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. 2018 Oct 8;172(12):1161–1168. doi: 10.1001/jamapediatrics.2018.2530

Table 1. Clinical and Economic Inputs.

Characteristic Tisagenlecleucel Clofarabine
Clinical Input
Response
Percentage of patients achieving response (complete or partial) 84.48 30.017
Percentage of patients dead before assessment of response 7.48 25.017
Percentage of patients achieving no response 8.28 45.017
Stem cell transplantation
Percentage of patients who receive stem cell transplantation 10.5 14.8
Adverse events
Cytokine release syndrome (grade III or higher) 49 0
Hypogammaglobulinemia (B-cell aplasia) 43 0
Utilities
Alive and not responding to treatment 0.7518 0.7518
Alive and responding to treatment 0.9118 0.9118
Long-term survivor—alive 0.9118 0.9118
Cytokine release syndrome (grade III or higher) 014 for 8 d19 014 for 8 d19
Disutilities
Chemotherapy −0.42 for duration of treatment20 –0.42 for duration of treatment20
Stem cell transplantation −0.57 for duration of treatment20 −0.57 for duration of treatment20
Economic Inputs, 2017 US $
Treatment acquisition cost (with markup)$
Tisagenlecleucel 575 000 575 000
Clofarabine 264 per mg/mL21 264 per mg/mL21
Methotrexate 0.09 per mg/mL21 0.09 per mg/mL21
Fludarabine 3.70 per mg/mL21 3.70 per mg/mL21
Cyclophosphamide 0.74 per mg/mL21 0.74 per mg/mL21
Cytarabine 0.02 per mg/mL21 0.02 per mg/mL21
Etoposide 0.09 per mg/mL21 0.09 per mg/mL21
Tocilizumab 7.69 per mg/mL21 7.69 per mg/mL21
Intravenous immunoglobulin 0.14 per mg/mL21 0.14 per mg/mL21
Healthcare use costs, $
Cost per hospital day 404922 404922
Cost per day in intensive care unit 404922 404922
Office visit 7423 7423
Intravenous treatment administration (first hour) 14023 14023
Intravenous treatment administration (each additional hour) 2923 2923
Intravenous treatment administration (each additional sequence or drug) 6623 6623
Hematology panel 1123 1123
Liver function test 823 823