Acute Promyelocytic Leukemia With PML-RARA, t(15;17)
Conditions
Brief summary
This clinical trial studies how well simplified patient care strategy works in decreasing early death in patients with acute promyelocytic leukemia. Implementing simplified acute promyelocytic leukemia guidelines along with support from acute promyelocytic leukemia experts may decrease deaths and improve survival.
Detailed description
PRIMARY OBJECTIVES: I. To evaluate if the proposed patient care strategy, that includes use of simplified guidelines along with acute promyelocytic leukemia (APL) expert support, decreases the one-month induction mortality rate from 30% to under 15%. SECONDARY OBJECTIVES: I. To assess the overall survival 1 year after accrual is completed. II. To assess incidence and severity of differentiation syndrome. III. To correlate outcomes with time to initiation of all-trans retinoic acid (ATRA) from diagnosis or suspicion of diagnosis. IV. To compare outcomes between academic and community centers separately. V. To evaluate factors associated with outcome. OUTLINE: Patients receive standard of care treatment for APL. Patients and doctors regularly discuss with an APL expert to identify and mange treatment. After diagnosis, patients are followed up for 1 year.
Interventions
OTHERSimplified Patient Care Strategy
Treatment per Acute Promyelocytic Leukemia (APL) Treatment Guidelines plus discussion between doctors and APL expert
OTHERLaboratory Biomarker Analysis
Correlative studies
Sponsors
ECOG-ACRIN Cancer Research Group
National Cancer Institute (NCI)
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Confirmed to have a diagnosis of APL, which is defined as: * Positive t(15:17) by fluorescence in situ hybridization (FISH) or conventional karyotype * Positive promyelocytic leukemia/retinoic acid receptor PML/RAR alpha by polymerase chain reaction (PCR) * Patients must accept treatment and supportive care guidelines * Referrals must be made as early as possible but no later than 5 calendar days after ATRA therapy is initiated; consent can be obtained up till day 7 or earlier * Co-management can be started as soon as referral is made including weekends; the physician at the outlying facility should make every effort to call the APL expert at the first suspicion of APL
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| One-month Mortality Rate | Assessed at one month | Proportion of patients died within one month from the first date of induction therapy. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Survival Rate at One Year | Assessed at one year | Overall survival is defined as the time from the first date of induction therapy to death or date last known alive. Proportion of patients alive at one year was estimated using the method of Kaplan and Meier. |
| One-month Survival Rate by Lead Academic Sites and Community Sites | Assessed at one month | Proportion of patients who are alive at one month since the first date of induction therapy by lead academic sites and community sites |
| Differentiation Syndrome Rate | Assessed up to 1 year | The proportion of patients with differentiation syndrome (measured based on the CTC version 4.0) will be evaluated. |
| Association Between One-month Mortality Rate and Time From Diagnosis to Initiation of All-trans Retinoic Acid (ATRA) | Assessed at one month | The time from diagnosis and initiation of ATRA will be assessed and dichotomized as "short' vs. "long" using the median value. One-month mortality rate will be compared in these two groups (short vs. long) using the Fisher's exact test. |
| Association Between One-month Survival and Baseline Factors | Assessed at baseline and one month | The associations between one-month survival and baseline factors will be evaluated. |
Countries
Puerto Rico, United States
Contacts
PRINCIPAL_INVESTIGATORAnand Jillella
ECOG-ACRIN Cancer Research Group
Participant flow
Recruitment details
This study was activated on August 16, 2017, and closed to accrual on July 2, 2021. A total of 202 patients were enrolled.
Participants by arm
| Arm | Count |
|---|---|
| Simplified Patient Care Strategy Plus APL Expert Support All evaluable patients are included in this analysis. | 201 |
| Total | 201 |
Baseline characteristics
| Characteristic | Simplified Patient Care Strategy Plus APL Expert Support |
|---|---|
| Age, Continuous | 53 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 36 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 161 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 3 Participants |
| Race (NIH/OMB) Asian | 11 Participants |
| Race (NIH/OMB) Black or African American | 23 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 5 Participants |
| Race (NIH/OMB) White | 158 Participants |
| Sex: Female, Male Female | 96 Participants |
| Sex: Female, Male Male | 105 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 22 / 201 |
| other Total, other adverse events | 152 / 199 |
| serious Total, serious adverse events | 115 / 199 |
Outcome results
Primary
One-month Mortality Rate
Proportion of patients died within one month from the first date of induction therapy.
Time frame: Assessed at one month
Population: All evaluable patients are included in this analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Simplified Patient Care Strategy plus APL Expert Support | One-month Mortality Rate | 0.03 Proportion of participants |
Secondary
Association Between One-month Mortality Rate and Time From Diagnosis to Initiation of All-trans Retinoic Acid (ATRA)
The time from diagnosis and initiation of ATRA will be assessed and dichotomized as short' vs. long using the median value. One-month mortality rate will be compared in these two groups (short vs. long) using the Fisher's exact test.
Time frame: Assessed at one month
Secondary
Association Between One-month Survival and Baseline Factors
The associations between one-month survival and baseline factors will be evaluated.
Time frame: Assessed at baseline and one month
Secondary
Differentiation Syndrome Rate
The proportion of patients with differentiation syndrome (measured based on the CTC version 4.0) will be evaluated.
Time frame: Assessed up to 1 year
Secondary
One-month Survival Rate by Lead Academic Sites and Community Sites
Proportion of patients who are alive at one month since the first date of induction therapy by lead academic sites and community sites
Time frame: Assessed at one month
Population: All evaluable patients are included in this analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Simplified Patient Care Strategy plus APL Expert Support | One-month Survival Rate by Lead Academic Sites and Community Sites | 0.952 Proportion of participants |
| Patients accrued by community sites | One-month Survival Rate by Lead Academic Sites and Community Sites | 0.978 Proportion of participants |
Secondary
Survival Rate at One Year
Overall survival is defined as the time from the first date of induction therapy to death or date last known alive. Proportion of patients alive at one year was estimated using the method of Kaplan and Meier.
Time frame: Assessed at one year
Population: All evaluable patients are included in this analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Simplified Patient Care Strategy plus APL Expert Support | Survival Rate at One Year | 0.945 Proportion of participants |