Non-tuberculous Mycobacterial Diseases (Including MAC Disease), Tuberculosis
Conditions
Brief summary
The objective of this surveillance is to collect information about 1) adverse drug reaction not expected from the LPD (unknown adverse drug reaction), 2) the incidence of adverse drug reactions in this surveillance, and 3)factors considered to affect the safety and/or efficacy of this drug.
Detailed description
All the patients whom an investigator prescribes the first Mycobutin® should be registered consecutively until the number of subjects reaches target number in order to extract patients enrolled into the investigation at random.
Interventions
DRUGrifabutin
Mycobutin® capsules150mg depending on the Investigator prescription. Frequency and duration are according to Package Insert as follows. 1.Tuberculosis : The usual adult dosage for oral use is 150 mg to 300 mg of rifabutin once daily.For the treatment of multiple-drug resistance tuberculosis, the usual dosage for oral use is 300 to 450 mg of rifabutin once daily. 2.Treatment of non-tuberculous mycobacterial diseases (including MAC disease) : The usual adult dosage for oral use is 300 mg of rifabutin once daily.
Sponsors
Pfizer
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients need to be administered Mycobutin® in order to be enrolled in the surveillance.
Exclusion criteria
* Patients not administered Mycobutin®.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-Related Adverse Events | 1 year | A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator. |
| Number of Participants With Treatment-Related Adverse Events Unexpected From Japanese Package Insert | 1 year | A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Expectedness of the adverse event was determined according to the Japanese package insert. Relatedness to Mycobutin was assessed by the physician/investigator. |
| Number of Participants With Treatment-Related Adverse Events by Diagnosis | 1 year | A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator. Participants with treatment related adverse events were counted by diagnosis to assess whether they were risk factors for the treatment related adverse events. |
| Number of Participants With Treatment-Related Adverse Events by Gender | 1 year | A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator. Participants with treatment related adverse events were counted by gender to assess whether they were risk factors for the treatment related adverse events. |
| Number of Participants With Treatment-Related Adverse Events by Age | 1 year | A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator. Participants with treatment related adverse events were counted by age to assess whether they were risk factors for the treatment related adverse events. |
| Clinical Efficacy Rate | 1 year | Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values. |
| Clinical Efficacy Rate by Diagnosis | 1 year | Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values. Participants achieved clinical effectiveness by diagnosis were counted to assess whether they contribute to the clinical effectiveness. |
| Clinical Efficacy Rate by Gender | 1 year | Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values. Participants achieved clinical effectiveness by gender were counted to assess whether they contribute to the clinical effectiveness. |
| Clinical Efficacy Rate by Age | 1 year | Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values. Participants achieved clinical effectiveness by age were counted to assess whether they contribute to the clinical effectiveness. |
Participant flow
Pre-assignment details
A total 628 subjects were registered in this study. Of the 628 subjects, 594 subjects CRFs were collected and included in the study. Of the 594 subjects, 6 subjects were excluded from the safety analysis set (SAS). In total, 588 subjects were included in the SAS as the completed the study
Participants by arm
| Arm | Count |
|---|---|
| Mycobutin (Rifabutin) Participants who received Mycobutin as indicated in the approved local product document were observed for a period of 1 year. The dosage can be adjusted as per physician's discretion. | 588 |
| Total | 588 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | No Visit After First Day of Treatment | 3 |
| Overall Study | Protocol Violation | 3 |
Baseline characteristics
| Characteristic | Mycobutin (Rifabutin) |
|---|---|
| Age, Customized ≥15 and <65 years | 234 Participants |
| Age, Customized <15 years | 1 Participants |
| Age, Customized ≥65 years | 349 Participants |
| Age, Customized Unknown | 4 Participants |
| Diagnosis Coinfection | 5 Participants |
| Diagnosis Mycobacterium Avium Complex (MAC) | 428 Participants |
| Diagnosis Non-tuberculous Mycobacteria Other Than MAC | 19 Participants |
| Diagnosis Others | 2 Participants |
| Diagnosis Tuberculosis | 134 Participants |
| Sex: Female, Male Female | 363 Participants |
| Sex: Female, Male Male | 225 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 470 / 588 |
| serious Total, serious adverse events | 184 / 588 |
Outcome results
Primary
Clinical Efficacy Rate
Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values.
Time frame: 1 year
Population: The effectiveness analysis set comprised of participants in the safety analysis set who had effectiveness evaluation (overall evaluation by the physician/investigator based upon change in clinical symptoms and laboratory findings) at least once.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Mycobutin (Rifabutin) | Clinical Efficacy Rate | 62.7 Percentage of Participants |
Primary
Clinical Efficacy Rate by Age
Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values. Participants achieved clinical effectiveness by age were counted to assess whether they contribute to the clinical effectiveness.
Time frame: 1 year
Population: The effectiveness analysis set comprised of participants in the safety analysis set who had effectiveness evaluation (overall evaluation by the physician/investigator based upon change in clinical symptoms and laboratory findings) at least once. Participants with observed effectiveness data were included in table.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Age | ˂15 years | 0.0 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Age | ≥15 and <65 years | 71.8 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Age | ≥65 years | 57.1 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Age | Unknown | 50 Percentage of Participants |
Primary
Clinical Efficacy Rate by Diagnosis
Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values. Participants achieved clinical effectiveness by diagnosis were counted to assess whether they contribute to the clinical effectiveness.
Time frame: 1 year
Population: The effectiveness analysis set comprised of participants in the safety analysis set who had effectiveness evaluation (overall evaluation by the physician/investigator based upon change in clinical symptoms and laboratory findings) at least once. Participants with observed effectiveness data were included in table.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Diagnosis | Tuberculosis | 82.3 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Diagnosis | Mycobacterium Avium Complex (MAC) | 53.9 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Diagnosis | Non-tuberculous Mycobacteria Other Than MAC | 58.3 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Diagnosis | Coinfection | 100.0 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Diagnosis | Others | 0.0 Percentage of Participants |
Primary
Clinical Efficacy Rate by Gender
Clinical effectiveness rate, which was defined as the percentage of participants who achieved clinical effectiveness over the total number of assessable effectiveness analysis population, was presented. Clinical effectiveness of Mycobutin was assessed as effective, ineffective, or unassessable by the physician/investigator. Overall effectiveness of Mycobutin was determined by the physician/investigator based on clinical symptoms, laboratory values, and other examinations such as bacterial values. Participants achieved clinical effectiveness by gender were counted to assess whether they contribute to the clinical effectiveness.
Time frame: 1 year
Population: The effectiveness analysis set comprised of participants in the safety analysis set who had effectiveness evaluation (overall evaluation by the physician/investigator based upon change in clinical symptoms and laboratory findings) at least once. Participants with observed effectiveness data were included in table.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Gender | Male | 71.1 Percentage of Participants |
| Mycobutin (Rifabutin) | Clinical Efficacy Rate by Gender | Female | 55.7 Percentage of Participants |
Primary
Number of Participants With Treatment-Related Adverse Events
A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator.
Time frame: 1 year
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received Mycobutin at least once.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events | Treatment-Related Adverse Event | 387 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events | Treatment-Related Serious Adverse Event | 113 Participants |
Primary
Number of Participants With Treatment-Related Adverse Events by Age
A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator. Participants with treatment related adverse events were counted by age to assess whether they were risk factors for the treatment related adverse events.
Time frame: 1 year
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received Mycobutin at least once.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Age | <15 years | 1 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Age | ≥15 and <65 years | 149 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Age | ≥65 years | 235 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Age | Unknown | 2 Participants |
Comparison: The factor tested was age. The null hypothesis was that there was no association between age of participants and the number of participants with treatment-related adverse events.p-value: 0.587Fisher Exact
Comparison: The factor tested was age. The null hypothesis was that there was no ordinal trend in the number of participants with treatment-related adverse events across the age at baseline.p-value: 0.428Cochran-Armitage (EXACT)
Primary
Number of Participants With Treatment-Related Adverse Events by Diagnosis
A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator. Participants with treatment related adverse events were counted by diagnosis to assess whether they were risk factors for the treatment related adverse events.
Time frame: 1 year
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received Mycobutin at least once.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Diagnosis | Tuberculosis | 60 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Diagnosis | Mycobacterium Avium Complex (MAC) | 312 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Diagnosis | Non-tuberculous Mycobacteria Other Than MAC | 11 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Diagnosis | Coinfection | 4 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Diagnosis | Others | 0 Participants |
Comparison: The factor tested was diagnosis. The null hypothesis was that there was no association between diagnosis and the number of participants with treatment-related adverse events.p-value: <0.001Fisher Exact
Primary
Number of Participants With Treatment-Related Adverse Events by Gender
A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Relatedness to Mycobutin was assessed by the physician/investigator. Participants with treatment related adverse events were counted by gender to assess whether they were risk factors for the treatment related adverse events.
Time frame: 1 year
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received Mycobutin at least once.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Gender | Female | 264 Participants |
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events by Gender | Male | 123 Participants |
Comparison: The factor tested was gender. The null hypothesis was that there was no association between gender and the number of participants with treatment-related adverse events.p-value: <0.001Fisher Exact
Primary
Number of Participants With Treatment-Related Adverse Events Unexpected From Japanese Package Insert
A treatment-related adverse event was any untoward medical occurrence attributed to Mycobutin in a participant who received Mycobutin. Expectedness of the adverse event was determined according to the Japanese package insert. Relatedness to Mycobutin was assessed by the physician/investigator.
Time frame: 1 year
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received Mycobutin at least once.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Mycobutin (Rifabutin) | Number of Participants With Treatment-Related Adverse Events Unexpected From Japanese Package Insert | 71 Participants |