Waldenstrom's Disease Clinical Trial
— SérieProWMOfficial title:
Prognostic Analyses on a Validation Series of Patients With Waldenström's Disease: Validation of International Prognostic Indexes, Evaluation of Progression-free Survival as a Surrogate Endpoint for Overall Survival
Verified date | August 2023 |
Source | French Innovative Leukemia Organisation |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational [Patient Registry] |
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.
Status | Recruiting |
Enrollment | 500 |
Est. completion date | June 15, 2030 |
Est. primary completion date | June 15, 2030 |
Accepts healthy volunteers | |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - 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 Exclusion Criteria: - 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. |
Country | Name | City | State |
---|---|---|---|
France | AMIENS - CH Amiens Picardie Site Sud | Amiens | |
France | Angers Chu | Angers | |
France | Institut Bergonie | Bordeaux | |
France | Clermont-Ferrand - Chu Estaing | Clermont-Ferrand | |
France | Le Mans CH | Le Mans | |
France | LENS - GHT Artois | Lens | |
France | LIBOURNE - Hôpital Robert Boulin | Libourne | |
France | LILLE GHICL - Hôpital Saint Vincent de Paul | Lille | |
France | Institut Paoli Calmette | Marseille | |
France | APHP - Hôpital Pitié Salpêtrière - Hématologie | Paris | |
France | POITIERS - Hématologie et Thérapie Cellulaire | Poitiers | |
France | Reims Chu | Reims | |
France | Strasbourg - Icans | Strasbourg | |
France | Toulouse - IUCT Oncopole - Service d'Hématologie | Toulouse | |
France | VERSAILLES - Hôpital André Mignot | Versailles |
Lead Sponsor | Collaborator |
---|---|
French Innovative Leukemia Organisation |
France,
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. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Overall survival | Percentage of living patients | 5 years after WM diagnosis | |
Secondary | Progression free survival | percentage of living patients without disease progression | 1 year after WM diagnosis | |
Secondary | Tolerance to treatment | percentage of patients discontinuing WM treatment due to toxicity | 1 year after initiating WM treatment |
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