The REFRACT-LYMA cohort study: a French observational prospective cohort study of patients with mantle cell lymphoma

International audienceAbstractBackgroundMantle Cell Lymphoma (MCL) is often associated with progression, temporary response to therapy and a high relapse rate over time resulting in a poor long-term prognosis. Because MCL is classified as an incurable disease, therapeutic resistance is of great interest. However, knowledge about the biological mechanisms underlying resistance associated with MCL therapies and about associated predictors remains poor. The REFRACT-LYMA Cohort, a multicenter prospective cohort of patients with MCL, is set up to address this limitation. We here describe the study background, design and methods used for this cohort.Methods/DesignThe REFRACT-LYMA Cohort Study aims at including all patients (>18 years old) who are diagnosed with MCL in any stage of the disease and treated in specialized oncology centers in three public hospitals in Northwestern France. Any such patient providing a signed informed consent is included. All subjects are followed up indefinitely, until refusal to participate in the study, emigration or death. The REFRACT-LYMA follow-up is continuous and collects data on socio-economic status, medical status, MCL therapies and associated events (resistance, side effects). Participants also complete standardized quality of life (QOL) questionnaires. In addition, participants are asked to donate blood samples that will support ex vivo analysis of expression and functional assays required to uncover predictive biomarkers and companion diagnostics. If diagnostic biopsies are performed during the course of the disease, extracted biological samples are kept in a dedicated biobank.DiscussionTo our knowledge, the REFRACT-LYMA Cohort Study is the first prospective cohort of patients with MCL for whom “real-life” medical, epidemiological and QOL data is repeatedly collected together with biological samples during the course of the disease. The integrative cohort at mid-term will be unique at producing a large variety of data that can be used to conceive the most effective personalized therapy for MCL patients. Additionally, the REFRACT-LYMA Cohort puts the medical care of MCL patients in a health and pharmacoeconomic perspective

Long-term effectiveness and safety of interleukin-1 receptor antagonist (anakinra) in Schnitzler's syndrome: A French multicenter study.

Conflicts of interest: DL received a grant from Sobi (< 10,000 USD). EH received lecture fees from Sobi (< 10,000 USD). The other authors have no conflict of interest to disclose. Ethics approval: According to French legislation regarding retrospective observational studies using anonymous data, Ethics Committee approval was not required, which was confirmed by the chair of our Institutional Ethics Committee. The study was performed in accordance with the Declaration of Helsinki's guidelines for research in humans.International audienceThe aim of this study is to assess the long-term effectiveness and safety of IL1Ra in Schnitzler syndrome (SchS). Between 2010 and 2012, we performed a nationwide survey among French internal medicine departments to identify SchS patients. We retrospectively analyzed the long-term efficacy and safety of IL1Ra and the outcome of patients that did not receive this treatment. Forty-two patients were included in the study, 29 of whom received IL1Ra. The mean age at disease onset was 59.9years. Disease manifestations included urticaria (100%), fever (76%), bone/joint pain (86%), bone lesions (76%), anemia (67%), and weight loss (60%). The monoclonal gammopathy was overwhelmingly IgM kappa (83%). The mean follow-up was 9.5years (range: 1.6-35). Two patients developed Waldenström's macroglobulinemia and one developed AA amyloidosis. All of the 29 patients who received IL1Ra responded dramatically. After a median follow-up of 36months (range: 2-79), the effectiveness remained unchanged. All patients remained on anti-IL-1 therapy. Twenty-four patients (83%) went into complete remission and five (17%) into partial remission. Three patients experienced grade 3-4 neutropenia. Six patients developed severe infections. No lymphoproliferative diseases occurred while on IL1Ra. When last seen, all patients without anakinra had an active disease with variable impact on their quality of life. Their median corticosteroids dosage was 6mg/d (range: 5-25). IL1Ra is effective in SchS, with a sharp corticosteroid-sparing effect. Treatment failures should lead to reconsider the diagnosis. Long-term follow-up revealed no loss of effectiveness and a favorable tolerance profile. The long-term effects on the risk of hemopathy remain unknown

Consolidation anti-CD22 fractionated radioimmunotherapy with 90 Y-epratuzumab tetraxetan following R-CHOP in elderly patients with diffuse large B-cell lymphoma: a prospective, single group, phase 2 trial

International audienceRadioimmunotherapy represents a potential option as consolidation after chemoimmunotherapy in patients with diffuse large B-cell lymphoma who are not candidates for transplantation. We aimed to assess activity and toxicity of fractionated radioimmunotherapy using anti-CD22 90Y-epratuzumab tetraxetan as consolidation after front-line induction chemoimmunotherapy in untreated elderly patients with diffuse large B-cell lymphoma

Evaluation of Residual Disease and TKI Duration Are Critical Predictive Factors for Molecular Recurrence after Stopping Imatinib First-line in Chronic Phase CML Patients

International audienc