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 language | English (US) |
|---|---|
| Pages (from-to) | 170-175 |
| Number of pages | 6 |
| Journal | Pediatric Blood and Cancer |
| Volume | 45 |
| Issue number | 2 |
| DOIs | |
| State | Published - Aug 2005 |
| Externally published | Yes |
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Keywords
- Aplastic anemia
- Cytokines
- GM-CSF
- Immune-suppression
ASJC Scopus subject areas
- Cancer Research
- Pediatrics, Perinatology, and Child Health
- Hematology
Cite this
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 journal › Article
}
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 -