Granulocyte-macrophage colony stimulating factor and immunosuppression in the treatment of pediatric acquired severe aplastic anemia

Michael R. Jeng, Paula E. Naidu, Martha D. Rieman, Carlos Rodriguez-Galindo, Kerri A. Nottage, Donyell T. Thornton, Chin-Shang Li, Winfred C. Wiang

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

Background. Immunosuppressive therapy (IS) is effective in the treatment of patients with acquired severe aplastic anemia (SAA). An enhanced myeloid response and decreased infection risk may be possible with the addition of a hematopoietic cytokine. Published data on the combination of cytokines and IS in patients with SAA are limited. The addition of C-CSF to IS shortens the time to neutrophil count recovery, but may not improve overall survival. Because CM-CSF acts differently than C-CSF, its use in combination with IS may be different. Procedure. A retrospective chart review was performed on patients diagnosed with SAA and treated with IS and CM-CSF at St. Jude Children's Research Hospital. Hematologic recovery, prognostic factors, and infection data were collected. Results. Eighteen patients were included in this study. The median age at diagnosis was 7.2 years (range 1.8-17.0). Ten patients (56%) had a complete response, four (22%) a partial response, and four (22%) no response. Median time to erythrocyte and platelet transfusion independence were 90 (18,243) and 64 days (18-243), and to discontinuation of treatment 287 days (90-730). Median time to partial (ANC > 500) and full (ANC > 1,500) neutrophil recovery were 41 and 51 days, respectively. Seventeen documented discrete infections occurred in six patients over 36 patient years. Conclusions. CM-CSF, in addition to IS, may shorten time to neutrophil count recovery, may be beneficial in decreasing infection rates, and may improve platelet response in patients with SAA. However, consistent with studies utilizing C-CSF, CM-CSF probably does not affect overall response rate. To fully answer whether or not cytokine therapy is of added value to IS in pediatric patients, a multi-institutional randomized trial is needed.

Original languageEnglish (US)
Pages (from-to)170-175
Number of pages6
JournalPediatric Blood and Cancer
Volume45
Issue number2
DOIs
StatePublished - Aug 2005
Externally publishedYes

Fingerprint

Aplastic Anemia
Granulocyte-Macrophage Colony-Stimulating Factor
Immunosuppression
Immunosuppressive Agents
Pediatrics
Therapeutics
Neutrophils
Cytokines
Infection
Erythrocyte Transfusion
Platelet Transfusion
Blood Platelets
Survival

Keywords

  • Aplastic anemia
  • Cytokines
  • GM-CSF
  • Immune-suppression

ASJC Scopus subject areas

  • Cancer Research
  • Pediatrics, Perinatology, and Child Health
  • Hematology

Cite this

Jeng, M. R., Naidu, P. E., Rieman, M. D., Rodriguez-Galindo, C., Nottage, K. A., Thornton, D. T., ... Wiang, W. C. (2005). Granulocyte-macrophage colony stimulating factor and immunosuppression in the treatment of pediatric acquired severe aplastic anemia. Pediatric Blood and Cancer, 45(2), 170-175. https://doi.org/10.1002/pbc.20278

Granulocyte-macrophage colony stimulating factor and immunosuppression in the treatment of pediatric acquired severe aplastic anemia. / Jeng, Michael R.; Naidu, Paula E.; Rieman, Martha D.; Rodriguez-Galindo, Carlos; Nottage, Kerri A.; Thornton, Donyell T.; Li, Chin-Shang; Wiang, Winfred C.

In: Pediatric Blood and Cancer, Vol. 45, No. 2, 08.2005, p. 170-175.

Research output: Contribution to journalArticle

Jeng, MR, Naidu, PE, Rieman, MD, Rodriguez-Galindo, C, Nottage, KA, Thornton, DT, Li, C-S & Wiang, WC 2005, 'Granulocyte-macrophage colony stimulating factor and immunosuppression in the treatment of pediatric acquired severe aplastic anemia', Pediatric Blood and Cancer, vol. 45, no. 2, pp. 170-175. https://doi.org/10.1002/pbc.20278
Jeng, Michael R. ; Naidu, Paula E. ; Rieman, Martha D. ; Rodriguez-Galindo, Carlos ; Nottage, Kerri A. ; Thornton, Donyell T. ; Li, Chin-Shang ; Wiang, Winfred C. / Granulocyte-macrophage colony stimulating factor and immunosuppression in the treatment of pediatric acquired severe aplastic anemia. In: Pediatric Blood and Cancer. 2005 ; Vol. 45, No. 2. pp. 170-175.
@article{f85e0c5691504d268709edc60c921ead,
title = "Granulocyte-macrophage colony stimulating factor and immunosuppression in the treatment of pediatric acquired severe aplastic anemia",
abstract = "Background. Immunosuppressive therapy (IS) is effective in the treatment of patients with acquired severe aplastic anemia (SAA). An enhanced myeloid response and decreased infection risk may be possible with the addition of a hematopoietic cytokine. Published data on the combination of cytokines and IS in patients with SAA are limited. The addition of C-CSF to IS shortens the time to neutrophil count recovery, but may not improve overall survival. Because CM-CSF acts differently than C-CSF, its use in combination with IS may be different. Procedure. A retrospective chart review was performed on patients diagnosed with SAA and treated with IS and CM-CSF at St. Jude Children's Research Hospital. Hematologic recovery, prognostic factors, and infection data were collected. Results. Eighteen patients were included in this study. The median age at diagnosis was 7.2 years (range 1.8-17.0). Ten patients (56{\%}) had a complete response, four (22{\%}) a partial response, and four (22{\%}) no response. Median time to erythrocyte and platelet transfusion independence were 90 (18,243) and 64 days (18-243), and to discontinuation of treatment 287 days (90-730). Median time to partial (ANC > 500) and full (ANC > 1,500) neutrophil recovery were 41 and 51 days, respectively. Seventeen documented discrete infections occurred in six patients over 36 patient years. Conclusions. CM-CSF, in addition to IS, may shorten time to neutrophil count recovery, may be beneficial in decreasing infection rates, and may improve platelet response in patients with SAA. However, consistent with studies utilizing C-CSF, CM-CSF probably does not affect overall response rate. To fully answer whether or not cytokine therapy is of added value to IS in pediatric patients, a multi-institutional randomized trial is needed.",
keywords = "Aplastic anemia, Cytokines, GM-CSF, Immune-suppression",
author = "Jeng, {Michael R.} and Naidu, {Paula E.} and Rieman, {Martha D.} and Carlos Rodriguez-Galindo and Nottage, {Kerri A.} and Thornton, {Donyell T.} and Chin-Shang Li and Wiang, {Winfred C.}",
year = "2005",
month = "8",
doi = "10.1002/pbc.20278",
language = "English (US)",
volume = "45",
pages = "170--175",
journal = "Pediatric Blood and Cancer",
issn = "1545-5009",
publisher = "Wiley-Liss Inc.",
number = "2",

}

TY - JOUR

T1 - Granulocyte-macrophage colony stimulating factor and immunosuppression in the treatment of pediatric acquired severe aplastic anemia

AU - Jeng, Michael R.

AU - Naidu, Paula E.

AU - Rieman, Martha D.

AU - Rodriguez-Galindo, Carlos

AU - Nottage, Kerri A.

AU - Thornton, Donyell T.

AU - Li, Chin-Shang

AU - Wiang, Winfred C.

PY - 2005/8

Y1 - 2005/8

N2 - Background. Immunosuppressive therapy (IS) is effective in the treatment of patients with acquired severe aplastic anemia (SAA). An enhanced myeloid response and decreased infection risk may be possible with the addition of a hematopoietic cytokine. Published data on the combination of cytokines and IS in patients with SAA are limited. The addition of C-CSF to IS shortens the time to neutrophil count recovery, but may not improve overall survival. Because CM-CSF acts differently than C-CSF, its use in combination with IS may be different. Procedure. A retrospective chart review was performed on patients diagnosed with SAA and treated with IS and CM-CSF at St. Jude Children's Research Hospital. Hematologic recovery, prognostic factors, and infection data were collected. Results. Eighteen patients were included in this study. The median age at diagnosis was 7.2 years (range 1.8-17.0). Ten patients (56%) had a complete response, four (22%) a partial response, and four (22%) no response. Median time to erythrocyte and platelet transfusion independence were 90 (18,243) and 64 days (18-243), and to discontinuation of treatment 287 days (90-730). Median time to partial (ANC > 500) and full (ANC > 1,500) neutrophil recovery were 41 and 51 days, respectively. Seventeen documented discrete infections occurred in six patients over 36 patient years. Conclusions. CM-CSF, in addition to IS, may shorten time to neutrophil count recovery, may be beneficial in decreasing infection rates, and may improve platelet response in patients with SAA. However, consistent with studies utilizing C-CSF, CM-CSF probably does not affect overall response rate. To fully answer whether or not cytokine therapy is of added value to IS in pediatric patients, a multi-institutional randomized trial is needed.

AB - Background. Immunosuppressive therapy (IS) is effective in the treatment of patients with acquired severe aplastic anemia (SAA). An enhanced myeloid response and decreased infection risk may be possible with the addition of a hematopoietic cytokine. Published data on the combination of cytokines and IS in patients with SAA are limited. The addition of C-CSF to IS shortens the time to neutrophil count recovery, but may not improve overall survival. Because CM-CSF acts differently than C-CSF, its use in combination with IS may be different. Procedure. A retrospective chart review was performed on patients diagnosed with SAA and treated with IS and CM-CSF at St. Jude Children's Research Hospital. Hematologic recovery, prognostic factors, and infection data were collected. Results. Eighteen patients were included in this study. The median age at diagnosis was 7.2 years (range 1.8-17.0). Ten patients (56%) had a complete response, four (22%) a partial response, and four (22%) no response. Median time to erythrocyte and platelet transfusion independence were 90 (18,243) and 64 days (18-243), and to discontinuation of treatment 287 days (90-730). Median time to partial (ANC > 500) and full (ANC > 1,500) neutrophil recovery were 41 and 51 days, respectively. Seventeen documented discrete infections occurred in six patients over 36 patient years. Conclusions. CM-CSF, in addition to IS, may shorten time to neutrophil count recovery, may be beneficial in decreasing infection rates, and may improve platelet response in patients with SAA. However, consistent with studies utilizing C-CSF, CM-CSF probably does not affect overall response rate. To fully answer whether or not cytokine therapy is of added value to IS in pediatric patients, a multi-institutional randomized trial is needed.

KW - Aplastic anemia

KW - Cytokines

KW - GM-CSF

KW - Immune-suppression

UR - http://www.scopus.com/inward/record.url?scp=22244476435&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=22244476435&partnerID=8YFLogxK

U2 - 10.1002/pbc.20278

DO - 10.1002/pbc.20278

M3 - Article

C2 - 15593082

AN - SCOPUS:22244476435

VL - 45

SP - 170

EP - 175

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

SN - 1545-5009

IS - 2

ER -