Fungal Infection
Conditions
Brief summary
Examine the safety and effectiveness of Vfend \[voriconazole\] for prophylaxix use under general clinical practices.
Interventions
None listed
Sponsors
Pfizer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
0 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients undergoing HSCT (Hematopoietic Stem Cell Transplantation).
Exclusion criteria
* Patients who have been previously enrolled in this study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Drug Reactions | Maximum 3 years | An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. A serious ADR was an ADR resulting in any of the following outcomes or deemed significant for any other reason: death; life-threatening experience (immediate risk of dying); initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Relatedness to VFEND was assessed by the physician. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants With Adverse Drug Reactions by Age | Maximum 3 years | An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by age (\<15 years, ≥15 years) to assess whether it was a risk factor for the occurrence of ADRs. |
| Proportion of Participants With Adverse Drug Reactions by Reason for Use | Maximum 3 years | An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by reason for use to assess whether it was a risk factor for the occurrence of ADRs. |
| Proportion of Participants With Adverse Drug Reactions by Long-term Use | Maximum 3 years | An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by long-term use to assess whether it was a risk factor for the occurrence of ADRs. |
| Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) | Maximum 3 years | IFI rate, which was defined as the percentage of participants who developed invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician. In case of with onset, it was classified as proven diagnosis, probable diagnosis, or possible case according to the diagnostic criteria of the European Organization for Research and Treatment of Cancer/Mycoses Study Group (EORTC/MSG). Participants judged as proven diagnosis or probable diagnosis were counted as those who developed invasive fungal infections for the calculation of IFI rate. |
| Number of Participants With Adverse Drug Reactions Not Expected From the Approved Local Product Document (Unknown Adverse Drug Reactions) | Maximum 3 years | An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Expectedness of the adverse event was determined according to the Japanese package insert. Relatedness to VFEND was assessed by the physician. |
| Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Reason for Use | Maximum 3 years | IFI rate, which was defined as the percentage of participants who developed invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician. In case of with onset, it was classified as proven diagnosis, probable diagnosis, or possible case according to the diagnostic criteria of the European Organization for Research and Treatment of Cancer/Mycoses Study Group (EORTC/MSG). Participants judged as proven diagnosis or probable diagnosis were counted as those who developed invasive fungal infections for the calculation of IFI rate. Participants who developed invasive fungal infections were counted by reason for use to assess whether it contributes to the clinical effectiveness. |
| Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) | Maximum 3 years | Success rate, which was defined as the percentage of participants with successful prophylaxis of invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician, and participants judged as without onset were counted as those with successful prophylaxis of invasive fungal infections for the calculation of success rate. |
| Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Age | Maximum 3 years | Success rate, which was defined as the percentage of participants with successful prophylaxis of invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician, and participants judged as without onset were counted as those with successful prophylaxis of invasive fungal infections for the calculation of success rate. Participants with successful prophylaxis of invasive fungal infections were counted by age (\<15 years, ≥15 years) to assess whether it contributes to the clinical effectiveness. |
| Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Reason for Use | Maximum 3 years | Success rate, which was defined as the percentage of participants with successful prophylaxis of invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician, and participants judged as without onset were counted as those with successful prophylaxis of invasive fungal infections for the calculation of success rate. Participants with successful prophylaxis of invasive fungal infections were counted by reason for use to assess whether it contributes to the clinical effectiveness. |
| Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Age | Maximum 3 years | IFI rate, which was defined as the percentage of participants who developed invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician. In case of with onset, it was classified as proven diagnosis, probable diagnosis, or possible case according to the diagnostic criteria of the European Organization for Research and Treatment of Cancer/Mycoses Study Group (EORTC/MSG). Participants judged as proven diagnosis or probable diagnosis were counted as those who developed invasive fungal infections for the calculation of IFI rate. Participants who developed invasive fungal infections were counted by age (\<15 years, ≥15 years) to assess whether it contributes to the clinical effectiveness. |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| VFEND (Voriconazole) Participants who received VFEND as indicated in the approved local product document were observed for a period of 3 years at maximum. The dosage can be adjusted as per physician's discretion. | 233 |
| Total | 233 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 4 |
Baseline characteristics
| Characteristic | VFEND (Voriconazole) | — |
|---|---|---|
| Age, Customized ≥15 and <65 years | 128 Participants | — |
| Age, Customized <15 years | 79 Participants | — |
| Age, Customized ≥65 years | 26 Participants | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Reason for Use Others | 1 Participants | — |
| Reason for Use Primary prophylaxis | 216 Participants | — |
| Reason for Use Secondary prophylaxis | 16 Participants | — |
| Sex: Female, Male Female | 96 Participants | — |
| Sex: Female, Male Male | 137 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 19 / 233 |
| other Total, other adverse events | 153 / 233 |
| serious Total, serious adverse events | 60 / 233 |
Outcome results
Primary
Number of Participants With Adverse Drug Reactions
An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. A serious ADR was an ADR resulting in any of the following outcomes or deemed significant for any other reason: death; life-threatening experience (immediate risk of dying); initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Relatedness to VFEND was assessed by the physician.
Time frame: Maximum 3 years
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Number of Participants With Adverse Drug Reactions | ADR | 62 Participants |
| VFEND (Voriconazole) | Number of Participants With Adverse Drug Reactions | Serious ADR | 5 Participants |
Secondary
Number of Participants With Adverse Drug Reactions Not Expected From the Approved Local Product Document (Unknown Adverse Drug Reactions)
An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Expectedness of the adverse event was determined according to the Japanese package insert. Relatedness to VFEND was assessed by the physician.
Time frame: Maximum 3 years
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| VFEND (Voriconazole) | Number of Participants With Adverse Drug Reactions Not Expected From the Approved Local Product Document (Unknown Adverse Drug Reactions) | 6 Participants |
Secondary
Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate)
IFI rate, which was defined as the percentage of participants who developed invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician. In case of with onset, it was classified as proven diagnosis, probable diagnosis, or possible case according to the diagnostic criteria of the European Organization for Research and Treatment of Cancer/Mycoses Study Group (EORTC/MSG). Participants judged as proven diagnosis or probable diagnosis were counted as those who developed invasive fungal infections for the calculation of IFI rate.
Time frame: Maximum 3 years
Population: The efficacy analysis set (EAS) comprised of participants in the safety analysis set for whom the presence/absence of invasive fungal infections has been evaluated, excluding those with no information on effectiveness or non-target disease. The EAS was 199 participants. Those assessed as indeterminate (n=1) was excluded from the calculation.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) | 1.5 Percentage of Participants |
Secondary
Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Age
IFI rate, which was defined as the percentage of participants who developed invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician. In case of with onset, it was classified as proven diagnosis, probable diagnosis, or possible case according to the diagnostic criteria of the European Organization for Research and Treatment of Cancer/Mycoses Study Group (EORTC/MSG). Participants judged as proven diagnosis or probable diagnosis were counted as those who developed invasive fungal infections for the calculation of IFI rate. Participants who developed invasive fungal infections were counted by age (\<15 years, ≥15 years) to assess whether it contributes to the clinical effectiveness.
Time frame: Maximum 3 years
Population: The efficacy analysis set (EAS) comprised of participants in the safety analysis set for whom the presence/absence of invasive fungal infections has been evaluated, excluding those with no information on effectiveness or non-target disease. The EAS was 199 participants. Those assessed as indeterminate (n=1) was excluded from the calculation.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Age | <15 years | 1.5 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Age | ≥15 years | 1.5 Percentage of Participants |
Secondary
Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Reason for Use
IFI rate, which was defined as the percentage of participants who developed invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician. In case of with onset, it was classified as proven diagnosis, probable diagnosis, or possible case according to the diagnostic criteria of the European Organization for Research and Treatment of Cancer/Mycoses Study Group (EORTC/MSG). Participants judged as proven diagnosis or probable diagnosis were counted as those who developed invasive fungal infections for the calculation of IFI rate. Participants who developed invasive fungal infections were counted by reason for use to assess whether it contributes to the clinical effectiveness.
Time frame: Maximum 3 years
Population: The efficacy analysis set (EAS) comprised of participants in the safety analysis set for whom the presence/absence of invasive fungal infections has been evaluated, excluding those with no information on effectiveness or non-target disease. The EAS was 199 participants. Those assessed as indeterminate (n=1) was excluded from the calculation.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Reason for Use | Primary prophylaxis | 1.1 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants Who Developed Invasive Fungal Infections (IFI Rate) by Reason for Use | Secondary prophylaxis | 7.1 Percentage of Participants |
Secondary
Proportion of Participants With Adverse Drug Reactions by Age
An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by age (\<15 years, ≥15 years) to assess whether it was a risk factor for the occurrence of ADRs.
Time frame: Maximum 3 years
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants With Adverse Drug Reactions by Age | <15 years | 20.25 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants With Adverse Drug Reactions by Age | ≥15 years | 29.87 Percentage of Participants |
Secondary
Proportion of Participants With Adverse Drug Reactions by Long-term Use
An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by long-term use to assess whether it was a risk factor for the occurrence of ADRs.
Time frame: Maximum 3 years
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants With Adverse Drug Reactions by Long-term Use | <180 days | 24.56 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants With Adverse Drug Reactions by Long-term Use | ≥180 days | 32.26 Percentage of Participants |
Secondary
Proportion of Participants With Adverse Drug Reactions by Reason for Use
An adverse drug reaction (ADR) was any untoward medical occurrence attributed to VFEND in a participant who received VFEND. Relatedness to VFEND was assessed by the physician. Participants with ADRs were counted by reason for use to assess whether it was a risk factor for the occurrence of ADRs.
Time frame: Maximum 3 years
Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received VFEND at least once.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants With Adverse Drug Reactions by Reason for Use | Primary prophylaxis | 26.39 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants With Adverse Drug Reactions by Reason for Use | Secondary prophylaxis | 31.25 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants With Adverse Drug Reactions by Reason for Use | Others | 0.00 Percentage of Participants |
Secondary
Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate)
Success rate, which was defined as the percentage of participants with successful prophylaxis of invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician, and participants judged as without onset were counted as those with successful prophylaxis of invasive fungal infections for the calculation of success rate.
Time frame: Maximum 3 years
Population: The efficacy analysis set (EAS) comprised of participants in the safety analysis set for whom the presence/absence of invasive fungal infections has been evaluated, excluding those with no information on effectiveness or non-target disease. The EAS was 199 participants. Those assessed as indeterminate (n=1) was excluded from the calculation.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) | 97.5 Percentage of Participants |
Secondary
Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Age
Success rate, which was defined as the percentage of participants with successful prophylaxis of invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician, and participants judged as without onset were counted as those with successful prophylaxis of invasive fungal infections for the calculation of success rate. Participants with successful prophylaxis of invasive fungal infections were counted by age (\<15 years, ≥15 years) to assess whether it contributes to the clinical effectiveness.
Time frame: Maximum 3 years
Population: The efficacy analysis set (EAS) comprised of participants in the safety analysis set for whom the presence/absence of invasive fungal infections has been evaluated, excluding those with no information on effectiveness or non-target disease. The EAS was 199 participants. Those assessed as indeterminate (n=1) was excluded from the calculation.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Age | <15 years | 98.5 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Age | ≥15 years | 96.9 Percentage of Participants |
Secondary
Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Reason for Use
Success rate, which was defined as the percentage of participants with successful prophylaxis of invasive fungal infections over the total number of assessable effectiveness analysis population, was presented along with the corresponding 2-sided 95% CI. Presence or absence of invasive fungal infections was judged as without onset, with onset, or indeterminate by the physician, and participants judged as without onset were counted as those with successful prophylaxis of invasive fungal infections for the calculation of success rate. Participants with successful prophylaxis of invasive fungal infections were counted by reason for use to assess whether it contributes to the clinical effectiveness.
Time frame: Maximum 3 years
Population: The efficacy analysis set (EAS) comprised of participants in the safety analysis set for whom the presence/absence of invasive fungal infections has been evaluated, excluding those with no information on effectiveness or non-target disease. The EAS was 199 participants. Those assessed as indeterminate (n=1) was excluded from the calculation.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| VFEND (Voriconazole) | Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Reason for Use | Primary prophylaxis | 97.8 Percentage of Participants |
| VFEND (Voriconazole) | Proportion of Participants With Successful Prophylaxis of Invasive Fungal Infections (Success Rate) by Reason for Use | Secondary prophylaxis | 92.9 Percentage of Participants |