Table 5.
Multivariate analysis of the survival parameters in JMML
| Variable | N | EFS (%) | HR (95% CI) | P |
|---|---|---|---|---|
| PTPN11 status | ||||
| Mutation absent | 55 | 57.4 | 1 | |
| Mutation present | 38 | 27.2 | 2.57 (1.41–4.69) | 0.002 |
| Somatic alterations at diagnosis | ||||
| 0 or 1 | 61 | 56.4 | 1 | |
| 2 or more | 32 | 25.0 | 2.05 (1.15–3.67) | 0.015 |
| HSCT | ||||
| Yes | 45 | 54.1 | 1 | |
| No | 48 | 33.6 | 2.66 (1.46–4.84) | 0.001 |
| Mutation subtype | ||||
| Only one non-PTPN11 mutation | 41 | 60.9 | 1 | |
| Only one PTPN11 mutation | 20 | 47.8 | 1.63 (0.71–3.72) | 0.246 |
| Combined mutations without PTPN11 | 14 | 50.0 | 1.32 (0.53–3.31) | 0.556 |
| PTPN11 mutation with additional alterations | 18 | 5.6 | 3.88 (1.87–8.05) | < 0.0001 |
When a Cox multivariate regression model was applied, PTPN11 mutation and the number of somatic alterations remained independently prognostic of poor outcome after adjusting for the improvement of HSCT treatment. Then, the cohort was subdivided. PTPN11 mutation with additional alteration showed the poorest outcome in comparison with those with only one non-PTPN11 mutation, only one PTPN11 mutation, and combined mutations without PTPN11. HSCT could improve the outcome significantly. P values < 0.05% are shown in italics