Patient characteristics and comparison between G-CSF and PEGf
| Patient . | Characteristics of the patients . | G-CSF treatment . | PEGf treatment . | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Date of birth . | Age at diagnosis (mo) . | Type of mutation . | Dose (μg/kg per d) . | Length FUP (mo) . | Median ANC ×109/L . | IR . | Type of infection . | Growth percentile(weight/ height) . | Dose (μg/kg per d) . | Length FUP (mo) . | Median ANC ×109/L . | IR . | Type of infection . | Growth percentile (weight/ height) . | |
| 1/F | July 2002 | 10 | ELANE: Ex 5 | 10 | 38 | 0.82 (0-4) | 10.5 | Otitis | 10th/10th | 60/7 | 111 | 1.18 (0-34) | 6.3 | Otitis | 25th/10th |
| g.4988 T>A | Mastoiditis | ||||||||||||||
| c.704T>A | Skin abscesses | ||||||||||||||
| p.Val235Glu | |||||||||||||||
| 2/M | November 2004 | 2 | ELANE: Ex 2 | 10-25 | 25 | 0.2 (0-6.7) | 10.6 | Otitis | <5th/5th | 55/7 | 103 | 0.52 (0-7) | 5.1 | Mastoiditis | 10th/25th |
| g.1894T>C | Skin abscesses | ||||||||||||||
| c.176T>C | Periodontitis | ||||||||||||||
| p.Leu59Pro | Pneumonia | ||||||||||||||
| 3/F | February 2010 | 2 | ELANE: Ex 5 | 7.5-10 | 36 | 0.5 (0-3.4) | 11.1 | Otitis | 3th/10th | 100/12 | 18 | 0.69 (0-5.4) | 3.7 | Skin abscesses | 10th/10th |
| g.4901T>C | Skin abscesses | ||||||||||||||
| c.617T>C | Pneumonia | ||||||||||||||
| p.Leu206Ser | |||||||||||||||
| 4/M | October 2002 | 18 | HAX-1: c.409C>T | 5 | 89 | 2.2 (0.37-10.300) | 6.25 | Pneumonia | 10th/25th | 60/8 | 46 | 1.3 (0.34-8.3) | 5.1 | Periodontitis | 25th/50th |
| p.Gln137X | Periodontitis | ||||||||||||||
| 5/M | December 2014 | Birth | ELANE: Intr 4 | 5 | 0.25 | 0.16 (0.12-1.2) | – | Otitis | 10th/10th | 50/21 | 7 | 1.8 (0.23-19) | 4.7 | Otitis | 10th/25th |
| g.4716G>A (+1) | Mastoiditis | ||||||||||||||
| p.Val190_Phe199del | Skin abscesses | ||||||||||||||
| Patient . | Characteristics of the patients . | G-CSF treatment . | PEGf treatment . | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Date of birth . | Age at diagnosis (mo) . | Type of mutation . | Dose (μg/kg per d) . | Length FUP (mo) . | Median ANC ×109/L . | IR . | Type of infection . | Growth percentile(weight/ height) . | Dose (μg/kg per d) . | Length FUP (mo) . | Median ANC ×109/L . | IR . | Type of infection . | Growth percentile (weight/ height) . | |
| 1/F | July 2002 | 10 | ELANE: Ex 5 | 10 | 38 | 0.82 (0-4) | 10.5 | Otitis | 10th/10th | 60/7 | 111 | 1.18 (0-34) | 6.3 | Otitis | 25th/10th |
| g.4988 T>A | Mastoiditis | ||||||||||||||
| c.704T>A | Skin abscesses | ||||||||||||||
| p.Val235Glu | |||||||||||||||
| 2/M | November 2004 | 2 | ELANE: Ex 2 | 10-25 | 25 | 0.2 (0-6.7) | 10.6 | Otitis | <5th/5th | 55/7 | 103 | 0.52 (0-7) | 5.1 | Mastoiditis | 10th/25th |
| g.1894T>C | Skin abscesses | ||||||||||||||
| c.176T>C | Periodontitis | ||||||||||||||
| p.Leu59Pro | Pneumonia | ||||||||||||||
| 3/F | February 2010 | 2 | ELANE: Ex 5 | 7.5-10 | 36 | 0.5 (0-3.4) | 11.1 | Otitis | 3th/10th | 100/12 | 18 | 0.69 (0-5.4) | 3.7 | Skin abscesses | 10th/10th |
| g.4901T>C | Skin abscesses | ||||||||||||||
| c.617T>C | Pneumonia | ||||||||||||||
| p.Leu206Ser | |||||||||||||||
| 4/M | October 2002 | 18 | HAX-1: c.409C>T | 5 | 89 | 2.2 (0.37-10.300) | 6.25 | Pneumonia | 10th/25th | 60/8 | 46 | 1.3 (0.34-8.3) | 5.1 | Periodontitis | 25th/50th |
| p.Gln137X | Periodontitis | ||||||||||||||
| 5/M | December 2014 | Birth | ELANE: Intr 4 | 5 | 0.25 | 0.16 (0.12-1.2) | – | Otitis | 10th/10th | 50/21 | 7 | 1.8 (0.23-19) | 4.7 | Otitis | 10th/25th |
| g.4716G>A (+1) | Mastoiditis | ||||||||||||||
| p.Val190_Phe199del | Skin abscesses | ||||||||||||||
The comparison between the median ANC and IR values in the whole cohort if compared with the overall G-CSF period and the PEGf period was not significant, but the same comparison done for PEGf administered every 7-8 d vs 9-12 d was statistically significant for ANC (P = .002) and IR (P = .029).