This is a non-interventional, observational, retrospective and prospective multicenter Italian study, to describe treatment management and outcomes of Waldenström's Macroglobulinemia (WM) patients treated according to the Italian Compassionate Use Program (CUP) and receiving zanubrutinib following its commercial approval.
Study Type
OBSERVATIONAL
Enrollment
212
Patients will be treated with Zanubrutinib as per routine clinical practice.
A.O. SS. Antonio e Biagio e Cesare Arrigo - S.C.D.U. Ematologia
Alessandria, Italy
A.O.U. Ospedali Riuniti - Clinica di Ematologia
Ancona, Italy
Ospedale C. e G. Mazzoni - U.O.C. di Ematologia
Ascoli Piceno, Italy
AOU Policlinico Consorziale - U.O. Ematologia con Trapianto
Bari, Italy
Nuovo Ospedale degli Infermi - SSD Ematologia
Biella, Italy
Cumulative incidence of treatment discontinuation due to toxicity
Cumulative incidence of treatment discontinuation due to toxicity
Time frame: From the date of first zanubrutinib administration to the date of definitive treatment discontinuation or dose reduction for any cause or death from any cause, from october 2020 up to 24 months since study start
Incidence of adverse events (AEs)
Incidence of adverse events (AEs)
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Incidence of adverse events related to zanubrutinib.
Incidence of adverse events related to zanubrutinib.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Incidence of relevant adverse events.
Incidence of relevant adverse events.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Incidence of serious adverse events (SAEs).
Incidence of serious adverse events (SAEs).
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Incidence of adverse events leading to death.
Incidence of adverse events leading to death.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Incidence of adverse events leading to treatment discontinuation.
Incidence of adverse events leading to treatment discontinuation.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Incidence of adverse events leading to dose reduction/interruption.
Incidence of adverse events leading to dose reduction/interruption.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Time to first onset of relevant adverse event.
Time to first onset of relevant adverse event.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
AEs ≥ G3 Hematological and non-hematological.
AEs ≥ G3 Hematological and non-hematological.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Impact of patients' characteristics and comorbidities on G3 or higher AEs and relevant adverse events development.
Impact of patients' characteristics and comorbidities on G3 or higher AEs and relevant adverse events development.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies.
Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Major response rate (MRR)
Major response rate (MRR) (≥PR) (best reported response).
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Overall response rate (ORR)
Overall response rate (ORR, defined as MRR plus minor response rate)
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Best response
Best response (best reported response).
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Progression-free survival (PFS)
Progression-free survival (PFS) including 6, 12, and 24-month PFS rate
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Overall survival (OS)
Overall survival (OS) including 6, 12, and 24-month OS rate.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Time to treatment failure
Time to treatment failure (any treatment definitive discontinuation, incl. patient or investigator decision, toxicity, progression or death).
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Duration of response (DOR)
Duration of response (DOR)
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Change of IgM levels until end of zanubrutinib treatment.
Change of IgM levels until end of zanubrutinib treatment.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response.
Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
Hospital stays
Hospital stays comprises all planned and unplanned hospitalizations as well as emergency unit visits, regardless of whether there is an association with WM or not. All hospitalizations and emergency unit visits that started during therapy with zanubrutinib will be considered for hospital stays.
Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
EORTC-QLQ-C30
Quality of life (QoL) assessed with EORTC-QLQ-C30 questionnaire
Time frame: Prospective cohort: up to 24 months (duration of study)
EQ-5D-5L
Quality of life (QoL) assessed with EQ-5D-5L questionnaire
Time frame: Prospective cohort: up to 24 months (duration of study)
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Policlinico S. Orsola-Malpighi - Istituto di Ematologia "Seragnoli"
Bologna, Italy
Ospedale Centrale di Bolzano - Divisione di Ematologia e T.M.O.
Bolzano, Italy
Azienda Ospedaliera Universitaria Policlinico - S. Marco - UOC di Ematologia
Catania, Italy
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant'Anna - Ematologia e fisiopatologia della coagulazione
Ferrara, Italy
Azienda Ospedaliera Universitaria Careggi - Unità funzionale di Ematologia
Florence, Italy
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