Table 2.
Comparisons between groups based on genetic evidence or spontaneous recovery of ANC.
| Comparison 1 | Comparison 2 | |||||
|---|---|---|---|---|---|---|
| Group 1–1 | Group 1–2 | Statistical significance | Group 2–1 | Group 2–2 | Statistical significance | |
| Congenital neutropenia with causal variants (n = 7) | Neutropenia without pathogenic genetic evidence (n = 9) | No spontaneous recovery of ANC (n = 8) | Spontaneous recovery of ANC (n = 8) | |||
| Age at initial presentation* (month) | 3.0 (0.5–28.0) | 10.0 (6.0–17.0) | P = 0.222 | 3.5 (0.6–25.0) | 10.0 (5.0–17.0) | P = 0.399 |
| Sex, n (%) | P < .01 | P < .01 | ||||
| Male | 7 (100.0) | 1 (11.1) | 7 (87.5) | 1 (12.5) | ||
| Female | 0 (0.0) | 8 (88.9) | 1 (12.5) | 7 (87.5) | ||
| Family history, n(%) | P = 1.000 | P = 1.000 | ||||
| Yes | 3 (42.9) | 4 (44.4) | 4 (50.0) | 3 (37.5) | ||
| No | 4 (57.1) | 5 (55.5) | 4 (50.0) | 5 (62.5) | ||
| Organomegaly, n (%) | P = 1.000 | P = 1.000 | ||||
| Yes | 3 (42.9) | 3 (33.3) | 3 (37.5) | 3 (37.5) | ||
| No | 4 (57.1) | 6 (66.7) | 5 (62.5) | 5 (62.5) | ||
| Extra-hematopoietic feature, n (%) | P = 0.106 | P = 0.282 | ||||
| Yes | 4 (57.1) | 1 (11.1) | 4 (50.0) | 1 (12.5) | ||
| No | 3 (42.9) | 8 (88.9) | 4 (50.0) | 7 (87.5) | ||
| Severe infection, n(%) | P < .01 | P < .01 | ||||
| Yes | 6 (85.7) | 1 (11.1) | 6 (75.0) | 1 (12.5) | ||
| No | 1 (14.3) | 8 (88.9) | 2 (25.0) | 7 (87.5) | ||
| Fever pattern, n (%)† | P = 0.592 | P = 1.000 | ||||
| Neutropenic fever only | 5 (71.4) | 3 (33.3) | 5 (62.5) | 3 (37.5) | ||
| Neutropenic fever and non-neutropenic fever | 2 (28.6) | 4 (44.4) | 3 (37.5) | 3 (37.5) | ||
| Neutropenia pattern, n (%) | P < .01 | P < .05 | ||||
| Permanent | 5 (71.4) | 0 (0.0) | 5 (62.5) | 0 (0.0) | ||
| Intermittent | 2 (28.6) | 9 (100.0) | 3 (37.5) | 8 (100.0) | ||
| G-CSF responder, n (%)‡ | P = 0.242 | P = 0.072 | ||||
| Yes | 1 (14.3) | 4 (44.4) | 1 (12.5) | 4 (50.0) | ||
| No | 5 (71.4) | 2 (22.2) | 6 (75.0) | 1 (12.5) | ||
| Initial ANC* | 102 (0–958) | 146 (32–540) | P = 0.872 | 124 (0–917) | 157 (16–682) | P = 0.915 |
| Last ANC* | 0 (0–69) | 1797 (1366–3892) | P < .01 | 26 (0–567) | 2246 (1652–3946) | P < .01 |
| M:E ratio*§ | 1.6 (0.6–3.4) | 2.7 (1.3–4.1) | P = 0.194 | 1.9 (0.8–3.2) | 2.4 (1.2–4.3) | P = 0.354 |
| MPO-positive cell (%)*¶ | 42.38 (22.06–45.78) | 42.65 (32.21–50.64) | P = 0.439 | 42.38 (26.45–46.05) | 42.7 (28.5–50.6) | P = 0.259 |
| MPO grade, n (%)*¶ | P = 0.138 | P = 0.103 | ||||
| Grade 0, 1 or 2 | 5 (71.4) | 3 (33.3) | 6 (0.75) | 2 (25.0) | ||
| Grade 3 | 1 (14.3) | 5 (55.5) | 1 (12.5) | 5 (62.5) | ||
| Myelokathexis, n (%) | P = 0.063 | P = 0.200 | ||||
| Yes | 3 (42.9) | 0 (0.0) | 3 (37.5) | 0 (0.0) | ||
| No | 4 (57.1) | 9 (100.0) | 5 (62.5) | 8 (100.0) | ||
| Maturation arrest, n (%) | P < .05 | P = 0.143 | ||||
| Early stage | 3 (42.9) | 0 (0.0) | 3 (37.5) | 1 (12.5) | ||
| Late stage | 0 (0.0) | 5 (55.5) | 0 (0.0) | 4 (50.0) | ||
*Values presented as medians (interquartile ranges).
†Two patients who did not suffer from infection or fever during the follow-up period were excluded.
‡Three (18.8%) patients in whom G-CSF was not administered were not included. Also, one patient who could not assess the response to G-CSF were excluded.
§One patient with diluted BM aspiration in which reliable differential count could not be made was excluded.
¶Two patients were not included due to inadequate BM section quality or retrospectively unavailable BM paraffin block for MPO stain.
Abbreviations: BM, bone marrow; G-CSF, granulocyte-colony stimulating factor; M:E, myeloid to erythroid; MPO, myeloperoxidase.