Trial Outcomes & Findings for "Retrospective Study to Identify Clinical Factors Related to a High Benefit of Axitinib in mRCC" (NCT NCT03538717)

Data was collected and observed retrospectively for approximately 1 year, of metastatic renal cell carcinoma (mRCC) participants who received axitinib in the daily clinical practice as second and later lines prior to inclusion in study and met inclusion/exclusion criteria.

1 participant had an erroneous birth date data, hence data was not included for that participant.

ECOG: participant's performance status was measured on a 6-point scale: 0= fully active/able to carry on all pre-disease activities without restriction; 1= restricted in physically strenuous activity but ambulatory and able to carry out work of a light and sedentary nature; 2= ambulatory and capable of all self-care, but unable to carry out any work activities, up and about more than 50 percent (%) of waking hours; 3= capable of only limited self-care, confined to bed/chair \>50% of waking hours; 4= completely disabled, cannot carry on any self-care, totally confined to bed/chair: 5= dead. In this outcome measure, data for ECOG status (0, 1, \>=2: before first line treatment initiation), was compared between long responders and refractory participants.

ECOG: participant's performance status was measured on a 6-point scale: 0= fully active/able to carry on all pre-disease activities without restriction; 1= restricted in physically strenuous activity but ambulatory and able to carry out work of a light and sedentary nature; 2= ambulatory and capable of all self-care, but unable to carry out any work activities, up and about more than 50 percent (%) of waking hours; 3= capable of only limited self-care, confined to bed/chair \>50% of waking hours; 4= completely disabled, cannot carry on any self-care, totally confined to bed/chair: 5= dead. In this outcome measure, data for ECOG status (0, 1, \>=2: at initiation of axitinib), was compared between long responders and refractory participants.

ECOG: participant's performance status was measured on a 6-point scale: 0= fully active/able to carry on all pre-disease activities without restriction; 1= restricted in physically strenuous activity but ambulatory and able to carry out work of a light and sedentary nature; 2= ambulatory and capable of all self-care, but unable to carry out any work activities, up and about more than 50 percent (%) of waking hours; 3= capable of only limited self-care, confined to bed/chair \>50% of waking hours; 4= completely disabled, cannot carry on any self-care, totally confined to bed/chair: 5= dead. In this outcome measure, data for ECOG status (0, 1, \>=2: on axitinib discontinuation), was compared between long responders and refractory participants.

Nephrectomy is a surgical removal of kidney. Data for participants was categorized as yes and no to depict their nephrectomy status before axitinib treatment initiation and comparison was done between long responders and refractory participants.

MSKCC risk system stratifies participants with mRCC into poor, intermediate and favorable risk groups based on number of adverse clinical and laboratory parameters present. Favorable-risk group: participants had no poor prognostic factors. Intermediate-risk group: participants had 1 or 2 adverse prognostic factors. Poor-risk group: participants had more than 2 poor prognostic factors. Poor prognostic factors included a Karnofsky performance status (KPS) of less than (\<) 80 (KPS score quantify participant's general well-being and activities of daily life, based on their functional impairment. KPS score ranges between 0= death to 100= no evidence of disease; higher score means higher ability to perform daily tasks), time from diagnosis to treatment for more than 12 months, serum lactate dehydrogenase (LDH) \>1.5\*upper limit of normal (ULN), corrected serum calcium level \>10.0 milligram per deciliter (mg/dL) and hemoglobin \< lower limit of normal (LLN).

MSKCC risk system stratifies participants with mRCC into poor, intermediate and favorable risk groups based on number of adverse clinical and laboratory parameters present. Favorable-risk group: participants had no poor prognostic factors. Intermediate-risk group: participants had 1 or 2 adverse prognostic factors. Poor-risk group: participants had more than 2 poor prognostic factors. Poor prognostic factors included a KPS of \<80 (KPS score quantify participant's general well-being and activities of daily life, based on their functional impairment. KPS score ranges between 0= death to 100= no evidence of disease; higher score means higher ability to perform daily tasks), time from diagnosis to treatment for more than 12 months, serum LDH \>1.5\*ULN, corrected serum calcium level \>10.0 mg/dL and hemoglobin \< LLN.

MSKCC risk system stratifies participants with mRCC into poor, intermediate and favorable risk groups based on number of adverse clinical and laboratory parameters present. Favorable-risk group: participants had no poor prognostic factors. Intermediate-risk group: participants had 1 or 2 adverse prognostic factors. Poor-risk group: participants had more than 2 poor prognostic factors. Poor prognostic factors included a KPS of \<80 (KPS score quantify participant's general well-being and activities of daily life, based on their functional impairment. KPS score ranges between 0= death to 100= no evidence of disease; higher score means higher ability to perform daily tasks), time from diagnosis to treatment for more than 12 months, serum LDH \>1.5\*ULN, corrected serum calcium level \>10.0 mg/dL and hemoglobin \< LLN.

IMDC risk group stratifies participants with mRCC into poor, intermediate and favorable risk groups based on number of adverse clinical and laboratory parameters present. Favorable-risk group: participants had no poor prognostic factors. Intermediate-risk group: participants had 1 or 2 poor prognostic factors. Poor-risk group: participants had 3 to 6 poor prognostic factors. Poor prognostic factors included KPS score of \<80 at the initiation of treatment, time from diagnosis to metastasis treatment of \<12 months, anemia, hypercalcemia (corrected calcium \>10 mg/dL), neutrophilia and thrombocythemia. In this outcome measure, IMDC risk group (favorable, intermediate, poor: at initiation of axitinib) was compared between long responders and refractory participants.

IMDC risk group stratifies participants with mRCC into poor, intermediate and favorable risk groups based on number of adverse clinical and laboratory parameters present. Favorable-risk group: participants had no poor prognostic factors. Intermediate-risk group: participants had 1 or 2 poor prognostic factors. Poor-risk group: participants had 3 to 6 poor prognostic factors. Poor prognostic factors included KPS score of \<80 at the initiation of treatment, time from diagnosis to metastasis treatment of \<12 months, anemia, hypercalcemia (corrected calcium \>10 mg/dL), neutrophilia and thrombocythemia. In this outcome measure, IMDC risk group (favorable, intermediate, poor: on discontinuation of axitinib) was compared between long responders and refractory participants.

In this outcome measure, data for participants who had renal cells with different type of histology as 100% clear cells, 100% non-clear cells, majority component of clear cells and majority component of non-clear cells was compared between long responders and refractory participants.

Duration of first line treatment with TKI was stratified into 0-3 months, 3-6 months, 6-9 months, 9-12 months and \>12 months and compared between long responders and refractory participants.

In this outcome measure, data for participants with their best response as complete response (CR) and partial response (PR), stable disease (SD) or progressive-disease (PD) to the first line treatment with TKI, was compared between long responders and refractory participants. As per response evaluation criteria in solid tumors (RECIST) 1.1 criteria: CR = disappearance of all target lesions. PR = greater than equal to (\>=) 30% decrease in sum of target lesions taking as reference baseline sum diameters. PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 millimeter (mm), or the appearance of \>=1 new lesions. SD =neither shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of diameters during treatment.

In this outcome measure, data for participants with different metastatic sites as lymph nodes, central nervous system (CNS), hepatic, pulmonary, bone and another site of metastasis at diagnosis of advance or mRCC before axitinib treatment initiation, was compared between long responders and refractory participants.

In this outcome measure, data for different laboratory parameters at initiation of first line treatment: LDH level \>1.5\*ULN, haemoglobin (Hgb) levels \<=LLN, corrected Ca levels \>10 mg/dL, neutrophil levels \>ULN, platelet levels \>ULN and neutrophil-to-lymphocyte ratio of \<=3 was compared between long responders and refractory participants.

In this outcome measure, data for smoking habit of participants was compared between long responders and refractory participants.

PFS was defined as the time from the start of axitinib treatment to disease progression or death by any cause. If there was no progression or death, the case was censored as PFS at date of latest follow-up. PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 mm, or the appearance of \>=1 new lesions.

TTP was defined as the time from the start of axitinib treatment to date of disease progression. If there was no progression, the case was censored as TTP at latest follow-up. As per RECIST 1.1, PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 mm, or the appearance of \>=1 new lesions.

OS was defined as the time from the date of start of axitinib treatment to the date of death due to any cause. If there was no death, the case was censored as OS at latest follow-up.

In this outcome measure, data for number of participants with best response to axitinib as second line of treatment and subsequent treatment was collected. Best response is CR and PR, SD or PD. As per RECIST1.1 criteria: CR = disappearance of all target lesions; PR = \>= 30% decrease in sum of target lesions taking as reference baseline sum diameters; PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 mm, or the appearance of \>=1 new lesions; SD =neither shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference smallest sum of diameters while on treatment. Subsequent treatment referred to further treatment after second line of treatment.

PFS was defined as the time from the start of axitinib as second line of treatment and subsequent treatment to disease progression or death by any cause. If there was no progression or death, the case was censored as PFS at date of latest follow-up. As per RECIST 1.1, PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 mm, or the appearance of \>=1 new lesions.

ORR was defined as the number of participants with CR or PR. As per RECIST1.1 criteria: CR = disappearance of all target lesions; PR = \>= 30% decrease in sum of target lesions taking as reference baseline sum diameters.

CBR was defined as the frequency of participants with CR, PR or SD. As per RECIST1.1 criteria: CR = disappearance of all target lesions; PR = \>= 30% decrease in sum of target lesions taking as reference baseline sum diameters; PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 mm, or the appearance of \>=1 new lesions; SD =neither shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference smallest sum of diameters while on treatment.

TTP was defined as the time from the start of second line of treatment and subsequent treatment to date of disease progression. If there was no progression, the case was censored as TTP at latest follow-up. PD: \>=20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 mm, or the appearance of \>=1 new lesions.

OS was defined as the time from the date of start of second line of treatment and subsequent treatment till the date of death due to any cause. If there was no death, the case was censored as OS at latest follow-up.

Duration of treatment with axitinib was the time from date of start of axitinib treatment to date of end of axitinib treatment or of latest follow-up if not suspended.

Percentage of participants whose axitinib dose was titrated and increased to more than 5mg dose at least 1 time in 12 hours (twice daily), are reported in this outcome measure.

Percentage of participants whose axitinib dose was reduced 5 mg dose at least 1 time in 12 hours (twice daily) are reported in this outcome measure.

An AE was any untoward medical occurrence in a participant who received investigational product without regard to possibility of causal relationship. Serious adverse events (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. AEs included both SAEs and non-SAEs.

Best response is CR and PR, SD or PD. As per RECIST1.1 criteria: CR = disappearance of all target lesions; PR = \>= 30% decrease in sum of target lesions taking as reference baseline sum diameters; PD: \>= 20% increase in sum of diameters of target lesions, taking as a reference smallest sum on treatment, the sum must also demonstrate an absolute increase of at least 5 mm, or the appearance of \>=1 new lesions; SD =neither shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference smallest sum of diameters while on treatment.

In this outcome measure, participants who stopped subsequent treatments after axitinib discontinuation due to any reasons like PD, toxicity and others are reported.

OS was defined as the time from the date of randomization to treatment up to the date of death due to any cause.