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PADIMAC trial Multiple myeloma, autologous stem cell transplantation, high dose therapy, myeloma therapy Keywords: multiple myeloma; autologous stem cell transplantation; high dose therapy; myeloma therapy EN multiple myeloma autologous stem cell transplantation high dose therapy myeloma therapy e33 e37 5 02/14/22 20220215 NES 220215 Current Phase III trials demonstrate the benefit of up-front autologous stem cell transplant (ASCT) as standard of care for transplant-eligible newly diagnosed patients with multiple myeloma.1-3 Deeper responses and achievement of minimal residual disease (MRD) negativity with current triplet and quadruplet treatments4 has questioned timing and role of ASCT with deferral being preferable for some.5,6 Long-term analysis of the EMN02 trial shows that high-risk patients gain most survival benefit from up-front ASCT3 and such risk-stratified approach to ASCT with standard risk patients receiving deferred ASCT, may be acceptable.7 We previously reported primary results of the Phase II PADIMAC trial, which demonstrated a median progression-free survival (PFS) of 17-0 months [95% confidence interval (CI): 10-5-23-2] for 63 patients who achieved a very good partial response (VGPR) or better post induction and stopped treatment until disease progression.8 MRD-positive patients at day 100 had a median PFS of 9-9 months (95% CI: 5-8-23-2) compared to 24-8 months (95% CI: 18-3-34-2) for MRD-negative patients. [Extracted from the article]

Keywords: Castleman disease; interleukins; POEMS; KSHV/HHV8; HIV; TAFRO EN Castleman disease interleukins POEMS KSHV/HHV8 HIV TAFRO 328 337 10 11/01/21 20211101 NES 211101 Methodology This "Good Practice Paper" was compiled according to the British Society for Haematology (BSH) process at (http://www.b-s-h.org.uk/guidelines). Despite the success of IL-6-signalling blockade, there remains a significant unmet clinical need not only for patients who are intolerant of anti-IL-6 therapy, but also for a reliable disease-modifying therapy.50 Tocilizumab is a monoclonal antibody that antagonises the IL-6 receptor (IL-6R) to block signal transduction.57 Tocilizumab is licensed in Japan, but not in the UK, for the treatment of iMCD-TAFRO. Survival rates for MCD are not clear given changes in in diagnostic criteria, reporting and therapy.7 Nevertheless, the 5-year OS rate for MCD have been estimated at ˜65%.4 Human herpesvirus 8-associated MCD (HHV8-MCD) A proportion of cases of MCD have been attributed to infection with HHV8. The choice of therapy in MCD depends on the presentation of the patient as well as identification of drivers of disease: HHV8-MCD, POEMS-MCD, iMCD (NOS and TAFRO). [Extracted from the article]

Background: Patients with multiple myeloma, an incurable haematological cancer, often receive palliative care only late in their trajectory. Criteria for early referral are lacking. Aim: To identify which patients might benefit from early integration, by identifying trajectories of health-related quality of life and the determinants for declining or poor Health related quality of life. Design: Prospective, longitudinal cohort study. Participants: Multiple myeloma patients at all stages (newly diagnosed, first-line or second-line treatment, early or later treatment-free interval, refractory disease) from in- and outpatient units at 14 hospitals in England were recruited. In addition to clinical information and standardised Health related quality of life and psychological aspects, the Myeloma Patient Outcome Scale (MyPOS) measured palliative care concerns. Results: A total of 238 patients were recruited, on average 3.5 years (SD : 3.4) post-diagnosis. Latent mixture growth models identified four Health related quality of life trajectories. Classes 3 and 4 represent trajectories of stable poor Health related quality of life or declining Health related quality of life over an 8-month period. The strongest predictors of poor outcome at the end of follow-up were general symptom level (odds ratio (OR): 1.3, 95% CI: 1.0–1.6, p = 0.028), presence of clinically relevant anxiety (OR: 1.2, 95% confidence interval (CI): 1.0–1.4, p = 0.019), and presence of pain (OR: 1.02, 95% CI: 1.0–1.1, p = 0.018), all being more predictive than demographic or clinical characteristics. Conclusion: General symptom level, pain and presence of anxiety predict declining Health related quality of life in multiple myeloma. Identification of patients with palliative care needs should focus on assessing patient-reported symptoms and psychosocial well-being for identifying those at risk of deterioration. [ABSTRACT FROM AUTHOR]

Objectives Multiple myeloma ( MM) is an incurable haematological disease. Due to novel agents, overall survival has improved in this group, yet there are no systematic reviews to understand the symptom profiles resulting from disease and treatment-related toxicities. We aimed to synthesise data on the prevalence of symptoms in patients with MM. Methods A systematic database and grey literature search were conducted in six databases. Random-effects meta-analysis with inverse variance weighting to pool prevalence data was performed. Results Thirty-six studies were included of which 34 studies ( N = 3023) provided data for meta-analysis. Twenty-seven distinct symptoms were reported, with the majority of studies focusing on pain ( n = 27), fatigue ( n = 19) and problems with functioning ( n = 15). The most prevalent symptoms were fatigue (98.8%, 95% CI 98.1-99.2%), pain (73%, 39.9-91.7), constipation (65.2%, 22.9-92.2) and tingling in the hands/feet with 53.4% (0.4-99.7). The most common problems were decreased physical functioning (98.9%, 98.2-99.3), decreased cognitive functioning (80.2%, 40-96.1) and financial difficulties (78.4%, 39.1-95.4). These problems were present in newly diagnosed to advanced disease stage. Conclusions Optimal quality of life and good symptom management in this incurable disease can only be achieved by routinely assessing symptoms throughout the disease trajectory. [ABSTRACT FROM AUTHOR]

Background: Multiple myeloma, the second most common haematological cancer, remains incurable. Its incidence is rising due to population ageing. Despite the impact of the disease and its treatment, not much is known on who is most in need of supportive and palliative care. This study aimed to (a) assess symptom severity, palliative care concerns and health-related quality of life (HRQOL) in patients with multiple myeloma, and (b) to determine which factors are associated with a lower quality of life. We further wanted to know (c) whether general symptom level has a stronger influence on HRQOL than disease characteristics.Methods: This multi-centre cross-sectional study sampled two cohorts of patients with multiple myeloma from 18 haematological cancer centres in the UK. The Myeloma Patient Outcome Scale (MyPOS) was used to measure symptoms and concerns. Measures of quality of life included the EORTC QLQ-C30, its myeloma module and the EuroQoL EQ-5D. Data were collected on socio-demographic, disease and treatment characteristics and phase of illness. Point prevalence of symptoms and concerns was determined. Multiple regression models quantified relationships between independent factors and the MyPOS, EORTC global quality of life item and EQ5D Index.Results: Five-hundred-fifty-seven patients, on average 3.5 years (SD: 3.4) post-diagnosis, were recruited. 18.2 % had newly diagnosed disease, 47.9 % were in a treatment-free interval and 32.7 % had relapsed/progressive disease phase. Patients reported a mean of 7.2 symptoms (SD: 3.3) out of 15 potential symptoms. The most common symptoms were pain (72 %), fatigue (88 %) and breathlessness (61 %). Those with relapsed/progressive disease reported the highest mean number of symptoms and the highest overall palliative care concerns (F = 9.56, p < 0.001). Factors associated with high palliative care concerns were a general high symptom level, presence of pain, anxiety, low physical function, younger age, and being in the advanced stages of disease.Conclusion: Patients with multiple myeloma have a high symptom burden and low HRQOL, in the advanced and the earlier stages of disease. Identification of patients in need of supportive care should focus on assessing patient-reported outcomes such as symptoms and functioning regularly in clinical practice, complementary to traditional biomedical markers. [ABSTRACT FROM AUTHOR]

Background: Multiple myeloma is an incurable cancer with a rising incidence globally. Less toxic treatments are increasingly available, so patients are living longer and treatment decisions are increasingly guided by QOL concerns. There is no QOL assessment tool designed specifically for use in the clinical care of people with myeloma. This study aimed to develop and test the psychometric properties of a new myeloma-specific QOL questionnaire designed specifically for use in the clinical setting - the MyPOS. Methods: The MyPOS was developed using findings from a previously reported literature review and qualitative study. The prototype MyPOS was pretested using cognitive interviews in a purposive sample of myeloma patients and refined prior to field testing. The psychometric properties of the MyPOS were evaluated in a multi-centre, cross sectional survey of myeloma patients recruited from 14 hospital trusts across England. Results: The prototype MyPOS contained 33 structured and open questions. These were refined using cognitive interviews with 12 patients, and the final MyPOS contained 30 items taken forward for field-testing. The cross-sectional survey recruited 380 patients for the MyPOS validation. Mean time to complete was 7 minutes 19 seconds with 0.58% missing MyPOS items overall. Internal consistency was high (α = 0.89). Factor analysis confirmed three subscales: Symptoms & Function; Emotional Response and Healthcare Support. MyPOS total scores were higher (worse QOL) in those with active disease compared to those in the stable or plateau phase (F = 11.89, p < 0.001) and were worse in those currently receiving chemotherapy (t = 3.42, p = 0.001). Scores in the Symptoms & Function subscale were higher (worse QOL) in those with worse ECOG performance status (F = 31.33, p < 0.001). Good convergent and discriminant validity were demonstrated. Conclusions: The MyPOS is the first myeloma-specific QOL questionnaire designed specifically for use in the clinical setting. The MyPOS is based on qualitative enquiry and the issues most important to patients. It is a brief, comprehensive and acceptable tool that is reliable and valid on psychometric testing. The MyPOS can now be used to support clinical decision making in the routine care of myeloma patients. [ABSTRACT FROM AUTHOR]

Background: Multiple myeloma is an incurable haematological cancer that affects physical, psychological and social domains of quality of life (QOL). Treatment decisions are increasingly guided by QOL issues, creating a need to monitor QOL within clinical practice. The development of myeloma-specific QOL questionnaires has been limited by a paucity of research to fully characterise QOL in this group. Aims of the present study are to (1) explore the issues important to QOL from the perspective of people with multiple myeloma, and (2) explore the views of patients and clinical staff on existing QOL questionnaires and their use in clinical practice. Methods: The 'Issues Interviews' were semi-structured qualitative interviews to explore the issues important to QOL in a purposive sample of myeloma patients (n = 20). The 'Questionnaire Interviews' were semi-structured qualitative interviews in a separate purposive sample of myeloma patients (n = 20) to explore views on existing QOL questionnaires and their clinical use. Two patient focus groups (n = 7, n = 4) and a focus group of clinical staff (n = 6) complemented the semi-structured interviews. Thematic content analysis resulted in the development of a theoretical model of QOL in myeloma. Results: Main themes important to QOL were Biological Status, Treatment Factors, Symptoms Status, Activity & Participation, Emotional Status, Support Factors, Expectations, Adaptation & Coping and Spirituality. Symptoms had an indirect effect on QOL, only affecting overall QOL if they impacted upon Activity & Participation, Emotional Status or Support Factors. This indirect relationship has implications for the design of QOL questionnaires, which often focus on symptom status. Health-service factors emerged as important but are often absent from QOL questionnaires. Sexual function was important to patients and difficult for clinicians to discuss, so inclusion in clinical QOL tools may flag hidden problems and facilitate better care. Patients and staff expressed preferences for questionnaires to be no more than 2 pages long and to include a mixture of structured and open questions to focus the goals of care on what is most important to patients. Conclusion: Existing QOL questionnaires developed and validated for use in myeloma do not capture all that is important to patients and may not be well suited to clinical use. [ABSTRACT FROM AUTHOR]