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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).