The future of autologous stem cell transplantation in myeloma

Rhee, F. Van; Giralt, Sergío; Barlogie, Bart

doi:10.1182/blood-2014-03-561985

Cited by 42 publications

(33 citation statements)

References 48 publications

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“…Although real‐world methods for measuring high‐quality response are likely to be less rigorous than those used in clinical trials, we found that physician‐evaluated TTP correlated with depth of response. Furthermore, in accordance with published reports (van Rhee et al , 2014), patients who had undergone SCT were more likely to achieve CR than those who had not. Moreover, efficacy outcomes were broadly similar to those seen in clinical trials (Singhal et al , 1999; Richardson et al , 2005; Dimopoulos et al , 2007; Weber et al , 2007; San Miguel et al , 2013).…”

Section: Discussionsupporting

confidence: 90%

Multiple myeloma: patient outcomes in real‐world practice

et al. 2016

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SummaryWith 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.

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Section: Discussionsupporting

confidence: 90%

Multiple myeloma: patient outcomes in real‐world practice

et al. 2016

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“…Although novel agents substantially improved the outcome of first-line therapy, the use of ASCT has not declined over the past years. Importantly, despite data showing that overall survival does not differ comparing upfront and delayed ASCT (13), the following arguments still favour ASCT for eligible patients: The long treatment-free interval, the high quality of life following ASCT, and the possibility for long-term disease-free survival beyond 14 years (14). In consequence, only 8% of patients in the current survey received a delayed ASCT procedure (13,15).…”

Section: Discussionmentioning

confidence: 99%

Diagnostic and therapeutic approaches to multiple myeloma patients: ‘Real-world’ data from representative multicentre treatment surveys in Germany between 2008 and 2011

Michel

Merz

Kellermann³

et al. 2016

Oncology Letters

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A survey was conducted to investigate the standard of care for multiple myeloma in Germany, in order to clarify the status of implementation of international and national treatment guidelines. In addition, the changes in disease management over time were investigated by comparison with surveys conducted in 2008 and 2009. The survey captured a representative sample of 478 myeloma patients with a mean age of 67.9 years across various stages of the disease. Diagnostic approaches, prognostic aspects and treatment decisions were evaluated based on a survey conducted in 2011 in 58 representative centres in Germany, including university and non-university hospitals and office-based haematologists. Data were collected from chart reviews and were analysed retrospectively. Over time, an increasing number of patients were investigated for cytogenetic abnormalities (53%). Age <69 years and lack of comorbid conditions were major determinants for cytogenetic testing. Bortezomib/chemotherapy-based regimens have become the preferred first-line treatments independent of planning autologous blood stem cell transplantation (ASCT) in first-line therapy. Thalidomide- and lenalidomide-based combination therapies are typically used as second-line treatments in 31% of patients. Compared with previous reviews, the frequency of ASCT was stable, at ~30% of patients. Younger age and indicators of more severe disease, such as the presence of CRAB criteria, influenced the decision in favour of performing ASCT. Compared to previous surveys, the requirement for erythropoietin and granulocyte colony-stimulating factor, as well as transfusions of red blood cells and platelets, respectively, have decreased considerably. In summary, novel agents have led to a substantial change in the first-line and relapsed treatment approaches. Age and comorbidities remain major factors influencing treatment decisions, but cytogenetic testing to investigate myeloma-related risk profiles is increasingly integrated. The use of novel agents has affected supportive care, with reduced necessity for substitute blood products and reduced administration of bone marrow-stimulating factors.

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“…These results suggest not only that MRD kinetics is more informative than single time-point assessments, but also that this information may be useful to address specific clinical questions (eg, early vs delayed HDT/ASCT for CR patients after induction). 96 Maintenance therapy represents another illustrating example. In the Gruppo Italiano Malattie EMatologiche dell'Adulto (GIMEMA) VEL-03-096 study, Ladetto et al reported PFS rates at median followup of 100% vs 57% for patients in molecular-CR vs MRD-positive cases, respectively.…”

Section: Mrd and Treatment Schemamentioning

confidence: 99%

New criteria for response assessment: role of minimal residual disease in multiple myeloma

Paiva

Dongen

Órfão

2015

Blood

263

271

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Assessment of minimal residual disease (MRD) is becoming standard diagnostic care for potentially curable neoplasms such as acute lymphoblastic leukemia. In multiple myeloma (MM), the majority of patients will inevitably relapse despite achievement of progressively higher complete remission (CR) rates. Novel treatment protocols with inclusion of antibodies and small molecules might well be able to further increase remission rates and potentially also cure rates. Therefore, MRD diagnostics becomes essential to assess treatment effectiveness. This review summarizes reports from the past 2 decades, which demonstrate that persistent MRD by multiparameter flow cytometry, polymerase chain reaction, next-generation sequencing, and positron emission tomography/computed tomography, predicts significantly inferior survival among CR patients. We describe the specific features of currently available techniques for MRD monitoring and outline the arguments favoring new criteria for response assessment that incorporate MRD levels. Extensive data indicate that MRD information can potentially be used as biomarker to evaluate the efficacy of different treatment strategies, help on treatment decisions, and act as surrogate for overall survival. The time has come to address within clinical trials the exact role of baseline risk factors and MRD monitoring for tailored therapy in MM, which implies systematic usage of highly sensitive, cost-effective, readily available, and standardized MRD techniques.

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The future of autologous stem cell transplantation in myeloma

Cited by 42 publications

References 48 publications

Multiple myeloma: patient outcomes in real‐world practice

Multiple myeloma: patient outcomes in real‐world practice

Diagnostic and therapeutic approaches to multiple myeloma patients: ‘Real-world’ data from representative multicentre treatment surveys in Germany between 2008 and 2011

New criteria for response assessment: role of minimal residual disease in multiple myeloma

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