Essential Thrombocythemia (ET)
Conditions
Brief summary
The objective of this survey is to collect data to evaluate the safety and efficacy of anagrelide hydrochloride in the post-marketing phase in participants diagnosed with Essential Thrombocythemia (ET).
Detailed description
Anagrelide hydrochloride Drug Use-Result Survey
Interventions
None listed
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
\- All cases who received treatment with Agrylin.
Exclusion criteria
\-
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Had One or More Adverse Drug Reactions | From start of study drug administration up to 12 months | AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. Adverse drug reaction refers to AE related to administered drug. |
| Number of Participants Who Had One or More Adverse Events | From start of study drug administration up to 12 months | An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. |
| Number of Participants Who Had One or More Serious Adverse Event | From start of study drug administration up to 12 months | A serious AE is any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability / incapacity, is a congenital anomaly / birth defect or is medically important due to other reasons than the above mentioned criteria. |
| Percentage of Participants With Normalization in Platelet Count | From 3 months after the start of study drug administration, up to 12 months | Percentage of participants with normalization in platelet count was assessed. Normalization was defined as platelet counts to \<40 x10\^4 platelets/mcrL beyond 3 months after the start of study drug administration. |
| Number of Participants Who Had One or More Serious Adverse Drug Reactions | From start of study drug administration up to 12 months | A serious AE is any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability / incapacity, is a congenital anomaly / birth defect or is medically important due to other reasons than the above mentioned criteria. Serious adverse drug reaction refers to serious AE that are related to administered drug. |
| Percentage of Participants Who Responded in Platelet Count | From 3 months after the start of study drug administration, up to 12 months | Percentage of participants who responded in platelet count was assessed. A response was defined as platelet counts to \<60 x10\^4 platelet/mcrL beyond 3 months after the start of study drug administration. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With at Least 50% Reduction in Platelet Count | From start of study drug administration up to 12 months | Participants who achieved at least 50% reduction in platelet count from their baseline during the 1-year observation period was assessed. |
Countries
Japan
Participant flow
Recruitment details
Participant s took part in the survey at 513 investigative sites in Japan, from 25 November 2014 to 11 March 2021.
Pre-assignment details
Participants with a historical diagnosis of essential thrombocythemia were enrolled. Participants received anagrelide hydrochloride as part of a routine medical care.
Participants by arm
| Arm | Count |
|---|---|
| Anagrelide Hydrochloride Participants who received treatment with Anagrelide hydrochloride will be evaluated for this study. Participants will receive interventions as part of routine medical care. | 1,241 |
| Total | 1,241 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol Violation | 585 |
Baseline characteristics
| Characteristic | Anagrelide Hydrochloride | — |
|---|---|---|
| Age, Customized ≤ 17 | 7 Participants | — |
| Age, Customized 18 - 64 | 443 Participants | — |
| Age, Customized ≥ 65 | 790 Participants | — |
| Age, Customized Unknown | 1 Participants | — |
| History of Essential Thrombocythemia | 5.7 years STANDARD_DEVIATION 6.1 | — |
| Janus kinase (JAK)-2 Status Negative | 276 Participants | — |
| Janus kinase (JAK)-2 Status Not Done | 584 Participants | — |
| Janus kinase (JAK)-2 Status Positive | 374 Participants | — |
| Janus kinase (JAK)-2 Status Unknown | 7 Participants | — |
| Medical History/Complication Had Medical History/Complication | 796 Participants | — |
| Medical History/Complication Had No Medical History/Complication | 445 Participants | — |
| Platelet Count at Registration | 97.7 10^4 platelets/µL STANDARD_DEVIATION 45.63 | — |
| Pregnancy Test at Baseline Negative | 4 Participants | — |
| Pregnancy Test at Baseline Not Done/Unknown | 701 Participants | — |
| Pregnancy Test at Baseline Positive | 0 Participants | — |
| Prior Thrombocythemia Therapy Had No Prior Thrombocythemia Therapy (Treatment Naïve) | 242 Participants | — |
| Prior Thrombocythemia Therapy Had Prior Thrombocythemia Therapy | 999 Participants | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Region of Enrollment Japan | 1241 participants | — |
| Sex: Female, Male Female | 705 Participants | — |
| Sex: Female, Male Male | 536 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 47 / 1,241 |
| other Total, other adverse events | 477 / 1,241 |
| serious Total, serious adverse events | 234 / 1,241 |
Outcome results
Primary
Number of Participants Who Had One or More Adverse Drug Reactions
AE is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. Adverse drug reaction refers to AE related to administered drug.
Time frame: From start of study drug administration up to 12 months
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Anagrelide Hydrochloride | Number of Participants Who Had One or More Adverse Drug Reactions | 628 Participants |
Primary
Number of Participants Who Had One or More Adverse Events
An adverse event (AE) is defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.
Time frame: From start of study drug administration up to 12 months
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Anagrelide Hydrochloride | Number of Participants Who Had One or More Adverse Events | 776 Participants |
Primary
Number of Participants Who Had One or More Serious Adverse Drug Reactions
A serious AE is any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability / incapacity, is a congenital anomaly / birth defect or is medically important due to other reasons than the above mentioned criteria. Serious adverse drug reaction refers to serious AE that are related to administered drug.
Time frame: From start of study drug administration up to 12 months
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Anagrelide Hydrochloride | Number of Participants Who Had One or More Serious Adverse Drug Reactions | 100 Participants |
Primary
Number of Participants Who Had One or More Serious Adverse Event
A serious AE is any untoward medical occurrence or effect that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability / incapacity, is a congenital anomaly / birth defect or is medically important due to other reasons than the above mentioned criteria.
Time frame: From start of study drug administration up to 12 months
Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Anagrelide Hydrochloride | Number of Participants Who Had One or More Serious Adverse Event | 234 Participants |
Primary
Percentage of Participants Who Responded in Platelet Count
Percentage of participants who responded in platelet count was assessed. A response was defined as platelet counts to \<60 x10\^4 platelet/mcrL beyond 3 months after the start of study drug administration.
Time frame: From 3 months after the start of study drug administration, up to 12 months
Population: Efficacy assessment population, The efficacy assessment population was defined as participants who completed the survey and had efficacy data at baseline and post-baseline time points available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Participants Who Responded in Platelet Count | 61.02 Percentage of Participants |
Primary
Percentage of Participants With Normalization in Platelet Count
Percentage of participants with normalization in platelet count was assessed. Normalization was defined as platelet counts to \<40 x10\^4 platelets/mcrL beyond 3 months after the start of study drug administration.
Time frame: From 3 months after the start of study drug administration, up to 12 months
Population: Efficacy assessment population, The efficacy assessment population was defined as participants who completed the survey and had efficacy data at baseline and post-baseline time points available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Participants With Normalization in Platelet Count | 28.97 Percentage of Participants |
Secondary
Percentage of Participants With at Least 50% Reduction in Platelet Count
Participants who achieved at least 50% reduction in platelet count from their baseline during the 1-year observation period was assessed.
Time frame: From start of study drug administration up to 12 months
Population: Efficacy assessment population, The efficacy assessment population was defined as participants who completed the survey and had efficacy data at baseline and post-baseline time points available.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Participants With at Least 50% Reduction in Platelet Count | 38.90 Percentage of Participants |