Table 3.

Clinical and genetic features by maximum level of therapy received

Maximum level of therapy	Total	Overall survivors, N	1-year survival estimate,* %	3-year survival estimate,* %	Overall survival estimate,* %	Associated trigger, %	Genetic profile, %
Observation/none	9	9	100	100	100	None: 22.2	fHLH: 0.0
						Autoimmune: 11.1	PIDD: 0.0
						Infection: 55.6	DIAP: 0.0
						Malignancy: 11.1	PIDD/DIAP: 0.0
							Other candidate defects: 11.1
							No genetic explanation: 88.9
Biologics/steroids	18	14	70	70.0	70	None: 11.1	fHLH: 0.0
						Autoimmune: 55.6	PIDD: 0.0
						Infection: 33.3	DIAP: 16.7
						Malignancy: 0.0†	PIDD/DIAP: 5.6
							Other candidate defects: 0.0
							No genetic explanation: 77.8
Immunochemotherapy	54	32	58	58	39	None: 18.5	fHLH: 9.3
						Autoimmune: 31.5	PIDD: 16.7
						Infection: 29.6	DIAP: 7.4
						Malignancy: 20.4	PIDD/DIAP: 1.9
							Other candidate defects: 3.7
							No genetic explanation: 61.1
HSCT	38	21	75	57	40	None: 47.4†	fHLH: 36.8†
						Autoimmune: 10.5†	PIDD: 0.0†
						Infection: 39.5	DIAP: 2.6
						Malignancy: 2.6†	PIDD/DIAP: 2.6
							Other candidate defects: 2.6
							No genetic explanation: 55.3

^*By Kaplan-Meier analysis.

^†Significantly different compared with HLH standard of care Immunochemotherapy group (P < .05).