Evidence map›Paper›PMID 33284944›Full record

Trial reportBlood advances2020

Zanubrutinib for the treatment of MYD88 wild-type Waldenström macroglobulinemia: a substudy of the phase 3 ASPEN trial.

Meletios Dimopoulos, Ramon Garcia Sanz, Hui-Peng Lee, Marek Trneny, Marzia Varettoni, Stephen Opat, Shirley D'Sa, Roger G Owen, Gavin Cull, Stephen Mulligan and 24 more

Open access · goldAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Blood advances, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.

0numbers the graph read from it
0cells of the map it votes in
43citing papers in PubMed
7.5field-weighted citation impact, top 2% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

43 citing papers in PubMed, 76 citations in OpenAlex.

  1. Trial
  2. Zanubrutinib Versus Ibrutinib in Symptomatic Waldenström Macroglobulinemia: Final Analysis From the Randomized Phase III ASPEN Study.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023
    Trial
  3. Single-Agent Ibrutinib for Rituximab-Refractory Waldenström Macroglobulinemia: Final Analysis of the Substudy of the Phase III InnovateClinical cancer research : an official journal of the American Association for Cancer Research · 2021
    Trial
  4. Trial
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  8. Review
  9. Article
  10. Novel Therapies in Primary Central Nervous System Lymphoma.Clinical pharmacology : advances and applications · 2025
    Review
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  14. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

34 authors at 20 institutions in 11 countries.

Meletios DimopoulosDepartment of Clinical Therapeutics, National and Kapodistrian University of Athens, Athens, Greece.
Ramon Garcia SanzHospital Universitario de Salamanca, Salamanca, Spain.
Hui-Peng LeeDepartment of Haematology, Flinders Medical Centre, Adelaide, SA, Australia.
Marek Trneny1st Department of Medicine, Faculty of Medicine 1, Charles University, General Hospital, Prague, Czech Republic.
Marzia VarettoniFondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico, San Matteo, Pavia, Italy.
Stephen OpatMonash Health and.
Shirley D'SaUniversity College London Hospital Foundation Trust, London, United Kingdom.
Roger G OwenSt James University Hospital, Leeds, United Kingdom.
Gavin CullSir Charles Gairdner Hospital, Perth, WA, Australia.
Stephen MulliganRoyal North Shore Hospital, Sydney, NSW, Australia.
Jaroslaw CzyzSzpital Uniwersytecki nr 2, Dr Jana Biziela, Kujawsko-pomorskie, Bydgoszcz, Poland.
Jorge J CastilloBing Center for Waldenström Macroglobulinemia, Dana-Farber Cancer Institute, Boston, MA.
Marina MottaAO Spedali Civili di Brescia, Lombardia, Italy.
Tanya SiddiqiCity of Hope National Medical Center, Duarte, CA.
Mercedes Gironella MesaHospital Universitario Vall d'Hebrón, Barcelona, Spain.
Miquel Granell GorrochateguiHospital de La Santa Creu i Sant Pau, Barcelona, Spain.
Dipti TalaulikarThe John Curtin School of Medical Research, Australian National University, Canberra, ACT, Australia.
Pier Luigi ZinzaniAzienda Ospedaliero-Universitaria di Bologna, Bologna, Italy.
Elham AskariHospital Universitario Fundación Jiménez Díaz, Madrid, Spain.
Sebastian GrosickiDepartment of Hematology and Cancer Prevention, Health Sciences Faculty, Medical University of Silesia, Katowice, Poland.
Albert OriolInstitut Català d'Oncologia-Hospital Universitari Germans Trias i Pujol, Barcelona, Spain.
Simon RulePlymouth Hospitals National Health Service (NHS) Trust, Derriford Hospital, Devon, United Kingdom.
Janusz KloczkoUniwersytecki Szpital Kliniczny w Bialymstoku, Podlaskie, Poland.
Alessandra TedeschiASST Grande Ospedale Metropolitano Niguarda, Milan, Italy.
Christian BuskeComprehensive Cancer Center (CCC) Ulm, Universitätsklinikum Ulm, Baden-Württemberg, Germany.
Veronique LeblondClinical Hematology Department, Sorbonne University, Pitié Salpêtrière Hospital, Paris, France.
Judith TrotmanConcord Repatriation General Hospital, Sydney, Concord, NSW, Australia.
Wai Y ChanBeiGene USA, Inc., San Mateo, CA.
Jan MichelBeiGene Switzerland GmbH, Basel, Switzerland.
Jingjing SchneiderBeiGene USA, Inc., San Mateo, CA.
Ziwen TanBeiGene (Beijing) Co., Ltd, Beijing, China.
Aileen CohenBeiGene USA, Inc., San Mateo, CA.
Jane HuangBeiGene USA, Inc., San Mateo, CA.
Constantine S TamPeter MacCallum Cancer Centre, Melbourne, VIC, Australia.
Australian National University · AUAzienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia · ITAzienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda · ITBeiGene (China) · CNCharles University · CZCity Of Hope National Medical Center · USComplejo Hospitalario de Salamanca · ESFlinders Medical Centre · AUHarvard University · USHospital de Sant Pau · ESHospital Universitario Fundación Jiménez Díaz · ESInstitut Català d'Oncologia · ESIstituti di Ricovero e Cura a Carattere Scientifico · ITIstituto di Ematologia di Bologna · ITMedical University of Silesia · PLMonash Health · AUNational and Kapodistrian University of Athens · GRNational Health Service · GBNicolaus Copernicus University · PLRoyal North Shore Hospital · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Patients with Waldenström macroglobulinemia (WM) lacking activating mutations in the MYD88 gene (MYD88WT) have demonstrated relatively poor outcomes to ibrutinib monotherapy, with no major responses reported in a phase 2 pivotal study. Zanubrutinib is a novel, selective Bruton tyrosine kinase (BTK) inhibitor designed to maximize BTK occupancy and minimize off-target activity. The ASPEN study consisted of a randomized comparison of zanubrutinib and ibrutinib efficacy and safety in patients with WM who have the MYD88 mutation, as well as a separate cohort of patients without MYD88 mutation (MYD88WT) or with unknown mutational status who received zanubrutinib. Results from the latter single-arm cohort are reported herein. Efficacy endpoints included overall, major and complete (CR) or very good partial response (VGPR) rates, progression-free survival (PFS), duration of response (DOR), and overall survival (OS). Twenty-eight patients (23 relapsed/refractory; 5 treatment-naïve) were enrolled, including 26 with centrally confirmed MYD88WT disease and 2 with unknown MYD88 mutational status. At a median follow-up of 17.9 months, 7 of 26 MYD88WT patients (27%) had achieved a VGPR and 50% a major response (partial response or better); there were no CRs. At 18 months, the estimated PFS and OS rates were 68% and 88%, respectively, while the median DOR had not been reached. Two patients discontinued zanubrutinib due to adverse events. Treatment-emergent hypertension, atrial fibrillation, and major hemorrhages were reported in 3, 1 and 2 patients (including 1 concurrent with enoxaparin therapy), respectively. Results of this substudy demonstrate that zanubrutinib monotherapy can induce high quality responses in patients with MYD88WT WM. This trial is registered on www.clinicaltrials.gov as NCT #03053440.

Indexed as

Myeloid Differentiation Factor 88Waldenstrom MacroglobulinemiaHumansPiperidinesPyrazolesPyrimidinesMyeloid Differentiation Factor 88PiperidinesPyrazolesPyrimidineszanubrutinib

Identifiers

PMID33284944
PMCPMC7724905
OpenAlexW3110781951

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.