Recombinant human granulocyte colony-stimulating factor promotes wound healing in a patient with congenital neutropenia

1992 ◽  
Vol 27 (3) ◽  
pp. 288-291 ◽  
Author(s):  
Gail E. Besner ◽  
Philip L. Glick ◽  
Melvyn P. Karp ◽  
Winfred C. Wang ◽  
Thom E. Lobe ◽  
...  
Keyword(s):  
Wound Healing ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Congenital Neutropenia ◽  
Stimulating Factor

scholarly journals Blood mononuclear cells from patients with severe congenital neutropenia are capable of producing granulocyte colony-stimulating factor

Blood ◽  
1991 ◽  
Vol 77 (6) ◽  
pp. 1234-1237 ◽  
Author(s):  
T Pietsch ◽  
C Buhrer ◽  
K Mempel ◽  
T Menzel ◽  
U Steffens ◽  
...  
Keyword(s):  
Mononuclear Cells ◽  
Leukemia Cell Line ◽  
Severe Neutropenia ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Severe Congenital Neutropenia ◽  
Congenital Neutropenia ◽  
Blood Mononuclear Cells ◽  
Stimulating Factor

Abstract Severe congenital neutropenia (SCN) is a disorder of myelopoiesis characterized by severe neutropenia or absence of blood neutrophils secondary to a maturational arrest at the level of promyelocytes. We examined peripheral blood mononuclear cells (PBMC) of SCN patients who demonstrated normalization of their blood neutrophil counts in a phase II clinical study with recombinant human granulocyte colony-stimulating factor (rhG-CSF). When stimulated in vitro with bacterial lipopolysaccharides (LPS), PBMC of those SCN patients produced G-CSF activity, as judged by proliferation induction of the murine leukemia cell line, NFS-60. Western and Northern blot analysis showed G-CSF protein and G-CSF-mRNA indistinguishable in size from those of normal controls. We conclude that PBMC of the SCN patients tested are capable of synthesizing and secreting biologically active G-CSF in vitro.

Spontaneous remission of granulocyte colony-stimulating factor–associated leukemia in a child with severe congenital neutropenia

Blood ◽  
2000 ◽  
Vol 96 (10) ◽  
pp. 3647-3649 ◽  
Author(s):  
Sima Jeha ◽  
Ka Wah Chan ◽  
Andrew G. Aprikyan ◽  
W. Keith Hoots ◽  
Steven Culbert ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Allogeneic Bone Marrow Transplantation ◽  
Spontaneous Remission ◽  
Myelogenous Leukemia ◽  
Allogeneic Bone ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Severe Congenital Neutropenia ◽  
Congenital Neutropenia ◽  
Stimulating Factor

Leukemia is observed with increased frequency in patients with severe congenital neutropenia (SCN). In the past decade, recombinant human granulocyte colony-stimulating factor (rh G-CSF) has prolonged the survival of patients with SCN increasingly reported to have leukemias. In this communication acute myelogenous leukemia (AML) associated with a mutation of the G-CSF receptor (G-CSF-R) developed in a patient with SCN maintained on long-term G-CSF therapy. The blast count in the blood and bone marrow fell to undetectable levels twice on withholding G-CSF and without chemotherapy administration, but the mutant G-CSF-R was detectable during this period. The patient subsequently underwent successful allogeneic bone marrow transplantation. After transplantation, the patient's neutrophil elastase (ELA-2) mutation and G-CSF-R mutation became undetectable by polymerase chain reaction. This report provides novel insights on leukemia developing in congenital neutropenia.

Sweet's syndrome induced by granulocyte colony-stimulating factor in a woman with congenital neutropenia

1996 ◽  
Vol 35 (4) ◽  
pp. 629-631 ◽  
Author(s):  
Marie Aleth Richard ◽  
Jean Jacques Grob ◽  
Renaud Laurans ◽  
Sylvie Hesse ◽  
Philippe Brunet ◽  
...  
Keyword(s):  
Sweet’S Syndrome ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Congenital Neutropenia ◽  
Sweet's Syndrome ◽  
Stimulating Factor

scholarly journals ASSESSMENT OF CONGENITAL NEUTROPENIA IN CHILDREN: COMMON CLINICAL SCENERIES AND CLUES FOR MANAGEMENT

2022 ◽  
Vol 14 (1) ◽  
pp. e2022008
Author(s):  
Ilaria Lazzareschi ◽  
Elena Rossi ◽  
Antonietta Curatola ◽  
Giovanna Capozio ◽  
Luca Benacquista ◽  
...  
Keyword(s):  
Hematologic Malignancy ◽  
Neutrophil Granulocytes ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Congenital Neutropenia ◽  
Cyclic Neutropenia ◽  
Hematological Diseases ◽  
Neutropenic Patients ◽  
The Many ◽  
Stimulating Factor

A disparate group of rare hematological diseases characterized by impaired maturation of neutrophil granulocytes defines congenital neutropenias. Neutropenic patients are prone to recurrent infections beginning in the first months of life. Of interest is “cyclic neutropenia”, an ultra-rare disorder revealed by sinusoidal variations of the neutrophil count and periodically-recurring infections every 21 days. Diagnosis of these disorders is frequently obscured by the multiple causes of recurrent fevers in children. Aim of this overview is to outline the physical assessment of children presenting with early-onset symptomatic neutropenia, identify the disease between the many medical conditions and even emergencies which should enter in differential diagnosis, hint at the potential management with granulocyte-colony stimulating factor, define the risk of evolution to hematologic malignancy, and summarize inter-professional team strategies for improving care coordination and outcomes of such patients.

scholarly journals Reduction of Granulocyte-Colony Stimulating Factor Requirement in the Course of Long-Term Treatment for More Than 5 Years in Congenital Neutropenia Patients Harbouring ELANE Mutations

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1399-1399
Author(s):  
Cornelia Zeidler ◽  
Anna Nickel ◽  
Ulrike A.H. Grote ◽  
Sabine Mellor-Heineke ◽  
Karl Welte
Keyword(s):  
Body Weight ◽  
Treatment Duration ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Congenital Neutropenia ◽  
Long Term Treatment ◽  
Stimulating Factor ◽  
Chronic Neutropenia

Abstract Congenital neutropenias include a heterogeneous group of diseases characterized by a decrease in circulating neutrophils and different underlying germ-line gene mutations. Since 1988 recombinant human G-CSF is available for the treatment of severe chronic neutropenia patients. In phase I/II/III studies in patients with severe congenital and cyclic neutropenia, treatment with recombinant human granulocyte colony-stimulating factor (r-metHuG-CSF) resulted in a rise in the absolute neutrophil counts (ANC) and a reduction in infections. The Severe Chronic Neutropenia International Registry collects clinical information on patients suffering from severe chronic neutropenia since 1994. 312 of 379 CN and 65 of 79 CyN patients receive long-term G-CSF treatment for a median duration of 8,26 years in CN and 9,37 in CyN. Median G-CSF doses vary by neutropenia subtype and gene mutation. Patients with congenital neutropenia revealing ELANE mutations require the highest G-CSF doses compared to other subtypes (median G-CSF dose 5 µg/kg/day in 88 patients). SCNIR follow-up data suggest that pediatric ELANE-CN patients were maintained at a particular G-CSF dose per kg body weight for longer than expected by the gain of body weight. We therefore analysed the reported yearly G-CSF doses in all treated ELANE-CN patients to evaluate the dose trend: From 88 G-CSF treated patients with ELANE-CN we excluded 16 patients with nonresponse (ANC remained below 0.5 x 109 /L) and partial response (ANC remained between 0.5 and 0.99 x 109 /L). Since the gain of body weight is highest during the first 5 years of life with and 10-fold increase we divided G-CSF good responders by age at G-CSF initiation (0-5 years vs above 6 years) and compared G-CSF doses at the end of the dose finding period with the last dose reported. 51 of the remaining 72 patients started G-CSF treatment between the first and 5th year of life with a median G-CSF treatment duration of 9.76 years. 37 of the 51 patients were treated for at least 5 years. In 21 of the 72 patients G-CSF treatment was initiated after their 10thbirthday with a median follow up of 20.45 years. 19 of the 21 patients were treated for at least 5 years. All patients with a treatment duration of less than 5 years were excluded from further analysis. In ELANE-CN patients with treatment start at an age of 0-5 years the mean G-CSF dose decreased significantly from 13.47 µg/kg/day at the time of ANC-response to 7.96 µg/kg/day at the last report (median G-CSF dose decreased from 6.85 µg/kg/day to 4.42 µg/kg/day) during a treatment duration of at least 5 years. In summary, a significant decrease in the individual G-CSF doses could be observed in ELANE-CN patients who started G-CSF treatment during the first five years of their lives suggesting an age dependent alleviation of the severity of the disease as judged by the response to G-CSF. Disclosures No relevant conflicts of interest to declare.

scholarly journals Increased serum levels of granulocyte colony-stimulating factor in patients with severe congenital neutropenia

Blood ◽  
1991 ◽  
Vol 77 (9) ◽  
pp. 1919-1922 ◽  
Author(s):  
K Mempel ◽  
T Pietsch ◽  
T Menzel ◽  
C Zeidler ◽  
K Welte
Keyword(s):  
Serum Levels ◽  
Granulocyte Colony Stimulating Factor ◽  
Colony Stimulating Factor ◽  
Severe Congenital Neutropenia ◽  
Congenital Neutropenia ◽  
Maturation Arrest ◽  
Kostmann Syndrome ◽  
Stimulating Factor ◽  
Normal Controls ◽  
Csf Production

Severe congenital neutropenia (SCN), also known as Kostmann Syndrome, is characterized by a maturation arrest of myelopoiesis at the level of promyelocytes with absence of neutrophils in bone marrow (BM) and blood. Hypotheses of the pathophysiology of SCN include (1) defective production of granulocyte colony-stimulating factor (G-CSF), and/or (2) defective response to G-CSF. To exclude defective G-CSF production we tested sera from patients with SCN for the presence of G-CSF using Western blot analysis and NFS-60 proliferation assay. Using these assays we were able to detect increased G-CSF serum levels in SCN patients (150 to 910 pg/mL) as compared with normal controls (between undetectable and 100 pg/mL). These results suggest that patients with SCN have no defect in G-CSF production but a defective response of neutrophil precursors to endogenous G-CSF.

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