Prognostic Analyses on a Validation Series of Patients With Waldenström's Disease

NCT05911802 | Recruiting DMC

• 1 other identifier: FILObs_SérieProWM
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 15

Brief Summary

Waldenström's macroglobulinemia (WM) is defined by the association of bone marrow lymphoplasmocytic infiltration and monoclonal immunoglobulin M (IgM). A mutation in the MYD88 gene is found in up to 90% of patients, and a mutation in the CXCR4 gene in approximately one third of patients. Treatment should be initiated in cases of cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM). However, approximately 30% of patients are diagnosed without any symptom and therefore they do not meet the criteria for initiating treatment. At the time of initiation of the first treatment, the prognosis is usually estimated with the International Prognostic Index (IPSSWM) which is based on five variables: age, platelet count, haemoglobin concentrations, β2-microglobulin and monoclonal component concentration. Serum albumin and lactate dehydrogénase (LDH) levels also retain a prognostic role and these two characteristics have been incorporated in a proposal for a revision of this index. Improving prognostic assessment at the time of the first treatment initiation and taking into account the prognostic impact of events occurring in the course of evolution, should improve the strength of treatment decision at the time of initial treatment and during the follow-up. It should also help to design clinical trial for fast and effective evaluation of new treatments. Our work should also help to adjust clinical monitoring of asymptomatic patients. Prospective and retrospective multicenter prognostic study with a descriptive objective, associated with a biological collection appropriately annotated and stored. A retrospective series including 470 patients with symptomatic WM is already available. The follow-up of these patients will be updated and an additional series of 250 symptomatic patients will be prospectively enrolled. 250 asymptomatic patients will be also enrolled.

Conditions

Waldenstrom's Disease; Prognostic Index

Outcome Measures

Primary Outcomes (1)

• Overall survival

  Percentage of living patients

  5 years after WM diagnosis

Secondary Outcomes (2)

• Progression free survival

  1 year after WM diagnosis

• Tolerance to treatment

  1 year after initiating WM treatment

Study Arms (2)

symptomatic WM

WM patients with symptom(s) : cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM)

asymptomatic WM

WM patients without any symptom

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

250 symptomatic patients and 250 asymptomatic patients will be prospectively enrolled

You may qualify if:

• Patient with WM, fulfilling the diagnostic criteria defined at the 2nd Workshop on WM.
• Patient in whom follow-up is available until at least 01/01/2020. Each participating center should not enroll more 10% of patients lost to follow-up.
• Patient for whom a minimum annual follow-up is planned until 2024.
• Having given their consent for this study

You may not qualify if:

• Patient with other chronic lymphoid malignancy. Special attention will be paid to exclude other lymphoplasmacytic proliferations, especially marginal zone lymphoma.
• Patient with histological transformation in a diffuse large B-cell lymphoma or any other lymphoma at the time of the initiation of the 1st treatment.
• No consent for this study.

Sponsors & Collaborators

• French Innovative Leukemia Organisation: lead

Study Sites (15)

AMIENS - CH Amiens Picardie Site Sud

Amiens, 80054, France

NOT YET RECRUITING

Angers Chu

Angers, 49933, France

NOT YET RECRUITING

Institut Bergonie

Bordeaux, 33076, France

NOT YET RECRUITING

Clermont-Ferrand - Chu Estaing

Clermont-Ferrand, 63000, France

NOT YET RECRUITING

Le Mans CH

Le Mans, France

NOT YET RECRUITING

LENS - GHT Artois

Lens, 62300, France

NOT YET RECRUITING

LIBOURNE - Hôpital Robert Boulin

Libourne, 33505, France

NOT YET RECRUITING

LILLE GHICL - Hôpital Saint Vincent de Paul

Lille, 59000, France

RECRUITING

Institut Paoli Calmette

Marseille, 130009, France

NOT YET RECRUITING

APHP - Hôpital Pitié Salpêtrière - Hématologie

Paris, 75651, France

NOT YET RECRUITING

POITIERS - Hématologie et Thérapie Cellulaire

Poitiers, 86021, France

NOT YET RECRUITING

Reims Chu

Reims, 51092, France

NOT YET RECRUITING

Strasbourg - Icans

Strasbourg, 67033, France

NOT YET RECRUITING

Toulouse - IUCT Oncopole - Service d'Hématologie

Toulouse, 31059, France

NOT YET RECRUITING

VERSAILLES - Hôpital André Mignot

Versailles, France

NOT YET RECRUITING

Related Publications (1)

• Royston P, Altman DG. External validation of a Cox prognostic model: principles and methods. BMC Med Res Methodol. 2013 Mar 6;13:33. doi: 10.1186/1471-2288-13-33.

  PMID: 23496923 BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

bone marrow samples for analysis like genetic analysis (MYD88 (L265P) and CXCR4 mutational status)

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 12, 2023
• First Posted: June 22, 2023
• Study Start: August 11, 2023
• Primary Completion (Estimated): June 15, 2030
• Study Completion (Estimated): June 15, 2030
• Last Updated: November 26, 2025

Record last verified: 2025-11

Locations

FR (15)