Evidence map›Paper›PMID 39020041›Full record

Observational studyAnnals of hematology2024

Three years follow-up of Venetoclax in advanced-stage, relapsed or refractory AL amyloidosis with cardiac involvement and t(11;14) with BCL2 expression.

Max J Rieger, Thomas Pabst, Barbara Jeker, Pamella Paul, Fabio Bergamini, Marco M Bühler, Adalgisa Condoluci, Andreas J Flammer, Davide Rossi, Georg Stussi and 2 more

Abstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Annals of hematology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
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

3 citing papers in PubMed.

  1. Review
  2. Article
  3. Review
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

12 authors.

Max J RiegerDepartment of Medical Oncology and Hematology, University and University Hospital of Zurich, Rämistrasse 100, 8091, Zurich, Switzerland. max.rieger@usz.ch.ORCID http://orcid.org/0000-0002-8441-4547
Thomas PabstDepartment of Medical Oncology, Inselspital, Bern University Hospital, Bern, Switzerland.
Barbara JekerDepartment of Medical Oncology, Inselspital, Bern University Hospital, Bern, Switzerland.
Pamella PaulClinic of Hematology, Oncology Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona, Switzerland.
Fabio BergaminiClinic of Hematology, Oncology Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona, Switzerland.
Marco M BühlerDepartment of Pathology and Molecular Pathology, University Hospital and University of Zurich, Zurich, Switzerland.
Adalgisa CondoluciClinic of Hematology, Oncology Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona, Switzerland.
Andreas J FlammerUniversity Heart Center, University Hospital Zurich, Zurich, Switzerland.
Davide RossiClinic of Hematology, Oncology Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona, Switzerland.
Georg StussiClinic of Hematology, Oncology Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona, Switzerland.
Bernhard GerberClinic of Hematology, Oncology Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona, Switzerland.
Rahel SchwotzerDepartment of Medical Oncology and Hematology, University and University Hospital of Zurich, Rämistrasse 100, 8091, Zurich, Switzerland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Further line treatment of patients with advanced stage AL amyloidosis with cardiac involvement is challenging. Venetoclax is a promising option, especially in t(11;14) and BCL2 expression.In our multicentre observational study, we report the 3-year follow-up of Venetoclax treatment in 9 patients with advanced, relapsed or refractory AL amyloidosis with t(11;14) and BCL-2 expression in > 50% of plasma cells. At baseline, all patients had been previously treated with daratumumab, all had cardiac involvement with revised Mayo stage III or IV/ European modification of Mayo 2004 IIIA or IIIB (1/9 unclassified due to missing troponin T), 5/9 patients had renal involvement.After a median of 35 months (range 25-49) since the start of Venetoclax, 8/9 patients were still alive (OS 89%). First and best hematological responses were observed after a median of 26 days (11-125) and 106 days (35-659), overall response rate was 100% (7/9 CR, 2/9 VGPR). Where observed, organ response was documented within the first 6 months of therapy, including cardiac (6/9) and renal (3/5) improvements. Venetoclax was discontinued in 6/9 patients after a median of 15 months (11-48) due to toxicity (2/9), disease progression (2/9), fixed treatment duration (1/9), or safety concerns (1/9).In conclusion, Venetoclax induces a rapid and deep hematologic response with consistent improvement in organ function with an acceptable safety profile in patients with pretreated, advanced stage AL amyloidosis with cardiac involvement and BCL2 expression with and potentially without detected t(11:14), which warrants further investigation.

Indexed as

Bridged Bicyclo Compounds, HeterocyclicImmunoglobulin Light-chain AmyloidosisProto-Oncogene Proteins c-bcl-2SulfonamidesTranslocation, GeneticAgedAged, 80 and overAntineoplastic AgentsChromosomes, Human, Pair 11Chromosomes, Human, Pair 14FemaleFollow-Up StudiesHumansMaleMiddle AgedRecurrenceAntineoplastic AgentsBCL2 protein, humanBridged Bicyclo Compounds, HeterocyclicProto-Oncogene Proteins c-bcl-2SulfonamidesvenetoclaxAL AmyloidosisBCL-2Revised Mayo Stage III/IVt(11;14)Venetoclax

Identifiers

PMID39020041
PMCPMC11512835

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Registered trials

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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.