Healthcare resource utilization and costs among patients with relapsed and/or refractory multiple myeloma treated with proteasome inhibitors in real-world clinical practice in Germany.

AIMS: To assess the real-world healthcare resource utilization (HRU) and costs associated with different proteasome inhibitors (PIs) for the treatment of patients with relapsed and/or refractory multiple myeloma (RRMM) in Germany. METHODS: We conducted a retrospective medical chart review of treatment patterns, outcomes, and HRU for patients with RRMM treated with bortezomib, carfilzomib, or ixazomib in second- or third-line (2L or 3L) therapy in Germany. Data were collected between 1 January 2017 and 30 June 2017. Costs were calculated based on drug prices and unit costs in Germany. RESULTS: Physicians provided data on 302 patients. Mean monthly total direct costs per patient receiving PI-based therapy were €7,925 and €10,693 for 2L and 3L, respectively, of which approximately 90% was anti-myeloma drug costs. Overall, the highest costs were associated with patients receiving 3L therapy. Regardless of treatment line, costs were higher for patients who had received a stem cell transplant (SCT) in a previous treatment line than for those who had not; the data suggest that this reflects the use of triplet regimens following a SCT. Patients with a complete response (CR) experienced no unplanned hospitalizations during the study period, whereas patients with progressive disease experienced the highest number of unplanned and planned hospitalizations. In 2L therapy, the highest proportion of patients with a CR was observed in those receiving carfilzomib (12% carfilzomib; 4% bortezomib; 0% ixazomib). LIMITATIONS: Patients with missing or incomplete follow-up data were included in the study and were accounted for using monthly cost estimates. CONCLUSIONS: Anti-myeloma drugs were the main contributor to total HRU costs associated with RRMM in Germany. Improved treatment response was associated with lower costs and reduced hospitalizations.

Bone complications in patients with multiple myeloma in five European countries: a retrospective patient chart review.

BACKGROUND: Bone complications (pathologic fracture, spinal cord compression, surgery to bone and radiation to bone) are a common problem in patients with multiple myeloma (MM). We set out to provide insights into the real-world burden of bone complications in patients with newly diagnosed MM (NDMM). METHODS: We conducted a retrospective review of medical charts of patients with NDMM whose disease had progressed following first-line treatment in the 3 months before data collection in 2016 in five European countries (France, Germany, Italy, Spain and the United Kingdom). RESULTS: The aggregated study population included 813 patients. Bone pain commonly led to MM diagnosis (63%) and 74% of all patients had two or more bone lesions at initiation of first-line treatment. Furthermore, 26% of patients experienced a new bone complication between MM diagnosis and disease progression following first-line treatment, despite 75% of individuals receiving bisphosphonates. Most bone complications (52%) occurred in the period before initiation of first-line treatment (mean duration: 2.3 months) and more than half of patients (56%) who experienced a new bone complication were hospitalised. Analgesics were used more frequently in patients with bone complications than in those without them (76% vs 50%, respectively). Furthermore, 51% of patients had renal impairment by the time first-line treatment was started. Overall, 25% of patients did not receive bisphosphonates for prevention of bone complications and one in four of those with renal impairment at initiation of first-line treatment did not receive bisphosphonates. CONCLUSIONS: Bone complications are common in patients with NDMM. They are frequently associated with hospitalization and analgesic use. Data from this study, conducted in the era of novel anti-myeloma therapies and before the approval of denosumab for use in patients with MM, suggest that although most patients (75%) received bisphosphonates, use of anti-resorptive therapy for prevention of bone complications may be suboptimal in patients with NDMM, irrespective of renal function.

Understanding mortality in multiple myeloma: Findings of a European retrospective chart review.

OBJECTIVES: This study aimed to provide real-world data on the characteristics and treatment of patients with multiple myeloma (MM) at the time of death. METHODS: The study was a retrospective patient chart review across France, Germany, Italy, Spain and the UK during 2016, and included patients who had died in the 3 months before the index date. RESULTS: Data from 786 patients were reviewed. At the time of death, 37% of patients were receiving active treatment, 12% were in a treatment-free interval and 51% were receiving only supportive care. Death before and during active first-line treatment was not uncommon (6% and 24% of patients, respectively) but these deaths were often not solely due to disease progression; factors such as renal failure and infection frequently played a role (in 30% and 20% of patients at first-line, respectively). Most deaths at later lines were due to progressive disease. Cox model results suggested that early deaths were associated with advanced disease stage, high-risk cytogenetics and poor response and relapse profiles. CONCLUSIONS: These real-world data could be used to help develop strategies for improving survival in patients with MM and to support management tailored to the stage of disease.

Real­world multiple myeloma management practice patterns and outcomes in selected Central and Eastern European countries.

Introduction Multiple myeloma (MM) treatment has evolved substantially in recent years. Solid data on the impact of treatment strategies on patient outcomes beyond clinical trials are scarce, especially in budget­restricted environments with limited access to new treatments. Objectives This study investigated treatment practices, patterns, and outcomes in real­world clinical practice in Bulgaria, Croatia, Czech Republic, Poland, Romania, and Slovakia. Patients and methods This was a noninterventional, observational chart review comprising a cross­sectional and a retrospective longitudinal phase observing adult patients with symptomatic MM at all stages of therapy. Results The study revealed structural differences in clinical practice compared with a similarly designed study previously conducted in 7 Western European countries. Stem cell transplantation was performed in less than half of newly diagnosed eligible patients. The most frequently used first­line regimens were bortezomib based, with frequent bortezomib retreatment after the first relapse. Lenalidomide­based regimens were predominant in the third and subsequent lines of therapy. Depth of response decreased with each treatment line, with half of patients achieving at least very good partial response (≥VGPR) in the first line, while only one­fourth achieved ≥VGPR in the third or subsequent lines. Time to progression was longer in patients with better response levels. Conclusions Inadequate access to advanced antimyeloma regimens and-in some countries-stem cell transplantation highlights the challenges of MM treatment in the region. Information on real­world patient management and its outcomes can provide valuable input for decision makers to effectively allocate limited resources.

Healthcare resource utilization among patients with relapsed multiple myeloma in the UK, France, and Italy.

AIMS: To assess the real-world healthcare resource utilization (HRU) and costs associated with different treatment regimens used in the management of patients with relapsed multiple myeloma in the UK, France, and Italy. METHODS: Retrospective medical chart review of characteristics, time to progression, level of response, HRU during treatment, and adverse events (AEs). Data collection started on June 1, 2015 and was completed on July 15, 2015. In the 3 months before record abstraction, eligible patients had either disease progression after receiving one of their country's most commonly prescribed regimens or had received the best supportive care and died. Costs were calculated based on HRU and country-specific diagnosis-related group and/or unit reference costs, amongst other standard resources. RESULTS: Physicians provided data for 1,282 patients (387 in the UK, 502 in France, 393 in Italy) who met the inclusion criteria. Mean [median] total healthcare costs associated with a single line of treatment were €51,717 [35,951] in the UK, €37,009 [32,538] for France, and €34,496 [42,342] for Italy, driven largely by anti-myeloma medications costs (contributing 95.0%, 90.0%, and 94.2% of total cost, respectively). During active treatment, the highest costs were associated with lenalidomide- and pomalidomide-based regimens. Mean cost per month was lowest for patients achieving a very good partial response or better. Unscheduled events (i.e. not considered part of routine management, whether or not related to multiple myeloma, such as unscheduled hospitalization, AEs, fractures) accounted for 1-9% of total costs and were highest for bendamustine. LIMITATIONS: The use of retrospective data means that clinical practice (e.g. use of medical procedures, evaluation of treatment response) is not standardized across participating countries/centers, and some data (e.g. low-grade AEs) may be incomplete or differently adjudicated/reported. The centers involved may not be fully representative of national practice. CONCLUSIONS: Drug costs are the main contributor to total HRU costs associated with multiple myeloma. The duration of active treatment may influence the average total costs, as well as response, associated with a single line of therapy. Improved treatment outcomes, and reductions in unscheduled events and concomitant medication use may, therefore, reduce the overall HRU and related costs of care in multiple myeloma.

Making decisions on resectability and injectability status of lesions in the management of advanced melanoma in Germany, France and the UK.

There is anecdotal evidence of variation in the treatment of patients with metastatic melanoma. We aimed to describe the decision-making process physicians use to define resectability and injectability in patients with metastatic melanoma, and to identify patient characteristics associated with unresectable and injectable lesions. Physicians in Germany, France and the UK who manage patients with metastatic melanoma completed a questionnaire and case report forms on lesion resectability and injectability. In total, 122 physicians participated in the study, collecting data on 1,193 patients. Physicians' resection history was the main factor impacting their resection decisions; those who had frequently performed resections in the past were more likely to consider a lesion resectable than those who had rarely performed resections. A physician's decision to resect varied according to field of expertise; 46% of oncologists rarely performed resections, but this was the case for only 10% of dermatologists and 26% of dermato-oncologists. Another important factor affecting resectability status was the number of in-transit lesions; 49% of patients with three or more in-transit lesions were considered resectable compared with 73% of patients with fewer than three in-transit lesions. Lesion location impacted on injectability status; cutaneous and regional lymph node lesions were often considered injectable, whereas distant lesions in the bone, brain, lung, and liver were considered uninjectable. Assessment of resectability status was influenced by physicians' resection history; this varied according to field of expertise, and may reflect the lack of clear guidance on resection for patients with advanced melanoma.

Multiple myeloma: patient outcomes in real-world practice.

With increasing number of therapies available for the treatment of multiple myeloma, it is timely to examine the course of patients' journeys. We investigated patient characteristics, treatment durations and outcomes, and symptom burden across the treatment pathway in Belgium, France, Germany, Italy, Spain, Switzerland and the UK. In total, 435 physicians retrospectively reviewed 4997 patient charts. Profiles of patients diagnosed with multiple myeloma during the last 12 months were similar across countries; bone pain was the most common presentation. Median duration of first-line therapy was 6 months, followed by a median treatment-free interval of 10 months; both these decreased with increasing lines of therapy, as did time to progression. Depth of response, as assessed by the treating physician, also decreased with each additional line of therapy: 74% of patients achieved at least a very good partial response at first line, compared with only 11% at fifth line. Deeper responses were associated with longer time to progression, although these were physician-judged. Toxicities and co-morbidities increased with later treatment lines, and were more likely to have led to discontinuation of treatment. These real-world data provide an insight into patient outcomes and treatment decisions being made in clinical practice.

Multiple myeloma: practice patterns across Europe.

Real-world data describing management of patients with multiple myeloma are limited. A European (Belgium, France, Germany, Italy, Spain, Switzerland, UK) observational chart review was conducted to address this. Physicians completed questionnaires for every patient seen during a 2-4-week observation period, regardless of treatment status. A total of 435 physicians completed 7635 cross-sectional chart reviews. Overall, 47% of patients were undergoing anti-tumour drug treatment, 42% had previously received ≥1 line of treatment and 12% had never received anti-tumour drug treatment. Of the patients treated by oncologists, onco-haematologists or internists, 95% received, or were expected to receive, at least one line of anti-tumour drug treatment, 61% received ≥2 lines of therapy and 38% received ≥3 lines. Except in the UK, the most commonly used induction therapies contained bortezomib (48%); lenalidomide was the most commonly used first-line maintenance therapy (45%) and second- and third-line agent overall (60% and 52% of patients at those lines, respectively). Bortezomib retreatment was used in 47% of patients who received it first line. Treatment patterns became more diverse with subsequent treatment lines. This study provides insight into real-world treatment patterns in Europe. While treatment practices are broadly similar across countries, some notable differences in the agents used exist.