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Multiple myeloma: patient outcomes in real-world practice.

ABSTRACT: 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.

SUBMITTER: Yong K

PROVIDER: S-EPMC5096152 | biostudies-literature | 2016 Oct

REPOSITORIES: biostudies-literature

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Multiple myeloma: patient outcomes in real-world practice.

Yong Kwee K Delforge Michel M Driessen Christoph C Fink Leah L Flinois Alain A Gonzalez-McQuire Sebastian S Safaei Reza R Karlin Lionel L Mateos Maria-Victoria MV Raab Marc S MS Schoen Paul P Cavo Michele M

British journal of haematology 20160713 2

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 acro ...[more]

PMID: 27411022

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Project description:Most patients cannot be included in randomized clinical trials. We report real-world outcomes of all Danish patients with multiple myeloma (MM) treated with daratumumab-based regimens until 1 January 2019.MethodsInformation of 635 patients treated with daratumumab was collected retrospectively and included lines of therapy (LOT), hematologic responses according to the International Myeloma Working Group recommendations, time to next treatment (TNT) and the cause of discontinuation of treatment. Baseline characteristics were acquired from the validated Danish Multiple Myeloma Registry (DMMR).ResultsDaratumumab was administrated as monotherapy (Da-mono) in 27.7%, in combination with immunomodulatory drugs (Da-IMiD) in 57.3%, in combination with proteasome inhibitors (Da-PI) in 11.2% and in other combinations (Da-other) in 3.8% of patients. The median number of lines of therapy given before daratumumab was 5 for Da-mono, 3 for Da-IMiD, 4 for Da-PI, and 2 for Da-other. In Da-mono, overall response rate (ORR) was 44.9% and median time to next treatment (mTNT) was 4.9 months. In Da-IMiD, ORR was 80.5%, and mTNT was 16.1 months. In Da-PI, OOR was 60.6% and mTNT was 5.3 months. In patients treated with Da-other, OOR was 54,2% and mTNT was 5.6 months. The use of daratumumab in early LOT was associated with longer TNT (p<0.0001). Patients with amplification 1q had outcome comparable to standard risk patients, while patients with t(4;14), t(14;16) or del17p had worse outcome (p = 0.0001). Multivariate analysis indicated that timing of treatment (timing of daratumumab in the sequence of all LOT that the patients received throughout the course of their disease) was the most important factor for outcome (p<0.0001).ConclusionThe real-world outcomes of multiple myeloma patients treated with daratumumab are worse than the results of clinical trials. Outcomes achieved with daratumumab were best when daratumumab was used in combination with IMIDs and in early LOT. Patients with high-risk CA had worse outcomes, but patients with amp1q had similar outcomes to standard-risk patients.

Project description:This study describes how health care providers approach canagliflozin for the treatment of patients with type 2 diabetes mellitus (T2DM) in the real world.An Internet-based questionnaire was completed by 101 endocrinologists, 101 primary care physicians, and 100 nurse practitioners/physician assistants (NP/PAs). Health care providers were required to have experience prescribing or managing patients using canagliflozin to be included in the study. Health care providers compared canagliflozin with other T2DM medication classes on clinical characteristics, costs, and patient satisfaction. Confidence in canagliflozin was also measured. Health care providers reported their canagliflozin prescribing experience and good candidate characteristics for treatment. Finally, providers reported on patient outcomes among those receiving canagliflozin. All variables were compared across provider type.Health care providers reported higher favorability for canagliflozin for blood pressure and body weight compared with dipeptidyl peptidase-4 (DPP-4) inhibitors and higher favorability for effect on blood pressure, body weight, treatment satisfaction, and glycosylated hemoglobin (HbA1c) compared with sulfonylureas (SUs), with differences observed for effect on blood pressure. Health care providers reported being very/extremely confident (55%-74%) with canagliflozin as a second-to fourth-line treatment. The top 3 characteristics reported by the providers, in terms of describing a good candidate for canagliflozin, include those concerned about their weight, insurance coverage/affordability, and avoiding injectable treatments. Finally, providers reported often/always observing patients' lowering or controlling HbA1c (82%-88%) and improvement in overall quality of life (QoL; 50%-53%) with canagliflozin treatment. No differences were observed across provider type for confidence, good candidate characteristics, or patient outcomes.Health care providers reported favorable experiences with canagliflozin and witnessed improvements in patients' clinical outcomes and QoL.

Project description:IntroductionThe objective of this study was to describe the treatment patterns among patients with newly diagnosed multiple myeloma (MM) who had not received autologous stem cell transplantation (ASCT). It further compares the safety and clinical outcomes across different frontline regimens as well as explores whether treatment duration predicts outcomes.MethodsPatients with MM (> 45 years) who had not received ASCT were retrospectively identified from the US SEER-Medicare (Jan 2007-Dec 2016) and Optum (Jan 2007-Sep 2018) databases. Cox proportional hazard models were used to compare overall survival (OS) among bortezomib + lenalidomide + dexamethasone regimen (VRd), lenalidomide + dexamethasone regimen (Rd), cyclophosphamide + bortezomib + dexamethasone regimen (CyBorD), bortezomib + dexamethasone regimen (Vd), and other bortezomib-containing therapies based on propensity score matching. To address immortal time bias, time-fixed and time-dependent Cox models were employed to estimate the association of longer frontline treatment exposure with outcomes.ResultsMean (standard deviation; SD) age was 71 (9.8) years; and 49.51% were women. Bortezomib and lenalidomide-based combinations were the most common treatment modalities. After matching, the HR (95% CI) of OS by frontline therapies comparing VRd with Vd was 0.76 (0.66, 0.86), CyBorD was 0.87 (0.75, 1.05), for other bortezomib-based therapies was 0.56 (0.49, 0.64), Rd was 0.83 (0.73, 0.95), and for other therapies was 0.70 (0.61, 0.80). Longer frontline treatment duration was associated with better OS for overall frontline [HR (95% CI) 0.86 (0.82, 0.90)]; Vd [0.81 (0.74, 0.89)]; CyBorD [0.79 (0.64, 0.98)] and Rd [0.86 (0.78, 0.95)].ConclusionResults demonstrated that the frontline therapies prescribed to most patients who did not receive ASCT for MM in the United States were consistent with the NCCN guideline recommendations. Longer frontline treatment duration was associated with improved OS.

Dataset Information

Multiple myeloma: patient outcomes in real-world practice.

Publications

Multiple myeloma: patient outcomes in real-world practice.

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