Publications by authors named "Laurent Frenzel"

Inflammation in Waldenström Macroglobulinemia (iWM) predicts outcomes after immuno-chemotherapy and BTK inhibitors, but its origin is unknown. Here, we unravel increased clonal hematopoiesis in iWM patients (61% versus 23% in non-inflammatory WM), suggesting a contribution of environmental cells to iWM.

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  • Fidanacogene elaparvovec is a genetic therapy for hemophilia B that showed promising results in a phase 1-2a study, maintaining high levels of factor IX activity.* -
  • In a phase 3 study involving men aged 18 to 65 with severe hemophilia B, the therapy led to a significant 71% reduction in bleeding episodes over 15 months compared to standard prophylactic treatment.* -
  • Despite some participants needing glucocorticoids for side effects, the therapy was generally safe, with no serious adverse infusion events reported.*
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  • Advanced systemic mastocytosis (AdvSM) is a complex condition linked to poor outcomes, and while midostaurin is the first approved treatment, its long-lasting effectiveness is limited.
  • Various prognostic scoring systems like MARS, IPSM, and GPSM have been developed to assess patients' outcomes, but it's essential to tailor these scores to specific AdvSM subtypes for better accuracy.
  • A study of patients treated with midostaurin revealed that MARS and AdvSM subtype significantly predict overall survival, identifying five distinct patient subgroups with varying median survival times, highlighting the need for personalized management strategies.
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Background: Bidirectional interactions between eosinophils and mast cells (MCs) have been reported in various allergic diseases. Bone marrow (BM) eosinophilia, and to a lesser extent blood eosinophilia, is common in systemic mastocytosis (SM), but its significance remains unknown.

Objective: We described blood and BM eosinophil characteristics in SM.

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A second autologous stem-cell transplantation (ASCT2) is considered for relapsed multiple myeloma (RMM) patients showing prolonged response after a first ASCT. However, given breakthrough treatments like anti-CD38 and immunotherapy, its role remains debated. We conducted a real-life study in 10 French centers (1996-2017) involving 267 RMM patients receiving ASCT2.

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  • CD38-targeting immunotherapy combined with lenalidomide and dexamethasone is the current best standard of care for newly diagnosed multiple myeloma patients who can't undergo transplants.
  • A phase 3 study involving 270 patients tested the effectiveness of adding weekly bortezomib to this regimen, comparing the outcomes of the combination (Isa-VRd) against the standard (IsaRd).
  • Results showed a significantly higher rate of minimal residual disease negativity at 18 months and better response rates in the Isa-VRd group, suggesting it could become the new standard of care for these patients.
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Multiple myeloma is characterized by a huge heterogeneity at the molecular level. The RAS/RAF pathway is the most frequently mutated, in ∼50% of the patients. However, these mutations are frequently subclonal, suggesting a secondary event.

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Background: Emicizumab, a bispecific monoclonal antibody, bridges activated factor (F) IX and FX, mimicking the function of missing or deficient activated FVIII in people with hemophilia A (HA).

Objectives: To evaluate the long-term efficacy and safety of emicizumab prophylaxis in people with HA without FVIII inhibitors in the HAVEN 3 and 4 studies.

Methods: HAVEN 3 and 4 were phase 3 open-label studies.

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Background And Aims: Systemic mastocytosis (SM) is characterized by the accumulation of atypical mast cells (MCs) in organs. Liver histology of SM has been marginally described and accurate histological classification is critical, given the consequences of aggressive SM diagnosis. We aimed to describe the histological features associated with liver SM using updated tools.

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  • Bispecific antibodies (BsAbs) are used to treat relapsed or refractory multiple myeloma, but frequent infections were noted during clinical trials, indicating a need for further real-world data on infection risks and outcomes.
  • A multicenter retrospective study involving 229 patients in France from December 2020 to February 2023 found a high incidence of infections, with 234 recorded cases, primarily affecting the respiratory system, and a hospitalization rate of 56%.
  • The study identified corticosteroids used for managing cytokine release syndrome as a risk factor for infections, while using GPRC5D-targeting BsAbs and antibacterial prophylaxis was associated with a reduced risk.
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Background: Hemophilia A (HA) is predominantly associated with males due to X-linked inheritance. Males and females with HA have shared unmet medical needs, highlighting the necessity for comprehensive care irrespective of sex.

Objectives: This analysis investigated the efficacy and safety of emicizumab prophylaxis in 3 females with HA.

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  • Recent approvals of gene therapies for haemophilia A and B highlight the need for educational materials to help the community understand this new approach and make informed healthcare decisions.
  • The aim of the review is to provide an illustrated summary of AAV-based gene therapy, covering everything from fundamental concepts to real-world implementation.
  • The review includes eight sections that discuss the basics of haemophilia, treatment options, AAV principles, trial results, challenges, and practical application, ultimately aiding healthcare professionals in their understanding and patient education.
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Venous thromboembolism (VTE) remains a critical issue in the management of patients with multiple myeloma (MM), particularly when immunomodulatory drugs (IMiDs) combined with dexamethasone therapy are being prescribed as first-line and relapse therapy. One possible explanation for the persistent high rates of VTE, is the use of inappropriate thromboprophylaxis strategies for patients starting antimyeloma treatment. To tackle the issue, the Intergroupe francophone du myélome (IFM) offered convenient guidance for VTE thromboprophylaxis in MM patients initiating systemic therapy.

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Background: Mastocytosis and monoclonal mast cell (MC) activation syndrome (MMAS) are heterogeneous conditions characterized by the accumulation of atypical MCs. Despite the recurrent involvement of KIT mutations, the pathophysiologic origin of mastocytosis and MMAS is unclear. Although hereditary α-tryptasemia (HαT, related to TPSAB1 gene duplication) is abnormally frequent in these diseases, it is not known whether the association is coincidental or causal.

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