Long survival in patients with Waldenström macroglobulinaemia diagnosed at a young age

Babwah, Amaara; Gustine, Joshua; Meid, Kirsten; Dubeau, Toni; Liu, Xu; Yang, Guang; Hunter, Zachary; Treon, Steven P.; Castillo, Jorge J.

doi:10.1111/bjh.15634

Cited by 4 publications

(5 citation statements)

References 10 publications

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“…Historically seen as a condition of older Caucasian males, the RMR has highlighted a significant proportion of younger patients (37% aged ≤60; 13% aged ≤50). This demographic heterogeneity impacts on treatment choices and highlights the need for personalised management plans 13 …”

Section: Discussionmentioning

confidence: 99%

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The Rory Morrison WMUK Registry for Waldenström macroglobulinaemia: The growth of a national registry for a rare disorder

et al. 2023

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Summary National registries are used globally to characterise patient demographics, treatment choices and mortality to inform and improve clinical management. Waldenström macroglobulinaemia (WM) is a rare, treatment‐responsive B‐cell lymphoproliferative disorder with diverse clinical features and variable outcomes. To prospectively chart changes in the management of WM in the UK, the Rory Morrison Registry (RMR) was developed to systematically collect real‐world data. Here we describe the development of the RMR, demonstrate its feasibility and describe preliminary observations. The RMR was devised as a collaborative project between patients and clinicians, under the auspices of the UK Charity for WM in 2016. Patients may be registered after the point of diagnosis and those with historic diagnosis were also eligible. Data collection fields were compiled by focus groups of clinicians, patients, industry and commissioning partners. The RMR launched in November 2017 and as of March 2022, there were 22 participating centres and 1305 patients registered. Median follow‐up was 6.4 years, five‐year overall survival 90.7% (95% confidence interval [CI] 88.4%–92.5%) and 10‐year overall survival 79.3% (95% CI 75.7%–82.4%). There has been a clear evolution in treatments including a rapid growth in the use of Bruton's tyrosine kinase inhibitors in relapsed disease since their availability in the UK.

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Section: Discussionmentioning

confidence: 99%

“…This demographic heterogeneity impacts on treatment choices and highlights the need for personalised management plans. 13 The RMR has shown a clear evolution in treatments, both in first line and after first relapse. At present the combinations of DRC and BR appear to be the preferred choices at front-line in the UK.…”

Section: Wm Landscapementioning

confidence: 99%

The Rory Morrison WMUK Registry for Waldenström macroglobulinaemia: The growth of a national registry for a rare disorder

et al. 2023

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“…Age is the most powerful predictor of outcome. The 10 year survival of patients age 45 years or younger is 86% 63 …”

Section: Risk Stratificationmentioning

confidence: 99%

“…57 Lactate dehydrogenase was absent from the International Prognostic Scoring System for Waldenström Macroglobulinemia. 58 In a revision of the prognostic scoring system age (≤65 vs 66-75 vs ≥76 years), β2-microglobulin ≥4 mg/L, serum albumin <3.5 g/dL, and LDH ≥250 IU/L (ULN < 225) was able to stratify patients in five different prognostic groups and identify a very-low risk as well as a very-high risk group with a 3-year WM-related death rate of 0%, 10%, 14%, 38%, and 48% (P < 0.001) and 10-year survival rate of 84%, 59%, 37%, 19%, and 9% (P < 0.001). 59 So, LDH is a predictor of early mortality in this disease.…”

Section: Diagnosismentioning

confidence: 99%

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Waldenström macroglobulinemia: 2021 update on diagnosis, risk stratification, and management

Gertz

2021

American J Hematol

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Disease Overview: Waldenström macroglobulinemia (WM) is a lymphoplasmacytic lymphoma with immunoglobulin M (IgM) monoclonal protein. Clinical features include anemia, thrombocytopenia, hepatosplenomegaly, lymphadenopathy, and rarely hyperviscosity. Diagnosis: Presence of IgM monoclonal protein associated with ≥10% clonal lymphoplasmacytic cells in bone marrow confirms the diagnosis. The L265P mutation in MYD88 is detectable in more than 90% of patients and is found in the majority of IgM MGUS patients. Risk Stratification: Age, hemoglobin level, platelet count, β 2 microglobulin, LDH and monoclonal IgM concentrations are characteristics that are predictive of outcomes. Risk-Adapted Therapy: Not all patients who fulfill WM criteria require therapy; these patients can be observed until symptoms develop. Rituximab-monotherapy is inferior to regimens that combine it with bendamustine, an alkylating agent, a proteosome inhibitor, or ibrutinib. Purine nucleoside analogues are active but usage is declining in favor of less toxic alternatives. The preferred Mayo Clinic induction is rituximab and bendamustine. Management of Refractory Disease: Bortezomib, fludarabine, thalidomide, everolimus, Bruton Tyrosine Kinase inhibitors, carfilzomib, lenalidomide, and bendamustine have all been shown to have activity in relapsed WM. Given WM's natural history, reduction of therapy toxicity is an important part of treatment selection. PATIENT A 55-year-old male was found to have an IgM monoclonal gammopathy in January 2001. He was observed until November 2006 when his IgM level climbed to 9135 mg/dL. He was treated with rituximab, bortezomib and dexamethasone for 4 months. Treatment was interrupted due to neuropathy, but the IgM level fell to 1150 mg/dL. He was observed until April 2011. At the time of progression his IgM was 11 500 mg/dL and he was treated with single agent rituximab. At that time he was found to have acquired von Willebrand disease. Rituximab failed to produce a minor response so he was placed on a trial of bendamustine, lenalidomide and rituximab. His response lasted less than 1 year. In 2014 he was placed on an experimental trial of oprozomib. This resulted in a response of 5 years duration. At relapse in August of 2019 he was placed on a trial of ixazomib and ibrutinib. Ixazomib was poorly tolerated due to diarrhea and he continued on ibrutinib alone. He has now been on ibrutinib 15 months and has achieved a very good partial response.

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Complete Response of a Young Woman With MYD88WT Bing-Neel Syndrome on Ibrutinib Monotherapy Following a Single Cycle of B Hyper-CVAD/IT MTX

Pabon

Neff

Forns

et al. 2020

Clinical Lymphoma Myeloma and Leukemia

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Long survival in patients with Waldenström macroglobulinaemia diagnosed at a young age

Cited by 4 publications

References 10 publications

The Rory Morrison WMUK Registry for Waldenström macroglobulinaemia: The growth of a national registry for a rare disorder

The Rory Morrison WMUK Registry for Waldenström macroglobulinaemia: The growth of a national registry for a rare disorder

Waldenström macroglobulinemia: 2021 update on diagnosis, risk stratification, and management

Complete Response of a Young Woman With MYD88WT Bing-Neel Syndrome on Ibrutinib Monotherapy Following a Single Cycle of B Hyper-CVAD/IT MTX

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