Japanese post-marketing surveillance

14 evidence topics · 1 source

Objective

Real-world safety and effectiveness objective

“This post-marketing surveillance evaluated the safety and effectiveness of tirabrutinib in patients with relapsed/refractory primary central nervous system lymphoma (r/r PCNSL) in a real-world setting in Japan.” (opens the source at this quote in a new tab)

Location and study date

Registration period and observation duration

“All patients with r/r PCNSL who started treatment with tirabrutinib between May 20 and 31 October 2020, were registered.” (opens the source at this quote in a new tab)
“Adverse drug reactions (ADRs) and effectiveness were assessed over 52-weeks from the start of tirabrutinib treatment.” (opens the source at this quote in a new tab)

Study design

Observational surveillance design

“This was a prospective, non-interventional, observational PMS which evaluated the safety and effectiveness of tirabrutinib over the 52-week period from the first dose in patients with r/r PCNSL in Japan.” (opens the source at this quote in a new tab)

Eligibility criteria

Registered population

“All patients with r/r PCNSL who started treatment with tirabrutinib between May 20 and 31 October 2020, were registered.” (opens the source at this quote in a new tab)

Treatment

Routine-care treatment

“No invasive or interventional procedures were performed on the patients for the purpose of this surveillance; only clinical information was collected from routine medical practice” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using adverse drug reactions as the safety outcome

Safety and effectiveness observation

“Adverse drug reactions (ADRs) and effectiveness were assessed over 52-weeks from the start of tirabrutinib treatment.” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Safety and effectiveness population sizes

“Of the 189 patients assessed for safety, 52.4% were male, the median age was 70.0 years (range 30-88), and 49.2% of patients had a Karnofsky performance status score of ≤60.” (opens the source at this quote in a new tab)
“Among the 121 patients assessed for effectiveness, the overall response rate evaluated by the treating physicians was 61.2%, and the 365-day survival rate was 69.8%.” (opens the source at this quote in a new tab)

Description of analysis sets

Analysis-set construction

“After combining 10 transferred patients with the original cases and excluding 43 whose consent for disclosure was not obtained from patients or facilities, 189 patients were included in the safety analysis set.” (opens the source at this quote in a new tab)
“These 68 patients were excluded from the effectiveness analysis set, hence a total of 121 patients were included in the effectiveness analysis set.” (opens the source at this quote in a new tab)

Descriptive analysis and confidence intervals

“Descriptive statistics were used to summarize all patients’ baseline characteristics, treatment status, and safety and effectiveness parameters including ADRs and tumor response.” (opens the source at this quote in a new tab)
“The 95% confidence intervals were determined by the Clopper-Pearson exact method.” (opens the source at this quote in a new tab)

Results

Participant disposition

Registration and case report forms

“A total of 244 patients who started treatment with tirabrutinib between May 20 and 31 October 2020, were registered in this surveillance; case report forms were collected from 242 of these patients” (opens the source at this quote in a new tab)

Baseline characteristics

Safety population baseline characteristics

“Of the 189 patients assessed for safety, 52.4% were male, the median age was 70.0 years (range 30-88), and 49.2% of patients had a Karnofsky performance status score of ≤60.” (opens the source at this quote in a new tab)

Efficacy results

Physician-assessed response

“the overall response rate evaluated by the treating physicians was 61.2%” (opens the source at this quote in a new tab)

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

Adverse drug reactions

“ADRs of any grade and grade ≥3 were observed in 55.6% and 24.3%” (opens the source at this quote in a new tab)

Study limitations

Summary

This prospective observational surveillance had no randomized comparator. Safety was assessed in 189 participants and effectiveness in 121, so the response rate must not be interpreted using the safety-population denominator. Response was assessed by treating physicians. The 52-week observation period limits assessment of later outcomes.