Real-life effectiveness of carfilzomib in patients with relapsed multiple myeloma receiving treatment in the context of early access: The CARMYN study.

The real-life retrospective observational study CARMYN aimed at investigating the long-term efficacy and safety of carfilzomib in combination with dexamethasone and lenalidomide (KRd, 159 patients). These patients (62% in first and 38% in second relapse, median age 62 yo) were treated between 02/2014 and 02/2017. Most had been pre-exposed to bortezomib (98.2%) and to an IMID (75.4%). At the time of collection, 90% had permanently discontinued carfilzomib. Data collection was conducted from January to July 2021 in 27 participating sites, after a median of 39 months follow-up. For patients treated with KRd, an overall response rate of 78.4% translated in a median progression free survival (PFS) of 24.0 months (95% CI 18.8-27.6) and a median overall survival (OS) of 51.1 months (95% CI 41.3-not reached). Results were poorer but difficult to interpret in the small cohort of Kd recipients. The study is one of the longest real-life studies of carfilzomib treatment in patients in first or second relapse. CARMYN confirmed the real-life long-term efficacy of carfilzomib in combination with lenalidomide and dexamethasone with results similar to those of clinical trials. The KRd regimen is thus an option to consider for late relapses in the current context of MM management.

Dialysis Reimbursement: What Impact Do Different Models Have on Clinical Choices?

Allowing patients to live for decades without the function of a vital organ is a medical miracle, but one that is not without cost both in terms of morbidity and quality of life and in economic terms. Renal replacement therapy (RRT) consumes between 2% and 5% of the overall health care expenditure in countries where dialysis is available without restrictions. While transplantation is the preferred treatment in patients without contraindications, old age and comorbidity limit its indications, and low organ availability may result in long waiting times. As a consequence, 30⁻70% of the patients depend on dialysis, which remains the main determinant of the cost of RRT. Costs of dialysis are differently defined, and its reimbursement follows different rules. There are three main ways of establishing dialysis reimbursement. The first involves dividing dialysis into a series of elements and reimbursing each one separately (dialysis itself, medications, drugs, transportation, hospitalisation, etc.). The second, known as the capitation system, consists of merging these elements in a per capita reimbursement, while the third, usually called the bundle system, entails identifying a core of procedures intrinsically linked to treatment (e.g., dialysis sessions, tests, intradialyitc drugs). Each one has advantages and drawbacks, and impacts differently on the organization and delivery of care: payment per session may favour fragmentation and make a global appraisal difficult; a correct capitation system needs a careful correction for comorbidity, and may exacerbate competition between public and private settings, the latter aiming at selecting the least complex cases; a bundle system, in which the main elements linked to the dialysis sessions are considered together, may be a good compromise but risks penalising complex patients, and requires a rapid adaptation to treatment changes. Retarding dialysis is a clinical and economical goal, but the incentives for predialysis care are not established and its development may be unfavourable for the provider. A closer cooperation between policymakers, economists and nephrologists is needed to ensure a high quality of dialysis care.

Where Are You Going, Nephrology? Considerations on Models of Care in an Evolving Discipline.

Nephrology is a complex discipline, including care of kidney disease, dialysis, and transplantation. While in Europe, about 1:10 individuals is affected by chronic kidney disease (CKD), 1:1000 lives thanks to dialysis or transplantation, whose costs are as high as 2% of all the health care budget. Nephrology has important links with surgery, bioethics, cardiovascular and internal medicine, and is, not surprisingly, in a delicate balance between specialization and comprehensiveness, development and consolidation, cost constraints, and competition with internal medicine and other specialties. This paper proposes an interpretation of the different systems of nephrology care summarising the present choices into three not mutually exclusive main models ("scientific", "pragmatic", "holistic", or "comprehensive"), and hypothesizing an "ideal-utopic" prevention-based fourth one. The so-called scientific model is built around kidney transplantation and care of glomerulonephritis and immunologic diseases, which probably pose the most important challenges in our discipline, but do not mirror the most common clinical problems. Conversely, the pragmatic one is built around dialysis (the most expensive and frequent mode of renal replacement therapy) and pre-dialysis treatment, focusing attention on the most common diseases, the holistic, or comprehensive, model comprehends both, and is integrated by several subspecialties, such as interventional nephrology, obstetric nephrology, and the ideal-utopic one is based upon prevention, and early care of common diseases. Each model has strength and weakness, which are commented to enhance discussion on the crucial issue of the philosophy of care behind its practical organization. Increased reflection and research on models of nephrology care is urgently needed if we wish to rise to the challenge of providing earlier and better care for older and more complex kidney patients with acute and chronic kidney diseases, with reduced budgets.

Meningeal involvement in multiple myeloma.

A patient with multiple myeloma with a mass in the nasopharyngeal was diagnosed. He received melphalan autograft and radiotherapy, and obtained complete remission. He relapsed 3 months later, with meningeal involvement and without systemic relapse. He received intrathecal and systemic chemotherapy, without neurological improvement and died 4 weeks after relapse.