SOHO State of the Art Updates and Next Questions: Targeted therapies and emerging novel treatment approaches for Waldenström Macroglobulinemia

Sermer, David; Sarosiek, Shayna; Branagan, Andrew R.; Treon, Steven P.; Castillo, Jorge J.

doi:10.1016/j.clml.2022.02.005

Cited by 6 publications

(3 citation statements)

References 65 publications

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“…Tirabrutinib is an oral second-generation BTK inhibitor with higher BTK selectivity and fewer off-target effects compared to ibrutinib [ 3 ]. Tirabrutinib was approved in Japan in August 2020 for the treatment of WM and LPL, subsequent to approval for treatment of relapsed/refractory primary central nervous system lymphoma (PCNSL) [ 4 ]. A Japanese phase 2 trial administering 480mg/day of tirabrutinib to 27 Waldenström macroglobulinemia (WM) patients reported a major response rate of 93%, 2-year PFS rate of 92.6%, and 2-year OS of 100% [ 3 ].…”

Section: Discussionmentioning

confidence: 99%

Anti-myelin-associated-glycoprotein neuropathy successfully treated with tirabrutinib

Yasuda

Tomizawa

Harada

et al. 2022

Heliyon

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Section: Discussionmentioning

confidence: 99%

Anti-myelin-associated-glycoprotein neuropathy successfully treated with tirabrutinib

Yasuda

Tomizawa

Harada

et al. 2022

Heliyon

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“…Intra-tumor heterogeneity is of particular interest in WM as it increases the difficulty of defining targets sufficiently broadly expressed on tumor cells ( 364 ). Especially young patients, who might tolerate the potential side effects of a CAR-T-cell therapy better, could profit from the curative therapeutic approach ( 382 ).…”

Section: Discussionmentioning

confidence: 99%

Broadening the horizon: potential applications of CAR-T cells beyond current indications

Karsten,

Matrisch,

Cichutek

et al. 2023

Front. Immunol.

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Engineering immune cells to treat hematological malignancies has been a major focus of research since the first resounding successes of CAR-T-cell therapies in B-ALL. Several diseases can now be treated in highly therapy-refractory or relapsed conditions. Currently, a number of CD19- or BCMA-specific CAR-T-cell therapies are approved for acute lymphoblastic leukemia (ALL), diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), multiple myeloma (MM), and follicular lymphoma (FL). The implementation of these therapies has significantly improved patient outcome and survival even in cases with previously very poor prognosis. In this comprehensive review, we present the current state of research, recent innovations, and the applications of CAR-T-cell therapy in a selected group of hematologic malignancies. We focus on B- and T-cell malignancies, including the entities of cutaneous and peripheral T-cell lymphoma (T-ALL, PTCL, CTCL), acute myeloid leukemia (AML), chronic myeloid leukemia (CML), chronic lymphocytic leukemia (CLL), classical Hodgkin-Lymphoma (HL), Burkitt-Lymphoma (BL), hairy cell leukemia (HCL), and Waldenström’s macroglobulinemia (WM). While these diseases are highly heterogenous, we highlight several similarly used approaches (combination with established therapeutics, target depletion on healthy cells), targets used in multiple diseases (CD30, CD38, TRBC1/2), and unique features that require individualized approaches. Furthermore, we focus on current limitations of CAR-T-cell therapy in individual diseases and entities such as immunocompromising tumor microenvironment (TME), risk of on-target-off-tumor effects, and differences in the occurrence of adverse events. Finally, we present an outlook into novel innovations in CAR-T-cell engineering like the use of artificial intelligence and the future role of CAR-T cells in therapy regimens in everyday clinical practice.

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“…Several other therapies are currently being investigated in clinical trials for WM, some of which have reported encouraging results [ 76 ]. Venetoclax, a first-in-class, selective B-cell lymphoma-2 (BCL-2) inhibitor demonstrated a major response rate of 84% and median PFS of 30 months in a phase 2 study of 32 patients with previously treated WM (16 of which received prior BTK inhibitor therapy) [ 77 ].…”

Section: Treatment Options For Waldenström Macroglobulinemiamentioning

confidence: 99%

A Canadian Perspective on the Treatment of Waldenström Macroglobulinemia

et al. 2022

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Waldenström macroglobulinemia (WM) is a slowly progressing B-cell non-Hodgkin lymphoma characterized by monoclonal IgM gammopathy in the blood and infiltration of the bone marrow by clonal lymphoplasmacytic cells. As an incurable disease, the goals for therapy for WM are to relieve symptoms, slow disease progression, prevent organ damage, and maintain quality of life. However, given the rarity of WM, clinical trials comparing treatments for WM are limited and there is no definitive standard of care. The selection of first-line WM therapy is thus based on patient factors, disease characteristics, and drug access, with bendamustine-rituximab and Bruton’s tyrosine kinase (BTK) inhibitor therapy considered preferred treatments. Other treatments such as proteasome inhibitor- or purine analogue-based therapy, alternative chemoimmunotherapy, and autologous stem cell transplantation are generally reserved for the relapsed setting but may be used in rare circumstances in earlier lines of therapy. This paper summarizes the efficacy and safety of these WM therapies and discusses considerations for treatment from a Canadian perspective.

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SOHO State of the Art Updates and Next Questions: Targeted therapies and emerging novel treatment approaches for Waldenström Macroglobulinemia

Cited by 6 publications

References 65 publications

Anti-myelin-associated-glycoprotein neuropathy successfully treated with tirabrutinib

Anti-myelin-associated-glycoprotein neuropathy successfully treated with tirabrutinib

Broadening the horizon: potential applications of CAR-T cells beyond current indications

A Canadian Perspective on the Treatment of Waldenström Macroglobulinemia

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