Cost utility and budget impact analysis of dexamethasone compared with bortezomib and lenalidomide for the treatment of second line multiple myeloma from a South African public health perspective

Matsela, Lineo; Cleary, Susan; Wilkinson, Thomas

doi:10.1186/s12962-022-00399-4

Cited by 2 publications

(1 citation statement)

References 32 publications

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“…New therapies pioneered during the past two decades have improved both progression-free survival (PFS) and overall survival (OS) for MM patients [ 2 , 22 ]. Although there has been an improvement in survival, contemporary outcomes for unselected MM patients have been difficult to determine precisely, and many of these newer therapies are costly and not readily available for patients in the public health sector in South Africa [ 23 ].…”

Section: Introductionmentioning

confidence: 99%

Profile and outcome of multiple myeloma with and without HIV treated at a tertiary hospital in KwaZulu-Natal, South Africa

Chili,

Mackraj,

Rapiti

2023

PLoS ONE

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Objectives To profile the outcome of multiple myeloma (MM) patients treated at a South African tertiary hospital in KwaZulu-Natal (KZN) and to compare MM in HIV-negative patients and MM in people living with HIV (PLWH). Methods A retrospective analysis of patients with MM was conducted over 5 years (2015–2020). Patient demographics, presenting complaints, symptom duration, disease stage, molecular profile, treatment, and survival data were captured. Statistical analysis was conducted using R Statistical software of the R Core Team, 2020, version 3.6.3. Results 135 patients; 79% (n = 106) HIV-negative and 21% (n = 29) PLWH were investigated. 54% (n = 74) females and 57% (n = 76) 51–70-year-olds. The 40-50-year-old patient group had a significantly higher proportion of PLWH (p = 0.032). Pathological fractures were the commonest presenting complaint, 47% (n = 57 and 49% (n = 49) had International Staging System, stage III disease. Fluorescent in-situ hybridization (FISH) MM profiling was completed in 58% (n = 78). Positivity for del 11q22 was found in 23.7% (n = 14) with significantly more HIV-negative patients having the mutation (p = 0.027). Overall, 42.2% (n = 57) achieved 2-year overall survival (OS). There were no significant differences in treatment (p = 0.926) and 2-year survival outcome (p = 0.792) between the two groups. Conclusion The incidence of HIV in newly diagnosed MM patients in KZN was increasing. KZN patient profile differed from other reports by showing female predominance but was similar in advanced-stage presentation and bone fracture predominance. Statistically significant differences between the HIV-negative patients and PLWH were observed in age distribution and mutational landscape. Further studies are required in this area.

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Section: Introductionmentioning

confidence: 99%

Profile and outcome of multiple myeloma with and without HIV treated at a tertiary hospital in KwaZulu-Natal, South Africa

Chili,

Mackraj,

Rapiti

2023

PLoS ONE

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A Systematic Review of Modeling Approaches to Evaluate Treatments for Relapsed Refractory Multiple Myeloma: Critical Review and Considerations for Future Health Economic Models

Freitag,

Sarri,

et al. 2024

PharmacoEconomics

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Background and Objective Multiple myeloma is a rare incurable hematological cancer in which most patients relapse or become refractory to treatment. This systematic literature review aimed to critically review the existing economic models used in economic evaluations of systemic treatments for relapsed/refractory multiple myeloma and to summarize how the models addressed differences in the line of therapy and exposure to prior treatment. Methods Following a pre-approved protocol, literature searches were conducted on 17 February, 2023, in relevant databases for models published since 2014. Additionally, key health technology assessment agency websites were manually searched for models published as part of submission dossiers since 2018. Reported information related to model conceptualization, structure, uncertainty, validation, and transparency were extracted into a pre-defined extraction sheet. Results In total, 49 models assessing a wide range of interventions across multiple lines of therapy were included. Only five models specific to heavily pre-treated patients and/or those who were refractory to multiple treatment classes were identified. Most models followed a conventional simple methodology, such as partitioned survival ( n = 28) or Markov models ( n = 9). All included models evaluated specific interventions rather than the whole treatment sequence. Where subsequent therapies were included in the model, these were generally only considered from a cost and resource use perspective. The models generally used overall and progression-free survival as model inputs, although data were often immature. Sensitivity analyses were frequently reported ( n = 41) whereas validation was only considered in less than half ( n = 19) of the models. Conclusions Published economic models in relapsed/refractory multiple myeloma rarely followed an individual patient approach, mainly owing to the higher need for complex data assumptions compared with simpler modeling approaches. As many patients experience disease progression on multiple treatment lines, there is a growing need for modeling complex treatment strategies, leading to more sophisticated approaches in the future. Maintaining transparency, high reporting standards, and thorough analyses of uncertainty are crucial to support these advancements. Supplementary Information The online version contains supplementary material available at 10.1007/s40273-024-01399-3.

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Cost utility and budget impact analysis of dexamethasone compared with bortezomib and lenalidomide for the treatment of second line multiple myeloma from a South African public health perspective

Cited by 2 publications

References 32 publications

Profile and outcome of multiple myeloma with and without HIV treated at a tertiary hospital in KwaZulu-Natal, South Africa

Profile and outcome of multiple myeloma with and without HIV treated at a tertiary hospital in KwaZulu-Natal, South Africa

A Systematic Review of Modeling Approaches to Evaluate Treatments for Relapsed Refractory Multiple Myeloma: Critical Review and Considerations for Future Health Economic Models

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