Table 1 |. Univariable and multivariable analysis for overall survival in the biological validation cohort.

An univariable analysis including age at diagnosis, platelet count at diagnosis, XGBoost Cluster, MethylSeq Cluster, somatic PTPN11 mutations, sex, number of somatic mutations and HbF status was conducted for predictors of overall survival (OS). P-values lower than 0.05 were considered as statistically significant. A multivariable analysis including the significant features was calculated thereafter including either the XGBoost or MethylSeq methylation cluster assignments. (N = Number of pts; HR = Hazard ratio; CI = Confidence interval).

Univariable Analysis	OS from date of diagnosis
	N	HR	95% CI	p
Age at diagnosis (months)
≤12 months	27	1
>12 months	20	2.75	1.08–7.01	0.03
Platelet count at diagnosis ×109
≤50	17	1
>50	30	0.35	0.14–0.89	0.026
XGBoost Cluster
Low	16	1
Intermediate	10	2.96	0.48–18.22	0.0026
High	21	8.07	1.81–36.01
MethylSeq Cluster
Low	19	1
Intermediate	8	6.76	1.20–38.01	0.0015
High	20	8.93	1.99–40.0
Somatic PTPN11 mutation
No	26	1
Yes	21	1.79	0.72–44.45	0.21
Sex
Male	34	1
Female	13	0.84	0.28–2.57	0.76
Somatic Mutations at Diagnosis
<=1	29	1
>1	18	2.33	0.93–5.79	0.068
HbF at diagnosis
Not elevated for age	10	1
Elevated for age	35	1.36	0.39–4.73	0.61
Multivariable Analysis (XGBoost)
Age at diagnosis (months)
≤12 months	27	1
>12 months	20	0.61	0.15–2.47	0.51
Platelet count at diagnosis ×109
≤50	17	1
>50	30	0.49	0.19–1.25	0.13
XGBoost Cluster
Low	16	1
Intermediate	10	3.09	0.49–19.65	0.046
High	21	10.82	1.56–74.84
Multivariable Analysis (MethylSeq)
Age at diagnosis (months)
≤12 months	27	1
>12 months	20	0.85	0.20–3.53	0.82
Platelet count at diagnosis ×109
≤50	17	1
>50	30	0.52	0.20–1.31	0.16
MethylSeq Cluster
Low	19	1
Intermediate	8	6.17	1.06–35.94	0.039
High	20	8.92	1.25–63.48