":[{"given":"Stanley","family":"Calderwood","sequence":"first","affiliation":[{"name":"From the City of Hope\/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the Division of Immunologic and Infectious Diseases and the Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; the Division of Hematology, The Hospital for Sick Children, Toronto, Ontario, Canada; and the Division of Pediatrics, Duke University Medical Center, Durham, NC."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laurie","family":"Kilpatrick","sequence":"additional","affiliation":[{"name":"From the City of Hope\/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the Division of Immunologic and Infectious Diseases and the Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; the Division of Hematology, The Hospital for Sick Children, Toronto, Ontario, Canada; and the Division of Pediatrics, Duke University Medical Center, Durham, NC."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Steven D.","family":"Douglas","sequence":"additional","affiliation":[{"name":"From the City of Hope\/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the Division of Immunologic and Infectious Diseases and the Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; the Division of Hematology, The Hospital for Sick Children, Toronto, Ontario, Canada; and the Division of Pediatrics, Duke University Medical Center, Durham, NC."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Melvin","family":"Freedman","sequence":"additional","affiliation":[{"name":"From the City of Hope\/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the Division of Immunologic and Infectious Diseases and the Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; the Division of Hematology, The Hospital for Sick Children, Toronto, Ontario, Canada; and the Division of Pediatrics, Duke University Medical Center, Durham, NC."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kim","family":"Smith-Whitley","sequence":"additional","affiliation":[{"name":"From the City of Hope\/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the Division of Immunologic and Infectious Diseases and the Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; the Division of Hematology, The Hospital for Sick Children, Toronto, Ontario, Canada; and the Division of Pediatrics, Duke University Medical Center, Durham, NC."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martha","family":"Rolland","sequence":"additional","affiliation":[{"name":"From the City of Hope\/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the Division of Immunologic and Infectious Diseases and the Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; the Division of Hematology, The Hospital for Sick Children, Toronto, Ontario, Canada; and the Division of Pediatrics, Duke University Medical Center, Durham, NC."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joanne","family":"Kurtzberg","sequence":"additional","affiliation":[{"name":"From the City of Hope\/Samaritan Bone Marrow Transplant Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the Division of Immunologic and Infectious Diseases and the Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; the Division of Hematology, The Hospital for Sick Children, Toronto, Ontario, Canada; and the Division of Pediatrics, Duke University Medical Center, Durham, NC."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"234","reference":[{"key":"2020012418222453200_B1","first-page":"425","article-title":"Glycogen storage disease.","volume-title":"The Metabolic Basis of Inherited Disease.","author":"Hers","year":"1989"},{"key":"2020012418222453200_B2","doi-asserted-by":"crossref","first-page":"218","DOI":"10.7326\/0003-4819-120-3-199402010-00008","article-title":"Glycogen storage 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Pediatr."},{"key":"2020012418222453200_B6","doi-asserted-by":"crossref","first-page":"438","DOI":"10.1203\/00006450-198605000-00012","article-title":"Impaired chemotaxis and neutrophil (polymorphonuclear leukocyte) function in glycogenosis type 1B.","volume":"20","author":"Koven","year":"1986","journal-title":"Pediatr Res."},{"key":"2020012418222453200_B7","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1093\/infdis\/143.3.447","article-title":"Recurrent infection in glycogenosis type 1B: abnormal neutrophil motility related to impaired redistribution of adhesion sites.","volume":"143","author":"Anderson","year":"1981","journal-title":"J Infect Dis."},{"key":"2020012418222453200_B8","doi-asserted-by":"crossref","first-page":"196","DOI":"10.1172\/JCI114684","article-title":"Impaired metabolic function and signaling defects in phagocytic cells in glycogen storage disease type 1B.","volume":"86","author":"Kilpatrick","year":"1990","journal-title":"J Clin Invest."},{"key":"2020012418222453200_B9","doi-asserted-by":"crossref","first-page":"S39","DOI":"10.1007\/BF02072086","article-title":"Impairment of calcium mobilization in phagocytic cells in glycogen storage disease type 1b.","volume":"152(suppl 1)","author":"Korchak","year":"1993","journal-title":"Eur J Pediatr."},{"key":"2020012418222453200_B10","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1203\/00006450-199401000-00017","article-title":"In vitro and in vivo effects of granulocyte colony stimulating factor on neutrophils in glycogen storage disease type 1b: granulocyte colony stimulating factor therapy corrects the neutropenia and the defects in respiratory burst activity and Ca2+ mobilization.","volume":"35","author":"McCawley","year":"1994","journal-title":"Ped Res."},{"key":"2020012418222453200_B11","doi-asserted-by":"crossref","first-page":"1508","DOI":"10.1182\/blood.V69.5.1508.1508","article-title":"Activities of four purified growth factors on highly enriched 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1b.","volume":"5","author":"Wang","year":"1991","journal-title":"Leukemia."},{"key":"2020012418222453200_B15","doi-asserted-by":"crossref","first-page":"2496","DOI":"10.1182\/blood.V81.10.2496.2496","article-title":"A randomized controlled phase III trial of recombinant human granulocyte colony-stimulating factor (filgrastim) for treatment of severe chronic neutropenia.","volume":"81","author":"Dale","year":"1993","journal-title":"Blood."},{"key":"2020012418222453200_B16","doi-asserted-by":"crossref","first-page":"266","DOI":"10.1203\/00006450-199309000-00005","article-title":"Interferon-gamma corrects the respiratory burst defect in vitro in monocyte-derived macrophages from glycogen storage disease type 1b patients.","volume":"34","author":"McCawley","year":"1993","journal-title":"Pediatric Research."},{"key":"2020012418222453200_B17","first-page":"24","article-title":"Examination of the marrow.","volume-title":"Hematology.","author":"Williams","year":"1990","edition":"4th ed."},{"key":"2020012418222453200_B18","doi-asserted-by":"crossref","first-page":"1666","DOI":"10.1056\/NEJM199206183262504","article-title":"Brief report: treatment of chronic inflammatory bowel disease in glycogen storage disease type 1B with colony-stimulating factors.","volume":"326","author":"Roe","year":"1992","journal-title":"New Engl J Med."},{"key":"2020012418222453200_B19","doi-asserted-by":"crossref","first-page":"3422","DOI":"10.4049\/jimmunol.139.10.3422","article-title":"Effects of recombinant human granulocyte and macrophage colony-stimulating factors on signal transduction pathways in human granulocytes.","volume":"139","author":"Sullivan","year":"1987","journal-title":"J Immunol."},{"key":"2020012418222453200_B20","first-page":"576","article-title":"Recombinant human G-CSF and GM-CSF prime human neutrophils for superoxide production through different signal transduction mechanisms.","volume":"118","author":"Balazovich","year":"1991","journal-title":"J Lab Clin 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