Macroglobulinemia

Waldenström, Jan

doi:10.1016/b978-1-4831-6750-3.50008-1

Cited by 32 publications

(7 citation statements)

References 52 publications

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“…Our analysis showed that despite the higher costs of ibrutinib pathway vs. CTP, the ICERs both in the Base Case and in DSA were under the threshold of €60,000/LY indicated as acceptable for Italy, except in the scenario with an increased price of +20% per ibrutinib and a 10-year time-horizon (Table 6). However, if we consider an equal value for LYGs and QALYs, due to a lack of evidence on quality of life data for Italian patients, with a WTP threshold for orphan drugs of £50,000/QALY (€68,885/QALY), 3 as reported by Drummond et al [32], the result is that in every scenario ibrutinib is cost-effective. Also the PSA confirmed the good pharmacoeconomic results of ibrutinib vs. CTP, with a probability of being cost-effective in 81% of the simulations at a WTP threshold of €60,000/LYG (Figure 2).…”

Section: Discussionmentioning

confidence: 97%

“…Waldenström Macroglobulinemia (WM) is a B-cell lymphoproliferative disorder characterized by high levels of immunoglobulin M (IgM [macroglobulin]) in peripheral blood and histological bone marrow with evidence of at least 10% lymphoplasmacytic, associated or not with lymphadenopathy and/or splenomegaly [1][2][3][4][5][6]. High levels of IgM may be responsible for hyperviscosity syndrome but are generally associated with typical fundoscopic findings [2].…”

Section: Introductionmentioning

confidence: 99%

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Cost-effectiveness analysis of ibrutinib in patients with Waldenström macroglobulinemia in Italy

Aiello

D’Ausilio

Muto

et al. 2017

Journal of Market Access & Health Policy

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Background and Objective: Ibrutinib has recently been approved in Europe for Waldenström Macroglobulinemia (WM) in symptomatic patients who have received at least one prior therapy, or in first-line treatment for patients unsuitable for chemo-immunotherapy. The aim of the study is to estimate the incremental cost-effectiveness ratio (ICER) of ibrutinib in relapse/refractory WM, compared with the Italian current therapeutic pathways (CTP). Methods: A Markov model was adapted for Italy considering the National Health System perspective. Input data from literature as well as global trials were used. The percentage use of therapies, and healthcare resources consumption were estimated according to expert panel advice. Drugs ex-factory prices and national tariffs were used for estimating costs. The model had a 15-year time horizon, with a 3.0% discount rate for both clinical and economic data. Deterministic and probabilistic sensitivity analyses were performed to test the results strength. Results: Ibrutinib resulted in increased Life Years Gained (LYGs) and increased costs compared to CTP, with an ICER of €52,698/LYG. Sensitivity analyses confirmed the results of the BaseCase. Specifically, in the probabilistic analysis, at a willingness to pay threshold of €60,000/LYG ibrutinib was cost-effective in 84% of simulations. Conclusions: Ibrutinib has demonstrated a positive cost-effectiveness profile in Italy.

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Section: Discussionmentioning

confidence: 97%

Section: Introductionmentioning

confidence: 99%

Cost-effectiveness analysis of ibrutinib in patients with Waldenström macroglobulinemia in Italy

Aiello

D’Ausilio

Muto

et al. 2017

Journal of Market Access & Health Policy

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“…Still an other group of diseases, described a long time before, now found a common origin: the ''malignant lymphomas'', that is multiple myeloma, macroglobulinaemia and Bence Jones proteinuria [179,187]. They all exhibit excessive production of abnormal proteins which are closely related to Igs, as shown with immunochemical methods [92].…”

Section: Aberrant Antibody Formation: Clinical Observationsmentioning

confidence: 99%

The discovery of agammaglobulinaemia in 1952

Hitzig¹

2003

Eur J Pediatr

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Fifty years ago a new disease, agammaglobulinaemia, was described. This was made possible by a great number of preceding technical innovations and theories on different fields of research, in particular haematology, microbiology/immunology and basic sciences. The widely used name ''Bruton disease'' credits one single man with a new observation which, however, was simultaneously made by several physicians. Agammaglobulinaemia was the first example of an immunodeficiency syndrome (IDS). Based on new facts, new ideas emerged which in turn gave rise to innovative research, concerning both clinical observations and basic problems. Many similar diseases, usually resulting from a genetic defect, were described. Since 1970, an International Committee appointed by the WHO, has, with periodic reassessments, been working on the classification of the syndromes. All participants of an efficient immune reaction can be deficient in individual patients, that is: antibodies, lymphocytes, phagocytes and complement. Basic scientists presented striking results concerning the structure and action of all elements mentioned above. Conclusion: mutual stimuli coming from observations in families and from gene technology have resulted in the elucidation of the genetic defects of most IDS. Recent results of genetic engineering seem to justify some hope for success in therapy. Keywords Agammaglobulinaemia AE Bruton disease AE ReviewAbbreviations APC antigen presenting cells AE CGD chronic granulomatous disease AE IDS immunodeficiency syndrome AE MHC major histocompatability antigens AE MIG monoclonal immunoglobulin AE SCID severe combined immunodeficiency AE TCR T-cell receptor AE XLA X-linked agammaglobulinaemia Bruton's description and his forerunnersFifty years ago the first patients with complete lack of gammaglobulin in the blood were described: Ogden Bruton was a paediatrician at the Walter Reed Army Hospital in Washington. His paper [26] published in 1952 is still worth reading because of its precise content and tight phrasing; he observed a boy who was admitted to hospital 19 times between the ages of 4 and 9 years for recurring infections. Nine times a pneumococcal bacteraemia was documented which promptly responded to antibiotic treatment. Antibodies against pneumococci, however, were never present! After futile wanderings through every conceivable test, Bruton heard rumours of a new piece of laboratory equipment called the ''Tiselius' moving boundary machine''. The result was surprising and unexpected for everybody: no gammaglobulin gradient was detectable; this had never been seen before. Later on Bruton [27] recalled: ''things began to click then: no gammaglobulins; can't build antibodies....This seems so simple now that it seems to me hardly worth repeating, but I thought, well, if he doesn't have any gammaglobulins, maybe we could try treating him with gammaglobulin'' . Pure gammaglobulin was at that time available. There was a spectacular improvement in the clinical picture after the first injection, and the electrophoresis ...

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“…Five to 10% of patients with macroglobulinemia develop peripheral neuropathy ( 40). Macroglobulinemia should always be considered in the differential diagnosis of elderly patients with polyneuropathy of unknown cause ( 41).…”

Section: Waldenström's Macroglobulinemiamentioning

confidence: 99%