Table 1.

Definitions of ruxolitinib failure used in phase 2 and 3 clinical trials

Failure feature*DefinitionPhase 2/3 trial(s)
Ruxolitinib duration At least 14 d (less if intolerability or allergy) JAKARTA II (NCT01523171) 
At least 28 d SIMPLIFY 2 (NCT02101268) 
≥3 mo (if initial response was observed) or ≥28 d (if transfusion, hemorrhage, or cytopenias) PAC203 (NCT03165734) 
Ruxolitinib dose <20 mg twice daily SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Spleen response Increase in spleen volume of ≥25% from nadir, splenic irradiation or splenectomy COMFORT II (NCT00934544) 
<10% spleen volume reduction by magnetic resonance imaging or <30% decrease from baseline in spleen length by physical examination or regrowth to these parameters after an initial response PAC203 (NCT03165734) 
Red blood cell transfusions Required red blood cell transfusions (at least 2 U/mo for 2 mo in PAC203) PAC203 (NCT03165734) 
SIMPLIFY 2 (NCT02101268) 
Thrombocytopenia CTCAE grade ≥3 SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Anemia CTCAE grade ≥3 SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Hemorrhage or hematoma CTCAE grade ≥3 (or bleeding) SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Progression Peripheral blood blast percentage ≥20% sustained for ≥8 wk or bone marrow blast count ≥20% COMFORT II (NCT00934544) 
Failure feature*DefinitionPhase 2/3 trial(s)
Ruxolitinib duration At least 14 d (less if intolerability or allergy) JAKARTA II (NCT01523171) 
At least 28 d SIMPLIFY 2 (NCT02101268) 
≥3 mo (if initial response was observed) or ≥28 d (if transfusion, hemorrhage, or cytopenias) PAC203 (NCT03165734) 
Ruxolitinib dose <20 mg twice daily SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Spleen response Increase in spleen volume of ≥25% from nadir, splenic irradiation or splenectomy COMFORT II (NCT00934544) 
<10% spleen volume reduction by magnetic resonance imaging or <30% decrease from baseline in spleen length by physical examination or regrowth to these parameters after an initial response PAC203 (NCT03165734) 
Red blood cell transfusions Required red blood cell transfusions (at least 2 U/mo for 2 mo in PAC203) PAC203 (NCT03165734) 
SIMPLIFY 2 (NCT02101268) 
Thrombocytopenia CTCAE grade ≥3 SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Anemia CTCAE grade ≥3 SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Hemorrhage or hematoma CTCAE grade ≥3 (or bleeding) SIMPLIFY 2 (NCT02101268) 
PAC203 (NCT03165734) 
Progression Peripheral blood blast percentage ≥20% sustained for ≥8 wk or bone marrow blast count ≥20% COMFORT II (NCT00934544) 

CTCAE, Common Terminology Criteria for Adverse Events.

*

Inadequate or recurrent symptom response criteria not defined in previous clinical trials, although symptom response has been defined as ≥50% reduction in the pretreatment Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score.21 

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