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Survival, health care resource utilization and expenditures of first-line treatments for multiple myeloma patients ineligible for transplant in Taiwan.

BackgroundWe aimed to provide real-world information on survival, health care resource utilization (HCRU), and expenditures related to various first lines of therapy (1LOTs) in newly diagnosed multiple myeloma (NDMM) patients who were transplant ineligible (TI).Patients and methodsFrom the Taiwan National Health Insurance Database (2008-2016), we identified 1,511 NDMM-TI patients who had received 1LOT since June 2012. We categorized 1LOT regimens into four groups: bortezomib (V)+thalidomide (T), V, T, and non-V/T. Patients' characteristics were collected. The overall survival (OS), event-free

Treatment Satisfaction and Burden of Illness in Patients with Newly Diagnosed Multiple Myeloma.

ObjectivesThis observational study identified attributes of patient-reported satisfaction with therapy for multiple myeloma (MM), described the treatment-related time burden and indirect costs, and investigated the effect of administration route (oral vs. injectable) on these outcomes among patients with newly diagnosed MM (NDMM) and among caregivers.MethodsPatients residing in the USA with a self-reported diagnosis of NDMM were recruited from PatientsLikeMe, MyelomaCrowd, and Facebook (16 December 2016 and 6 July 2017) to complete an electronic survey including questions on treatment experien

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… Options for therapy include combinations of proteasome inhibitors (bortezomib, carfilzomib, ixazomib), immunomodulatory drugs (lenalidomide, thalidomide, pomalidomide), monoclonal antibodies (daratumumab, elotuzumab), alkylating agents, and steroids combined as doublets and triplets [ 4 ]. … Triplet combinations in relapsed/refractory myeloma: update on recent phase 3 trials. Expert Rev Hematol. 2017;10(3):207–215. doi: 10.1080/17474086.2017.1285694. …

How I manage frontline transplant-ineligible multiple myeloma.

The Multiple Myeloma (MM) is a plasma cells hematological malignancy with a median age of 69 years at diagnosis. The autologous stem cell transplantation is the standard of care for this disease but less than half of newly diagnosed patients are assessed for this treatment due to comorbidities or complications of disease. The management of transplant ineligible MM patients is based on the balance safety and efficacy of the new available regimen and a careful assessment of the frailty status is mandatory to define the goals. In this review we discuss of the clinical dilemmas in the management a

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… Another association of new drugs with MP is the triplet bortezomib – melphalan – prednisone (VMP). … daratumumab are not available According to the regimens in use, the frailty stratification and the personalized goal of therapy, the following treatment selection could be proposed ( Figure 1 ): fit patients should achieve a complete response and MRD negativity with an increased PFS and OS by full dose triplets …

Известная эволюция полимер-связывающего пептида LCI для улучшения связывания полипропилена.

Функционализация поверхностей полимеров с помощью полимерных связывающих пептидов предоставляет значительные возможности для направленногоImmobilization ферментов, биоактивных пептидов и антигенов. Применение полимерных связывающих пептидов в качестве адгезивов требует надежного и стабильного связывания при условиях процесса. Молекулярные режимы взаимодействия между материалами поверхностей, пептидами и растворителем часто не понимаются на уровне, который позволяет (semi-) рационально проектировать полимерные связывающие пептиды, что мешает полному использованию их потенциала. Концепция направ

Isatuximab, bortezomib, lenalidomide и dexamethason for multieloma: динамика негатива MRD в исследовании IMROZ.

Квадруплетная терапия с Isa-VRd (изатуксимаб, велкад [бортезомиб], релимид [леналидомид] и дексаметасон), за которой последовала Иса-Рд в ходе случайного этапа 3 исследования IMROZ, дала значительную возможность выжить без прогрессирования пациентам, имеющим право на трансплантацию с недавно диагностированным множественным миэломой (NDMM). В ходе первичного анализа большее число пациентов, прошедших лечение Isa-VRd/Isa-Rd, достигли минимальной остаточной негативности (MRD) и полной реакции (MRD-отрицательной реакции) в любой момент времени, чем пациенты, получающие VRd, за которыми следует Rd.

Supporting treatment decision-making for patients with multiple myeloma post-DRd in Italy: a multi-criteria decision framework.

BACKGROUND: A substantial unmet medical need exists for patients with Multiple Myeloma ineligible for autologous stem cell transplantation who relapse after first-line therapy with daratumumab, lenalidomide and dexamethasone (DRd). Three therapeutic options recommended for lenalidomide-refractory patients from EHA-ESMO guidelines are approved in Europe and reimbursed in Italy: carfilzomib and dexamethasone (Kd); pomalidomide, bortezomib and dexamethasone (PVd); selinexor, bortezomib and dexamethasone (SVd). This study aimed to identify key decision criteria and their relevance for assessing th

Maintenance therapy for cytogenetically high-risk multiple myeloma: landscape in the era of novel drugs.

Although the significant strides in novel therapeutic approaches have prolonged the survival of multiple myeloma (MM) patients, the unfavorable prognosis of cytogenetically high-risk newly diagnosed MM (NDMM) remains intractable with the lack of consensus regarding the choice of maintenance regimens. Therefore, this study was initiated with the aim of examining the effectiveness of various maintenance treatments for this group of patients in jeopardy. Overall, 17 studies with 1937 high-risk NDMM patients were included in the network meta-analysis. Combination therapies involving novel drugs pr

Waldenström macroglobulinemia: biology, genetics, and therapy.

Waldenström macroglobulinemia (WM) is a distinct clinicopathologic entity characterized by the presence of a lymphoplasmacytic lymphoma, a non-Hodgkin lymphoma, and IgM monoclonal gammopathy. WM is an indolent, uncommon malignancy mostly affecting the elderly. Patient outcomes have modestly improved since the introduction of rituximab to conventional cytotoxic chemotherapy more than 20 years ago. However, the pivotal discovery of the somatic MYD88 L265P mutation, harbored by most patients with WM, and the somatic CXCR4 WHIM mutations, similar to germline CXCR4 mutations seen in the warts, hypo

Intra-apheresis Cycling to Improve the Clinical Efficacy of Peripheral Blood Stem Cell Donations.

Peripheral blood stem cell (PBSC) donation is the primary procedure used to collect haemopoietic stem cells (HSCs) for transplantation in individuals with haematological malignancies. More than 90,000 HSC transplants take place globally each year, and there is an increasing need to guarantee HSC mobilisation, improve tolerability to apheresis, and optimise immune reconstitution. Currently, mobilisation of HSCs depends upon pharmacological agents, with donors inactive during their subsequent apheresis. A successful yield of HSCs is not always achieved, and greater efficiency of collection proce

Множественная миелома высокого риска: от молекулярной классификации к новаторскому лечению с помощью Моноклональных антител и режиссёрской терапии Т-Келл.

Большие генетические миеломы по-прежнему являются серьезной терапевтической проблемой, поскольку пациенты, имеющие неблагоприятные генетические аномалии, такие, как дель(17p), TP53 мутации и biallelic del(1p32), по-прежнему сталкиваются с плохими результатами, несмотря на недавние терапевтические достижения. В рамках этого обзора изучаются меняющиеся определения и молекулярные характеристики HRMM с уделением особого внимания последним обновленным данным о стратегиях стратификации и лечения рисков. Новая генетическая классификация, предложенная на 2025 Совещание ЕММА обеспечивает более высокую

AL Amyloidosis: Current Chemotherapy and Immune Therapy Treatment Strategies: JACC: CardioOncology State-of-the-Art Review.

Immunoglobulin light chain (AL) amyloidosis is an incurable plasma cell disorder characterized by deposition of fibrils of misfolded immunoglobulin free light chains (FLC) in target organs, leading to failure. Cardiac involvement is common in AL amyloidosis and represents the single most adverse prognostic feature. A high index of clinical suspicion with rapid tissue diagnosis and commencement of combinatorial, highly effective cytoreductive therapy is crucial to arrest the process of amyloid deposition and preserve organ function. The clinical use of molecularly targeted drugs, such as protea

Нацеливание на Икароса и Айолоса: следующие циклоны Цереблона Е3 Ligase Modulars в MM.

Множественная миелома (ММ) остается неизлечимой нечистотой плазменных клеток, характеризующейся повторяющимися рецидивами и, в конечном итоге, рефракторностью стандартных агентов, включая протеазонные ингибиторы (ИП), иммунодуляторные препараты (ИМИД), и anti-CD38 Моноклональные антитела. В связи с тем, что прифронтовые терапевтические стратегии все чаще предусматривают использование четырехструнных индукционных режимов и продолжительное техническое обслуживание линалидоидной системы, все более широко распространено сопротивление традиционным IMID, что создает срочную потребность в модуляторах

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… Beyond the doublet backbone, iberdomide has also been explored in several rationally designed triplet combinations. In the IberDd cohort—iberdomide, daratumumab, and dexamethasone—an overall response rate of 46% was achieved among 43 patients at a median follow‐up of 4.2 months. … Across all triplets, non‐hematologic grade > 3 toxicities were uncommon. Rash, fatigue, or gastrointestinal events were generally mild, suggesting that the enhanced immunomodulatory profile of iberdomide is not accompanied by disproportionate off‐target systemic toxicity [ 36 ]. …

Клиническое и экономическое бремя лиц, получающих пособия по уходу за больными, с множественными миеломами и почечными расстройствами: исследование наблюдений.

Данные о реальных клинических и экономических результатах лечения пациентов с множественными миеломой (ММ) и почечными расстройствами (РИ) в Соединенных Штатах ограничены. Цель этого ретроспективного исследования состояла в том, чтобы подготовить обновленную всеобъемлющую оценку клинических и экономических результатов лечения пациентов с РИ с использованием поддающихся идентификации данных исследования " Медикэр " с увязкой с частью D, что могло бы помочь в оценке общего клинического и социально-экономического бремени этих пациентов с высокой степенью риска и труднодоступным лечением

Teclistamab for Multiple Myeloma: Clinical Insights and Practical Considerations for a First-in-Class Bispecific Antibody.

Teclistamab is a BCMAxCD3 bispecific antibody, the first approved for the treatment of relapsed or refractory multiple myeloma. Given its impressive efficacy in heavily pretreated patients and better accessibility compared to BCMA-directed CAR T cells, teclistamab is sure to become a staple of relapsed/refractory multiple myeloma therapy. Teclistamab carries a set of notable adverse effects including cytokine release syndrome (CRS), infections, and neurotoxicity for which providers must take unique precautions and prophylactic measures. Here, we review the preclinical and clinical data, which

Ixazomib with cyclophosphamide and dexamethasone in relapsed or refractory myeloma: MUKeight phase II randomised controlled trial results.

The all-oral combination of ixazomib, cyclophosphamide, and dexamethasone (ICD) is well tolerated and effective in newly diagnosed and relapsed multiple myeloma (MM). We carried out MUKeight, a randomised, controlled, open, parallel group, multi-centre phase II trial in patients with relapsed MM after prior treatment with thalidomide, lenalidomide, and a proteasome inhibitor (ISRCTN58227268), with the primary objective to test whether ICD has improved clinical activity compared to cyclophosphamide and dexamethasone (CD) in terms of progression-free survival (PFS). Between January 2016 and Dece

Quality of life analyses in patients with multiple myeloma: results from the Selinexor (KPT-330) Treatment of Refractory Myeloma (STORM) phase 2b study.

BACKGROUND: Selinexor is an oral, selective nuclear export inhibitor. STORM was a phase 2b, single-arm, open-label, multicenter trial of selinexor with low dose dexamethasone in patients with penta-exposed relapsed/refractory multiple myeloma (RRMM) that met its primary endpoint, with overall response of 26% (95% confidence interval [CI], 19 to 35%). Health-related quality of life (HRQoL) was a secondary endpoint measured using the Functional Assessment of Cancer Therapy - Multiple Myeloma (FACT-MM). This study examines impact of selinexor treatment on HRQoL of patients treated in STORM and re

Selinexor, bortezomib, and dexamethasone versus bortezomib and dexamethasone in previously treated multiple myeloma: Outcomes by cytogenetic risk.

In the phase 3 BOSTON study, patients with multiple myeloma (MM) after 1-3 prior regimens were randomized to once-weekly selinexor (an oral inhibitor of exportin 1 [XPO1]) plus bortezomib-dexamethasone (XVd) or twice-weekly bortezomib-dexamethasone (Vd). Compared with Vd, XVd was associated with significant improvements in median progression-free survival (PFS), overall response rate (ORR), and lower rates of peripheral neuropathy, with trends in overall survival (OS) favoring XVd. In BOSTON, 141 (35.1%) patients had MM with high-risk (presence of del[17p], t[4;14], t[14;16], or ≥4 copies of a

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… Moreover, the use of weekly bortezomib in the XVd arm reflects the more common clinical practice when using bortezomib‐based triplets. … Given that most patients now receive lenalidomide‐based therapy in the frontline setting, a non‐IMiD based, simple triplet regimen with reduced long‐term toxicity (e.g., peripheral neuropathy) and without the need for intravenous or prolonged subcutaneous infusions makes it a viable option in the treatment …

Risk Stratification in Multiple Myeloma: Putting the Pieces Together.

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Melflufen in Multiple Myeloma: Clinical Limitations, Biological Rationale, and Future Perspectives.

Despite transformative therapeutic advances, multiple myeloma (MM) remains an incurable malignancy characterized by sequential relapses and progressive treatment resistance. Patients with heavily pretreated relapsed or refractory MM continue to face limited therapeutic options and poor outcomes. Melflufen (melphalan flufenamide) is a peptide-drug conjugate that enhances intracellular delivery of alkylating agents via aminopeptidase-mediated activation. Early clinical studies demonstrated encouraging activity in advanced MM, leading to accelerated approval by the U.S. Food and Drug Administrati

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… Combination regimens incorporating these agents, often administered as triplets or quadruplets, now represent the standard of care in both newly diagnosed and relapsed disease settings [ 8 , 9 ]. … Although the triplet regimen demonstrated improved response rates (ORR 59% vs. 30%), the study was prematurely terminated following a partial clinical hold requested by the U.S. Food and Drug Administration during the regulatory reassessment of melflufen ( Table 1 ). 5. …

Attrition rates and treatment outcomes in multiple myeloma: real-world data over a 40-year period.

The treatment landscape of multiple myeloma (MM) has evolved significantly over four decades, driven by novel therapies and optimized supportive care. However, the attrition rate (AR), defined as the proportion of patients who die without advancing to the next line of therapy (LOT) after treatment failure, remains a major challenge. To assess how treatment patterns and outcomes have evolved, we analyzed 1,297 MM patients treated between 1980 and 2020, stratified by diagnosis period and age. ARs declined from 38-55% in the 1980s to 15-20% in 2010-2020, but remained high in older patients, with

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… In fourth-line therapy, no significant PFS differences were found, and the use of K-based, CD38-based, and triplet regimens was minimal (Supplementary Fig. 2 ). … While PFS improvements remain concentrated in first-line treatment, introducing triplets and novel agents has extended disease control and OS across successive lines. …