Leukemia, Myeloid, Acute
Conditions
Keywords
AML, Mylotarg, gemtuzumab
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.
Interventions
DRUGGemtuzumab Ozogamicin
Gemtuzumab Ozogamicin (Mylotarg) administered in any form or combination
Sponsors
Pfizer
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
United States
Participant flow
Pre-assignment details
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).
Participants by arm
| Arm | Count |
|---|---|
| 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. | 32 |
| Total | 32 |
Baseline characteristics
| Characteristic | Mylotarg |
|---|---|
| Age, Continuous | 58.9 Years STANDARD_DEVIATION 14.1 |
| AML Stage at Index Date De Novo | 22 Participants |
| AML Stage at Index Date Secondary | 8 Participants |
| AML Stage at Index Date Undocumented | 2 Participants |
| Body Mass Index (BMI) | 29.7 Kilogram/meters^2 STANDARD_DEVIATION 5.88 |
| Body Weight | 185.1 Pounds STANDARD_DEVIATION 52.71 |
| Height of Participants | 67.5 Inches STANDARD_DEVIATION 3.73 |
| Insurance Status Private Insurance, No Public Insurance | 4 Participants |
| Insurance Status Public and Private Insurance | 5 Participants |
| Insurance Status Public Insurance, No Private Insurance | 1 Participants |
| Insurance Status Unknown/Undocumented | 22 Participants |
| Mean Charlson Comorbidity Index of Participants | 1.1 Units on a scale STANDARD_DEVIATION 0.27 |
| Number of Participants With Comorbidities Congestive Heart Failure | 1 Participants |
| Number of Participants With Comorbidities Connective Tissue Disease | 2 Participants |
| Number of Participants With Comorbidities Diabetes | 11 Participants |
| Number of Participants With Comorbidities Myocardial Infarction | 1 Participants |
| Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) 0 | 4 Participants |
| Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) 1 | 5 Participants |
| Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) 2 | 3 Participants |
| Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) 3 | 1 Participants |
| Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) 4 | 0 Participants |
| Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) 5 | 0 Participants |
| Number of Participants With Eastern Cooperative Oncology Group Performance Status (ECOG PS) Undocumented ECOG | 19 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis -5 or del(5q); -7; -17/abn(17p) Testing No | 31 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis -5 or del(5q); -7; -17/abn(17p) Testing Yes | 1 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis Complex karyotype Testing No | 31 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis Complex karyotype Testing Yes | 1 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 No | 29 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 | 3 Participants |
| 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 | 30 Participants |
| 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 | 2 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis Monosomal karyotype Testing No | 31 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis Monosomal karyotype Testing Yes | 1 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(6;9)(p23:q34.1) Testing No | 32 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(6;9)(p23:q34.1) Testing Yes | 0 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(8;21)(q22;q22.1) Testing No | 29 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(8;21)(q22;q22.1) Testing Yes | 3 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(9;22)(q34.1;q11.2) Testing No | 32 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(9;22)(q34.1;q11.2) Testing Yes | 0 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(v;11q23.3) Testing No | 32 Participants |
| Number of Participants With Results for Cytogenetic/FISH Testing at Initial AML Diagnosis t(v;11q23.3) Testing Yes | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race (NIH/OMB) White | 26 Participants |
| Region of Residence Midwest | 5 Participants |
| Region of Residence Northeast | 1 Participants |
| Region of Residence South | 4 Participants |
| Region of Residence Undocumented Region | 20 Participants |
| Region of Residence West | 2 Participants |
| Sex: Female, Male Female | 11 Participants |
| Sex: Female, Male Male | 21 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 0 |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
Number of Participants With First Positive Response
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)
Population: Analysis population included all participants included in this study. Here, 'number analyzed' signifies participants evaluable for each specified category.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Mylotarg | Number of Participants With First Positive Response | First line (1L) Therapy | CR | 9 Participants |
| Mylotarg | 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 | 7 Participants |
| Mylotarg | Number of Participants With First Positive Response | First line (1L) Therapy | PR | 0 Participants |
| Mylotarg | Number of Participants With First Positive Response | Second line (2L) Therapy | CR | 2 Participants |
| Mylotarg | 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 |
| Mylotarg | Number of Participants With First Positive Response | Second line (2L) Therapy | PR | 1 Participants |
| Mylotarg | Number of Participants With First Positive Response | Third line (3L) Therapy | CR | 0 Participants |
| Mylotarg | 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 |
| Mylotarg | Number of Participants With First Positive Response | Third line (3L) Therapy | PR | 3 Participants |
Primary
Real-World Event-Free Survival (rwEFS)
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)
Population: Analysis population included all participants included in this study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Mylotarg | Real-World Event-Free Survival (rwEFS) | 1.66 months |
Primary
Real-World Overall Survival (rwOS)
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)
Population: Analysis population included all participants included in this study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Mylotarg | Real-World Overall Survival (rwOS) | 6.15 months |
Primary
Real-World Relapse Free Survival (rwRFS)
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)
Population: Analysis population included all participants included in this study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Mylotarg | Real-World Relapse Free Survival (rwRFS) | 4.87 months |
Other Pre-specified
Duration of Therapy
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)
Population: Analysis population included all participants included in this study. Here, 'number analyzed' signifies participants evaluable for each specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Mylotarg | Duration of Therapy | Mylotarg in 1L therapy | 1.6 months | Standard Deviation 2.13 |
| Mylotarg | Duration of Therapy | Mylotarg in 2L therapy | 1.2 months | Standard Deviation 1.36 |
| Mylotarg | Duration of Therapy | Mylotarg in 3L therapy | 1.0 months | Standard Deviation 1.99 |
Post Hoc
rwEFS in De-Novo AML Participants Using a Closer-to-Typical Combination of Mylotarg + Chemotherapy as 1L Therapy
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)
Population: Analysis population included all participants included in this study. Here, 'overall number of participants analyzed' signifies participants evaluable for this outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Mylotarg | rwEFS in De-Novo AML Participants Using a Closer-to-Typical Combination of Mylotarg + Chemotherapy as 1L Therapy | 6.15 months |