Trial Outcomes & Findings for A Study Of Treatment Patterns And Clinical Outcomes In Patients Diagnosed With Acute Myeloid Leukemia Who Received Mylotarg in the Real-World (NCT NCT04337138)
NCT ID: NCT04337138
Last Updated: 2021-08-09
Results Overview
rwEFS defined as time from treatment initiation date (for first line of Mylotarg-containing therapy) to date of treatment failure (TF), relapse from complete response (CR) or better, death from any cause, whichever came first. TF defined as failure to achieve CR or better following up to 3 cycles of Mylotarg. Time origin for rwEFS was start of Mylotarg in first line of therapy in which it was used. Terminal event for analysis of rwEFS was earlier of treatment failure, relapse from CR or better, death. CR is defined as having less than (\<) 5% blasts in the bone marrow and 0% blasts in the peripheral blood. Real-world setting signifies participants treated in clinical practice and in a non-trial setting.
COMPLETED
32 participants
From treatment initiation date to date of TF, relapse from CR or better, death from any cause, whichever came first (maximum duration of 3 years)
2021-08-09
Participant Flow
In this study, data for participants was collected retrospectively through the United States oncology electronic medical record data available to Concerto HealthAI, including data from CancerLinQ-affiliated practices (referred as definitive oncology dataset).
Participant milestones
| Measure |
Mylotarg
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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Overall Study
STARTED
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32
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Overall Study
COMPLETED
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32
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Here 'number analyzed" signifies participants evaluable for this baseline measure.
Baseline characteristics by cohort
| Measure |
Mylotarg
n=32 Participants
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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Age, Continuous
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58.9 Years
STANDARD_DEVIATION 14.10 • n=32 Participants
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Sex: Female, Male
Female
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11 Participants
n=32 Participants
|
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Sex: Female, Male
Male
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21 Participants
n=32 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=32 Participants
|
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Race (NIH/OMB)
Asian
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0 Participants
n=32 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=32 Participants
|
|
Race (NIH/OMB)
Black or African American
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2 Participants
n=32 Participants
|
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Race (NIH/OMB)
White
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26 Participants
n=32 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=32 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
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4 Participants
n=32 Participants
|
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Body Mass Index (BMI)
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29.7 Kilogram/meters^2
STANDARD_DEVIATION 5.88 • n=32 Participants
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Insurance Status
Private Insurance, No Public Insurance
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4 Participants
n=32 Participants
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Insurance Status
Public Insurance, No Private Insurance
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1 Participants
n=32 Participants
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Insurance Status
Public and Private Insurance
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5 Participants
n=32 Participants
|
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Insurance Status
Unknown/Undocumented
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22 Participants
n=32 Participants
|
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Height of Participants
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67.5 Inches
STANDARD_DEVIATION 3.73 • n=30 Participants • Here 'number analyzed" signifies participants evaluable for this baseline measure.
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Body Weight
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185.1 Pounds
STANDARD_DEVIATION 52.71 • n=32 Participants
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Region of Residence
Midwest
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5 Participants
n=32 Participants
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Region of Residence
Northeast
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1 Participants
n=32 Participants
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Region of Residence
South
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4 Participants
n=32 Participants
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Region of Residence
West
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2 Participants
n=32 Participants
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Region of Residence
Undocumented Region
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20 Participants
n=32 Participants
|
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Number of Participants With Comorbidities
Congestive Heart Failure
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1 Participants
n=15 Participants • Here "number analyzed" signifies participants evaluable for this baseline measure.
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Number of Participants With Comorbidities
Connective Tissue Disease
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2 Participants
n=15 Participants • Here "number analyzed" signifies participants evaluable for this baseline measure.
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Number of Participants With Comorbidities
Diabetes
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11 Participants
n=15 Participants • Here "number analyzed" signifies participants evaluable for this baseline measure.
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Number of Participants With Comorbidities
Myocardial Infarction
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1 Participants
n=15 Participants • Here "number analyzed" signifies participants evaluable for this baseline measure.
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Mean Charlson Comorbidity Index of Participants
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1.1 Units on a scale
STANDARD_DEVIATION 0.27 • n=14 Participants • Here "number analyzed" signifies participants evaluable for this baseline measure.
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Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS)
0
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4 Participants
n=32 Participants
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Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS)
1
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5 Participants
n=32 Participants
|
|
Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS)
2
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3 Participants
n=32 Participants
|
|
Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS)
3
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1 Participants
n=32 Participants
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|
Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS)
4
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0 Participants
n=32 Participants
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|
Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS)
5
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0 Participants
n=32 Participants
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|
Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS)
Undocumented ECOG
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19 Participants
n=32 Participants
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AML Stage at Index Date
De Novo
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22 Participants
n=32 Participants
|
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AML Stage at Index Date
Secondary
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8 Participants
n=32 Participants
|
|
AML Stage at Index Date
Undocumented
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2 Participants
n=32 Participants
|
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(6;9)(p23:q34.1) Testing · Yes
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0 Participants
n=32 Participants
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(6;9)(p23:q34.1) Testing · No
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32 Participants
n=32 Participants
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(v;11q23.3) Testing · Yes
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0 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(v;11q23.3) Testing · No
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32 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(9;22)(q34.1;q11.2) Testing · Yes
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0 Participants
n=32 Participants
|
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(9;22)(q34.1;q11.2) Testing · No
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32 Participants
n=32 Participants
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
inv(3)(q21.3q26.2)³ or t(3;3)(q21.3;q26.2) Testing · Yes
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2 Participants
n=32 Participants
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
inv(3)(q21.3q26.2)³ or t(3;3)(q21.3;q26.2) Testing · No
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30 Participants
n=32 Participants
|
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
-5 or del(5q); -7; -17/abn(17p) Testing · Yes
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1 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
-5 or del(5q); -7; -17/abn(17p) Testing · No
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31 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
Complex karyotype Testing · Yes
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1 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
Complex karyotype Testing · No
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31 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
Monosomal karyotype Testing · Yes
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1 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
Monosomal karyotype Testing · No
|
31 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(8;21)(q22;q22.1) Testing · Yes
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3 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
t(8;21)(q22;q22.1) Testing · No
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29 Participants
n=32 Participants
|
|
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
inv(16)(p13.1q22)¹ or t(16;16)(p13.1;q22) Testing · Yes
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3 Participants
n=32 Participants
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Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis
inv(16)(p13.1q22)¹ or t(16;16)(p13.1;q22) Testing · No
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29 Participants
n=32 Participants
|
PRIMARY outcome
Timeframe: From treatment initiation date to date of TF, relapse from CR or better, death from any cause, whichever came first (maximum duration of 3 years)Population: Analysis population included all participants included in this study.
rwEFS defined as time from treatment initiation date (for first line of Mylotarg-containing therapy) to date of treatment failure (TF), relapse from complete response (CR) or better, death from any cause, whichever came first. TF defined as failure to achieve CR or better following up to 3 cycles of Mylotarg. Time origin for rwEFS was start of Mylotarg in first line of therapy in which it was used. Terminal event for analysis of rwEFS was earlier of treatment failure, relapse from CR or better, death. CR is defined as having less than (\<) 5% blasts in the bone marrow and 0% blasts in the peripheral blood. Real-world setting signifies participants treated in clinical practice and in a non-trial setting.
Outcome measures
| Measure |
Mylotarg
n=32 Participants
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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Real-World Event-Free Survival (rwEFS)
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1.66 months
Interval 0.9 to 4.8
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PRIMARY outcome
Timeframe: From the treatment initiation date (during the first line of Mylotarg-containing therapy) to the date of a relapse event, or death from any cause, whichever came first (maximum duration of 3 years)Population: Analysis population included all participants included in this study.
rwRFS was defined as the time from the treatment initiation date (during the first line of Mylotarg-containing therapy) to the date of a relapse event, or death from any cause, whichever came first. The time origin for rwRFS was the start of Mylotarg in the first line of therapy in which it was used. Real-world setting signifies participants treated in clinical practice and in a non-trial setting.
Outcome measures
| Measure |
Mylotarg
n=32 Participants
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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Real-World Relapse Free Survival (rwRFS)
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4.87 months
Interval 2.6 to 8.2
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PRIMARY outcome
Timeframe: From the start of the first Mylotarg use till death (maximum duration of 3 years)Population: Analysis population included all participants included in this study.
rwOS was defined as the time from the start of the first Mylotarg use till the date of death. Participants who were not indicated to be deceased in clinical records or Social Security Disability Insurance (SSDI) records were censored for rwOS analysis as of the later of 1) the latest date known alive within the clinical record, 2) 4 months prior to the date of most recent SSDI update. Real-world setting signifies participants treated in clinical practice and in a non-trial setting.
Outcome measures
| Measure |
Mylotarg
n=32 Participants
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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Real-World Overall Survival (rwOS)
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6.15 months
Interval 3.1 to
The upper limit of 95% confidence interval was not estimable due to small sample size as a result of censoring.
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PRIMARY outcome
Timeframe: From the first qualifying Mylotarg-containing line of therapy to the end of the third line of therapy or the end of record, whichever occurs first (maximum duration of 3 years)Population: Analysis population included all participants included in this study. Here, 'number analyzed' signifies participants evaluable for each specified category.
First positive response was assessed by physician as first response from any of the following: CR, partial response (PR), stable disease (SD) and progressive disease (PD). CR is defined as having \< 5% blasts in the bone marrow and 0% blasts in the peripheral blood. PR is defined as having 5 to 25% bone marrow blasts with \> 50% reduction in blasts and peripheral blood count recovery. SD is defined as having no change in bone marrow blasts. PD is defined as having relapse following response. Participant for whom record did not indicate one of these classification classed as not evaluable (NE).
Outcome measures
| Measure |
Mylotarg
n=32 Participants
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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Number of Participants With First Positive Response
First line (1L) Therapy · CR
|
9 Participants
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Number of Participants With First Positive Response
First line (1L) Therapy · NA-Participant had SD, PD, NE, no documentation of response assessment or other negative response
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7 Participants
|
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Number of Participants With First Positive Response
First line (1L) Therapy · PR
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0 Participants
|
|
Number of Participants With First Positive Response
Second line (2L) Therapy · CR
|
2 Participants
|
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Number of Participants With First Positive Response
Second line (2L) Therapy · NA-Participant had SD, PD, NE, no documentation of response assessment or other negative response
|
5 Participants
|
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Number of Participants With First Positive Response
Second line (2L) Therapy · PR
|
1 Participants
|
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Number of Participants With First Positive Response
Third line (3L) Therapy · CR
|
0 Participants
|
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Number of Participants With First Positive Response
Third line (3L) Therapy · NA-Participant had SD, PD, NE, no documentation of response assessment or other negative response
|
5 Participants
|
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Number of Participants With First Positive Response
Third line (3L) Therapy · PR
|
3 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: From the initiation of Mylotarg to the last date of Mylotarg therapy (maximum duration of 3 years)Population: Analysis population included all participants included in this study. Here, 'number analyzed' signifies participants evaluable for each specified category.
Duration of therapy was time from the initiation of Mylotarg to the last date of Mylotarg therapy, regardless of line of therapy (duration could continue into subsequent lines of therapy), study end date, or death, whichever came first.
Outcome measures
| Measure |
Mylotarg
n=32 Participants
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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Duration of Therapy
Mylotarg in 1L therapy
|
1.6 months
Standard Deviation 2.13
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Duration of Therapy
Mylotarg in 2L therapy
|
1.2 months
Standard Deviation 1.36
|
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Duration of Therapy
Mylotarg in 3L therapy
|
1.0 months
Standard Deviation 1.99
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POST_HOC outcome
Timeframe: From treatment initiation date to date of TF, relapse from CR or better, death from any cause, whichever came first (maximum duration of 3 years)Population: Analysis population included all participants included in this study. Here, 'overall number of participants analyzed' signifies participants evaluable for this outcome measure.
rwEFS defined as time from treatment initiation date (for first line of Mylotarg-containing therapy) to date of TF, relapse from CR or better, death from any cause, whichever came first. TF defined as failure to achieve CR or better following up to 3 cycles of Mylotarg. Time origin for rwEFS was start of Mylotarg in first line of therapy in which it was used. Terminal event for analysis of rwEFS was earlier of treatment failure, relapse from CR or better, death. CR is defined as having \< 5% blasts in the bone marrow and 0% blasts in the peripheral blood. Real-world setting signifies participants treated in clinical practice and in a non-trial setting.
Outcome measures
| Measure |
Mylotarg
n=7 Participants
Participants with confirmed diagnosis of Acute Myeloid Leukemia (AML) on or after 01 December 2014, who received Mylotarg (Gemtuzumab ozogamicin \[mean number of doses of gemtuzumab administered = 1 per participant\]) from 2017 to 2020 in real-world clinical setting (i.e. treated in clinical practice and in a non-trial setting) were included in this retrospective study and their data from definitive oncology dataset was retrospectively assessed in this study of up to 4 months.
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|---|---|
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rwEFS in De-Novo AML Participants Using a Closer-to-Typical Combination of Mylotarg + Chemotherapy as 1L Therapy
|
6.15 months
Interval 0.5 to
95% confidence interval upper limit was not estimable due to very less number of participants.
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Adverse Events
Mylotarg
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER