A Study Of Treatment Patterns And Clinical Outcomes In Patients Diagnosed With Acute Myeloid Leukemia Who Received Mylotarg in the Real-World

• Sponsor: Pfizer (Industry)
• Overall Status: Completed
• CT.gov ID: NCT04337138
• Collaborator: (none)
• Enrollment: 32
• Location: 1
• Actual Duration (Months): 3.8
• Patients Per Site Per Month: 8.5

Study Details

Study Description

Brief Summary

The aim of this observational study is to describe treatment patterns and effectiveness outcomes in a sample of oncology patients treated for AML with Mylotarg through up to two additional relapsed/refractory (R/R)-based lines of therapy (through third-line therapy). The study will use United States oncology electronic medical record (EMR) data. All study data are secondary data and will have been collected retrospectively from existing clinical data originally collected as part of routine care.

Condition or Disease	Intervention/Treatment	Phase
Leukemia, Myeloid, Acute	Drug: Gemtuzumab Ozogamicin

Study Design

• Study Type: Observational
• Actual Enrollment: 32 participants
• Observational Model: Cohort
• Time Perspective: Retrospective
• Official Title: Characteristics, Treatment Patterns, and Clinical Outcomes in Acute Myeloid Leukemia (AML) Patients Using Mylotarg - a US Real-World Study Using Electronic Medical Record Data
• Actual Study Start Date: Apr 7, 2020
• Actual Primary Completion Date: Jul 31, 2020
• Actual Study Completion Date: Jul 31, 2020

Outcome Measures

Primary Outcome Measures

1. Real-World Event-Free Survival (rwEFS) [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)]

   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.

2. Real-World Relapse Free Survival (rwRFS) [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)]

   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.

3. Real-World Overall Survival (rwOS) [From the start of the first Mylotarg use till death (maximum duration of 3 years)]

   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.

4. Number of Participants With First Positive Response [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)]

   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).

Other Outcome Measures

1. Duration of Therapy [From the initiation of Mylotarg to the last date of Mylotarg therapy (maximum duration of 3 years)]

   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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Confirmed diagnosis of acute myeloid leukemia (AML) on or after 01 December 2014 through Clinical Research Nurse (CRN) review of provider documentation of AML diagnosis in the medical record;

• Receipt of Mylotarg at any point during first three lines of therapy following initial AML diagnosis;

• Age greater than or equal to 18 years at initial diagnosis of AML.

Exclusion Criteria:

• Record of 1 or more of the following confounding diagnoses at any point before or after AML diagnosis: Acute lymphoblastic leukemia; acute promyelocytic leukemia, aggressive systemic mastocytosis; hypereosinophilic syndrome and/or chronic eosinophilic leukemia; dermatofibrosarcoma protuberans; gastrointestinal stromal tumors.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Pfizer Investigational Site	New York	New York	United States	10017

Sponsors and Collaborators

• Pfizer

Investigators

• Study Director: Pfizer CT.gov Call Center, Pfizer

Study Documents (Full-Text)

• Study Protocol - Feb 24, 2020
• Statistical Analysis Plan - Sep 9, 2020

More Information

Additional Information:

• To obtain contact information for a study center near you, click here.

Publications

None provided.

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT04337138
• Other Study ID Numbers: B1761033
• First Posted: Apr 7, 2020
• Last Update Posted: Aug 9, 2021
• Last Verified: Jul 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Pfizer: AML; Mylotarg; gemtuzumab
• Additional relevant MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Gemtuzumab

Study Results

Participant Flow

Pre-assignment Detail	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).

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Period Title: Overall Study
STARTED	32
COMPLETED	32
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Overall Participants	32
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	58.9 (14.10)
Sex: Female, Male (Count of Participants)
Female	11 34.4%
Male	21 65.6%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	2 6.3%
White	26 81.3%
More than one race	0 0%
Unknown or Not Reported	4 12.5%
Body Mass Index (BMI) (Kilogram/meters^2) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Kilogram/meters^2]	29.7 (5.88)
Insurance Status (Count of Participants)
Private Insurance, No Public Insurance	4 12.5%
Public Insurance, No Private Insurance	1 3.1%
Public and Private Insurance	5 15.6%
Unknown/Undocumented	22 68.8%
Height of Participants (Inches) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Inches]	67.5 (3.73)
Body Weight (Pounds) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Pounds]	185.1 (52.71)
Region of Residence (Count of Participants)
Midwest	5 15.6%
Northeast	1 3.1%
South	4 12.5%
West	2 6.3%
Undocumented Region	20 62.5%
Number of Participants With Comorbidities (Count of Participants)
Congestive Heart Failure	1 3.1%
Connective Tissue Disease	2 6.3%
Diabetes	11 34.4%
Myocardial Infarction	1 3.1%
Mean Charlson Comorbidity Index of Participants (Units on a scale) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Units on a scale]	1.1 (0.27)
Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) (Count of Participants)
0	4 12.5%
1	5 15.6%
2	3 9.4%
3	1 3.1%
4	0 0%
5	0 0%
Undocumented ECOG	19 59.4%
AML Stage at Index Date (Count of Participants)
De Novo	22 68.8%
Secondary	8 25%
Undocumented	2 6.3%
Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis (Count of Participants)
Yes	0 0%
No	32 100%
Yes	0 0%
No	32 100%
Yes	0 0%
No	32 100%
Yes	2 6.3%
No	30 93.8%
Yes	1 3.1%
No	31 96.9%
Yes	1 3.1%
No	31 96.9%
Yes	1 3.1%
No	31 96.9%
Yes	3 9.4%
No	29 90.6%
Yes	3 9.4%
No	29 90.6%

Outcome Measures

1. Primary Outcome

Title	Real-World Event-Free Survival (rwEFS)
Description	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.
Time Frame	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)

Outcome Measure Data

Analysis Population Description

Analysis population included all participants included in this study.

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Measure Participants	32
Median (95% Confidence Interval) [months]	1.66

2. Primary Outcome

Title	Real-World Relapse Free Survival (rwRFS)
Description	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.
Time Frame	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)

Outcome Measure Data

Analysis Population Description

Analysis population included all participants included in this study.

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Measure Participants	32
Median (95% Confidence Interval) [months]	4.87

3. Primary Outcome

Title	Real-World Overall Survival (rwOS)
Description	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.
Time Frame	From the start of the first Mylotarg use till death (maximum duration of 3 years)

Outcome Measure Data

Analysis Population Description

Analysis population included all participants included in this study.

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Measure Participants	32
Median (95% Confidence Interval) [months]	6.15

4. Primary Outcome

Title	Number of Participants With First Positive Response
Description	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).
Time Frame	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)

Outcome Measure Data

Analysis Population Description

Analysis population included all participants included in this study. Here, 'number analyzed' signifies participants evaluable for each specified category.

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Measure Participants	32
CR	9 28.1%
NA-Participant had SD, PD, NE, no documentation of response assessment or other negative response	7 21.9%
PR	0 0%
CR	2 6.3%
NA-Participant had SD, PD, NE, no documentation of response assessment or other negative response	5 15.6%
PR	1 3.1%
CR	0 0%
NA-Participant had SD, PD, NE, no documentation of response assessment or other negative response	5 15.6%
PR	3 9.4%

5. Other Pre-specified Outcome

Title	Duration of Therapy
Description	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.
Time Frame	From the initiation of Mylotarg to the last date of Mylotarg therapy (maximum duration of 3 years)

Outcome Measure Data

Analysis Population Description

Analysis population included all participants included in this study. Here, 'number analyzed' signifies participants evaluable for each specified category.

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Measure Participants	32
Mylotarg in 1L therapy	1.6 (2.13)
Mylotarg in 2L therapy	1.2 (1.36)
Mylotarg in 3L therapy	1.0 (1.99)

6. Post-Hoc Outcome

Title	rwEFS in De-Novo AML Participants Using a Closer-to-Typical Combination of Mylotarg + Chemotherapy as 1L Therapy
Description	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.
Time Frame	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)

Outcome Measure Data

Analysis Population Description

Analysis population included all participants included in this study. Here, 'overall number of participants analyzed' signifies participants evaluable for this outcome measure.

Arm/Group Title	Mylotarg
Arm/Group Description	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.
Measure Participants	7
Median (95% Confidence Interval) [months]	6.15

Adverse Events

Time Frame	Not applicable as adverse events not collected during the study
Adverse Event Reporting Description	This study involved data that existed as structured data by the time of study start. In these data sources, individual participant data were not retrieved or validated, and it was not possible to link a particular product and medical event for any individual. Thus, the minimum criteria for reporting an adverse event (i.e., identifiable participant, identifiable reporter, a suspect product, and event) was not met, hence, safety data was not planned to be collected and reported.
Arm/Group Title	Mylotarg
Arm/Group Description	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.
All Cause Mortality
	Mylotarg
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)
Serious Adverse Events
	Mylotarg
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)
Other (Not Including Serious) Adverse Events
	Mylotarg
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.