Trial Outcomes & Findings for Non-interventional Study for Real-world Data of Afatinib Treatment in First-line Setting and of Subsequent Therapies for Patients With Advanced Epidermal Growth Factor Receptor (EGFR) Mutation-positive Lung Adenocarcinoma (NCT NCT04795245)

NCT ID: NCT04795245

Last Updated: 2024-12-27

Results Overview

Time on Treatment (TOT) with afatinib in first-line TOT (TOT1). This was assessed as the time from the start of afatinib as first-line treatment until the end of afatinib treatment or death date by any cause, whichever occurs first. If patients did not discontinue first-line treatment with afatinib and did not die at the data extraction, they were censored on the date they were last verified to have been on first-line treatment with afatinib. The survival probability rate (95% confidence interval) against time to first-line treatment failure at 18 months and at 36 months is reported.

Recruitment status

COMPLETED

Target enrollment

805 participants

Primary outcome timeframe

From start of afatinib as first line treatment up to 18 months and up to 36 months.

Results posted on

2024-12-27

Participant Flow

Non-interventional, multi-center study from existing data of patients treated with afatinib as the first-line treatment. 40 study sites in Japan were planned for participating. Recruitment of patients was stopped at all sites when it was determined that a sufficient number of patients have been enrolled. A maximum of 50 patients were limited for enrolment per site to avoid differential study site influence on study results. Data extraction started on 01 April 2021 and ended on 07 November 2022.

Only subjects that met all inclusion and none of the exclusion criteria were included.

Participant milestones

Participant milestones
Measure
Patients Treated With Afatinib
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Overall Study
STARTED
805
Overall Study
COMPLETED
85
Overall Study
NOT COMPLETED
720

Reasons for withdrawal

Reasons for withdrawal
Measure
Patients Treated With Afatinib
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Overall Study
Unkown reason
7
Overall Study
Death
17
Overall Study
Other than listed
19
Overall Study
Adverse Event
143
Overall Study
Progressive disease
512
Overall Study
Unknown if patients were still on treatment at end of observation period
22

Baseline Characteristics

Non-interventional Study for Real-world Data of Afatinib Treatment in First-line Setting and of Subsequent Therapies for Patients With Advanced Epidermal Growth Factor Receptor (EGFR) Mutation-positive Lung Adenocarcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Patients Treated With Afatinib
n=805 Participants
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Age, Continuous
65.8 Years
STANDARD_DEVIATION 10.5 • n=5 Participants
Sex: Female, Male
Female
440 Participants
n=5 Participants
Sex: Female, Male
Male
365 Participants
n=5 Participants
Race/Ethnicity, Customized
Japanese
799 Participants
n=5 Participants
Race/Ethnicity, Customized
Other Asian
4 Participants
n=5 Participants
Race/Ethnicity, Customized
Non-Asian
2 Participants
n=5 Participants

PRIMARY outcome

Timeframe: From start of afatinib as first line treatment up to 18 months and up to 36 months.

Population: Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.

Time on Treatment (TOT) with afatinib in first-line TOT (TOT1). This was assessed as the time from the start of afatinib as first-line treatment until the end of afatinib treatment or death date by any cause, whichever occurs first. If patients did not discontinue first-line treatment with afatinib and did not die at the data extraction, they were censored on the date they were last verified to have been on first-line treatment with afatinib. The survival probability rate (95% confidence interval) against time to first-line treatment failure at 18 months and at 36 months is reported.

Outcome measures

Outcome measures
Measure
Patients Treated With Afatinib
n=805 Participants
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Time on Treatment (TOT) With Afatinib in First-line TOT (TOT1)
At 18 months
0.39 Proportion of participants
Interval 0.35 to 0.42
Time on Treatment (TOT) With Afatinib in First-line TOT (TOT1)
At 36 months
0.15 Proportion of participants
Interval 0.12 to 0.17

SECONDARY outcome

Timeframe: From start of afatinib up to 18 months and up to 36 months.

Population: Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.

Time on treatment from the start of afatinib until end of subsequent therapies in the second-line setting or death by any cause (TOT). If patients did not discontinue second-line treatment and did not die at the data extraction, they were censored on the date they are last verified to have been on second-line treatment. If patients were on first-line treatment and did not move to second-line treatment at the data extraction, ToT is same as first-line TOT (ToT1) for these patients. The survival probability rate (95% confidence interval) against time to treatment failure at 18 months and at 36 months is reported.

Outcome measures

Outcome measures
Measure
Patients Treated With Afatinib
n=805 Participants
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Time on Treatment From the Start of Afatinib Until End of Subsequent Therapies in the Second-line Setting or Death by Any Cause (TOT)
At 18 months
0.58 Proportion of participants
Interval 0.54 to 0.61
Time on Treatment From the Start of Afatinib Until End of Subsequent Therapies in the Second-line Setting or Death by Any Cause (TOT)
At 36 months
0.30 Proportion of participants
Interval 0.27 to 0.34

SECONDARY outcome

Timeframe: From start of afatinib as second line treatment up to 18 months and up to 36 months.

Population: Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.

Time on treatment from start of the second-line treatment until end of the second-line treatment or death by any cause (TOT2). If patients did not discontinue second-line treatment and did not die at the data extraction, they were censored on the date they are last verified to have been on second-line treatment. The survival probability rate (95% confidence interval) against time to second-line treatment failure at 18 months and at 36 months is reported.

Outcome measures

Outcome measures
Measure
Patients Treated With Afatinib
n=805 Participants
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Time on Treatment From Start of the Second-line Treatment Until End of the Second-line Treatment or Death by Any Cause (TOT2)
At 18 months
0.17 Proportion of participants
Interval 0.14 to 0.2
Time on Treatment From Start of the Second-line Treatment Until End of the Second-line Treatment or Death by Any Cause (TOT2)
At 36 months
0.09 Proportion of participants
Interval 0.07 to 0.12

SECONDARY outcome

Timeframe: From start of afatinib up to 18 months and up to 36 months.

Population: Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.

If patients did not die at the data extraction, they were censored on the date they are last verified to be alive. The survival probability rate (95% confidence interval) at 18 months and at 36 months is reported.

Outcome measures

Outcome measures
Measure
Patients Treated With Afatinib
n=805 Participants
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Overall Survival
At 18 months
0.80 Proportion of participants
Interval 0.78 to 0.83
Overall Survival
At 36 months
0.54 Proportion of participants
Interval 0.51 to 0.58

SECONDARY outcome

Timeframe: From start of afatinib up to 18 months and up to 36 months.

Population: Participants included in the full analysis set and who had received afatinib at initial dose of 40 mg.

Time to initial dose reduction of afatinib. If patients did not reduce initial dose of afatinib at the data extraction, they were censored on the date they were last verified to have been on the initial dose of afatinib or increased dose of afatinib. The survival probability rate (95% confidence interval) against time to initial dose reduction of afatinib at 18 months and 36 months is reported.

Outcome measures

Outcome measures
Measure
Patients Treated With Afatinib
n=594 Participants
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Time to Initial Dose Reduction of Afatinib
At 18 months
0.22 Proportion of participants
Interval 0.19 to 0.26
Time to Initial Dose Reduction of Afatinib
At 36 months
0.20 Proportion of participants
Interval 0.16 to 0.24

SECONDARY outcome

Timeframe: From start of data extraction until end of data extraction, up to 586 days.

Population: Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.

Percentage of participants with dose modifications of afatinib.

Outcome measures

Outcome measures
Measure
Patients Treated With Afatinib
n=805 Participants
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Percentage of Participants With Dose Modifications of Afatinib
73.7 Percentage of participants

Adverse Events

Patients Treated With Afatinib

Serious events: 8 serious events
Other events: 43 other events
Deaths: 486 deaths

Serious adverse events

Serious adverse events
Measure
Patients Treated With Afatinib
n=805 participants at risk
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Cardiac disorders
Acute myocardial infarction
0.12%
1/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.
Gastrointestinal disorders
Diarrhoea
0.25%
2/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.
Immune system disorders
Anaphylactic reaction
0.12%
1/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.
Injury, poisoning and procedural complications
Fall
0.12%
1/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.
Metabolism and nutrition disorders
Decreased appetite
0.12%
1/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.
Respiratory, thoracic and mediastinal disorders
Pneumonitis
0.12%
1/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
0.25%
2/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.
Skin and subcutaneous tissue disorders
Rash
0.12%
1/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.

Other adverse events

Other adverse events
Measure
Patients Treated With Afatinib
n=805 participants at risk
Patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) treated with afatinib as the first-line treatment during the period from launch of afatinib (7 May 2014) to at least 20 months prior to the time point of enrolment of patients (first round) and follow-up data was extracted for the same patients one year after completion of first round during the study period (second round). Patients were treated with 20, 30, 40 or 50 milligram (mg) tablet of afatinib once daily as indicated in the approved label of afatinib (Giotrif®).
Gastrointestinal disorders
Nasopharyngitis
5.3%
43/805 • Adverse events and deaths were collected for approximately 8.5 years.
Final analysis set: All patients who were enrolled in this study and were not excluded due to violations.

Additional Information

Boehringer Ingelheim, Call Center

Boehringer Ingelheim

Phone: 1-800-243-0127

Results disclosure agreements

  • Principal investigator is a sponsor employee Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.
  • Publication restrictions are in place

Restriction type: OTHER