Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Other |
Patients' treatment expectation and satisfaction |
Patients' treatment expectation and satisfaction (assessed via project specific questionnaire) will be analyzed by time point, using absolute and relative frequencies. |
Baseline, 3 months after treatment start with zanubrutinib, end of treatment |
|
| Other |
Physicians' treatment expectation and satisfaction |
Physicians' treatment expectation and satisfaction (assessed via project specific questionnaire) will be analyzed by time point, using absolute and relative frequencies. |
Baseline, 3 months after treatment start with zanubrutinib, end of treatment |
|
| Other |
Collection of biomarker test results (according to clinical routine) |
Number of patients with biomarker testing as well as sample types, test methods and test results of biomarker testing per biomarker will be provided including patients with resistance mechanism testing before treatment decision with zanubrutinib. Information on the testing of MYD88 and CXCR4 is mandatory. |
Baseline, up to 5 years |
|
| Other |
Patient management during SARS-Covid-19 pandemic |
The patient management during SARS-Covid-19 pandemic (assessed via project specific questionnaire) (patient supervised by oncologist or additionally by family doctor) will be presented by frequency tables. |
Baseline and end of zanubrutnib treatment, up to 5 years |
|
| Primary |
Medical resource utilization |
Frequency of hospitalizations, i.e. number of hospital stays plus number of emergency unit visits (without hospitalization) per patient |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Global health-related quality of life (QoL) collected via EORTC QLQ-C30 during course of treatment and follow-up |
Course of QoL during treatment and follow-up (collected via European Organisation for research and treatment of cancer quality of life in cancer patient questionnaire (EORTC QLQ-C30). Scoring of the questionnaire will be performed according to the respective manual. |
During zanubrutinib treatment and follow-up, up to 5 years |
|
| Secondary |
Global health-related quality of life (QoL) collected via EQ-5D-5L during course of treatment and follow-up |
Course of QoL during treatment and follow-up (collected via European quality of life 5 dimension 5 level version (EQ-5D-5L)). Scoring of the questionnaire will be performed according to the respective manual. |
During zanubrutinib treatment and follow-up, up to 5 years |
|
| Secondary |
Incidence of (serious) adverse events ((S)AEs) |
Incidence of (S)AEs; (S)AEs will be summarized by the most recent Medical Dictionary for Regulatory Activities (MedDRA) system organ class and preferred term. |
Start of zanubrutinib treatment until 30 days after end of zanubrutinib treatment |
|
| Secondary |
Incidence of (serious) adverse drug reactions ((S)ADRs) |
Incidence of (S)ADRs related to zanubrutinib |
Start of zanubrutinib treatment until end of study, up to 5 years |
|
| Secondary |
Incidence of adverse events of special interest (AESIs) |
Incidence of AESIs |
Start of zanubrutinib treatment until 30 days after end of zanubrutinib treatment |
|
| Secondary |
Time to AESIs |
The time to first onset of AESIs. |
Start of zanubrutinib treatment until 30 days after end of zanubrutinib treatment |
|
| Secondary |
Time to neutropenia |
The time to first onset of neutropenia grade =3 (MedDRA terms: neutropenia and neutrophil count decrease). |
Start of zanubrutinib treatment until 30 days after end of zanubrutinib treatment |
|
| Secondary |
Rate of neutropenia grade =3 |
Rate of patients with neutropenia grade =3 during zanubrutinib treatment. Neutropenia incorporates the MedDRA terms: neutropenia and neutrophil count decrease. |
Start of zanubrutinib treatment until 30 days after end of zanubrutinib treatment |
|
| Secondary |
Proportion of patients with complete response (CR) or very good partial response (VGPR) (best reported response) |
The proportion of patients with a best overall response of CR or VGPR. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
In the WM cohort only: Major response rate (MRR) |
MRR is defined as the proportion of patients with a best overall response = PR (partial response) |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
In the WM cohort only: Best response |
Best response is defined as best reported response during study treatment CR (complete response) or VGPR (very good partial response). |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
In the WM cohort only: IgM levels |
Change of IgM levels until end of zanubrutinib treatment for WM cohort. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Progression-free Survival (PFS) |
PFS is defined as the time from treatment start until progression or death from any cause, whichever comes first. |
Treatment start with zanubrutinib until end of study, up to 5 years |
|
| Secondary |
6-, 12-, 18- and 24-month PFS rate |
Percentage of patients without disease progression or death from any cause at 6, 12, and 24 months after start of zanubrutinib treatment. |
6, 12, and 24 months after start of zanubrutinib treatment |
|
| Secondary |
Overall Survival (OS) |
OS is defined as the time from treatment start until death. |
Treatment start with zanubrutinib until end of study, up to 5 years |
|
| Secondary |
6-, 12-, 18- and 24-month OS rate |
Percentage of patients alive at 6, 12, and 24 months after start of zanubrutinib treatment. |
6, 12, and 24 months after start of zanubrutinib treatment |
|
| Secondary |
WM Cohort: Overall response rate (ORR) |
For the WM cohort, overall response rate is defined as CR, VGPR and PR (partial response). |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
WM Cohort: Best overall response rate (ORR) |
For the WM cohort, best overall response rate is defined as CR, VGPR, PR (partial response), MR (minor response), SD (stable disease) or PD (progressive disease). |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
CLL Cohort: Overall response rate (ORR) |
For the CLL cohort, overall response rate is defined as CR and PR. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
CLL Cohort: Best overall response rate (ORR) |
For the CLL cohort, best overall response rate is defined as CR, PR, SD or PD. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
MZL Cohort: Overall response rate (ORR) |
For the MZL cohort, overall response rate is defined as CR, pMRD (probable minimal residue disease), PR and rRD (responding residual disease). |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
MZL Cohort: Best overall response rate (ORR) |
For the MZL cohort, best overall response rate is defined as CR, pMRD, PR, rRD, No change/SD or PD |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
FL Cohort: Overall response rate (ORR) |
For the FL cohort, overall response rate is defined as CR or PR. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
FL Cohort: Best overall response rate (ORR) |
For the FL cohort, best overall response rate is defined as CR, PR, MR, SD or PD. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Time to treatment failure (TTF) |
TTF is defined as time interval from treatment start to discontinuation of treatment because of disease progression, treatment toxicity, switch of therapy of any reason, and death. |
Treatment start with zanubrutinib until end of treatment, up to 5 years |
|
| Secondary |
Frequency of blood product transfusion |
The number of patients receiving blood product transfusion, the number of transfusions per patient and the kind of transfusion (e.g., erythrocytes, thrombocytes). |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
WM Cohort: Change of IgM levels until end of zanubrutinib treatment |
Difference between the baseline value and the end of treatment value of the IgM level. |
Baseline and end of zanubrutinib treatment, up to 5 years |
|
| Secondary |
Therapy decision making |
Frequency and weighting of distinct parameters affecting therapy choice of the treating physician assessed by project specific questionnaire |
Baseline |
|
| Secondary |
Time from first diagnosis of WM, CLL, MZL or FL to zanubrutinib treatment start |
Time from first diagnosis of WM, CLL, MZL or FL to zanubrutinib treatment start including timing and duration of possible watch & wait strategy. |
Baseline |
|
| Secondary |
Previous therapies |
Key details of previous therapies (including plasmapheresis for WM, transplantations for WM, CLL and FL, radiotherapy for CLL, MZL and FL and surgery for CLL, MZL and FL). |
Baseline |
|
| Secondary |
Daily dose of zanubrutinib |
Frequency tables including the daily dose (mg) will be given using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
FL cohort: Daily dose of obinutuzumab |
Frequency tables including the daily dose (mg) will be given using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Dose modifications of zanubrutinib |
Frequency tables will be provided including therapy modifications (reduction and increase) with reasons using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
FL cohort: Dose modifications of obinutuzumab |
Frequency tables will be provided including therapy modifications (reduction and increase) with reasons using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Therapy interruptions of zanubrutinib |
Frequency tables will be provided including treatment interruptions with reasons as well as reasons for treatment termination. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
FL cohort: Therapy interruptions of obinutuzumab |
Frequency tables will be provided including treatment interruptions with reasons as well as reasons for treatment termination. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Dose intensity of zanubrutinib |
Frequency tables will be provided including the dose intensity (absolute and relative) using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
FL cohort: Dose intensity of obinutuzumab |
Frequency tables will be provided including the dose intensity (absolute and relative) using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Treatment duration with zanubrutinib |
Frequency tables will be provided including the treatment duration will be given using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
FL cohort: Treatment duration with obinutuzumab |
Frequency tables will be provided including the treatment duration will be given using descriptive statistics. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Subsequent antineoplastic therapies |
Key details of subsequent antineoplastic therapies after zanubrutinib (including plasmapheresis for WM, stem cell transplantations for WM, CLL and FL, radiotherapy for CLL, MZL and FL and surgery for CLL, MZL and FL): duration (descriptive statistics), number, substances and reason for end of subsequent treatments (frequencies). |
End of zanubrutinib treatment until end of study, up to 5 years |
|
| Secondary |
Frequency for concomitant medication during zanubrutinib treatment |
Substances (WHO-ATC level 2), ongoing status and indication (frequencies) |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Frequency of antibiotic use for prophylactic reasons during zanubrutinib treatment |
Proportion of patients with at least one-time antibiotic use for prophylactic reasons during zanubrutinib treatment. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Frequency of antibiotic use for treatment of AEs during zanubrutinib treatment |
Proportion of patients taking at least one-time antibiotics for treatment of AEs during zanubrutinib treatment. |
During zanubrutinib treatment, up to 5 years |
|
| Secondary |
Frequency of antibiotic use in patients with neutropenia during zanubrutinib treatment |
Proportion of patients presenting with neutropenia during zanubrutinib treatment taking at least one-time antibiotics. |
During zanubrutinib treatment, up to 5 years |
|
